Parkinson's Disease
Conditions
Brief summary
Primary Objectives: * Part 1: To determine the safety and tolerability of 4, 8, and 15 milligrams of GZ/SAR402671 (venglustat) administered orally for 4 weeks, as compared to placebo in participants with early-stage Parkinson's disease (PD) carrying a glucocerebrosidase gene (GBA) mutation or other pre-specified variants. * Part 2: To determine the efficacy of GZ/SAR402671 administered orally daily, as compared to placebo in participants with early-stage PD carrying a GBA mutation or other pre-specified variants. Secondary Objectives: Part 1: * To assess the pharmacokinetic (PK) profile of oral dosing of GZ/SAR402671 in plasma when administered in early-stage PD participants carrying a GBA mutation. * To assess the exposure of GZ/SAR402671 in cerebrospinal fluid (CSF) when administered in early-stage PD participants carrying a GBA mutation. Part 2: * To demonstrate overall safety and tolerability of GZ/SAR402671 administered orally for 52 weeks in early-stage PD participants carrying a GBA mutation as compared to placebo. * To assess the pharmacodynamic response to daily oral dosing of GZ/SAR402671 in plasma and CSF as measured by glucosylceramide (GL-1) when administered in early-stage PD participants carrying a GBA mutation over a 52-week period.
Detailed description
Part 1: the total duration was as following: i) Rest of the world (ROW): up to approximately 50 weeks (8.5 weeks of screening, maximum of 36 weeks of treatment and 6 weeks follow-up). ii) Japan only: up to approximately 66 weeks (8.5 weeks of screening, maximum of 52 weeks of treatment and 6 weeks follow-up). Part 2: the total duration was up to approximately 224 weeks that consisted of 8.5 weeks of screening period, 52 weeks of treatment period, 156 weeks of long-term follow-up (LTFU) period and 8 weeks of post-treatment period. At the end of a 52-week main placebo-controlled treatment period, all participants were evaluated for possibility to transition to receive active treatment for 156 weeks plus 8-week post-treatment observation.
Interventions
Pharmaceutical form: capsule Route of administration: oral
DRUGPlacebo
Pharmaceutical form: capsule Route of administration: oral
Sponsors
Genzyme, a Sanofi Company
Study design
Allocation
RANDOMIZED
Intervention model
PARALLEL
Primary purpose
TREATMENT
Masking
TRIPLE (Subject, Investigator, Outcomes Assessor)
Eligibility
Sex/Gender
ALL
Age
18 Years to 80 Years
Healthy volunteers
No
Inclusion criteria
* Male and female adults with a diagnosis of PD and who were heterozygous carriers of a GBA mutation associated with PD. * Participants carrying known sequence variants associated with GBA-PD must had rapid eye movement (REM) sleep behavior disorder (RBD) confirmed by historically documented polysomnography or by questionnaire. * Age greater than or equal to (\>=) 18 years to 80 years inclusive at the time of informed consent signing (FOR JAPANESE PARTICIPANTS ONLY: Age \>=20 years to 80 years, inclusive, at the time of signing the informed consent. Note: Japanese participants refers only to Japanese participants enrolled and living in Japan). * Had symptoms of PD \>=2 years. * Hoehn and Yahr (H and Y) stage of 2 or lower at baseline. * Stable medication regimen of PD drugs for at least 30 days (at least 60 days for rasagiline) prior to randomization. * The participant was willing to abstain from grapefruit containing products for 72 hours prior to administration of the first dose of GZ/SAR402671 and for the duration of the entire treatment period (Part 1 and Part 2, Periods 2 and 3). * Signed written consent.
Exclusion criteria
* Parkinsonism due to drug(s) or toxin(s). * Participants carrying the LRRK2 G2019S mutation. * Participants with Gaucher disease (GD) as defined by clinical signs and symptoms (i.e., hepatosplenomegaly, cytopenia, skeletal disease) and/or marked deficiency of GCase activity compatible with GD. * Montreal Cognitive Assessment score less than 20. * Participants with prior surgical history of deep brain stimulation (DBS). * Participants with baseline brain MRI without contrast showing a structural abnormality that is a possible cause of their PD signs or symptoms. * Hepatic insufficiency with liver function tests (LFT) greater than (\>) 2 times upper limit of normal at Screening Visit. * The participant had a documented diagnosis, as per local regulations, of any of the following infections: hepatitis B, hepatitis C, human immunodeficiency virus 1 or 2. * Renal insufficiency as defined by creatine \>1.5 times normal at Screening Visit. * The participant had received strong or moderate inducers or inhibitors of CYP3A4 within 30 days or 5 half-lives prior to randomization, whichever is longer. * The participant had, according to World Health Organization (WHO) Grading, a cortical cataract \> one-quarter the lens circumference (grade cortical catact-2 \[COR-2\]) or a posterior subcapsular cataract \>2 millimeters (grade posterior subscapsular cataract \[PSC-2\]). Participant with nuclear cataracts would not be excluded. * The participant was currently receiving potentially cataractogenic medications, including chronic regimen (more frequently than every 2 weeks) of any dose or route of corticosteroids or any medication that could cause cataract or worsen the vision of participants with cataract (eg, glaucoma medications) according to the Prescribing Information. * If female, pregnant (defined as positive beta-human chorionic gonadotrophin \[Beta-HCG\] blood test) or lactating or breast-feeding. * Any medical disorders and/or clinically relevant findings that, in the opinion of the Investigator, could interfere with study-related procedures. This included condition(s) that precluded the safe performance of routine lumbar punctures, such as prohibitive spinal diseases, bleeding diasthesis, or clinically significant coagulopathy or thrombocytopenia. * Current participation in another investigational interventional study. * Any medications specifically used for treating memory dysfunction, such as, but not limited to cholinesterase inhibitors or memantine, within 30 days or 5 half-lives of these medications prior to randomization, whichever was longer. The above information was not intended to contain all considerations relevant to a participant's potential participation in a clinical trial.
Design outcomes
Primary
| Measure | Time frame | Description |
|---|---|---|
| Part 2: Change From Baseline to Week 52 in Movement Disorder Society-Unified Parkinson's Disease Rating Scale (MDS-UPDRS) Part II+III Total Score | Baseline to Week 52 | MDS-UPDRS is a multimodal scale consisting of 4 parts. Part II assessed motor experiences of daily living (total score range: 0 to 52). It contained 13 questions completed by the participant. Part III assessed the motor signs of PD and was administered by the rater (total score range: 0 to 132). Part III contained 33 scores based on 18 items. In both parts, higher score indicated more severe symptoms. For each question in both parts, numeric score was assigned between 0 to 4, where 0=Normal, 1=Slight, 2=Mild, 3=Moderate, 4=Severe. MDS-UPDRS Total Part II and III score = sum of Part II and III scores with score ranged from 0 (no symptom) to 184 (severe symptoms), where higher scores reflected more severe symptoms of PD. Data for this OM was not planned to be collected and analyzed for Part 1, Part 2: DB period re-randomized participants and Part 2 LTFU period, as pre-specified in protocol. |
| Part 1: Number of Participants With Potentially Clinically Significant Vital Signs Abnormalities | From the first IMP administration up to 6 weeks after the last administration of the IMP (i.e., up to 42 weeks for ROW and up to 58 weeks for Japanese participants) | Criteria for PCSA: Systolic blood pressure (SBP) supine: \<=95 millimeters of mercury (mmHg) and DFB \>=20 mmHg; \>=160 mmHg and increase from baseline (IFB) \>=20 mmHg; Diastolic blood pressure (DBP) supine: \<=45 mmHg and DFB \>=10 mmHg; \>=110 mmHg and IFB \>=10 mmHg; SBP (Orthostatic): \<=-20 mmHg; DBP (Orthostatic): \<=-10 mmHg; Heart rate (HR) supine: \<=50 beats per minute (bpm) and DFB \>=20 bpm; \>=120 bpm and IFB \>=20 bpm; Weight: \>=5% DFB; \>=5% IFB. |
| Part 1: Number of Participants With Potentially Clinically Significant Ophthalmological Abnormalities | From the first IMP administration up to 6 weeks after the last administration of the IMP (i.e., up to 42 weeks for ROW and up to 58 weeks for Japanese participants) | Clinically significant observations in left eye, right eye and any eye (any eye among left and right) were assessed by the investigator based on methods like visual acuity, slit lamp examination, examination of the cornea, lens, and retina. Any abnormal clinically significant ophthalmological examination finding (which was present at screening or not) on any eye during the treatment-emergent period corresponded to cornea verticillata, cataract and abnormal overall evaluation |
| Part 1: Number of Participants With Potentially Clinically Significant Electrocardiogram Abnormalities | From the first IMP administration up to 6 weeks after the last administration of the IMP (i.e., up to 42 weeks for ROW and up to 58 weeks for Japanese participants) | Criteria for PCSA: HR: \<50 bpm; \<50 bpm and DFB \>=20 bpm, \<40 bpm; \>90 bpm, \<90 bpm and IFB \>=20 bpm, \>100 bpm; PR Interval: \>200 milliseconds (msec), \>200 msec and IFB \>=25%, \>220 msec; QRS Interval: \>110 msec, \>110 msec and IFB \>=25%, \>120 msec; QT Interval: \>500 msec; QTc Bazett (QTcB) interval: \>450 msec, \>480 msec, IFB \>30 and \<=60 msec, IFB \>60 msec; QTc Fridericia (QTc F): \>450 msec, \>480 msec, IFB \>30 and \<=60 msec, IFB \>60 msec. |
| Part 1: Number of Participants With Treatment-Emergent Adverse Events (TEAEs), and Treatment-Emergent Serious Adverse Events (TESAEs) | From the first IMP administration up to 6 weeks after the last administration of the IMP (i.e., up to 42 weeks for ROW and up to 58 weeks for Japanese participants) | An adverse event (AE) was defined as any untoward medical occurrence in a participant who received study drug and does not necessarily had to have a causal relationship with the treatment. Serious AEs (SAEs) were any untoward medical occurrence that resulted in any of the following outcomes: death, life-threatening, required initial or prolonged in-patient hospitalization, persistent or significant disability/incapacity, congenital anomaly/birth defect, or considered as medically important event. TEAEs were the AEs that developed or worsened or became serious during the TEAE period (defined as the period from the time of first investigational medicinal product \[IMP\] administration up of 6 weeks after the last administration of the IMP). |
| Part 1: Number of Participants With Abnormal Physical Examination Findings | From the first IMP administration up to 6 weeks after the last administration of the IMP (i.e., up to 42 weeks for ROW and up to 58 weeks for Japanese participants) | Physical examination included following observations/measurements: general appearance; heart, skin, respiratory auscultation; head, eyes, ears, nose, and throat, extremities/joints, and abdomen. New onset of abnormal physical examination was defined as a normal physical examination at Baseline and an abnormal physical examination during the treatment-emergent (TE) period (defined as the period from the time of first IMP administration up of 6 weeks after the last administration of the IMP). Abnormalities in physical examination were based on investigator's evaluation. |
| Part 1: Number of Participants With Abnormal Neurological Examination Findings | From the first IMP administration up to 6 weeks after the last administration of the IMP (i.e., up to 42 weeks for ROW and up to 58 weeks for Japanese participants) | Neurological examination included at least assessments of the participant's cranial nerves, motor system (including muscle atrophy, tone, and power), mental status, deep tendon reflex, sensation, and cerebellar function. Abnormalities in neurological examination were based on investigator's evaluation. |
| Part 1: Number of Participants With Potentially Clinically Significant Laboratory Abnormalities: Hematology | From the first IMP administration up to 6 weeks after the last administration of the IMP (i.e., up to 42 weeks for ROW and up to 58 weeks for Japanese participants) | Criteria for potentially clinically significant abnormalities (PCSA): Hemoglobin: less than or equal to (\<=) 115 grams per liter (g/L) (Male\[M\]) or \<=95 g/L (Female\[F\]), greater than or equal to (\>=) 185 g/L (M) or \>=165 g/L (F), Decrease from baseline (DFB) \>=20 g/L; Hematocrit: \<=0.37 volume/volume (v/v) (M) or \<=0.32 v/v (F), \>=0.55 v/v (M) or \>=0.5 v/v (F); Red blood cells (RBC): \>=6 Tera/L; Platelets: less than (\<) 100 Giga/L, \>=700 Giga/L; White blood cells (WBC): \<3.0 Giga/L (Non-Black \[NB\]) or \<2.0 Giga/L (Black \[B\]), \>=16.0 Giga/L; Neutrophils: \<1.5 Giga/L (NB) or \<1.0 Giga/L (B); Lymphocytes: \<lower limit of normal (LLN), greater than (\>) 4.0 Giga/L; Monocytes: \<LLN, \>0.7 Giga/L; Basophils: \>0.1 Giga/L; Eosinophils: \>0.5 Giga/L or \>upper limit of normal (ULN) (if ULN \>=0.5 Giga/L). |
| Part 1: Number of Participants With Potentially Clinically Significant Laboratory Abnormalities: Liver Function Parameters | From the first IMP administration up to 6 weeks after the last administration of the IMP (i.e., up to 42 weeks for ROW and up to 58 weeks for Japanese participants) | Criteria for PCSA: Alanine Aminotransferase (ALT): \>3 ULN, \>5 ULN; Aspartate aminotransferase (AST): \>3 ULN; Alkaline phosphatase: \>1.5 ULN; Total Bilirubin: \>1.5 ULN; ALT and Bilirubin: \>3 ULN and \>2 ULN; Direct Bilirubin and Bilirubin: \>35% and \>1.5 ULN. |
| Part 1: Number of Participants With Potentially Clinically Significant Laboratory Abnormalities: Renal Parameters | From the first IMP administration up to 6 weeks after the last administration of the IMP (i.e., up to 42 weeks for ROW and up to 58 weeks for Japanese participants) | Criteria for PCSA: Creatinine: \>=150 micromoles per liter (mcmol/L) (adults), \>=30% change from baseline, \>=100% change from baseline; Blood urea nitrogen: \>=17 millimoles (mmol)/L. |
| Part 1: Number of Participants With Potentially Clinically Significant Laboratory Abnormalities: Metabolic Parameters | From the first IMP administration up to 6 weeks after the last administration of the IMP (i.e., up to 42 weeks for ROW and up to 58 weeks for Japanese participants) | Criteria for PCSA: Glucose: \<=3.9 mmol/L and \<LLN; \>=11.1 mmol/L (unfasted \[unfas\]) or \>=7 mmol/L (fasted \[fas\]); Albumin: \<=25 g/L. |
| Part 1: Number of Participants With Potentially Clinically Significant Laboratory Abnormalities: Electrolytes Parameters | From the first IMP administration up to 6 weeks after the last administration of the IMP (i.e., up to 42 weeks for ROW and up to 58 weeks for Japanese participants) | Criteria for PCSA: Sodium: \<=129 mmol/L, \>=160 mmol/L; Potassium: \<3 mmol/L, \>=5.5 mmol/L and Chloride: \<80 mmol/L, \>115 mmol/L. |
Secondary
| Measure | Time frame | Description |
|---|---|---|
| Part 2: Change From Baseline to Week 52 in Movement Disorder Society-Unified Parkinson's Disease Rating Scale (MDS-UPDRS) Part I+ II+III Score | Baseline to Week 52 | MDS-UPDRS is a multimodal scale consisting of 4 parts. Part I assessed non-motor experiences of daily living and has 2 components (total score range: 0 to 52): Part IA contained 6 questions and was assessed by the examiner (total score range: 0 to 24). Part IB contained 7 questions on non-motor experiences of daily living which was completed by the participant (total score range: 0 to 28). Part II (13 questions completed by the participant) assessed motor experiences of daily living (total score range: 0 to 52). Part III assessed motor signs of PD and was administered by the rater (total score range: 0 to 132). Part III contained 33 scores based on 18 items. In all parts, higher score indicated more symptoms. For each question, numeric score was assigned between 0 to 4, where 0=Normal, 1=Slight, 2=Mild, 3=Moderate, 4=Severe. MDS-UPDRS total score = sum of Parts I, II, and III (Range: 0 to 236). Higher score = more severe symptoms of PD. |
| Part 2: Change From Baseline to Week 52 in Hoehn and Yahr (H and Y) Score | Baseline to Week 52 | H and Y scale measured how Parkinson's symptoms progress and the level of disability. Scale allocated stage scores were from 0 to 5 to indicate relative level of disability as: Stage 0: no symptoms; Stage 1: symptoms on one side of the body only; Stage 2: symptoms on both sides of the body, without impairment of balance; Stage 3: Mild to moderate bilateral disease; some postural instability; physically independent; Stage 4: Severe disability; still able to walk or stand unassisted and Stage 5: Wheelchair bound or bedridden unless aided, where higher stage score described an increased severity of disease. |
| Part 2: Change From Baseline to Week 52 in Parkinson's Disease Cognitive Rating Scale (PD-CRS) Total Score | Baseline to Week 52 | The PD-CRS detects early cognitive impairment in Parkinson's disease. It is composed of 2 scales, the fronto-subcortical scale (items: sustained attention, working memory, alternating and action verbal fluency, clock drawing, immediate, and delayed free recall verbal memory) and the posterior-cortical scale (items: confrontation naming and clock copying). The total score of the fronto-subcortical scale (sum of all items) ranged from 0 (worst) to 104 (maximum score indicates better) and the total score of the posterior-cortical scale (sum of all items) ranged from 0 (worst) to 30 (maximum score indicates better). The PD-CRS Total score = the sum of PD-CRS fronto-subcortical score and the PD-CRS posterior-cortical score, which ranged from 0 to 134, where higher score = less impairment. |
Countries
Austria, Canada, France, Germany, Greece, Israel, Italy, Japan, Norway, Portugal, Singapore, Spain, Sweden, Taiwan, United Kingdom, United States
Participant flow
Recruitment details
Study was conducted at 52 sites in 16 countries. A total of 273 participants were enrolled from 15-Dec-2016 to 18-Dec-2019. Study consisted of 2 Parts: Part 1 (dose escalation period) & Part 2 (double-blind \[DB\] treatment period + long-term follow-up \[LTFU\]) period.
Pre-assignment details
Post part 1 completion, 23 eligible & willing participants were re-randomized in Part 2 and were counted again in total enrollment number (273). i.e.,250 unique participants (29 in Part 1+221 in Part 2)+23 re-randomized in Part 2; these 23 participants are displayed only in 'participant flow' & 'adverse events' sections but not in any other section.
Participants by arm
| Arm | Count |
|---|---|
| Part 1: Placebo (ROW) Participants from rest of world (ROW, except Japan), received placebo (matched to venglustat) capsule orally once daily (QD) for up to 36 weeks in Part 1. | 4 |
| Part 1: Venglustat 4 mg (ROW) Participants from ROW (except Japan), received venglustat 4 milligrams (mg) capsule orally QD for up to 36 weeks in Part 1. | 4 |
| Part 1: Venglustat 8 mg (ROW) Participants from ROW (except Japan), received venglustat 8 mg capsule orally QD for up to 36 weeks in Part 1. | 5 |
| Part 1: Venglustat 15 mg (ROW) Participants from ROW (except Japan), received venglustat 15 mg capsule orally QD for up to 36 weeks in Part 1. | 4 |
| Part 1: Placebo (Japan Only) Japanese participants received placebo (matched to venglustat) capsule orally QD for up to 52 weeks in Part 1. | 3 |
| Part 1: Venglustat 4 mg (Japan Only) Japanese participants received venglustat 4 mg capsule orally QD for up to 52 weeks in Part 1. | 3 |
| Part 1: Venglustat 8 mg (Japan Only) Japanese participants received venglustat 8 mg capsule orally QD for up to 52 weeks in Part 1. | 3 |
| Part 1: Venglustat 15 mg (Japan Only) Japanese participants received venglustat 15 mg capsule orally QD for up to 52 weeks in Part 1. | 3 |
| Part 2, DB Period: Placebo Participants received placebo (matched to venglustat) capsule orally QD for 52 weeks in Part 2 DB period. | 111 |
| Part 2, DB Period: Venglustat 15 mg Participants received venglustat 15 mg capsule orally QD for 52 weeks in Part 2 DB period. | 110 |
| Total Title | 250 |
| Total | 500 |
Withdrawals & dropouts
| Period | Reason | FG000 | FG001 | FG002 | FG003 | FG004 | FG005 | FG006 | FG007 | FG008 | FG009 | FG010 | FG011 | FG012 | FG013 | FG014 | FG015 |
|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|
| Part 1: Dose Escalation Period | Adverse Event | 0 | 1 | 0 | 1 | 0 | 0 | 0 | 0 | 0 | 0 | 0 | 0 | 0 | 0 | 0 | 0 |
| Part 2: Double-blind Treatment Period | Adverse Event | 0 | 0 | 0 | 0 | 0 | 0 | 0 | 0 | 6 | 15 | 0 | 0 | 0 | 0 | 0 | 0 |
| Part 2: Double-blind Treatment Period | Other | 0 | 0 | 0 | 0 | 0 | 0 | 0 | 0 | 6 | 10 | 0 | 0 | 0 | 0 | 0 | 0 |
| Part 2: Long-term Follow-up Period | Adverse Event | 0 | 0 | 0 | 0 | 0 | 0 | 0 | 0 | 0 | 0 | 0 | 0 | 10 | 4 | 0 | 0 |
| Part 2: Long-term Follow-up Period | Lack of Efficacy | 0 | 0 | 0 | 0 | 0 | 0 | 0 | 0 | 0 | 0 | 0 | 0 | 1 | 1 | 0 | 0 |
| Part 2: Long-term Follow-up Period | Other | 0 | 0 | 0 | 0 | 0 | 0 | 0 | 0 | 0 | 0 | 0 | 0 | 13 | 8 | 3 | 2 |
| Part 2: Long-term Follow-up Period | Poor Compliance to Protocol | 0 | 0 | 0 | 0 | 0 | 0 | 0 | 0 | 0 | 0 | 0 | 0 | 1 | 4 | 0 | 0 |
| Part 2: Long-term Follow-up Period | Progressive Disease | 0 | 0 | 0 | 0 | 0 | 0 | 0 | 0 | 0 | 0 | 0 | 0 | 7 | 4 | 0 | 1 |
| Part 2: Long-term Follow-up Period | Study terminated by sponsor | 0 | 0 | 0 | 0 | 0 | 0 | 0 | 0 | 0 | 0 | 0 | 0 | 67 | 64 | 7 | 8 |
Baseline characteristics
| Characteristic | Part 1: Placebo (ROW) | Total Title | Part 2, DB Period: Venglustat 15 mg | Part 2, DB Period: Placebo | Part 1: Venglustat 15 mg (Japan Only) | Part 1: Venglustat 8 mg (Japan Only) | Part 1: Venglustat 4 mg (Japan Only) | Part 1: Placebo (Japan Only) | Part 1: Venglustat 15 mg (ROW) | Part 1: Venglustat 8 mg (ROW) | Part 1: Venglustat 4 mg (ROW) |
|---|---|---|---|---|---|---|---|---|---|---|---|
| Age, Continuous | 53.0 years STANDARD_DEVIATION 8.8 | 58.7 years STANDARD_DEVIATION 9.1 | 58.2 years STANDARD_DEVIATION 9.8 | 59.8 years STANDARD_DEVIATION 8.5 | 46.7 years STANDARD_DEVIATION 8.1 | 56.7 years STANDARD_DEVIATION 4 | 61.7 years STANDARD_DEVIATION 5.9 | 52.0 years STANDARD_DEVIATION 10.4 | 63.8 years STANDARD_DEVIATION 8.7 | 61.0 years STANDARD_DEVIATION 6.9 | 55.0 years STANDARD_DEVIATION 3.9 |
| Race (NIH/OMB) American Indian or Alaska Native | 0 Participants | 0 Participants | 0 Participants | 0 Participants | 0 Participants | 0 Participants | 0 Participants | 0 Participants | 0 Participants | 0 Participants | 0 Participants |
| Race (NIH/OMB) Asian | 0 Participants | 29 Participants | 10 Participants | 7 Participants | 3 Participants | 3 Participants | 3 Participants | 3 Participants | 0 Participants | 0 Participants | 0 Participants |
| Race (NIH/OMB) Black or African American | 0 Participants | 0 Participants | 0 Participants | 0 Participants | 0 Participants | 0 Participants | 0 Participants | 0 Participants | 0 Participants | 0 Participants | 0 Participants |
| Race (NIH/OMB) More than one race | 0 Participants | 0 Participants | 0 Participants | 0 Participants | 0 Participants | 0 Participants | 0 Participants | 0 Participants | 0 Participants | 0 Participants | 0 Participants |
| Race (NIH/OMB) Native Hawaiian or Other Pacific Islander | 0 Participants | 0 Participants | 0 Participants | 0 Participants | 0 Participants | 0 Participants | 0 Participants | 0 Participants | 0 Participants | 0 Participants | 0 Participants |
| Race (NIH/OMB) Unknown or Not Reported | 0 Participants | 0 Participants | 0 Participants | 0 Participants | 0 Participants | 0 Participants | 0 Participants | 0 Participants | 0 Participants | 0 Participants | 0 Participants |
| Race (NIH/OMB) White | 4 Participants | 221 Participants | 100 Participants | 104 Participants | 0 Participants | 0 Participants | 0 Participants | 0 Participants | 4 Participants | 5 Participants | 4 Participants |
| Sex: Female, Male Female | 1 Participants | 97 Participants | 45 Participants | 43 Participants | 1 Participants | 1 Participants | 3 Participants | 0 Participants | 1 Participants | 1 Participants | 1 Participants |
| Sex: Female, Male Male | 3 Participants | 153 Participants | 65 Participants | 68 Participants | 2 Participants | 2 Participants | 0 Participants | 3 Participants | 3 Participants | 4 Participants | 3 Participants |
Adverse events
| Event type | EG000 affected / at risk | EG001 affected / at risk | EG002 affected / at risk | EG003 affected / at risk | EG004 affected / at risk | EG005 affected / at risk | EG006 affected / at risk | EG007 affected / at risk | EG008 affected / at risk | EG009 affected / at risk | EG010 affected / at risk | EG011 affected / at risk | EG012 affected / at risk | EG013 affected / at risk | EG014 affected / at risk | EG015 affected / at risk |
|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|
| deaths Total, all-cause mortality | 0 / 4 | 0 / 4 | 0 / 5 | 0 / 4 | 0 / 3 | 0 / 3 | 0 / 3 | 0 / 3 | 0 / 111 | 1 / 110 | 0 / 10 | 0 / 13 | 1 / 87 | 0 / 75 | 0 / 10 | 0 / 11 |
| other Total, other adverse events | 4 / 4 | 4 / 4 | 4 / 5 | 4 / 4 | 2 / 3 | 3 / 3 | 3 / 3 | 2 / 3 | 87 / 111 | 95 / 110 | 8 / 10 | 11 / 13 | 53 / 87 | 39 / 75 | 7 / 10 | 8 / 11 |
| serious Total, serious adverse events | 0 / 4 | 0 / 4 | 0 / 5 | 0 / 4 | 0 / 3 | 0 / 3 | 0 / 3 | 0 / 3 | 12 / 111 | 12 / 110 | 0 / 10 | 3 / 13 | 15 / 87 | 9 / 75 | 3 / 10 | 1 / 11 |
Outcome results
Primary
Part 1: Number of Participants With Abnormal Neurological Examination Findings
Neurological examination included at least assessments of the participant's cranial nerves, motor system (including muscle atrophy, tone, and power), mental status, deep tendon reflex, sensation, and cerebellar function. Abnormalities in neurological examination were based on investigator's evaluation.
Time frame: From the first IMP administration up to 6 weeks after the last administration of the IMP (i.e., up to 42 weeks for ROW and up to 58 weeks for Japanese participants)
Population: Analysis was performed on safety population. This OM was planned to be analyzed and reported for Part 1 only and not for Part 2, as pre-specified in protocol.
| Arm | Measure | Group | Value (COUNT_OF_PARTICIPANTS) |
|---|---|---|---|
| Part 1: Placebo (ROW) | Part 1: Number of Participants With Abnormal Neurological Examination Findings | Abnormal sensory examination | 0 Participants |
| Part 1: Placebo (ROW) | Part 1: Number of Participants With Abnormal Neurological Examination Findings | Abnormal coordination examination | 2 Participants |
| Part 1: Placebo (ROW) | Part 1: Number of Participants With Abnormal Neurological Examination Findings | Abnormal strength examination | 1 Participants |
| Part 1: Placebo (ROW) | Part 1: Number of Participants With Abnormal Neurological Examination Findings | Abnormal cranial nerve examination | 1 Participants |
| Part 1: Placebo (ROW) | Part 1: Number of Participants With Abnormal Neurological Examination Findings | Abnormal motor examination | 3 Participants |
| Part 1: Placebo (ROW) | Part 1: Number of Participants With Abnormal Neurological Examination Findings | Abnormal reflex examination | 0 Participants |
| Part 1: Placebo (ROW) | Part 1: Number of Participants With Abnormal Neurological Examination Findings | Abnormal gait and coordination | 3 Participants |
| Part 1: Venglustat 4 mg (ROW) | Part 1: Number of Participants With Abnormal Neurological Examination Findings | Abnormal gait and coordination | 4 Participants |
| Part 1: Venglustat 4 mg (ROW) | Part 1: Number of Participants With Abnormal Neurological Examination Findings | Abnormal sensory examination | 2 Participants |
| Part 1: Venglustat 4 mg (ROW) | Part 1: Number of Participants With Abnormal Neurological Examination Findings | Abnormal motor examination | 4 Participants |
| Part 1: Venglustat 4 mg (ROW) | Part 1: Number of Participants With Abnormal Neurological Examination Findings | Abnormal strength examination | 4 Participants |
| Part 1: Venglustat 4 mg (ROW) | Part 1: Number of Participants With Abnormal Neurological Examination Findings | Abnormal reflex examination | 1 Participants |
| Part 1: Venglustat 4 mg (ROW) | Part 1: Number of Participants With Abnormal Neurological Examination Findings | Abnormal cranial nerve examination | 1 Participants |
| Part 1: Venglustat 4 mg (ROW) | Part 1: Number of Participants With Abnormal Neurological Examination Findings | Abnormal coordination examination | 1 Participants |
| Part 1: Venglustat 8 mg (ROW) | Part 1: Number of Participants With Abnormal Neurological Examination Findings | Abnormal sensory examination | 0 Participants |
| Part 1: Venglustat 8 mg (ROW) | Part 1: Number of Participants With Abnormal Neurological Examination Findings | Abnormal gait and coordination | 4 Participants |
| Part 1: Venglustat 8 mg (ROW) | Part 1: Number of Participants With Abnormal Neurological Examination Findings | Abnormal reflex examination | 1 Participants |
| Part 1: Venglustat 8 mg (ROW) | Part 1: Number of Participants With Abnormal Neurological Examination Findings | Abnormal motor examination | 4 Participants |
| Part 1: Venglustat 8 mg (ROW) | Part 1: Number of Participants With Abnormal Neurological Examination Findings | Abnormal cranial nerve examination | 1 Participants |
| Part 1: Venglustat 8 mg (ROW) | Part 1: Number of Participants With Abnormal Neurological Examination Findings | Abnormal coordination examination | 1 Participants |
| Part 1: Venglustat 8 mg (ROW) | Part 1: Number of Participants With Abnormal Neurological Examination Findings | Abnormal strength examination | 3 Participants |
| Part 1: Venglustat 15 mg (ROW) | Part 1: Number of Participants With Abnormal Neurological Examination Findings | Abnormal reflex examination | 1 Participants |
| Part 1: Venglustat 15 mg (ROW) | Part 1: Number of Participants With Abnormal Neurological Examination Findings | Abnormal gait and coordination | 2 Participants |
| Part 1: Venglustat 15 mg (ROW) | Part 1: Number of Participants With Abnormal Neurological Examination Findings | Abnormal motor examination | 3 Participants |
| Part 1: Venglustat 15 mg (ROW) | Part 1: Number of Participants With Abnormal Neurological Examination Findings | Abnormal coordination examination | 3 Participants |
| Part 1: Venglustat 15 mg (ROW) | Part 1: Number of Participants With Abnormal Neurological Examination Findings | Abnormal strength examination | 3 Participants |
| Part 1: Venglustat 15 mg (ROW) | Part 1: Number of Participants With Abnormal Neurological Examination Findings | Abnormal cranial nerve examination | 2 Participants |
| Part 1: Venglustat 15 mg (ROW) | Part 1: Number of Participants With Abnormal Neurological Examination Findings | Abnormal sensory examination | 1 Participants |
| Part 1: Placebo (Japan Only) | Part 1: Number of Participants With Abnormal Neurological Examination Findings | Abnormal reflex examination | 0 Participants |
| Part 1: Placebo (Japan Only) | Part 1: Number of Participants With Abnormal Neurological Examination Findings | Abnormal cranial nerve examination | 1 Participants |
| Part 1: Placebo (Japan Only) | Part 1: Number of Participants With Abnormal Neurological Examination Findings | Abnormal motor examination | 3 Participants |
| Part 1: Placebo (Japan Only) | Part 1: Number of Participants With Abnormal Neurological Examination Findings | Abnormal strength examination | 1 Participants |
| Part 1: Placebo (Japan Only) | Part 1: Number of Participants With Abnormal Neurological Examination Findings | Abnormal sensory examination | 0 Participants |
| Part 1: Placebo (Japan Only) | Part 1: Number of Participants With Abnormal Neurological Examination Findings | Abnormal coordination examination | 1 Participants |
| Part 1: Placebo (Japan Only) | Part 1: Number of Participants With Abnormal Neurological Examination Findings | Abnormal gait and coordination | 3 Participants |
| Part 1: Venglustat 4 mg (Japan Only) | Part 1: Number of Participants With Abnormal Neurological Examination Findings | Abnormal reflex examination | 2 Participants |
| Part 1: Venglustat 4 mg (Japan Only) | Part 1: Number of Participants With Abnormal Neurological Examination Findings | Abnormal gait and coordination | 2 Participants |
| Part 1: Venglustat 4 mg (Japan Only) | Part 1: Number of Participants With Abnormal Neurological Examination Findings | Abnormal sensory examination | 1 Participants |
| Part 1: Venglustat 4 mg (Japan Only) | Part 1: Number of Participants With Abnormal Neurological Examination Findings | Abnormal strength examination | 2 Participants |
| Part 1: Venglustat 4 mg (Japan Only) | Part 1: Number of Participants With Abnormal Neurological Examination Findings | Abnormal coordination examination | 0 Participants |
| Part 1: Venglustat 4 mg (Japan Only) | Part 1: Number of Participants With Abnormal Neurological Examination Findings | Abnormal motor examination | 3 Participants |
| Part 1: Venglustat 4 mg (Japan Only) | Part 1: Number of Participants With Abnormal Neurological Examination Findings | Abnormal cranial nerve examination | 3 Participants |
| Part 1: Venglustat 8 mg (Japan Only) | Part 1: Number of Participants With Abnormal Neurological Examination Findings | Abnormal motor examination | 3 Participants |
| Part 1: Venglustat 8 mg (Japan Only) | Part 1: Number of Participants With Abnormal Neurological Examination Findings | Abnormal cranial nerve examination | 3 Participants |
| Part 1: Venglustat 8 mg (Japan Only) | Part 1: Number of Participants With Abnormal Neurological Examination Findings | Abnormal coordination examination | 1 Participants |
| Part 1: Venglustat 8 mg (Japan Only) | Part 1: Number of Participants With Abnormal Neurological Examination Findings | Abnormal sensory examination | 0 Participants |
| Part 1: Venglustat 8 mg (Japan Only) | Part 1: Number of Participants With Abnormal Neurological Examination Findings | Abnormal gait and coordination | 2 Participants |
| Part 1: Venglustat 8 mg (Japan Only) | Part 1: Number of Participants With Abnormal Neurological Examination Findings | Abnormal strength examination | 2 Participants |
| Part 1: Venglustat 8 mg (Japan Only) | Part 1: Number of Participants With Abnormal Neurological Examination Findings | Abnormal reflex examination | 2 Participants |
| Part 1: Venglustat 15 mg (Japan Only) | Part 1: Number of Participants With Abnormal Neurological Examination Findings | Abnormal gait and coordination | 2 Participants |
| Part 1: Venglustat 15 mg (Japan Only) | Part 1: Number of Participants With Abnormal Neurological Examination Findings | Abnormal reflex examination | 0 Participants |
| Part 1: Venglustat 15 mg (Japan Only) | Part 1: Number of Participants With Abnormal Neurological Examination Findings | Abnormal coordination examination | 2 Participants |
| Part 1: Venglustat 15 mg (Japan Only) | Part 1: Number of Participants With Abnormal Neurological Examination Findings | Abnormal cranial nerve examination | 2 Participants |
| Part 1: Venglustat 15 mg (Japan Only) | Part 1: Number of Participants With Abnormal Neurological Examination Findings | Abnormal strength examination | 1 Participants |
| Part 1: Venglustat 15 mg (Japan Only) | Part 1: Number of Participants With Abnormal Neurological Examination Findings | Abnormal sensory examination | 0 Participants |
| Part 1: Venglustat 15 mg (Japan Only) | Part 1: Number of Participants With Abnormal Neurological Examination Findings | Abnormal motor examination | 3 Participants |
Primary
Part 1: Number of Participants With Abnormal Physical Examination Findings
Physical examination included following observations/measurements: general appearance; heart, skin, respiratory auscultation; head, eyes, ears, nose, and throat, extremities/joints, and abdomen. New onset of abnormal physical examination was defined as a normal physical examination at Baseline and an abnormal physical examination during the treatment-emergent (TE) period (defined as the period from the time of first IMP administration up of 6 weeks after the last administration of the IMP). Abnormalities in physical examination were based on investigator's evaluation.
Time frame: From the first IMP administration up to 6 weeks after the last administration of the IMP (i.e., up to 42 weeks for ROW and up to 58 weeks for Japanese participants)
Population: Analysis was performed on safety population. This OM was planned to be analyzed and reported for Part 1 only and not for Part 2, as pre-specified in protocol.
| Arm | Measure | Value (COUNT_OF_PARTICIPANTS) |
|---|---|---|
| Part 1: Placebo (ROW) | Part 1: Number of Participants With Abnormal Physical Examination Findings | 0 Participants |
| Part 1: Venglustat 4 mg (ROW) | Part 1: Number of Participants With Abnormal Physical Examination Findings | 0 Participants |
| Part 1: Venglustat 8 mg (ROW) | Part 1: Number of Participants With Abnormal Physical Examination Findings | 1 Participants |
| Part 1: Venglustat 15 mg (ROW) | Part 1: Number of Participants With Abnormal Physical Examination Findings | 2 Participants |
| Part 1: Placebo (Japan Only) | Part 1: Number of Participants With Abnormal Physical Examination Findings | 0 Participants |
| Part 1: Venglustat 4 mg (Japan Only) | Part 1: Number of Participants With Abnormal Physical Examination Findings | 0 Participants |
| Part 1: Venglustat 8 mg (Japan Only) | Part 1: Number of Participants With Abnormal Physical Examination Findings | 0 Participants |
| Part 1: Venglustat 15 mg (Japan Only) | Part 1: Number of Participants With Abnormal Physical Examination Findings | 0 Participants |
Primary
Part 1: Number of Participants With Potentially Clinically Significant Electrocardiogram Abnormalities
Criteria for PCSA: HR: \<50 bpm; \<50 bpm and DFB \>=20 bpm, \<40 bpm; \>90 bpm, \<90 bpm and IFB \>=20 bpm, \>100 bpm; PR Interval: \>200 milliseconds (msec), \>200 msec and IFB \>=25%, \>220 msec; QRS Interval: \>110 msec, \>110 msec and IFB \>=25%, \>120 msec; QT Interval: \>500 msec; QTc Bazett (QTcB) interval: \>450 msec, \>480 msec, IFB \>30 and \<=60 msec, IFB \>60 msec; QTc Fridericia (QTc F): \>450 msec, \>480 msec, IFB \>30 and \<=60 msec, IFB \>60 msec.
Time frame: From the first IMP administration up to 6 weeks after the last administration of the IMP (i.e., up to 42 weeks for ROW and up to 58 weeks for Japanese participants)
Population: Analysis was performed on safety population. This OM was planned to be analyzed and reported for Part 1 only and not for Part 2, as pre-specified in protocol.
| Arm | Measure | Group | Value (COUNT_OF_PARTICIPANTS) |
|---|---|---|---|
| Part 1: Placebo (ROW) | Part 1: Number of Participants With Potentially Clinically Significant Electrocardiogram Abnormalities | QTcB interval: >450 msec | 0 Participants |
| Part 1: Placebo (ROW) | Part 1: Number of Participants With Potentially Clinically Significant Electrocardiogram Abnormalities | QTc F: IFB >30 and <=60 msec | 0 Participants |
| Part 1: Placebo (ROW) | Part 1: Number of Participants With Potentially Clinically Significant Electrocardiogram Abnormalities | HR: >100 bpm | 0 Participants |
| Part 1: Placebo (ROW) | Part 1: Number of Participants With Potentially Clinically Significant Electrocardiogram Abnormalities | HR: >90 bpm | 0 Participants |
| Part 1: Placebo (ROW) | Part 1: Number of Participants With Potentially Clinically Significant Electrocardiogram Abnormalities | QTcB interval: >480 msec | 0 Participants |
| Part 1: Placebo (ROW) | Part 1: Number of Participants With Potentially Clinically Significant Electrocardiogram Abnormalities | HR: >90 bpm and IFB >=20 bpm | 0 Participants |
| Part 1: Placebo (ROW) | Part 1: Number of Participants With Potentially Clinically Significant Electrocardiogram Abnormalities | QTc F: >480 msec | 0 Participants |
| Part 1: Placebo (ROW) | Part 1: Number of Participants With Potentially Clinically Significant Electrocardiogram Abnormalities | HR: <50 bpm | 0 Participants |
| Part 1: Placebo (ROW) | Part 1: Number of Participants With Potentially Clinically Significant Electrocardiogram Abnormalities | QRS Interval: >110 msec | 0 Participants |
| Part 1: Placebo (ROW) | Part 1: Number of Participants With Potentially Clinically Significant Electrocardiogram Abnormalities | PR Interval: >220 msec | 0 Participants |
| Part 1: Placebo (ROW) | Part 1: Number of Participants With Potentially Clinically Significant Electrocardiogram Abnormalities | QRS Interval: >110 msec and IFB >=25% | 0 Participants |
| Part 1: Placebo (ROW) | Part 1: Number of Participants With Potentially Clinically Significant Electrocardiogram Abnormalities | QT Interval: >500 msec | 0 Participants |
| Part 1: Placebo (ROW) | Part 1: Number of Participants With Potentially Clinically Significant Electrocardiogram Abnormalities | QTcB interval: IFB >30 and <=60 msec | 0 Participants |
| Part 1: Placebo (ROW) | Part 1: Number of Participants With Potentially Clinically Significant Electrocardiogram Abnormalities | QTc F: IFB >60 msec | 0 Participants |
| Part 1: Placebo (ROW) | Part 1: Number of Participants With Potentially Clinically Significant Electrocardiogram Abnormalities | HR: <50 bpm and DFB >=20 bpm | 0 Participants |
| Part 1: Placebo (ROW) | Part 1: Number of Participants With Potentially Clinically Significant Electrocardiogram Abnormalities | PR Interval: >200 msec and IFB >=25% | 0 Participants |
| Part 1: Placebo (ROW) | Part 1: Number of Participants With Potentially Clinically Significant Electrocardiogram Abnormalities | HR: <40 bpm | 0 Participants |
| Part 1: Placebo (ROW) | Part 1: Number of Participants With Potentially Clinically Significant Electrocardiogram Abnormalities | QRS Interval: >120 msec | 0 Participants |
| Part 1: Placebo (ROW) | Part 1: Number of Participants With Potentially Clinically Significant Electrocardiogram Abnormalities | QTcB interval: IFB >60 msec | 0 Participants |
| Part 1: Placebo (ROW) | Part 1: Number of Participants With Potentially Clinically Significant Electrocardiogram Abnormalities | PR Interval: >200 msec | 0 Participants |
| Part 1: Placebo (ROW) | Part 1: Number of Participants With Potentially Clinically Significant Electrocardiogram Abnormalities | QTc F: >450 msec | 0 Participants |
| Part 1: Venglustat 4 mg (ROW) | Part 1: Number of Participants With Potentially Clinically Significant Electrocardiogram Abnormalities | QRS Interval: >110 msec | 1 Participants |
| Part 1: Venglustat 4 mg (ROW) | Part 1: Number of Participants With Potentially Clinically Significant Electrocardiogram Abnormalities | QTcB interval: >450 msec | 0 Participants |
| Part 1: Venglustat 4 mg (ROW) | Part 1: Number of Participants With Potentially Clinically Significant Electrocardiogram Abnormalities | QRS Interval: >120 msec | 0 Participants |
| Part 1: Venglustat 4 mg (ROW) | Part 1: Number of Participants With Potentially Clinically Significant Electrocardiogram Abnormalities | QT Interval: >500 msec | 0 Participants |
| Part 1: Venglustat 4 mg (ROW) | Part 1: Number of Participants With Potentially Clinically Significant Electrocardiogram Abnormalities | HR: <40 bpm | 0 Participants |
| Part 1: Venglustat 4 mg (ROW) | Part 1: Number of Participants With Potentially Clinically Significant Electrocardiogram Abnormalities | QTc F: IFB >30 and <=60 msec | 0 Participants |
| Part 1: Venglustat 4 mg (ROW) | Part 1: Number of Participants With Potentially Clinically Significant Electrocardiogram Abnormalities | HR: >90 bpm | 0 Participants |
| Part 1: Venglustat 4 mg (ROW) | Part 1: Number of Participants With Potentially Clinically Significant Electrocardiogram Abnormalities | HR: <50 bpm | 1 Participants |
| Part 1: Venglustat 4 mg (ROW) | Part 1: Number of Participants With Potentially Clinically Significant Electrocardiogram Abnormalities | QTc F: >480 msec | 0 Participants |
| Part 1: Venglustat 4 mg (ROW) | Part 1: Number of Participants With Potentially Clinically Significant Electrocardiogram Abnormalities | HR: >90 bpm and IFB >=20 bpm | 0 Participants |
| Part 1: Venglustat 4 mg (ROW) | Part 1: Number of Participants With Potentially Clinically Significant Electrocardiogram Abnormalities | QTc F: >450 msec | 0 Participants |
| Part 1: Venglustat 4 mg (ROW) | Part 1: Number of Participants With Potentially Clinically Significant Electrocardiogram Abnormalities | HR: >100 bpm | 0 Participants |
| Part 1: Venglustat 4 mg (ROW) | Part 1: Number of Participants With Potentially Clinically Significant Electrocardiogram Abnormalities | PR Interval: >200 msec | 0 Participants |
| Part 1: Venglustat 4 mg (ROW) | Part 1: Number of Participants With Potentially Clinically Significant Electrocardiogram Abnormalities | QTc F: IFB >60 msec | 0 Participants |
| Part 1: Venglustat 4 mg (ROW) | Part 1: Number of Participants With Potentially Clinically Significant Electrocardiogram Abnormalities | QTcB interval: IFB >60 msec | 0 Participants |
| Part 1: Venglustat 4 mg (ROW) | Part 1: Number of Participants With Potentially Clinically Significant Electrocardiogram Abnormalities | PR Interval: >200 msec and IFB >=25% | 0 Participants |
| Part 1: Venglustat 4 mg (ROW) | Part 1: Number of Participants With Potentially Clinically Significant Electrocardiogram Abnormalities | QTcB interval: IFB >30 and <=60 msec | 0 Participants |
| Part 1: Venglustat 4 mg (ROW) | Part 1: Number of Participants With Potentially Clinically Significant Electrocardiogram Abnormalities | PR Interval: >220 msec | 0 Participants |
| Part 1: Venglustat 4 mg (ROW) | Part 1: Number of Participants With Potentially Clinically Significant Electrocardiogram Abnormalities | HR: <50 bpm and DFB >=20 bpm | 0 Participants |
| Part 1: Venglustat 4 mg (ROW) | Part 1: Number of Participants With Potentially Clinically Significant Electrocardiogram Abnormalities | QTcB interval: >480 msec | 0 Participants |
| Part 1: Venglustat 4 mg (ROW) | Part 1: Number of Participants With Potentially Clinically Significant Electrocardiogram Abnormalities | QRS Interval: >110 msec and IFB >=25% | 0 Participants |
| Part 1: Venglustat 8 mg (ROW) | Part 1: Number of Participants With Potentially Clinically Significant Electrocardiogram Abnormalities | HR: <50 bpm and DFB >=20 bpm | 0 Participants |
| Part 1: Venglustat 8 mg (ROW) | Part 1: Number of Participants With Potentially Clinically Significant Electrocardiogram Abnormalities | QTc F: IFB >60 msec | 0 Participants |
| Part 1: Venglustat 8 mg (ROW) | Part 1: Number of Participants With Potentially Clinically Significant Electrocardiogram Abnormalities | QTcB interval: IFB >60 msec | 0 Participants |
| Part 1: Venglustat 8 mg (ROW) | Part 1: Number of Participants With Potentially Clinically Significant Electrocardiogram Abnormalities | PR Interval: >200 msec | 0 Participants |
| Part 1: Venglustat 8 mg (ROW) | Part 1: Number of Participants With Potentially Clinically Significant Electrocardiogram Abnormalities | HR: >100 bpm | 0 Participants |
| Part 1: Venglustat 8 mg (ROW) | Part 1: Number of Participants With Potentially Clinically Significant Electrocardiogram Abnormalities | QRS Interval: >120 msec | 1 Participants |
| Part 1: Venglustat 8 mg (ROW) | Part 1: Number of Participants With Potentially Clinically Significant Electrocardiogram Abnormalities | HR: <40 bpm | 0 Participants |
| Part 1: Venglustat 8 mg (ROW) | Part 1: Number of Participants With Potentially Clinically Significant Electrocardiogram Abnormalities | QTc F: IFB >30 and <=60 msec | 1 Participants |
| Part 1: Venglustat 8 mg (ROW) | Part 1: Number of Participants With Potentially Clinically Significant Electrocardiogram Abnormalities | PR Interval: >200 msec and IFB >=25% | 0 Participants |
| Part 1: Venglustat 8 mg (ROW) | Part 1: Number of Participants With Potentially Clinically Significant Electrocardiogram Abnormalities | QTcB interval: IFB >30 and <=60 msec | 1 Participants |
| Part 1: Venglustat 8 mg (ROW) | Part 1: Number of Participants With Potentially Clinically Significant Electrocardiogram Abnormalities | QTcB interval: >450 msec | 1 Participants |
| Part 1: Venglustat 8 mg (ROW) | Part 1: Number of Participants With Potentially Clinically Significant Electrocardiogram Abnormalities | HR: <50 bpm | 1 Participants |
| Part 1: Venglustat 8 mg (ROW) | Part 1: Number of Participants With Potentially Clinically Significant Electrocardiogram Abnormalities | QT Interval: >500 msec | 0 Participants |
| Part 1: Venglustat 8 mg (ROW) | Part 1: Number of Participants With Potentially Clinically Significant Electrocardiogram Abnormalities | PR Interval: >220 msec | 0 Participants |
| Part 1: Venglustat 8 mg (ROW) | Part 1: Number of Participants With Potentially Clinically Significant Electrocardiogram Abnormalities | QTcB interval: >480 msec | 0 Participants |
| Part 1: Venglustat 8 mg (ROW) | Part 1: Number of Participants With Potentially Clinically Significant Electrocardiogram Abnormalities | QTc F: >480 msec | 0 Participants |
| Part 1: Venglustat 8 mg (ROW) | Part 1: Number of Participants With Potentially Clinically Significant Electrocardiogram Abnormalities | QRS Interval: >110 msec and IFB >=25% | 0 Participants |
| Part 1: Venglustat 8 mg (ROW) | Part 1: Number of Participants With Potentially Clinically Significant Electrocardiogram Abnormalities | HR: >90 bpm | 0 Participants |
| Part 1: Venglustat 8 mg (ROW) | Part 1: Number of Participants With Potentially Clinically Significant Electrocardiogram Abnormalities | HR: >90 bpm and IFB >=20 bpm | 0 Participants |
| Part 1: Venglustat 8 mg (ROW) | Part 1: Number of Participants With Potentially Clinically Significant Electrocardiogram Abnormalities | QTc F: >450 msec | 1 Participants |
| Part 1: Venglustat 8 mg (ROW) | Part 1: Number of Participants With Potentially Clinically Significant Electrocardiogram Abnormalities | QRS Interval: >110 msec | 1 Participants |
| Part 1: Venglustat 15 mg (ROW) | Part 1: Number of Participants With Potentially Clinically Significant Electrocardiogram Abnormalities | QTc F: >480 msec | 0 Participants |
| Part 1: Venglustat 15 mg (ROW) | Part 1: Number of Participants With Potentially Clinically Significant Electrocardiogram Abnormalities | HR: >100 bpm | 0 Participants |
| Part 1: Venglustat 15 mg (ROW) | Part 1: Number of Participants With Potentially Clinically Significant Electrocardiogram Abnormalities | HR: <50 bpm and DFB >=20 bpm | 0 Participants |
| Part 1: Venglustat 15 mg (ROW) | Part 1: Number of Participants With Potentially Clinically Significant Electrocardiogram Abnormalities | HR: <50 bpm | 0 Participants |
| Part 1: Venglustat 15 mg (ROW) | Part 1: Number of Participants With Potentially Clinically Significant Electrocardiogram Abnormalities | QTcB interval: IFB >60 msec | 0 Participants |
| Part 1: Venglustat 15 mg (ROW) | Part 1: Number of Participants With Potentially Clinically Significant Electrocardiogram Abnormalities | QT Interval: >500 msec | 0 Participants |
| Part 1: Venglustat 15 mg (ROW) | Part 1: Number of Participants With Potentially Clinically Significant Electrocardiogram Abnormalities | HR: <40 bpm | 0 Participants |
| Part 1: Venglustat 15 mg (ROW) | Part 1: Number of Participants With Potentially Clinically Significant Electrocardiogram Abnormalities | QRS Interval: >110 msec | 1 Participants |
| Part 1: Venglustat 15 mg (ROW) | Part 1: Number of Participants With Potentially Clinically Significant Electrocardiogram Abnormalities | QRS Interval: >120 msec | 0 Participants |
| Part 1: Venglustat 15 mg (ROW) | Part 1: Number of Participants With Potentially Clinically Significant Electrocardiogram Abnormalities | PR Interval: >200 msec | 0 Participants |
| Part 1: Venglustat 15 mg (ROW) | Part 1: Number of Participants With Potentially Clinically Significant Electrocardiogram Abnormalities | PR Interval: >220 msec | 0 Participants |
| Part 1: Venglustat 15 mg (ROW) | Part 1: Number of Participants With Potentially Clinically Significant Electrocardiogram Abnormalities | QRS Interval: >110 msec and IFB >=25% | 0 Participants |
| Part 1: Venglustat 15 mg (ROW) | Part 1: Number of Participants With Potentially Clinically Significant Electrocardiogram Abnormalities | QTcB interval: >480 msec | 0 Participants |
| Part 1: Venglustat 15 mg (ROW) | Part 1: Number of Participants With Potentially Clinically Significant Electrocardiogram Abnormalities | QTcB interval: IFB >30 and <=60 msec | 0 Participants |
| Part 1: Venglustat 15 mg (ROW) | Part 1: Number of Participants With Potentially Clinically Significant Electrocardiogram Abnormalities | HR: >90 bpm and IFB >=20 bpm | 0 Participants |
| Part 1: Venglustat 15 mg (ROW) | Part 1: Number of Participants With Potentially Clinically Significant Electrocardiogram Abnormalities | QTcB interval: >450 msec | 0 Participants |
| Part 1: Venglustat 15 mg (ROW) | Part 1: Number of Participants With Potentially Clinically Significant Electrocardiogram Abnormalities | QTc F: IFB >60 msec | 0 Participants |
| Part 1: Venglustat 15 mg (ROW) | Part 1: Number of Participants With Potentially Clinically Significant Electrocardiogram Abnormalities | PR Interval: >200 msec and IFB >=25% | 0 Participants |
| Part 1: Venglustat 15 mg (ROW) | Part 1: Number of Participants With Potentially Clinically Significant Electrocardiogram Abnormalities | QTc F: IFB >30 and <=60 msec | 0 Participants |
| Part 1: Venglustat 15 mg (ROW) | Part 1: Number of Participants With Potentially Clinically Significant Electrocardiogram Abnormalities | QTc F: >450 msec | 0 Participants |
| Part 1: Venglustat 15 mg (ROW) | Part 1: Number of Participants With Potentially Clinically Significant Electrocardiogram Abnormalities | HR: >90 bpm | 0 Participants |
| Part 1: Placebo (Japan Only) | Part 1: Number of Participants With Potentially Clinically Significant Electrocardiogram Abnormalities | QRS Interval: >110 msec and IFB >=25% | 0 Participants |
| Part 1: Placebo (Japan Only) | Part 1: Number of Participants With Potentially Clinically Significant Electrocardiogram Abnormalities | HR: <50 bpm | 1 Participants |
| Part 1: Placebo (Japan Only) | Part 1: Number of Participants With Potentially Clinically Significant Electrocardiogram Abnormalities | HR: <50 bpm and DFB >=20 bpm | 0 Participants |
| Part 1: Placebo (Japan Only) | Part 1: Number of Participants With Potentially Clinically Significant Electrocardiogram Abnormalities | HR: <40 bpm | 0 Participants |
| Part 1: Placebo (Japan Only) | Part 1: Number of Participants With Potentially Clinically Significant Electrocardiogram Abnormalities | HR: >90 bpm | 0 Participants |
| Part 1: Placebo (Japan Only) | Part 1: Number of Participants With Potentially Clinically Significant Electrocardiogram Abnormalities | HR: >90 bpm and IFB >=20 bpm | 0 Participants |
| Part 1: Placebo (Japan Only) | Part 1: Number of Participants With Potentially Clinically Significant Electrocardiogram Abnormalities | HR: >100 bpm | 0 Participants |
| Part 1: Placebo (Japan Only) | Part 1: Number of Participants With Potentially Clinically Significant Electrocardiogram Abnormalities | PR Interval: >200 msec | 0 Participants |
| Part 1: Placebo (Japan Only) | Part 1: Number of Participants With Potentially Clinically Significant Electrocardiogram Abnormalities | PR Interval: >200 msec and IFB >=25% | 0 Participants |
| Part 1: Placebo (Japan Only) | Part 1: Number of Participants With Potentially Clinically Significant Electrocardiogram Abnormalities | PR Interval: >220 msec | 0 Participants |
| Part 1: Placebo (Japan Only) | Part 1: Number of Participants With Potentially Clinically Significant Electrocardiogram Abnormalities | QRS Interval: >110 msec | 0 Participants |
| Part 1: Placebo (Japan Only) | Part 1: Number of Participants With Potentially Clinically Significant Electrocardiogram Abnormalities | QRS Interval: >120 msec | 0 Participants |
| Part 1: Placebo (Japan Only) | Part 1: Number of Participants With Potentially Clinically Significant Electrocardiogram Abnormalities | QT Interval: >500 msec | 0 Participants |
| Part 1: Placebo (Japan Only) | Part 1: Number of Participants With Potentially Clinically Significant Electrocardiogram Abnormalities | QTcB interval: >450 msec | 0 Participants |
| Part 1: Placebo (Japan Only) | Part 1: Number of Participants With Potentially Clinically Significant Electrocardiogram Abnormalities | QTcB interval: >480 msec | 0 Participants |
| Part 1: Placebo (Japan Only) | Part 1: Number of Participants With Potentially Clinically Significant Electrocardiogram Abnormalities | QTcB interval: IFB >30 and <=60 msec | 0 Participants |
| Part 1: Placebo (Japan Only) | Part 1: Number of Participants With Potentially Clinically Significant Electrocardiogram Abnormalities | QTcB interval: IFB >60 msec | 0 Participants |
| Part 1: Placebo (Japan Only) | Part 1: Number of Participants With Potentially Clinically Significant Electrocardiogram Abnormalities | QTc F: >450 msec | 0 Participants |
| Part 1: Placebo (Japan Only) | Part 1: Number of Participants With Potentially Clinically Significant Electrocardiogram Abnormalities | QTc F: >480 msec | 0 Participants |
| Part 1: Placebo (Japan Only) | Part 1: Number of Participants With Potentially Clinically Significant Electrocardiogram Abnormalities | QTc F: IFB >30 and <=60 msec | 0 Participants |
| Part 1: Placebo (Japan Only) | Part 1: Number of Participants With Potentially Clinically Significant Electrocardiogram Abnormalities | QTc F: IFB >60 msec | 0 Participants |
| Part 1: Venglustat 4 mg (Japan Only) | Part 1: Number of Participants With Potentially Clinically Significant Electrocardiogram Abnormalities | QRS Interval: >110 msec and IFB >=25% | 0 Participants |
| Part 1: Venglustat 4 mg (Japan Only) | Part 1: Number of Participants With Potentially Clinically Significant Electrocardiogram Abnormalities | HR: <50 bpm and DFB >=20 bpm | 0 Participants |
| Part 1: Venglustat 4 mg (Japan Only) | Part 1: Number of Participants With Potentially Clinically Significant Electrocardiogram Abnormalities | QTcB interval: >450 msec | 0 Participants |
| Part 1: Venglustat 4 mg (Japan Only) | Part 1: Number of Participants With Potentially Clinically Significant Electrocardiogram Abnormalities | QRS Interval: >110 msec | 0 Participants |
| Part 1: Venglustat 4 mg (Japan Only) | Part 1: Number of Participants With Potentially Clinically Significant Electrocardiogram Abnormalities | PR Interval: >220 msec | 0 Participants |
| Part 1: Venglustat 4 mg (Japan Only) | Part 1: Number of Participants With Potentially Clinically Significant Electrocardiogram Abnormalities | QTcB interval: >480 msec | 0 Participants |
| Part 1: Venglustat 4 mg (Japan Only) | Part 1: Number of Participants With Potentially Clinically Significant Electrocardiogram Abnormalities | PR Interval: >200 msec and IFB >=25% | 0 Participants |
| Part 1: Venglustat 4 mg (Japan Only) | Part 1: Number of Participants With Potentially Clinically Significant Electrocardiogram Abnormalities | QTcB interval: IFB >30 and <=60 msec | 0 Participants |
| Part 1: Venglustat 4 mg (Japan Only) | Part 1: Number of Participants With Potentially Clinically Significant Electrocardiogram Abnormalities | PR Interval: >200 msec | 2 Participants |
| Part 1: Venglustat 4 mg (Japan Only) | Part 1: Number of Participants With Potentially Clinically Significant Electrocardiogram Abnormalities | HR: <50 bpm | 0 Participants |
| Part 1: Venglustat 4 mg (Japan Only) | Part 1: Number of Participants With Potentially Clinically Significant Electrocardiogram Abnormalities | QTcB interval: IFB >60 msec | 0 Participants |
| Part 1: Venglustat 4 mg (Japan Only) | Part 1: Number of Participants With Potentially Clinically Significant Electrocardiogram Abnormalities | HR: >100 bpm | 0 Participants |
| Part 1: Venglustat 4 mg (Japan Only) | Part 1: Number of Participants With Potentially Clinically Significant Electrocardiogram Abnormalities | HR: >90 bpm and IFB >=20 bpm | 0 Participants |
| Part 1: Venglustat 4 mg (Japan Only) | Part 1: Number of Participants With Potentially Clinically Significant Electrocardiogram Abnormalities | QTc F: >450 msec | 0 Participants |
| Part 1: Venglustat 4 mg (Japan Only) | Part 1: Number of Participants With Potentially Clinically Significant Electrocardiogram Abnormalities | HR: >90 bpm | 0 Participants |
| Part 1: Venglustat 4 mg (Japan Only) | Part 1: Number of Participants With Potentially Clinically Significant Electrocardiogram Abnormalities | QTc F: IFB >60 msec | 0 Participants |
| Part 1: Venglustat 4 mg (Japan Only) | Part 1: Number of Participants With Potentially Clinically Significant Electrocardiogram Abnormalities | QTc F: >480 msec | 0 Participants |
| Part 1: Venglustat 4 mg (Japan Only) | Part 1: Number of Participants With Potentially Clinically Significant Electrocardiogram Abnormalities | HR: <40 bpm | 0 Participants |
| Part 1: Venglustat 4 mg (Japan Only) | Part 1: Number of Participants With Potentially Clinically Significant Electrocardiogram Abnormalities | QTc F: IFB >30 and <=60 msec | 0 Participants |
| Part 1: Venglustat 4 mg (Japan Only) | Part 1: Number of Participants With Potentially Clinically Significant Electrocardiogram Abnormalities | QRS Interval: >120 msec | 0 Participants |
| Part 1: Venglustat 4 mg (Japan Only) | Part 1: Number of Participants With Potentially Clinically Significant Electrocardiogram Abnormalities | QT Interval: >500 msec | 0 Participants |
| Part 1: Venglustat 8 mg (Japan Only) | Part 1: Number of Participants With Potentially Clinically Significant Electrocardiogram Abnormalities | QTc F: IFB >30 and <=60 msec | 0 Participants |
| Part 1: Venglustat 8 mg (Japan Only) | Part 1: Number of Participants With Potentially Clinically Significant Electrocardiogram Abnormalities | PR Interval: >200 msec | 0 Participants |
| Part 1: Venglustat 8 mg (Japan Only) | Part 1: Number of Participants With Potentially Clinically Significant Electrocardiogram Abnormalities | HR: <50 bpm and DFB >=20 bpm | 0 Participants |
| Part 1: Venglustat 8 mg (Japan Only) | Part 1: Number of Participants With Potentially Clinically Significant Electrocardiogram Abnormalities | HR: >100 bpm | 0 Participants |
| Part 1: Venglustat 8 mg (Japan Only) | Part 1: Number of Participants With Potentially Clinically Significant Electrocardiogram Abnormalities | QTc F: IFB >60 msec | 0 Participants |
| Part 1: Venglustat 8 mg (Japan Only) | Part 1: Number of Participants With Potentially Clinically Significant Electrocardiogram Abnormalities | QT Interval: >500 msec | 0 Participants |
| Part 1: Venglustat 8 mg (Japan Only) | Part 1: Number of Participants With Potentially Clinically Significant Electrocardiogram Abnormalities | QTcB interval: IFB >60 msec | 0 Participants |
| Part 1: Venglustat 8 mg (Japan Only) | Part 1: Number of Participants With Potentially Clinically Significant Electrocardiogram Abnormalities | QTcB interval: IFB >30 and <=60 msec | 0 Participants |
| Part 1: Venglustat 8 mg (Japan Only) | Part 1: Number of Participants With Potentially Clinically Significant Electrocardiogram Abnormalities | HR: >90 bpm and IFB >=20 bpm | 0 Participants |
| Part 1: Venglustat 8 mg (Japan Only) | Part 1: Number of Participants With Potentially Clinically Significant Electrocardiogram Abnormalities | QRS Interval: >120 msec | 0 Participants |
| Part 1: Venglustat 8 mg (Japan Only) | Part 1: Number of Participants With Potentially Clinically Significant Electrocardiogram Abnormalities | QTc F: >450 msec | 0 Participants |
| Part 1: Venglustat 8 mg (Japan Only) | Part 1: Number of Participants With Potentially Clinically Significant Electrocardiogram Abnormalities | HR: >90 bpm | 1 Participants |
| Part 1: Venglustat 8 mg (Japan Only) | Part 1: Number of Participants With Potentially Clinically Significant Electrocardiogram Abnormalities | QRS Interval: >110 msec | 0 Participants |
| Part 1: Venglustat 8 mg (Japan Only) | Part 1: Number of Participants With Potentially Clinically Significant Electrocardiogram Abnormalities | QTcB interval: >450 msec | 0 Participants |
| Part 1: Venglustat 8 mg (Japan Only) | Part 1: Number of Participants With Potentially Clinically Significant Electrocardiogram Abnormalities | QTc F: >480 msec | 0 Participants |
| Part 1: Venglustat 8 mg (Japan Only) | Part 1: Number of Participants With Potentially Clinically Significant Electrocardiogram Abnormalities | PR Interval: >220 msec | 0 Participants |
| Part 1: Venglustat 8 mg (Japan Only) | Part 1: Number of Participants With Potentially Clinically Significant Electrocardiogram Abnormalities | HR: <40 bpm | 0 Participants |
| Part 1: Venglustat 8 mg (Japan Only) | Part 1: Number of Participants With Potentially Clinically Significant Electrocardiogram Abnormalities | QTcB interval: >480 msec | 0 Participants |
| Part 1: Venglustat 8 mg (Japan Only) | Part 1: Number of Participants With Potentially Clinically Significant Electrocardiogram Abnormalities | PR Interval: >200 msec and IFB >=25% | 0 Participants |
| Part 1: Venglustat 8 mg (Japan Only) | Part 1: Number of Participants With Potentially Clinically Significant Electrocardiogram Abnormalities | QRS Interval: >110 msec and IFB >=25% | 0 Participants |
| Part 1: Venglustat 8 mg (Japan Only) | Part 1: Number of Participants With Potentially Clinically Significant Electrocardiogram Abnormalities | HR: <50 bpm | 0 Participants |
| Part 1: Venglustat 15 mg (Japan Only) | Part 1: Number of Participants With Potentially Clinically Significant Electrocardiogram Abnormalities | HR: <40 bpm | 0 Participants |
| Part 1: Venglustat 15 mg (Japan Only) | Part 1: Number of Participants With Potentially Clinically Significant Electrocardiogram Abnormalities | QTcB interval: IFB >30 and <=60 msec | 1 Participants |
| Part 1: Venglustat 15 mg (Japan Only) | Part 1: Number of Participants With Potentially Clinically Significant Electrocardiogram Abnormalities | QRS Interval: >110 msec | 0 Participants |
| Part 1: Venglustat 15 mg (Japan Only) | Part 1: Number of Participants With Potentially Clinically Significant Electrocardiogram Abnormalities | PR Interval: >200 msec | 0 Participants |
| Part 1: Venglustat 15 mg (Japan Only) | Part 1: Number of Participants With Potentially Clinically Significant Electrocardiogram Abnormalities | HR: >100 bpm | 0 Participants |
| Part 1: Venglustat 15 mg (Japan Only) | Part 1: Number of Participants With Potentially Clinically Significant Electrocardiogram Abnormalities | QRS Interval: >110 msec and IFB >=25% | 0 Participants |
| Part 1: Venglustat 15 mg (Japan Only) | Part 1: Number of Participants With Potentially Clinically Significant Electrocardiogram Abnormalities | QTc F: IFB >30 and <=60 msec | 1 Participants |
| Part 1: Venglustat 15 mg (Japan Only) | Part 1: Number of Participants With Potentially Clinically Significant Electrocardiogram Abnormalities | HR: <50 bpm and DFB >=20 bpm | 0 Participants |
| Part 1: Venglustat 15 mg (Japan Only) | Part 1: Number of Participants With Potentially Clinically Significant Electrocardiogram Abnormalities | QTcB interval: >450 msec | 0 Participants |
| Part 1: Venglustat 15 mg (Japan Only) | Part 1: Number of Participants With Potentially Clinically Significant Electrocardiogram Abnormalities | QTcB interval: IFB >60 msec | 0 Participants |
| Part 1: Venglustat 15 mg (Japan Only) | Part 1: Number of Participants With Potentially Clinically Significant Electrocardiogram Abnormalities | QTcB interval: >480 msec | 0 Participants |
| Part 1: Venglustat 15 mg (Japan Only) | Part 1: Number of Participants With Potentially Clinically Significant Electrocardiogram Abnormalities | HR: >90 bpm and IFB >=20 bpm | 0 Participants |
| Part 1: Venglustat 15 mg (Japan Only) | Part 1: Number of Participants With Potentially Clinically Significant Electrocardiogram Abnormalities | PR Interval: >220 msec | 0 Participants |
| Part 1: Venglustat 15 mg (Japan Only) | Part 1: Number of Participants With Potentially Clinically Significant Electrocardiogram Abnormalities | QRS Interval: >120 msec | 0 Participants |
| Part 1: Venglustat 15 mg (Japan Only) | Part 1: Number of Participants With Potentially Clinically Significant Electrocardiogram Abnormalities | QTc F: IFB >60 msec | 0 Participants |
| Part 1: Venglustat 15 mg (Japan Only) | Part 1: Number of Participants With Potentially Clinically Significant Electrocardiogram Abnormalities | PR Interval: >200 msec and IFB >=25% | 0 Participants |
| Part 1: Venglustat 15 mg (Japan Only) | Part 1: Number of Participants With Potentially Clinically Significant Electrocardiogram Abnormalities | QTc F: >480 msec | 0 Participants |
| Part 1: Venglustat 15 mg (Japan Only) | Part 1: Number of Participants With Potentially Clinically Significant Electrocardiogram Abnormalities | QTc F: >450 msec | 0 Participants |
| Part 1: Venglustat 15 mg (Japan Only) | Part 1: Number of Participants With Potentially Clinically Significant Electrocardiogram Abnormalities | HR: >90 bpm | 1 Participants |
| Part 1: Venglustat 15 mg (Japan Only) | Part 1: Number of Participants With Potentially Clinically Significant Electrocardiogram Abnormalities | HR: <50 bpm | 0 Participants |
| Part 1: Venglustat 15 mg (Japan Only) | Part 1: Number of Participants With Potentially Clinically Significant Electrocardiogram Abnormalities | QT Interval: >500 msec | 0 Participants |
Primary
Part 1: Number of Participants With Potentially Clinically Significant Laboratory Abnormalities: Electrolytes Parameters
Criteria for PCSA: Sodium: \<=129 mmol/L, \>=160 mmol/L; Potassium: \<3 mmol/L, \>=5.5 mmol/L and Chloride: \<80 mmol/L, \>115 mmol/L.
Time frame: From the first IMP administration up to 6 weeks after the last administration of the IMP (i.e., up to 42 weeks for ROW and up to 58 weeks for Japanese participants)
Population: Analysis was performed on safety population. This OM was planned to be analyzed and reported for Part 1 only and not for Part 2, as pre-specified in protocol.
| Arm | Measure | Group | Value (COUNT_OF_PARTICIPANTS) |
|---|---|---|---|
| Part 1: Placebo (ROW) | Part 1: Number of Participants With Potentially Clinically Significant Laboratory Abnormalities: Electrolytes Parameters | Chloride >115 mmol/L | 0 Participants |
| Part 1: Placebo (ROW) | Part 1: Number of Participants With Potentially Clinically Significant Laboratory Abnormalities: Electrolytes Parameters | Sodium <=129 mmol/L | 0 Participants |
| Part 1: Placebo (ROW) | Part 1: Number of Participants With Potentially Clinically Significant Laboratory Abnormalities: Electrolytes Parameters | Sodium >=160 mmol/L | 0 Participants |
| Part 1: Placebo (ROW) | Part 1: Number of Participants With Potentially Clinically Significant Laboratory Abnormalities: Electrolytes Parameters | Potassium <3 mmol/L | 0 Participants |
| Part 1: Placebo (ROW) | Part 1: Number of Participants With Potentially Clinically Significant Laboratory Abnormalities: Electrolytes Parameters | Chloride <80 mmol/L | 0 Participants |
| Part 1: Placebo (ROW) | Part 1: Number of Participants With Potentially Clinically Significant Laboratory Abnormalities: Electrolytes Parameters | Potassium >=5.5 mmol/L | 0 Participants |
| Part 1: Venglustat 4 mg (ROW) | Part 1: Number of Participants With Potentially Clinically Significant Laboratory Abnormalities: Electrolytes Parameters | Potassium <3 mmol/L | 0 Participants |
| Part 1: Venglustat 4 mg (ROW) | Part 1: Number of Participants With Potentially Clinically Significant Laboratory Abnormalities: Electrolytes Parameters | Sodium <=129 mmol/L | 0 Participants |
| Part 1: Venglustat 4 mg (ROW) | Part 1: Number of Participants With Potentially Clinically Significant Laboratory Abnormalities: Electrolytes Parameters | Potassium >=5.5 mmol/L | 0 Participants |
| Part 1: Venglustat 4 mg (ROW) | Part 1: Number of Participants With Potentially Clinically Significant Laboratory Abnormalities: Electrolytes Parameters | Chloride >115 mmol/L | 0 Participants |
| Part 1: Venglustat 4 mg (ROW) | Part 1: Number of Participants With Potentially Clinically Significant Laboratory Abnormalities: Electrolytes Parameters | Sodium >=160 mmol/L | 0 Participants |
| Part 1: Venglustat 4 mg (ROW) | Part 1: Number of Participants With Potentially Clinically Significant Laboratory Abnormalities: Electrolytes Parameters | Chloride <80 mmol/L | 0 Participants |
| Part 1: Venglustat 8 mg (ROW) | Part 1: Number of Participants With Potentially Clinically Significant Laboratory Abnormalities: Electrolytes Parameters | Chloride <80 mmol/L | 0 Participants |
| Part 1: Venglustat 8 mg (ROW) | Part 1: Number of Participants With Potentially Clinically Significant Laboratory Abnormalities: Electrolytes Parameters | Potassium <3 mmol/L | 0 Participants |
| Part 1: Venglustat 8 mg (ROW) | Part 1: Number of Participants With Potentially Clinically Significant Laboratory Abnormalities: Electrolytes Parameters | Sodium >=160 mmol/L | 0 Participants |
| Part 1: Venglustat 8 mg (ROW) | Part 1: Number of Participants With Potentially Clinically Significant Laboratory Abnormalities: Electrolytes Parameters | Potassium >=5.5 mmol/L | 0 Participants |
| Part 1: Venglustat 8 mg (ROW) | Part 1: Number of Participants With Potentially Clinically Significant Laboratory Abnormalities: Electrolytes Parameters | Chloride >115 mmol/L | 0 Participants |
| Part 1: Venglustat 8 mg (ROW) | Part 1: Number of Participants With Potentially Clinically Significant Laboratory Abnormalities: Electrolytes Parameters | Sodium <=129 mmol/L | 0 Participants |
| Part 1: Venglustat 15 mg (ROW) | Part 1: Number of Participants With Potentially Clinically Significant Laboratory Abnormalities: Electrolytes Parameters | Chloride <80 mmol/L | 0 Participants |
| Part 1: Venglustat 15 mg (ROW) | Part 1: Number of Participants With Potentially Clinically Significant Laboratory Abnormalities: Electrolytes Parameters | Sodium <=129 mmol/L | 0 Participants |
| Part 1: Venglustat 15 mg (ROW) | Part 1: Number of Participants With Potentially Clinically Significant Laboratory Abnormalities: Electrolytes Parameters | Sodium >=160 mmol/L | 0 Participants |
| Part 1: Venglustat 15 mg (ROW) | Part 1: Number of Participants With Potentially Clinically Significant Laboratory Abnormalities: Electrolytes Parameters | Potassium <3 mmol/L | 0 Participants |
| Part 1: Venglustat 15 mg (ROW) | Part 1: Number of Participants With Potentially Clinically Significant Laboratory Abnormalities: Electrolytes Parameters | Potassium >=5.5 mmol/L | 0 Participants |
| Part 1: Venglustat 15 mg (ROW) | Part 1: Number of Participants With Potentially Clinically Significant Laboratory Abnormalities: Electrolytes Parameters | Chloride >115 mmol/L | 0 Participants |
| Part 1: Placebo (Japan Only) | Part 1: Number of Participants With Potentially Clinically Significant Laboratory Abnormalities: Electrolytes Parameters | Chloride <80 mmol/L | 0 Participants |
| Part 1: Placebo (Japan Only) | Part 1: Number of Participants With Potentially Clinically Significant Laboratory Abnormalities: Electrolytes Parameters | Sodium >=160 mmol/L | 0 Participants |
| Part 1: Placebo (Japan Only) | Part 1: Number of Participants With Potentially Clinically Significant Laboratory Abnormalities: Electrolytes Parameters | Chloride >115 mmol/L | 0 Participants |
| Part 1: Placebo (Japan Only) | Part 1: Number of Participants With Potentially Clinically Significant Laboratory Abnormalities: Electrolytes Parameters | Sodium <=129 mmol/L | 0 Participants |
| Part 1: Placebo (Japan Only) | Part 1: Number of Participants With Potentially Clinically Significant Laboratory Abnormalities: Electrolytes Parameters | Potassium >=5.5 mmol/L | 0 Participants |
| Part 1: Placebo (Japan Only) | Part 1: Number of Participants With Potentially Clinically Significant Laboratory Abnormalities: Electrolytes Parameters | Potassium <3 mmol/L | 0 Participants |
| Part 1: Venglustat 4 mg (Japan Only) | Part 1: Number of Participants With Potentially Clinically Significant Laboratory Abnormalities: Electrolytes Parameters | Potassium <3 mmol/L | 0 Participants |
| Part 1: Venglustat 4 mg (Japan Only) | Part 1: Number of Participants With Potentially Clinically Significant Laboratory Abnormalities: Electrolytes Parameters | Potassium >=5.5 mmol/L | 0 Participants |
| Part 1: Venglustat 4 mg (Japan Only) | Part 1: Number of Participants With Potentially Clinically Significant Laboratory Abnormalities: Electrolytes Parameters | Sodium >=160 mmol/L | 0 Participants |
| Part 1: Venglustat 4 mg (Japan Only) | Part 1: Number of Participants With Potentially Clinically Significant Laboratory Abnormalities: Electrolytes Parameters | Chloride <80 mmol/L | 0 Participants |
| Part 1: Venglustat 4 mg (Japan Only) | Part 1: Number of Participants With Potentially Clinically Significant Laboratory Abnormalities: Electrolytes Parameters | Sodium <=129 mmol/L | 0 Participants |
| Part 1: Venglustat 4 mg (Japan Only) | Part 1: Number of Participants With Potentially Clinically Significant Laboratory Abnormalities: Electrolytes Parameters | Chloride >115 mmol/L | 0 Participants |
| Part 1: Venglustat 8 mg (Japan Only) | Part 1: Number of Participants With Potentially Clinically Significant Laboratory Abnormalities: Electrolytes Parameters | Potassium >=5.5 mmol/L | 0 Participants |
| Part 1: Venglustat 8 mg (Japan Only) | Part 1: Number of Participants With Potentially Clinically Significant Laboratory Abnormalities: Electrolytes Parameters | Chloride <80 mmol/L | 0 Participants |
| Part 1: Venglustat 8 mg (Japan Only) | Part 1: Number of Participants With Potentially Clinically Significant Laboratory Abnormalities: Electrolytes Parameters | Sodium <=129 mmol/L | 0 Participants |
| Part 1: Venglustat 8 mg (Japan Only) | Part 1: Number of Participants With Potentially Clinically Significant Laboratory Abnormalities: Electrolytes Parameters | Potassium <3 mmol/L | 0 Participants |
| Part 1: Venglustat 8 mg (Japan Only) | Part 1: Number of Participants With Potentially Clinically Significant Laboratory Abnormalities: Electrolytes Parameters | Chloride >115 mmol/L | 0 Participants |
| Part 1: Venglustat 8 mg (Japan Only) | Part 1: Number of Participants With Potentially Clinically Significant Laboratory Abnormalities: Electrolytes Parameters | Sodium >=160 mmol/L | 0 Participants |
| Part 1: Venglustat 15 mg (Japan Only) | Part 1: Number of Participants With Potentially Clinically Significant Laboratory Abnormalities: Electrolytes Parameters | Potassium >=5.5 mmol/L | 0 Participants |
| Part 1: Venglustat 15 mg (Japan Only) | Part 1: Number of Participants With Potentially Clinically Significant Laboratory Abnormalities: Electrolytes Parameters | Potassium <3 mmol/L | 0 Participants |
| Part 1: Venglustat 15 mg (Japan Only) | Part 1: Number of Participants With Potentially Clinically Significant Laboratory Abnormalities: Electrolytes Parameters | Chloride >115 mmol/L | 0 Participants |
| Part 1: Venglustat 15 mg (Japan Only) | Part 1: Number of Participants With Potentially Clinically Significant Laboratory Abnormalities: Electrolytes Parameters | Chloride <80 mmol/L | 0 Participants |
| Part 1: Venglustat 15 mg (Japan Only) | Part 1: Number of Participants With Potentially Clinically Significant Laboratory Abnormalities: Electrolytes Parameters | Sodium <=129 mmol/L | 0 Participants |
| Part 1: Venglustat 15 mg (Japan Only) | Part 1: Number of Participants With Potentially Clinically Significant Laboratory Abnormalities: Electrolytes Parameters | Sodium >=160 mmol/L | 0 Participants |
Primary
Part 1: Number of Participants With Potentially Clinically Significant Laboratory Abnormalities: Hematology
Criteria for potentially clinically significant abnormalities (PCSA): Hemoglobin: less than or equal to (\<=) 115 grams per liter (g/L) (Male\[M\]) or \<=95 g/L (Female\[F\]), greater than or equal to (\>=) 185 g/L (M) or \>=165 g/L (F), Decrease from baseline (DFB) \>=20 g/L; Hematocrit: \<=0.37 volume/volume (v/v) (M) or \<=0.32 v/v (F), \>=0.55 v/v (M) or \>=0.5 v/v (F); Red blood cells (RBC): \>=6 Tera/L; Platelets: less than (\<) 100 Giga/L, \>=700 Giga/L; White blood cells (WBC): \<3.0 Giga/L (Non-Black \[NB\]) or \<2.0 Giga/L (Black \[B\]), \>=16.0 Giga/L; Neutrophils: \<1.5 Giga/L (NB) or \<1.0 Giga/L (B); Lymphocytes: \<lower limit of normal (LLN), greater than (\>) 4.0 Giga/L; Monocytes: \<LLN, \>0.7 Giga/L; Basophils: \>0.1 Giga/L; Eosinophils: \>0.5 Giga/L or \>upper limit of normal (ULN) (if ULN \>=0.5 Giga/L).
Time frame: From the first IMP administration up to 6 weeks after the last administration of the IMP (i.e., up to 42 weeks for ROW and up to 58 weeks for Japanese participants)
Population: Analysis was performed on safety population. This OM was planned to be analyzed and reported for Part 1 only and not for Part 2, as pre-specified in protocol.
| Arm | Measure | Group | Value (COUNT_OF_PARTICIPANTS) |
|---|---|---|---|
| Part 1: Placebo (ROW) | Part 1: Number of Participants With Potentially Clinically Significant Laboratory Abnormalities: Hematology | Platelets: <100 Giga/L | 0 Participants |
| Part 1: Placebo (ROW) | Part 1: Number of Participants With Potentially Clinically Significant Laboratory Abnormalities: Hematology | Lymphocytes: <LLN | 0 Participants |
| Part 1: Placebo (ROW) | Part 1: Number of Participants With Potentially Clinically Significant Laboratory Abnormalities: Hematology | Hemoglobin >=185 g/L (M) or >=165 g/L (F) | 0 Participants |
| Part 1: Placebo (ROW) | Part 1: Number of Participants With Potentially Clinically Significant Laboratory Abnormalities: Hematology | Monocytes: <LLN | 0 Participants |
| Part 1: Placebo (ROW) | Part 1: Number of Participants With Potentially Clinically Significant Laboratory Abnormalities: Hematology | Neutrophils <1.5 Giga/L (NB) or <1.0 Giga/L (B) | 0 Participants |
| Part 1: Placebo (ROW) | Part 1: Number of Participants With Potentially Clinically Significant Laboratory Abnormalities: Hematology | Basophils: >0.1 Giga/L | 0 Participants |
| Part 1: Placebo (ROW) | Part 1: Number of Participants With Potentially Clinically Significant Laboratory Abnormalities: Hematology | Platelets: >=700 Giga/L | 0 Participants |
| Part 1: Placebo (ROW) | Part 1: Number of Participants With Potentially Clinically Significant Laboratory Abnormalities: Hematology | Hematocrit: <=0.37 v/v (M) or <=0.32 v/v (F) | 0 Participants |
| Part 1: Placebo (ROW) | Part 1: Number of Participants With Potentially Clinically Significant Laboratory Abnormalities: Hematology | Eosinophils >0.5 Giga/L or >ULN (ULN >=0.5 Giga/L) | 0 Participants |
| Part 1: Placebo (ROW) | Part 1: Number of Participants With Potentially Clinically Significant Laboratory Abnormalities: Hematology | RBC: >=6 Tera/L | 0 Participants |
| Part 1: Placebo (ROW) | Part 1: Number of Participants With Potentially Clinically Significant Laboratory Abnormalities: Hematology | Lymphocytes: >4.0 Giga/L | 0 Participants |
| Part 1: Placebo (ROW) | Part 1: Number of Participants With Potentially Clinically Significant Laboratory Abnormalities: Hematology | WBC: >=16.0 Giga/L | 0 Participants |
| Part 1: Placebo (ROW) | Part 1: Number of Participants With Potentially Clinically Significant Laboratory Abnormalities: Hematology | WBC: <3.0 Giga/L (NB) or <2.0 Giga/L (B) | 0 Participants |
| Part 1: Placebo (ROW) | Part 1: Number of Participants With Potentially Clinically Significant Laboratory Abnormalities: Hematology | Hemoglobin DFB >=20 g/L | 1 Participants |
| Part 1: Placebo (ROW) | Part 1: Number of Participants With Potentially Clinically Significant Laboratory Abnormalities: Hematology | Monocytes: >0.7 Giga/L | 0 Participants |
| Part 1: Placebo (ROW) | Part 1: Number of Participants With Potentially Clinically Significant Laboratory Abnormalities: Hematology | Hematocrit: >=0.55 v/v (M); >=0.5 v/v (F) | 0 Participants |
| Part 1: Placebo (ROW) | Part 1: Number of Participants With Potentially Clinically Significant Laboratory Abnormalities: Hematology | Hemoglobin <= 115 g/L (M); ≤ 95 g/L (F) | 0 Participants |
| Part 1: Venglustat 4 mg (ROW) | Part 1: Number of Participants With Potentially Clinically Significant Laboratory Abnormalities: Hematology | Hematocrit: >=0.55 v/v (M); >=0.5 v/v (F) | 0 Participants |
| Part 1: Venglustat 4 mg (ROW) | Part 1: Number of Participants With Potentially Clinically Significant Laboratory Abnormalities: Hematology | Lymphocytes: >4.0 Giga/L | 0 Participants |
| Part 1: Venglustat 4 mg (ROW) | Part 1: Number of Participants With Potentially Clinically Significant Laboratory Abnormalities: Hematology | Eosinophils >0.5 Giga/L or >ULN (ULN >=0.5 Giga/L) | 0 Participants |
| Part 1: Venglustat 4 mg (ROW) | Part 1: Number of Participants With Potentially Clinically Significant Laboratory Abnormalities: Hematology | Platelets: <100 Giga/L | 0 Participants |
| Part 1: Venglustat 4 mg (ROW) | Part 1: Number of Participants With Potentially Clinically Significant Laboratory Abnormalities: Hematology | Monocytes: <LLN | 0 Participants |
| Part 1: Venglustat 4 mg (ROW) | Part 1: Number of Participants With Potentially Clinically Significant Laboratory Abnormalities: Hematology | RBC: >=6 Tera/L | 0 Participants |
| Part 1: Venglustat 4 mg (ROW) | Part 1: Number of Participants With Potentially Clinically Significant Laboratory Abnormalities: Hematology | Hemoglobin >=185 g/L (M) or >=165 g/L (F) | 0 Participants |
| Part 1: Venglustat 4 mg (ROW) | Part 1: Number of Participants With Potentially Clinically Significant Laboratory Abnormalities: Hematology | Neutrophils <1.5 Giga/L (NB) or <1.0 Giga/L (B) | 0 Participants |
| Part 1: Venglustat 4 mg (ROW) | Part 1: Number of Participants With Potentially Clinically Significant Laboratory Abnormalities: Hematology | Lymphocytes: <LLN | 1 Participants |
| Part 1: Venglustat 4 mg (ROW) | Part 1: Number of Participants With Potentially Clinically Significant Laboratory Abnormalities: Hematology | Hemoglobin DFB >=20 g/L | 0 Participants |
| Part 1: Venglustat 4 mg (ROW) | Part 1: Number of Participants With Potentially Clinically Significant Laboratory Abnormalities: Hematology | WBC: >=16.0 Giga/L | 0 Participants |
| Part 1: Venglustat 4 mg (ROW) | Part 1: Number of Participants With Potentially Clinically Significant Laboratory Abnormalities: Hematology | Platelets: >=700 Giga/L | 0 Participants |
| Part 1: Venglustat 4 mg (ROW) | Part 1: Number of Participants With Potentially Clinically Significant Laboratory Abnormalities: Hematology | Basophils: >0.1 Giga/L | 0 Participants |
| Part 1: Venglustat 4 mg (ROW) | Part 1: Number of Participants With Potentially Clinically Significant Laboratory Abnormalities: Hematology | Hematocrit: <=0.37 v/v (M) or <=0.32 v/v (F) | 0 Participants |
| Part 1: Venglustat 4 mg (ROW) | Part 1: Number of Participants With Potentially Clinically Significant Laboratory Abnormalities: Hematology | WBC: <3.0 Giga/L (NB) or <2.0 Giga/L (B) | 0 Participants |
| Part 1: Venglustat 4 mg (ROW) | Part 1: Number of Participants With Potentially Clinically Significant Laboratory Abnormalities: Hematology | Hemoglobin <= 115 g/L (M); ≤ 95 g/L (F) | 0 Participants |
| Part 1: Venglustat 4 mg (ROW) | Part 1: Number of Participants With Potentially Clinically Significant Laboratory Abnormalities: Hematology | Monocytes: >0.7 Giga/L | 0 Participants |
| Part 1: Venglustat 8 mg (ROW) | Part 1: Number of Participants With Potentially Clinically Significant Laboratory Abnormalities: Hematology | RBC: >=6 Tera/L | 0 Participants |
| Part 1: Venglustat 8 mg (ROW) | Part 1: Number of Participants With Potentially Clinically Significant Laboratory Abnormalities: Hematology | Neutrophils <1.5 Giga/L (NB) or <1.0 Giga/L (B) | 0 Participants |
| Part 1: Venglustat 8 mg (ROW) | Part 1: Number of Participants With Potentially Clinically Significant Laboratory Abnormalities: Hematology | WBC: >=16.0 Giga/L | 0 Participants |
| Part 1: Venglustat 8 mg (ROW) | Part 1: Number of Participants With Potentially Clinically Significant Laboratory Abnormalities: Hematology | Hemoglobin >=185 g/L (M) or >=165 g/L (F) | 0 Participants |
| Part 1: Venglustat 8 mg (ROW) | Part 1: Number of Participants With Potentially Clinically Significant Laboratory Abnormalities: Hematology | Hemoglobin <= 115 g/L (M); ≤ 95 g/L (F) | 0 Participants |
| Part 1: Venglustat 8 mg (ROW) | Part 1: Number of Participants With Potentially Clinically Significant Laboratory Abnormalities: Hematology | Basophils: >0.1 Giga/L | 0 Participants |
| Part 1: Venglustat 8 mg (ROW) | Part 1: Number of Participants With Potentially Clinically Significant Laboratory Abnormalities: Hematology | Hemoglobin DFB >=20 g/L | 1 Participants |
| Part 1: Venglustat 8 mg (ROW) | Part 1: Number of Participants With Potentially Clinically Significant Laboratory Abnormalities: Hematology | Hematocrit: <=0.37 v/v (M) or <=0.32 v/v (F) | 1 Participants |
| Part 1: Venglustat 8 mg (ROW) | Part 1: Number of Participants With Potentially Clinically Significant Laboratory Abnormalities: Hematology | Monocytes: >0.7 Giga/L | 2 Participants |
| Part 1: Venglustat 8 mg (ROW) | Part 1: Number of Participants With Potentially Clinically Significant Laboratory Abnormalities: Hematology | Hematocrit: >=0.55 v/v (M); >=0.5 v/v (F) | 0 Participants |
| Part 1: Venglustat 8 mg (ROW) | Part 1: Number of Participants With Potentially Clinically Significant Laboratory Abnormalities: Hematology | Monocytes: <LLN | 0 Participants |
| Part 1: Venglustat 8 mg (ROW) | Part 1: Number of Participants With Potentially Clinically Significant Laboratory Abnormalities: Hematology | Eosinophils >0.5 Giga/L or >ULN (ULN >=0.5 Giga/L) | 0 Participants |
| Part 1: Venglustat 8 mg (ROW) | Part 1: Number of Participants With Potentially Clinically Significant Laboratory Abnormalities: Hematology | Lymphocytes: >4.0 Giga/L | 0 Participants |
| Part 1: Venglustat 8 mg (ROW) | Part 1: Number of Participants With Potentially Clinically Significant Laboratory Abnormalities: Hematology | Platelets: <100 Giga/L | 1 Participants |
| Part 1: Venglustat 8 mg (ROW) | Part 1: Number of Participants With Potentially Clinically Significant Laboratory Abnormalities: Hematology | Platelets: >=700 Giga/L | 0 Participants |
| Part 1: Venglustat 8 mg (ROW) | Part 1: Number of Participants With Potentially Clinically Significant Laboratory Abnormalities: Hematology | Lymphocytes: <LLN | 0 Participants |
| Part 1: Venglustat 8 mg (ROW) | Part 1: Number of Participants With Potentially Clinically Significant Laboratory Abnormalities: Hematology | WBC: <3.0 Giga/L (NB) or <2.0 Giga/L (B) | 0 Participants |
| Part 1: Venglustat 15 mg (ROW) | Part 1: Number of Participants With Potentially Clinically Significant Laboratory Abnormalities: Hematology | Platelets: <100 Giga/L | 0 Participants |
| Part 1: Venglustat 15 mg (ROW) | Part 1: Number of Participants With Potentially Clinically Significant Laboratory Abnormalities: Hematology | Eosinophils >0.5 Giga/L or >ULN (ULN >=0.5 Giga/L) | 0 Participants |
| Part 1: Venglustat 15 mg (ROW) | Part 1: Number of Participants With Potentially Clinically Significant Laboratory Abnormalities: Hematology | Hematocrit: >=0.55 v/v (M); >=0.5 v/v (F) | 0 Participants |
| Part 1: Venglustat 15 mg (ROW) | Part 1: Number of Participants With Potentially Clinically Significant Laboratory Abnormalities: Hematology | Hematocrit: <=0.37 v/v (M) or <=0.32 v/v (F) | 2 Participants |
| Part 1: Venglustat 15 mg (ROW) | Part 1: Number of Participants With Potentially Clinically Significant Laboratory Abnormalities: Hematology | Lymphocytes: <LLN | 0 Participants |
| Part 1: Venglustat 15 mg (ROW) | Part 1: Number of Participants With Potentially Clinically Significant Laboratory Abnormalities: Hematology | Monocytes: >0.7 Giga/L | 0 Participants |
| Part 1: Venglustat 15 mg (ROW) | Part 1: Number of Participants With Potentially Clinically Significant Laboratory Abnormalities: Hematology | Hemoglobin <= 115 g/L (M); ≤ 95 g/L (F) | 0 Participants |
| Part 1: Venglustat 15 mg (ROW) | Part 1: Number of Participants With Potentially Clinically Significant Laboratory Abnormalities: Hematology | Neutrophils <1.5 Giga/L (NB) or <1.0 Giga/L (B) | 0 Participants |
| Part 1: Venglustat 15 mg (ROW) | Part 1: Number of Participants With Potentially Clinically Significant Laboratory Abnormalities: Hematology | Platelets: >=700 Giga/L | 0 Participants |
| Part 1: Venglustat 15 mg (ROW) | Part 1: Number of Participants With Potentially Clinically Significant Laboratory Abnormalities: Hematology | Hemoglobin DFB >=20 g/L | 1 Participants |
| Part 1: Venglustat 15 mg (ROW) | Part 1: Number of Participants With Potentially Clinically Significant Laboratory Abnormalities: Hematology | WBC: <3.0 Giga/L (NB) or <2.0 Giga/L (B) | 0 Participants |
| Part 1: Venglustat 15 mg (ROW) | Part 1: Number of Participants With Potentially Clinically Significant Laboratory Abnormalities: Hematology | Basophils: >0.1 Giga/L | 0 Participants |
| Part 1: Venglustat 15 mg (ROW) | Part 1: Number of Participants With Potentially Clinically Significant Laboratory Abnormalities: Hematology | Lymphocytes: >4.0 Giga/L | 0 Participants |
| Part 1: Venglustat 15 mg (ROW) | Part 1: Number of Participants With Potentially Clinically Significant Laboratory Abnormalities: Hematology | Hemoglobin >=185 g/L (M) or >=165 g/L (F) | 0 Participants |
| Part 1: Venglustat 15 mg (ROW) | Part 1: Number of Participants With Potentially Clinically Significant Laboratory Abnormalities: Hematology | RBC: >=6 Tera/L | 0 Participants |
| Part 1: Venglustat 15 mg (ROW) | Part 1: Number of Participants With Potentially Clinically Significant Laboratory Abnormalities: Hematology | Monocytes: <LLN | 0 Participants |
| Part 1: Venglustat 15 mg (ROW) | Part 1: Number of Participants With Potentially Clinically Significant Laboratory Abnormalities: Hematology | WBC: >=16.0 Giga/L | 0 Participants |
| Part 1: Placebo (Japan Only) | Part 1: Number of Participants With Potentially Clinically Significant Laboratory Abnormalities: Hematology | Neutrophils <1.5 Giga/L (NB) or <1.0 Giga/L (B) | 0 Participants |
| Part 1: Placebo (Japan Only) | Part 1: Number of Participants With Potentially Clinically Significant Laboratory Abnormalities: Hematology | Hemoglobin <= 115 g/L (M); ≤ 95 g/L (F) | 0 Participants |
| Part 1: Placebo (Japan Only) | Part 1: Number of Participants With Potentially Clinically Significant Laboratory Abnormalities: Hematology | Hemoglobin >=185 g/L (M) or >=165 g/L (F) | 0 Participants |
| Part 1: Placebo (Japan Only) | Part 1: Number of Participants With Potentially Clinically Significant Laboratory Abnormalities: Hematology | Hemoglobin DFB >=20 g/L | 0 Participants |
| Part 1: Placebo (Japan Only) | Part 1: Number of Participants With Potentially Clinically Significant Laboratory Abnormalities: Hematology | Hematocrit: <=0.37 v/v (M) or <=0.32 v/v (F) | 0 Participants |
| Part 1: Placebo (Japan Only) | Part 1: Number of Participants With Potentially Clinically Significant Laboratory Abnormalities: Hematology | Hematocrit: >=0.55 v/v (M); >=0.5 v/v (F) | 0 Participants |
| Part 1: Placebo (Japan Only) | Part 1: Number of Participants With Potentially Clinically Significant Laboratory Abnormalities: Hematology | RBC: >=6 Tera/L | 0 Participants |
| Part 1: Placebo (Japan Only) | Part 1: Number of Participants With Potentially Clinically Significant Laboratory Abnormalities: Hematology | Platelets: <100 Giga/L | 0 Participants |
| Part 1: Placebo (Japan Only) | Part 1: Number of Participants With Potentially Clinically Significant Laboratory Abnormalities: Hematology | Platelets: >=700 Giga/L | 0 Participants |
| Part 1: Placebo (Japan Only) | Part 1: Number of Participants With Potentially Clinically Significant Laboratory Abnormalities: Hematology | WBC: <3.0 Giga/L (NB) or <2.0 Giga/L (B) | 0 Participants |
| Part 1: Placebo (Japan Only) | Part 1: Number of Participants With Potentially Clinically Significant Laboratory Abnormalities: Hematology | WBC: >=16.0 Giga/L | 0 Participants |
| Part 1: Placebo (Japan Only) | Part 1: Number of Participants With Potentially Clinically Significant Laboratory Abnormalities: Hematology | Lymphocytes: <LLN | 0 Participants |
| Part 1: Placebo (Japan Only) | Part 1: Number of Participants With Potentially Clinically Significant Laboratory Abnormalities: Hematology | Lymphocytes: >4.0 Giga/L | 0 Participants |
| Part 1: Placebo (Japan Only) | Part 1: Number of Participants With Potentially Clinically Significant Laboratory Abnormalities: Hematology | Monocytes: <LLN | 0 Participants |
| Part 1: Placebo (Japan Only) | Part 1: Number of Participants With Potentially Clinically Significant Laboratory Abnormalities: Hematology | Monocytes: >0.7 Giga/L | 0 Participants |
| Part 1: Placebo (Japan Only) | Part 1: Number of Participants With Potentially Clinically Significant Laboratory Abnormalities: Hematology | Basophils: >0.1 Giga/L | 0 Participants |
| Part 1: Placebo (Japan Only) | Part 1: Number of Participants With Potentially Clinically Significant Laboratory Abnormalities: Hematology | Eosinophils >0.5 Giga/L or >ULN (ULN >=0.5 Giga/L) | 0 Participants |
| Part 1: Venglustat 4 mg (Japan Only) | Part 1: Number of Participants With Potentially Clinically Significant Laboratory Abnormalities: Hematology | Platelets: <100 Giga/L | 0 Participants |
| Part 1: Venglustat 4 mg (Japan Only) | Part 1: Number of Participants With Potentially Clinically Significant Laboratory Abnormalities: Hematology | Neutrophils <1.5 Giga/L (NB) or <1.0 Giga/L (B) | 0 Participants |
| Part 1: Venglustat 4 mg (Japan Only) | Part 1: Number of Participants With Potentially Clinically Significant Laboratory Abnormalities: Hematology | Platelets: >=700 Giga/L | 0 Participants |
| Part 1: Venglustat 4 mg (Japan Only) | Part 1: Number of Participants With Potentially Clinically Significant Laboratory Abnormalities: Hematology | Lymphocytes: <LLN | 0 Participants |
| Part 1: Venglustat 4 mg (Japan Only) | Part 1: Number of Participants With Potentially Clinically Significant Laboratory Abnormalities: Hematology | Hemoglobin <= 115 g/L (M); ≤ 95 g/L (F) | 0 Participants |
| Part 1: Venglustat 4 mg (Japan Only) | Part 1: Number of Participants With Potentially Clinically Significant Laboratory Abnormalities: Hematology | Lymphocytes: >4.0 Giga/L | 0 Participants |
| Part 1: Venglustat 4 mg (Japan Only) | Part 1: Number of Participants With Potentially Clinically Significant Laboratory Abnormalities: Hematology | RBC: >=6 Tera/L | 0 Participants |
| Part 1: Venglustat 4 mg (Japan Only) | Part 1: Number of Participants With Potentially Clinically Significant Laboratory Abnormalities: Hematology | Hematocrit: >=0.55 v/v (M); >=0.5 v/v (F) | 0 Participants |
| Part 1: Venglustat 4 mg (Japan Only) | Part 1: Number of Participants With Potentially Clinically Significant Laboratory Abnormalities: Hematology | Monocytes: <LLN | 0 Participants |
| Part 1: Venglustat 4 mg (Japan Only) | Part 1: Number of Participants With Potentially Clinically Significant Laboratory Abnormalities: Hematology | Hematocrit: <=0.37 v/v (M) or <=0.32 v/v (F) | 0 Participants |
| Part 1: Venglustat 4 mg (Japan Only) | Part 1: Number of Participants With Potentially Clinically Significant Laboratory Abnormalities: Hematology | Eosinophils >0.5 Giga/L or >ULN (ULN >=0.5 Giga/L) | 0 Participants |
| Part 1: Venglustat 4 mg (Japan Only) | Part 1: Number of Participants With Potentially Clinically Significant Laboratory Abnormalities: Hematology | Monocytes: >0.7 Giga/L | 0 Participants |
| Part 1: Venglustat 4 mg (Japan Only) | Part 1: Number of Participants With Potentially Clinically Significant Laboratory Abnormalities: Hematology | Hemoglobin DFB >=20 g/L | 0 Participants |
| Part 1: Venglustat 4 mg (Japan Only) | Part 1: Number of Participants With Potentially Clinically Significant Laboratory Abnormalities: Hematology | Basophils: >0.1 Giga/L | 0 Participants |
| Part 1: Venglustat 4 mg (Japan Only) | Part 1: Number of Participants With Potentially Clinically Significant Laboratory Abnormalities: Hematology | Hemoglobin >=185 g/L (M) or >=165 g/L (F) | 0 Participants |
| Part 1: Venglustat 4 mg (Japan Only) | Part 1: Number of Participants With Potentially Clinically Significant Laboratory Abnormalities: Hematology | WBC: >=16.0 Giga/L | 0 Participants |
| Part 1: Venglustat 4 mg (Japan Only) | Part 1: Number of Participants With Potentially Clinically Significant Laboratory Abnormalities: Hematology | WBC: <3.0 Giga/L (NB) or <2.0 Giga/L (B) | 0 Participants |
| Part 1: Venglustat 8 mg (Japan Only) | Part 1: Number of Participants With Potentially Clinically Significant Laboratory Abnormalities: Hematology | Lymphocytes: >4.0 Giga/L | 0 Participants |
| Part 1: Venglustat 8 mg (Japan Only) | Part 1: Number of Participants With Potentially Clinically Significant Laboratory Abnormalities: Hematology | WBC: <3.0 Giga/L (NB) or <2.0 Giga/L (B) | 0 Participants |
| Part 1: Venglustat 8 mg (Japan Only) | Part 1: Number of Participants With Potentially Clinically Significant Laboratory Abnormalities: Hematology | Hematocrit: >=0.55 v/v (M); >=0.5 v/v (F) | 0 Participants |
| Part 1: Venglustat 8 mg (Japan Only) | Part 1: Number of Participants With Potentially Clinically Significant Laboratory Abnormalities: Hematology | Basophils: >0.1 Giga/L | 0 Participants |
| Part 1: Venglustat 8 mg (Japan Only) | Part 1: Number of Participants With Potentially Clinically Significant Laboratory Abnormalities: Hematology | Monocytes: <LLN | 0 Participants |
| Part 1: Venglustat 8 mg (Japan Only) | Part 1: Number of Participants With Potentially Clinically Significant Laboratory Abnormalities: Hematology | Hematocrit: <=0.37 v/v (M) or <=0.32 v/v (F) | 0 Participants |
| Part 1: Venglustat 8 mg (Japan Only) | Part 1: Number of Participants With Potentially Clinically Significant Laboratory Abnormalities: Hematology | WBC: >=16.0 Giga/L | 0 Participants |
| Part 1: Venglustat 8 mg (Japan Only) | Part 1: Number of Participants With Potentially Clinically Significant Laboratory Abnormalities: Hematology | Monocytes: >0.7 Giga/L | 0 Participants |
| Part 1: Venglustat 8 mg (Japan Only) | Part 1: Number of Participants With Potentially Clinically Significant Laboratory Abnormalities: Hematology | Hemoglobin DFB >=20 g/L | 0 Participants |
| Part 1: Venglustat 8 mg (Japan Only) | Part 1: Number of Participants With Potentially Clinically Significant Laboratory Abnormalities: Hematology | Neutrophils <1.5 Giga/L (NB) or <1.0 Giga/L (B) | 0 Participants |
| Part 1: Venglustat 8 mg (Japan Only) | Part 1: Number of Participants With Potentially Clinically Significant Laboratory Abnormalities: Hematology | Platelets: >=700 Giga/L | 0 Participants |
| Part 1: Venglustat 8 mg (Japan Only) | Part 1: Number of Participants With Potentially Clinically Significant Laboratory Abnormalities: Hematology | Hemoglobin <= 115 g/L (M); ≤ 95 g/L (F) | 0 Participants |
| Part 1: Venglustat 8 mg (Japan Only) | Part 1: Number of Participants With Potentially Clinically Significant Laboratory Abnormalities: Hematology | Platelets: <100 Giga/L | 0 Participants |
| Part 1: Venglustat 8 mg (Japan Only) | Part 1: Number of Participants With Potentially Clinically Significant Laboratory Abnormalities: Hematology | Lymphocytes: <LLN | 1 Participants |
| Part 1: Venglustat 8 mg (Japan Only) | Part 1: Number of Participants With Potentially Clinically Significant Laboratory Abnormalities: Hematology | RBC: >=6 Tera/L | 0 Participants |
| Part 1: Venglustat 8 mg (Japan Only) | Part 1: Number of Participants With Potentially Clinically Significant Laboratory Abnormalities: Hematology | Hemoglobin >=185 g/L (M) or >=165 g/L (F) | 0 Participants |
| Part 1: Venglustat 8 mg (Japan Only) | Part 1: Number of Participants With Potentially Clinically Significant Laboratory Abnormalities: Hematology | Eosinophils >0.5 Giga/L or >ULN (ULN >=0.5 Giga/L) | 0 Participants |
| Part 1: Venglustat 15 mg (Japan Only) | Part 1: Number of Participants With Potentially Clinically Significant Laboratory Abnormalities: Hematology | Monocytes: >0.7 Giga/L | 0 Participants |
| Part 1: Venglustat 15 mg (Japan Only) | Part 1: Number of Participants With Potentially Clinically Significant Laboratory Abnormalities: Hematology | Lymphocytes: >4.0 Giga/L | 0 Participants |
| Part 1: Venglustat 15 mg (Japan Only) | Part 1: Number of Participants With Potentially Clinically Significant Laboratory Abnormalities: Hematology | RBC: >=6 Tera/L | 0 Participants |
| Part 1: Venglustat 15 mg (Japan Only) | Part 1: Number of Participants With Potentially Clinically Significant Laboratory Abnormalities: Hematology | Hematocrit: >=0.55 v/v (M); >=0.5 v/v (F) | 0 Participants |
| Part 1: Venglustat 15 mg (Japan Only) | Part 1: Number of Participants With Potentially Clinically Significant Laboratory Abnormalities: Hematology | Hemoglobin >=185 g/L (M) or >=165 g/L (F) | 0 Participants |
| Part 1: Venglustat 15 mg (Japan Only) | Part 1: Number of Participants With Potentially Clinically Significant Laboratory Abnormalities: Hematology | Neutrophils <1.5 Giga/L (NB) or <1.0 Giga/L (B) | 0 Participants |
| Part 1: Venglustat 15 mg (Japan Only) | Part 1: Number of Participants With Potentially Clinically Significant Laboratory Abnormalities: Hematology | Eosinophils >0.5 Giga/L or >ULN (ULN >=0.5 Giga/L) | 0 Participants |
| Part 1: Venglustat 15 mg (Japan Only) | Part 1: Number of Participants With Potentially Clinically Significant Laboratory Abnormalities: Hematology | Basophils: >0.1 Giga/L | 0 Participants |
| Part 1: Venglustat 15 mg (Japan Only) | Part 1: Number of Participants With Potentially Clinically Significant Laboratory Abnormalities: Hematology | Platelets: >=700 Giga/L | 0 Participants |
| Part 1: Venglustat 15 mg (Japan Only) | Part 1: Number of Participants With Potentially Clinically Significant Laboratory Abnormalities: Hematology | Monocytes: <LLN | 0 Participants |
| Part 1: Venglustat 15 mg (Japan Only) | Part 1: Number of Participants With Potentially Clinically Significant Laboratory Abnormalities: Hematology | Hematocrit: <=0.37 v/v (M) or <=0.32 v/v (F) | 0 Participants |
| Part 1: Venglustat 15 mg (Japan Only) | Part 1: Number of Participants With Potentially Clinically Significant Laboratory Abnormalities: Hematology | Lymphocytes: <LLN | 0 Participants |
| Part 1: Venglustat 15 mg (Japan Only) | Part 1: Number of Participants With Potentially Clinically Significant Laboratory Abnormalities: Hematology | WBC: <3.0 Giga/L (NB) or <2.0 Giga/L (B) | 0 Participants |
| Part 1: Venglustat 15 mg (Japan Only) | Part 1: Number of Participants With Potentially Clinically Significant Laboratory Abnormalities: Hematology | Hemoglobin DFB >=20 g/L | 1 Participants |
| Part 1: Venglustat 15 mg (Japan Only) | Part 1: Number of Participants With Potentially Clinically Significant Laboratory Abnormalities: Hematology | Hemoglobin <= 115 g/L (M); ≤ 95 g/L (F) | 0 Participants |
| Part 1: Venglustat 15 mg (Japan Only) | Part 1: Number of Participants With Potentially Clinically Significant Laboratory Abnormalities: Hematology | WBC: >=16.0 Giga/L | 0 Participants |
| Part 1: Venglustat 15 mg (Japan Only) | Part 1: Number of Participants With Potentially Clinically Significant Laboratory Abnormalities: Hematology | Platelets: <100 Giga/L | 0 Participants |
Primary
Part 1: Number of Participants With Potentially Clinically Significant Laboratory Abnormalities: Liver Function Parameters
Criteria for PCSA: Alanine Aminotransferase (ALT): \>3 ULN, \>5 ULN; Aspartate aminotransferase (AST): \>3 ULN; Alkaline phosphatase: \>1.5 ULN; Total Bilirubin: \>1.5 ULN; ALT and Bilirubin: \>3 ULN and \>2 ULN; Direct Bilirubin and Bilirubin: \>35% and \>1.5 ULN.
Time frame: From the first IMP administration up to 6 weeks after the last administration of the IMP (i.e., up to 42 weeks for ROW and up to 58 weeks for Japanese participants)
Population: Analysis was performed on safety population. This OM was planned to be analyzed and reported for Part 1 only and not for Part 2, as pre-specified in protocol.
| Arm | Measure | Group | Value (COUNT_OF_PARTICIPANTS) |
|---|---|---|---|
| Part 1: Placebo (ROW) | Part 1: Number of Participants With Potentially Clinically Significant Laboratory Abnormalities: Liver Function Parameters | AST >3 ULN | 0 Participants |
| Part 1: Placebo (ROW) | Part 1: Number of Participants With Potentially Clinically Significant Laboratory Abnormalities: Liver Function Parameters | ALT >5 ULN | 0 Participants |
| Part 1: Placebo (ROW) | Part 1: Number of Participants With Potentially Clinically Significant Laboratory Abnormalities: Liver Function Parameters | ALT >3 ULN | 0 Participants |
| Part 1: Placebo (ROW) | Part 1: Number of Participants With Potentially Clinically Significant Laboratory Abnormalities: Liver Function Parameters | AST >5 ULN | 0 Participants |
| Part 1: Placebo (ROW) | Part 1: Number of Participants With Potentially Clinically Significant Laboratory Abnormalities: Liver Function Parameters | Alkaline Phosphatase >1.5 ULN | 0 Participants |
| Part 1: Placebo (ROW) | Part 1: Number of Participants With Potentially Clinically Significant Laboratory Abnormalities: Liver Function Parameters | Total Bilirubin > 1.5 ULN | 0 Participants |
| Part 1: Placebo (ROW) | Part 1: Number of Participants With Potentially Clinically Significant Laboratory Abnormalities: Liver Function Parameters | ALT and Bilirubin: >3 ULN and >2 ULN | 0 Participants |
| Part 1: Placebo (ROW) | Part 1: Number of Participants With Potentially Clinically Significant Laboratory Abnormalities: Liver Function Parameters | Direct Bilirubin and Bilirubin: >35% and >1.5 ULN | 0 Participants |
| Part 1: Venglustat 4 mg (ROW) | Part 1: Number of Participants With Potentially Clinically Significant Laboratory Abnormalities: Liver Function Parameters | Alkaline Phosphatase >1.5 ULN | 0 Participants |
| Part 1: Venglustat 4 mg (ROW) | Part 1: Number of Participants With Potentially Clinically Significant Laboratory Abnormalities: Liver Function Parameters | AST >3 ULN | 0 Participants |
| Part 1: Venglustat 4 mg (ROW) | Part 1: Number of Participants With Potentially Clinically Significant Laboratory Abnormalities: Liver Function Parameters | Total Bilirubin > 1.5 ULN | 0 Participants |
| Part 1: Venglustat 4 mg (ROW) | Part 1: Number of Participants With Potentially Clinically Significant Laboratory Abnormalities: Liver Function Parameters | ALT and Bilirubin: >3 ULN and >2 ULN | 0 Participants |
| Part 1: Venglustat 4 mg (ROW) | Part 1: Number of Participants With Potentially Clinically Significant Laboratory Abnormalities: Liver Function Parameters | ALT >3 ULN | 0 Participants |
| Part 1: Venglustat 4 mg (ROW) | Part 1: Number of Participants With Potentially Clinically Significant Laboratory Abnormalities: Liver Function Parameters | AST >5 ULN | 0 Participants |
| Part 1: Venglustat 4 mg (ROW) | Part 1: Number of Participants With Potentially Clinically Significant Laboratory Abnormalities: Liver Function Parameters | ALT >5 ULN | 0 Participants |
| Part 1: Venglustat 4 mg (ROW) | Part 1: Number of Participants With Potentially Clinically Significant Laboratory Abnormalities: Liver Function Parameters | Direct Bilirubin and Bilirubin: >35% and >1.5 ULN | 0 Participants |
| Part 1: Venglustat 8 mg (ROW) | Part 1: Number of Participants With Potentially Clinically Significant Laboratory Abnormalities: Liver Function Parameters | ALT and Bilirubin: >3 ULN and >2 ULN | 0 Participants |
| Part 1: Venglustat 8 mg (ROW) | Part 1: Number of Participants With Potentially Clinically Significant Laboratory Abnormalities: Liver Function Parameters | Alkaline Phosphatase >1.5 ULN | 0 Participants |
| Part 1: Venglustat 8 mg (ROW) | Part 1: Number of Participants With Potentially Clinically Significant Laboratory Abnormalities: Liver Function Parameters | Direct Bilirubin and Bilirubin: >35% and >1.5 ULN | 0 Participants |
| Part 1: Venglustat 8 mg (ROW) | Part 1: Number of Participants With Potentially Clinically Significant Laboratory Abnormalities: Liver Function Parameters | AST >3 ULN | 0 Participants |
| Part 1: Venglustat 8 mg (ROW) | Part 1: Number of Participants With Potentially Clinically Significant Laboratory Abnormalities: Liver Function Parameters | Total Bilirubin > 1.5 ULN | 0 Participants |
| Part 1: Venglustat 8 mg (ROW) | Part 1: Number of Participants With Potentially Clinically Significant Laboratory Abnormalities: Liver Function Parameters | AST >5 ULN | 0 Participants |
| Part 1: Venglustat 8 mg (ROW) | Part 1: Number of Participants With Potentially Clinically Significant Laboratory Abnormalities: Liver Function Parameters | ALT >3 ULN | 0 Participants |
| Part 1: Venglustat 8 mg (ROW) | Part 1: Number of Participants With Potentially Clinically Significant Laboratory Abnormalities: Liver Function Parameters | ALT >5 ULN | 0 Participants |
| Part 1: Venglustat 15 mg (ROW) | Part 1: Number of Participants With Potentially Clinically Significant Laboratory Abnormalities: Liver Function Parameters | Direct Bilirubin and Bilirubin: >35% and >1.5 ULN | 0 Participants |
| Part 1: Venglustat 15 mg (ROW) | Part 1: Number of Participants With Potentially Clinically Significant Laboratory Abnormalities: Liver Function Parameters | ALT >5 ULN | 0 Participants |
| Part 1: Venglustat 15 mg (ROW) | Part 1: Number of Participants With Potentially Clinically Significant Laboratory Abnormalities: Liver Function Parameters | ALT >3 ULN | 0 Participants |
| Part 1: Venglustat 15 mg (ROW) | Part 1: Number of Participants With Potentially Clinically Significant Laboratory Abnormalities: Liver Function Parameters | AST >5 ULN | 0 Participants |
| Part 1: Venglustat 15 mg (ROW) | Part 1: Number of Participants With Potentially Clinically Significant Laboratory Abnormalities: Liver Function Parameters | AST >3 ULN | 0 Participants |
| Part 1: Venglustat 15 mg (ROW) | Part 1: Number of Participants With Potentially Clinically Significant Laboratory Abnormalities: Liver Function Parameters | Alkaline Phosphatase >1.5 ULN | 0 Participants |
| Part 1: Venglustat 15 mg (ROW) | Part 1: Number of Participants With Potentially Clinically Significant Laboratory Abnormalities: Liver Function Parameters | Total Bilirubin > 1.5 ULN | 0 Participants |
| Part 1: Venglustat 15 mg (ROW) | Part 1: Number of Participants With Potentially Clinically Significant Laboratory Abnormalities: Liver Function Parameters | ALT and Bilirubin: >3 ULN and >2 ULN | 0 Participants |
| Part 1: Placebo (Japan Only) | Part 1: Number of Participants With Potentially Clinically Significant Laboratory Abnormalities: Liver Function Parameters | AST >5 ULN | 0 Participants |
| Part 1: Placebo (Japan Only) | Part 1: Number of Participants With Potentially Clinically Significant Laboratory Abnormalities: Liver Function Parameters | ALT and Bilirubin: >3 ULN and >2 ULN | 0 Participants |
| Part 1: Placebo (Japan Only) | Part 1: Number of Participants With Potentially Clinically Significant Laboratory Abnormalities: Liver Function Parameters | AST >3 ULN | 1 Participants |
| Part 1: Placebo (Japan Only) | Part 1: Number of Participants With Potentially Clinically Significant Laboratory Abnormalities: Liver Function Parameters | ALT >3 ULN | 1 Participants |
| Part 1: Placebo (Japan Only) | Part 1: Number of Participants With Potentially Clinically Significant Laboratory Abnormalities: Liver Function Parameters | Direct Bilirubin and Bilirubin: >35% and >1.5 ULN | 0 Participants |
| Part 1: Placebo (Japan Only) | Part 1: Number of Participants With Potentially Clinically Significant Laboratory Abnormalities: Liver Function Parameters | Alkaline Phosphatase >1.5 ULN | 1 Participants |
| Part 1: Placebo (Japan Only) | Part 1: Number of Participants With Potentially Clinically Significant Laboratory Abnormalities: Liver Function Parameters | Total Bilirubin > 1.5 ULN | 0 Participants |
| Part 1: Placebo (Japan Only) | Part 1: Number of Participants With Potentially Clinically Significant Laboratory Abnormalities: Liver Function Parameters | ALT >5 ULN | 0 Participants |
| Part 1: Venglustat 4 mg (Japan Only) | Part 1: Number of Participants With Potentially Clinically Significant Laboratory Abnormalities: Liver Function Parameters | Direct Bilirubin and Bilirubin: >35% and >1.5 ULN | 0 Participants |
| Part 1: Venglustat 4 mg (Japan Only) | Part 1: Number of Participants With Potentially Clinically Significant Laboratory Abnormalities: Liver Function Parameters | ALT >5 ULN | 0 Participants |
| Part 1: Venglustat 4 mg (Japan Only) | Part 1: Number of Participants With Potentially Clinically Significant Laboratory Abnormalities: Liver Function Parameters | AST >3 ULN | 0 Participants |
| Part 1: Venglustat 4 mg (Japan Only) | Part 1: Number of Participants With Potentially Clinically Significant Laboratory Abnormalities: Liver Function Parameters | ALT >3 ULN | 0 Participants |
| Part 1: Venglustat 4 mg (Japan Only) | Part 1: Number of Participants With Potentially Clinically Significant Laboratory Abnormalities: Liver Function Parameters | ALT and Bilirubin: >3 ULN and >2 ULN | 0 Participants |
| Part 1: Venglustat 4 mg (Japan Only) | Part 1: Number of Participants With Potentially Clinically Significant Laboratory Abnormalities: Liver Function Parameters | Total Bilirubin > 1.5 ULN | 0 Participants |
| Part 1: Venglustat 4 mg (Japan Only) | Part 1: Number of Participants With Potentially Clinically Significant Laboratory Abnormalities: Liver Function Parameters | Alkaline Phosphatase >1.5 ULN | 0 Participants |
| Part 1: Venglustat 4 mg (Japan Only) | Part 1: Number of Participants With Potentially Clinically Significant Laboratory Abnormalities: Liver Function Parameters | AST >5 ULN | 0 Participants |
| Part 1: Venglustat 8 mg (Japan Only) | Part 1: Number of Participants With Potentially Clinically Significant Laboratory Abnormalities: Liver Function Parameters | AST >3 ULN | 0 Participants |
| Part 1: Venglustat 8 mg (Japan Only) | Part 1: Number of Participants With Potentially Clinically Significant Laboratory Abnormalities: Liver Function Parameters | ALT >5 ULN | 0 Participants |
| Part 1: Venglustat 8 mg (Japan Only) | Part 1: Number of Participants With Potentially Clinically Significant Laboratory Abnormalities: Liver Function Parameters | AST >5 ULN | 0 Participants |
| Part 1: Venglustat 8 mg (Japan Only) | Part 1: Number of Participants With Potentially Clinically Significant Laboratory Abnormalities: Liver Function Parameters | Alkaline Phosphatase >1.5 ULN | 0 Participants |
| Part 1: Venglustat 8 mg (Japan Only) | Part 1: Number of Participants With Potentially Clinically Significant Laboratory Abnormalities: Liver Function Parameters | Total Bilirubin > 1.5 ULN | 0 Participants |
| Part 1: Venglustat 8 mg (Japan Only) | Part 1: Number of Participants With Potentially Clinically Significant Laboratory Abnormalities: Liver Function Parameters | Direct Bilirubin and Bilirubin: >35% and >1.5 ULN | 0 Participants |
| Part 1: Venglustat 8 mg (Japan Only) | Part 1: Number of Participants With Potentially Clinically Significant Laboratory Abnormalities: Liver Function Parameters | ALT and Bilirubin: >3 ULN and >2 ULN | 0 Participants |
| Part 1: Venglustat 8 mg (Japan Only) | Part 1: Number of Participants With Potentially Clinically Significant Laboratory Abnormalities: Liver Function Parameters | ALT >3 ULN | 1 Participants |
| Part 1: Venglustat 15 mg (Japan Only) | Part 1: Number of Participants With Potentially Clinically Significant Laboratory Abnormalities: Liver Function Parameters | Direct Bilirubin and Bilirubin: >35% and >1.5 ULN | 0 Participants |
| Part 1: Venglustat 15 mg (Japan Only) | Part 1: Number of Participants With Potentially Clinically Significant Laboratory Abnormalities: Liver Function Parameters | Total Bilirubin > 1.5 ULN | 0 Participants |
| Part 1: Venglustat 15 mg (Japan Only) | Part 1: Number of Participants With Potentially Clinically Significant Laboratory Abnormalities: Liver Function Parameters | Alkaline Phosphatase >1.5 ULN | 0 Participants |
| Part 1: Venglustat 15 mg (Japan Only) | Part 1: Number of Participants With Potentially Clinically Significant Laboratory Abnormalities: Liver Function Parameters | AST >5 ULN | 0 Participants |
| Part 1: Venglustat 15 mg (Japan Only) | Part 1: Number of Participants With Potentially Clinically Significant Laboratory Abnormalities: Liver Function Parameters | AST >3 ULN | 0 Participants |
| Part 1: Venglustat 15 mg (Japan Only) | Part 1: Number of Participants With Potentially Clinically Significant Laboratory Abnormalities: Liver Function Parameters | ALT >5 ULN | 0 Participants |
| Part 1: Venglustat 15 mg (Japan Only) | Part 1: Number of Participants With Potentially Clinically Significant Laboratory Abnormalities: Liver Function Parameters | ALT >3 ULN | 0 Participants |
| Part 1: Venglustat 15 mg (Japan Only) | Part 1: Number of Participants With Potentially Clinically Significant Laboratory Abnormalities: Liver Function Parameters | ALT and Bilirubin: >3 ULN and >2 ULN | 0 Participants |
Primary
Part 1: Number of Participants With Potentially Clinically Significant Laboratory Abnormalities: Metabolic Parameters
Criteria for PCSA: Glucose: \<=3.9 mmol/L and \<LLN; \>=11.1 mmol/L (unfasted \[unfas\]) or \>=7 mmol/L (fasted \[fas\]); Albumin: \<=25 g/L.
Time frame: From the first IMP administration up to 6 weeks after the last administration of the IMP (i.e., up to 42 weeks for ROW and up to 58 weeks for Japanese participants)
Population: Analysis was performed on safety population. This OM was planned to be analyzed and reported for Part 1 only and not for Part 2, as pre-specified in protocol.
| Arm | Measure | Group | Value (COUNT_OF_PARTICIPANTS) |
|---|---|---|---|
| Part 1: Placebo (ROW) | Part 1: Number of Participants With Potentially Clinically Significant Laboratory Abnormalities: Metabolic Parameters | Glucose <=3.9 mmol/L and <LLN | 0 Participants |
| Part 1: Placebo (ROW) | Part 1: Number of Participants With Potentially Clinically Significant Laboratory Abnormalities: Metabolic Parameters | Albumin <=25 g/L | 0 Participants |
| Part 1: Placebo (ROW) | Part 1: Number of Participants With Potentially Clinically Significant Laboratory Abnormalities: Metabolic Parameters | Glucose >=11.1 mmol/L (unfas) or >=7 mmol/L (fas) | 0 Participants |
| Part 1: Venglustat 4 mg (ROW) | Part 1: Number of Participants With Potentially Clinically Significant Laboratory Abnormalities: Metabolic Parameters | Albumin <=25 g/L | 0 Participants |
| Part 1: Venglustat 4 mg (ROW) | Part 1: Number of Participants With Potentially Clinically Significant Laboratory Abnormalities: Metabolic Parameters | Glucose >=11.1 mmol/L (unfas) or >=7 mmol/L (fas) | 0 Participants |
| Part 1: Venglustat 4 mg (ROW) | Part 1: Number of Participants With Potentially Clinically Significant Laboratory Abnormalities: Metabolic Parameters | Glucose <=3.9 mmol/L and <LLN | 0 Participants |
| Part 1: Venglustat 8 mg (ROW) | Part 1: Number of Participants With Potentially Clinically Significant Laboratory Abnormalities: Metabolic Parameters | Albumin <=25 g/L | 0 Participants |
| Part 1: Venglustat 8 mg (ROW) | Part 1: Number of Participants With Potentially Clinically Significant Laboratory Abnormalities: Metabolic Parameters | Glucose <=3.9 mmol/L and <LLN | 0 Participants |
| Part 1: Venglustat 8 mg (ROW) | Part 1: Number of Participants With Potentially Clinically Significant Laboratory Abnormalities: Metabolic Parameters | Glucose >=11.1 mmol/L (unfas) or >=7 mmol/L (fas) | 0 Participants |
| Part 1: Venglustat 15 mg (ROW) | Part 1: Number of Participants With Potentially Clinically Significant Laboratory Abnormalities: Metabolic Parameters | Glucose <=3.9 mmol/L and <LLN | 0 Participants |
| Part 1: Venglustat 15 mg (ROW) | Part 1: Number of Participants With Potentially Clinically Significant Laboratory Abnormalities: Metabolic Parameters | Glucose >=11.1 mmol/L (unfas) or >=7 mmol/L (fas) | 0 Participants |
| Part 1: Venglustat 15 mg (ROW) | Part 1: Number of Participants With Potentially Clinically Significant Laboratory Abnormalities: Metabolic Parameters | Albumin <=25 g/L | 0 Participants |
| Part 1: Placebo (Japan Only) | Part 1: Number of Participants With Potentially Clinically Significant Laboratory Abnormalities: Metabolic Parameters | Glucose >=11.1 mmol/L (unfas) or >=7 mmol/L (fas) | 0 Participants |
| Part 1: Placebo (Japan Only) | Part 1: Number of Participants With Potentially Clinically Significant Laboratory Abnormalities: Metabolic Parameters | Glucose <=3.9 mmol/L and <LLN | 0 Participants |
| Part 1: Placebo (Japan Only) | Part 1: Number of Participants With Potentially Clinically Significant Laboratory Abnormalities: Metabolic Parameters | Albumin <=25 g/L | 0 Participants |
| Part 1: Venglustat 4 mg (Japan Only) | Part 1: Number of Participants With Potentially Clinically Significant Laboratory Abnormalities: Metabolic Parameters | Glucose >=11.1 mmol/L (unfas) or >=7 mmol/L (fas) | 1 Participants |
| Part 1: Venglustat 4 mg (Japan Only) | Part 1: Number of Participants With Potentially Clinically Significant Laboratory Abnormalities: Metabolic Parameters | Glucose <=3.9 mmol/L and <LLN | 0 Participants |
| Part 1: Venglustat 4 mg (Japan Only) | Part 1: Number of Participants With Potentially Clinically Significant Laboratory Abnormalities: Metabolic Parameters | Albumin <=25 g/L | 0 Participants |
| Part 1: Venglustat 8 mg (Japan Only) | Part 1: Number of Participants With Potentially Clinically Significant Laboratory Abnormalities: Metabolic Parameters | Glucose >=11.1 mmol/L (unfas) or >=7 mmol/L (fas) | 0 Participants |
| Part 1: Venglustat 8 mg (Japan Only) | Part 1: Number of Participants With Potentially Clinically Significant Laboratory Abnormalities: Metabolic Parameters | Glucose <=3.9 mmol/L and <LLN | 0 Participants |
| Part 1: Venglustat 8 mg (Japan Only) | Part 1: Number of Participants With Potentially Clinically Significant Laboratory Abnormalities: Metabolic Parameters | Albumin <=25 g/L | 0 Participants |
| Part 1: Venglustat 15 mg (Japan Only) | Part 1: Number of Participants With Potentially Clinically Significant Laboratory Abnormalities: Metabolic Parameters | Glucose <=3.9 mmol/L and <LLN | 0 Participants |
| Part 1: Venglustat 15 mg (Japan Only) | Part 1: Number of Participants With Potentially Clinically Significant Laboratory Abnormalities: Metabolic Parameters | Glucose >=11.1 mmol/L (unfas) or >=7 mmol/L (fas) | 0 Participants |
| Part 1: Venglustat 15 mg (Japan Only) | Part 1: Number of Participants With Potentially Clinically Significant Laboratory Abnormalities: Metabolic Parameters | Albumin <=25 g/L | 0 Participants |
Primary
Part 1: Number of Participants With Potentially Clinically Significant Laboratory Abnormalities: Renal Parameters
Criteria for PCSA: Creatinine: \>=150 micromoles per liter (mcmol/L) (adults), \>=30% change from baseline, \>=100% change from baseline; Blood urea nitrogen: \>=17 millimoles (mmol)/L.
Time frame: From the first IMP administration up to 6 weeks after the last administration of the IMP (i.e., up to 42 weeks for ROW and up to 58 weeks for Japanese participants)
Population: Analysis was performed on safety population. This OM was planned to be analyzed and reported for Part 1 only and not for Part 2, as pre-specified in protocol.
| Arm | Measure | Group | Value (COUNT_OF_PARTICIPANTS) |
|---|---|---|---|
| Part 1: Placebo (ROW) | Part 1: Number of Participants With Potentially Clinically Significant Laboratory Abnormalities: Renal Parameters | Creatinine >=150 mcmol/L (Adults) | 0 Participants |
| Part 1: Placebo (ROW) | Part 1: Number of Participants With Potentially Clinically Significant Laboratory Abnormalities: Renal Parameters | Creatinine >=30% change from baseline | 0 Participants |
| Part 1: Placebo (ROW) | Part 1: Number of Participants With Potentially Clinically Significant Laboratory Abnormalities: Renal Parameters | Creatinine >=100% change from baseline | 0 Participants |
| Part 1: Placebo (ROW) | Part 1: Number of Participants With Potentially Clinically Significant Laboratory Abnormalities: Renal Parameters | Blood Urea Nitrogen >=17 mmol/L | 0 Participants |
| Part 1: Venglustat 4 mg (ROW) | Part 1: Number of Participants With Potentially Clinically Significant Laboratory Abnormalities: Renal Parameters | Blood Urea Nitrogen >=17 mmol/L | 0 Participants |
| Part 1: Venglustat 4 mg (ROW) | Part 1: Number of Participants With Potentially Clinically Significant Laboratory Abnormalities: Renal Parameters | Creatinine >=30% change from baseline | 0 Participants |
| Part 1: Venglustat 4 mg (ROW) | Part 1: Number of Participants With Potentially Clinically Significant Laboratory Abnormalities: Renal Parameters | Creatinine >=150 mcmol/L (Adults) | 0 Participants |
| Part 1: Venglustat 4 mg (ROW) | Part 1: Number of Participants With Potentially Clinically Significant Laboratory Abnormalities: Renal Parameters | Creatinine >=100% change from baseline | 0 Participants |
| Part 1: Venglustat 8 mg (ROW) | Part 1: Number of Participants With Potentially Clinically Significant Laboratory Abnormalities: Renal Parameters | Creatinine >=150 mcmol/L (Adults) | 0 Participants |
| Part 1: Venglustat 8 mg (ROW) | Part 1: Number of Participants With Potentially Clinically Significant Laboratory Abnormalities: Renal Parameters | Blood Urea Nitrogen >=17 mmol/L | 0 Participants |
| Part 1: Venglustat 8 mg (ROW) | Part 1: Number of Participants With Potentially Clinically Significant Laboratory Abnormalities: Renal Parameters | Creatinine >=30% change from baseline | 0 Participants |
| Part 1: Venglustat 8 mg (ROW) | Part 1: Number of Participants With Potentially Clinically Significant Laboratory Abnormalities: Renal Parameters | Creatinine >=100% change from baseline | 0 Participants |
| Part 1: Venglustat 15 mg (ROW) | Part 1: Number of Participants With Potentially Clinically Significant Laboratory Abnormalities: Renal Parameters | Creatinine >=150 mcmol/L (Adults) | 0 Participants |
| Part 1: Venglustat 15 mg (ROW) | Part 1: Number of Participants With Potentially Clinically Significant Laboratory Abnormalities: Renal Parameters | Creatinine >=30% change from baseline | 0 Participants |
| Part 1: Venglustat 15 mg (ROW) | Part 1: Number of Participants With Potentially Clinically Significant Laboratory Abnormalities: Renal Parameters | Creatinine >=100% change from baseline | 0 Participants |
| Part 1: Venglustat 15 mg (ROW) | Part 1: Number of Participants With Potentially Clinically Significant Laboratory Abnormalities: Renal Parameters | Blood Urea Nitrogen >=17 mmol/L | 0 Participants |
| Part 1: Placebo (Japan Only) | Part 1: Number of Participants With Potentially Clinically Significant Laboratory Abnormalities: Renal Parameters | Blood Urea Nitrogen >=17 mmol/L | 0 Participants |
| Part 1: Placebo (Japan Only) | Part 1: Number of Participants With Potentially Clinically Significant Laboratory Abnormalities: Renal Parameters | Creatinine >=100% change from baseline | 0 Participants |
| Part 1: Placebo (Japan Only) | Part 1: Number of Participants With Potentially Clinically Significant Laboratory Abnormalities: Renal Parameters | Creatinine >=30% change from baseline | 0 Participants |
| Part 1: Placebo (Japan Only) | Part 1: Number of Participants With Potentially Clinically Significant Laboratory Abnormalities: Renal Parameters | Creatinine >=150 mcmol/L (Adults) | 0 Participants |
| Part 1: Venglustat 4 mg (Japan Only) | Part 1: Number of Participants With Potentially Clinically Significant Laboratory Abnormalities: Renal Parameters | Blood Urea Nitrogen >=17 mmol/L | 0 Participants |
| Part 1: Venglustat 4 mg (Japan Only) | Part 1: Number of Participants With Potentially Clinically Significant Laboratory Abnormalities: Renal Parameters | Creatinine >=100% change from baseline | 0 Participants |
| Part 1: Venglustat 4 mg (Japan Only) | Part 1: Number of Participants With Potentially Clinically Significant Laboratory Abnormalities: Renal Parameters | Creatinine >=30% change from baseline | 0 Participants |
| Part 1: Venglustat 4 mg (Japan Only) | Part 1: Number of Participants With Potentially Clinically Significant Laboratory Abnormalities: Renal Parameters | Creatinine >=150 mcmol/L (Adults) | 0 Participants |
| Part 1: Venglustat 8 mg (Japan Only) | Part 1: Number of Participants With Potentially Clinically Significant Laboratory Abnormalities: Renal Parameters | Blood Urea Nitrogen >=17 mmol/L | 0 Participants |
| Part 1: Venglustat 8 mg (Japan Only) | Part 1: Number of Participants With Potentially Clinically Significant Laboratory Abnormalities: Renal Parameters | Creatinine >=150 mcmol/L (Adults) | 0 Participants |
| Part 1: Venglustat 8 mg (Japan Only) | Part 1: Number of Participants With Potentially Clinically Significant Laboratory Abnormalities: Renal Parameters | Creatinine >=100% change from baseline | 0 Participants |
| Part 1: Venglustat 8 mg (Japan Only) | Part 1: Number of Participants With Potentially Clinically Significant Laboratory Abnormalities: Renal Parameters | Creatinine >=30% change from baseline | 1 Participants |
| Part 1: Venglustat 15 mg (Japan Only) | Part 1: Number of Participants With Potentially Clinically Significant Laboratory Abnormalities: Renal Parameters | Creatinine >=30% change from baseline | 0 Participants |
| Part 1: Venglustat 15 mg (Japan Only) | Part 1: Number of Participants With Potentially Clinically Significant Laboratory Abnormalities: Renal Parameters | Creatinine >=100% change from baseline | 0 Participants |
| Part 1: Venglustat 15 mg (Japan Only) | Part 1: Number of Participants With Potentially Clinically Significant Laboratory Abnormalities: Renal Parameters | Blood Urea Nitrogen >=17 mmol/L | 0 Participants |
| Part 1: Venglustat 15 mg (Japan Only) | Part 1: Number of Participants With Potentially Clinically Significant Laboratory Abnormalities: Renal Parameters | Creatinine >=150 mcmol/L (Adults) | 0 Participants |
Primary
Part 1: Number of Participants With Potentially Clinically Significant Ophthalmological Abnormalities
Clinically significant observations in left eye, right eye and any eye (any eye among left and right) were assessed by the investigator based on methods like visual acuity, slit lamp examination, examination of the cornea, lens, and retina. Any abnormal clinically significant ophthalmological examination finding (which was present at screening or not) on any eye during the treatment-emergent period corresponded to cornea verticillata, cataract and abnormal overall evaluation
Time frame: From the first IMP administration up to 6 weeks after the last administration of the IMP (i.e., up to 42 weeks for ROW and up to 58 weeks for Japanese participants)
Population: Analysis was performed on safety population. This OM was planned to be analyzed and reported for Part 1 only and not for Part 2, as pre-specified in protocol.
| Arm | Measure | Group | Value (COUNT_OF_PARTICIPANTS) |
|---|---|---|---|
| Part 1: Placebo (ROW) | Part 1: Number of Participants With Potentially Clinically Significant Ophthalmological Abnormalities | Any eye | 3 Participants |
| Part 1: Placebo (ROW) | Part 1: Number of Participants With Potentially Clinically Significant Ophthalmological Abnormalities | Left eye | 3 Participants |
| Part 1: Placebo (ROW) | Part 1: Number of Participants With Potentially Clinically Significant Ophthalmological Abnormalities | Right eye | 3 Participants |
| Part 1: Venglustat 4 mg (ROW) | Part 1: Number of Participants With Potentially Clinically Significant Ophthalmological Abnormalities | Left eye | 3 Participants |
| Part 1: Venglustat 4 mg (ROW) | Part 1: Number of Participants With Potentially Clinically Significant Ophthalmological Abnormalities | Right eye | 3 Participants |
| Part 1: Venglustat 4 mg (ROW) | Part 1: Number of Participants With Potentially Clinically Significant Ophthalmological Abnormalities | Any eye | 3 Participants |
| Part 1: Venglustat 8 mg (ROW) | Part 1: Number of Participants With Potentially Clinically Significant Ophthalmological Abnormalities | Left eye | 4 Participants |
| Part 1: Venglustat 8 mg (ROW) | Part 1: Number of Participants With Potentially Clinically Significant Ophthalmological Abnormalities | Any eye | 4 Participants |
| Part 1: Venglustat 8 mg (ROW) | Part 1: Number of Participants With Potentially Clinically Significant Ophthalmological Abnormalities | Right eye | 4 Participants |
| Part 1: Venglustat 15 mg (ROW) | Part 1: Number of Participants With Potentially Clinically Significant Ophthalmological Abnormalities | Any eye | 2 Participants |
| Part 1: Venglustat 15 mg (ROW) | Part 1: Number of Participants With Potentially Clinically Significant Ophthalmological Abnormalities | Right eye | 2 Participants |
| Part 1: Venglustat 15 mg (ROW) | Part 1: Number of Participants With Potentially Clinically Significant Ophthalmological Abnormalities | Left eye | 2 Participants |
| Part 1: Placebo (Japan Only) | Part 1: Number of Participants With Potentially Clinically Significant Ophthalmological Abnormalities | Right eye | 2 Participants |
| Part 1: Placebo (Japan Only) | Part 1: Number of Participants With Potentially Clinically Significant Ophthalmological Abnormalities | Any eye | 2 Participants |
| Part 1: Placebo (Japan Only) | Part 1: Number of Participants With Potentially Clinically Significant Ophthalmological Abnormalities | Left eye | 2 Participants |
| Part 1: Venglustat 4 mg (Japan Only) | Part 1: Number of Participants With Potentially Clinically Significant Ophthalmological Abnormalities | Right eye | 2 Participants |
| Part 1: Venglustat 4 mg (Japan Only) | Part 1: Number of Participants With Potentially Clinically Significant Ophthalmological Abnormalities | Any eye | 3 Participants |
| Part 1: Venglustat 4 mg (Japan Only) | Part 1: Number of Participants With Potentially Clinically Significant Ophthalmological Abnormalities | Left eye | 3 Participants |
| Part 1: Venglustat 8 mg (Japan Only) | Part 1: Number of Participants With Potentially Clinically Significant Ophthalmological Abnormalities | Right eye | 3 Participants |
| Part 1: Venglustat 8 mg (Japan Only) | Part 1: Number of Participants With Potentially Clinically Significant Ophthalmological Abnormalities | Any eye | 3 Participants |
| Part 1: Venglustat 8 mg (Japan Only) | Part 1: Number of Participants With Potentially Clinically Significant Ophthalmological Abnormalities | Left eye | 3 Participants |
| Part 1: Venglustat 15 mg (Japan Only) | Part 1: Number of Participants With Potentially Clinically Significant Ophthalmological Abnormalities | Any eye | 0 Participants |
| Part 1: Venglustat 15 mg (Japan Only) | Part 1: Number of Participants With Potentially Clinically Significant Ophthalmological Abnormalities | Right eye | 0 Participants |
| Part 1: Venglustat 15 mg (Japan Only) | Part 1: Number of Participants With Potentially Clinically Significant Ophthalmological Abnormalities | Left eye | 0 Participants |
Primary
Part 1: Number of Participants With Potentially Clinically Significant Vital Signs Abnormalities
Criteria for PCSA: Systolic blood pressure (SBP) supine: \<=95 millimeters of mercury (mmHg) and DFB \>=20 mmHg; \>=160 mmHg and increase from baseline (IFB) \>=20 mmHg; Diastolic blood pressure (DBP) supine: \<=45 mmHg and DFB \>=10 mmHg; \>=110 mmHg and IFB \>=10 mmHg; SBP (Orthostatic): \<=-20 mmHg; DBP (Orthostatic): \<=-10 mmHg; Heart rate (HR) supine: \<=50 beats per minute (bpm) and DFB \>=20 bpm; \>=120 bpm and IFB \>=20 bpm; Weight: \>=5% DFB; \>=5% IFB.
Time frame: From the first IMP administration up to 6 weeks after the last administration of the IMP (i.e., up to 42 weeks for ROW and up to 58 weeks for Japanese participants)
Population: Analysis was performed on safety population. This OM was planned to be analyzed and reported for Part 1 only and not for Part 2, as pre-specified in protocol.
| Arm | Measure | Group | Value (COUNT_OF_PARTICIPANTS) |
|---|---|---|---|
| Part 1: Placebo (ROW) | Part 1: Number of Participants With Potentially Clinically Significant Vital Signs Abnormalities | HR (supine) >=120 bpm and IFB >=20 bpm | 0 Participants |
| Part 1: Placebo (ROW) | Part 1: Number of Participants With Potentially Clinically Significant Vital Signs Abnormalities | HR (supine) <=50 bpm and DFB >= 20 bpm | 0 Participants |
| Part 1: Placebo (ROW) | Part 1: Number of Participants With Potentially Clinically Significant Vital Signs Abnormalities | Weight >=5% DFB | 0 Participants |
| Part 1: Placebo (ROW) | Part 1: Number of Participants With Potentially Clinically Significant Vital Signs Abnormalities | DBP (supine) >=110 mmHg and IFB >=10 mmHg | 0 Participants |
| Part 1: Placebo (ROW) | Part 1: Number of Participants With Potentially Clinically Significant Vital Signs Abnormalities | SBP (supine) >=160 mmHg and IFB >=20 mmHg | 0 Participants |
| Part 1: Placebo (ROW) | Part 1: Number of Participants With Potentially Clinically Significant Vital Signs Abnormalities | DBP (Orthostatic): <=-10 mmHg | 0 Participants |
| Part 1: Placebo (ROW) | Part 1: Number of Participants With Potentially Clinically Significant Vital Signs Abnormalities | Weight >=5% IFB | 1 Participants |
| Part 1: Placebo (ROW) | Part 1: Number of Participants With Potentially Clinically Significant Vital Signs Abnormalities | SBP (Orthostatic): <=-20 mmHg | 0 Participants |
| Part 1: Placebo (ROW) | Part 1: Number of Participants With Potentially Clinically Significant Vital Signs Abnormalities | DBP (supine) <=45 mmHg and DFB >=10 mmHg | 0 Participants |
| Part 1: Placebo (ROW) | Part 1: Number of Participants With Potentially Clinically Significant Vital Signs Abnormalities | SBP (supine) <=95 mmHg and DFB >=20 mmHg | 0 Participants |
| Part 1: Venglustat 4 mg (ROW) | Part 1: Number of Participants With Potentially Clinically Significant Vital Signs Abnormalities | SBP (supine) >=160 mmHg and IFB >=20 mmHg | 0 Participants |
| Part 1: Venglustat 4 mg (ROW) | Part 1: Number of Participants With Potentially Clinically Significant Vital Signs Abnormalities | DBP (supine) <=45 mmHg and DFB >=10 mmHg | 0 Participants |
| Part 1: Venglustat 4 mg (ROW) | Part 1: Number of Participants With Potentially Clinically Significant Vital Signs Abnormalities | Weight >=5% DFB | 0 Participants |
| Part 1: Venglustat 4 mg (ROW) | Part 1: Number of Participants With Potentially Clinically Significant Vital Signs Abnormalities | SBP (Orthostatic): <=-20 mmHg | 0 Participants |
| Part 1: Venglustat 4 mg (ROW) | Part 1: Number of Participants With Potentially Clinically Significant Vital Signs Abnormalities | HR (supine) >=120 bpm and IFB >=20 bpm | 0 Participants |
| Part 1: Venglustat 4 mg (ROW) | Part 1: Number of Participants With Potentially Clinically Significant Vital Signs Abnormalities | SBP (supine) <=95 mmHg and DFB >=20 mmHg | 0 Participants |
| Part 1: Venglustat 4 mg (ROW) | Part 1: Number of Participants With Potentially Clinically Significant Vital Signs Abnormalities | DBP (supine) >=110 mmHg and IFB >=10 mmHg | 0 Participants |
| Part 1: Venglustat 4 mg (ROW) | Part 1: Number of Participants With Potentially Clinically Significant Vital Signs Abnormalities | DBP (Orthostatic): <=-10 mmHg | 0 Participants |
| Part 1: Venglustat 4 mg (ROW) | Part 1: Number of Participants With Potentially Clinically Significant Vital Signs Abnormalities | Weight >=5% IFB | 0 Participants |
| Part 1: Venglustat 4 mg (ROW) | Part 1: Number of Participants With Potentially Clinically Significant Vital Signs Abnormalities | HR (supine) <=50 bpm and DFB >= 20 bpm | 0 Participants |
| Part 1: Venglustat 8 mg (ROW) | Part 1: Number of Participants With Potentially Clinically Significant Vital Signs Abnormalities | HR (supine) <=50 bpm and DFB >= 20 bpm | 0 Participants |
| Part 1: Venglustat 8 mg (ROW) | Part 1: Number of Participants With Potentially Clinically Significant Vital Signs Abnormalities | DBP (Orthostatic): <=-10 mmHg | 0 Participants |
| Part 1: Venglustat 8 mg (ROW) | Part 1: Number of Participants With Potentially Clinically Significant Vital Signs Abnormalities | Weight >=5% IFB | 1 Participants |
| Part 1: Venglustat 8 mg (ROW) | Part 1: Number of Participants With Potentially Clinically Significant Vital Signs Abnormalities | SBP (supine) >=160 mmHg and IFB >=20 mmHg | 0 Participants |
| Part 1: Venglustat 8 mg (ROW) | Part 1: Number of Participants With Potentially Clinically Significant Vital Signs Abnormalities | Weight >=5% DFB | 1 Participants |
| Part 1: Venglustat 8 mg (ROW) | Part 1: Number of Participants With Potentially Clinically Significant Vital Signs Abnormalities | DBP (supine) <=45 mmHg and DFB >=10 mmHg | 0 Participants |
| Part 1: Venglustat 8 mg (ROW) | Part 1: Number of Participants With Potentially Clinically Significant Vital Signs Abnormalities | SBP (supine) <=95 mmHg and DFB >=20 mmHg | 1 Participants |
| Part 1: Venglustat 8 mg (ROW) | Part 1: Number of Participants With Potentially Clinically Significant Vital Signs Abnormalities | DBP (supine) >=110 mmHg and IFB >=10 mmHg | 0 Participants |
| Part 1: Venglustat 8 mg (ROW) | Part 1: Number of Participants With Potentially Clinically Significant Vital Signs Abnormalities | HR (supine) >=120 bpm and IFB >=20 bpm | 0 Participants |
| Part 1: Venglustat 8 mg (ROW) | Part 1: Number of Participants With Potentially Clinically Significant Vital Signs Abnormalities | SBP (Orthostatic): <=-20 mmHg | 0 Participants |
| Part 1: Venglustat 15 mg (ROW) | Part 1: Number of Participants With Potentially Clinically Significant Vital Signs Abnormalities | HR (supine) <=50 bpm and DFB >= 20 bpm | 0 Participants |
| Part 1: Venglustat 15 mg (ROW) | Part 1: Number of Participants With Potentially Clinically Significant Vital Signs Abnormalities | HR (supine) >=120 bpm and IFB >=20 bpm | 0 Participants |
| Part 1: Venglustat 15 mg (ROW) | Part 1: Number of Participants With Potentially Clinically Significant Vital Signs Abnormalities | DBP (supine) <=45 mmHg and DFB >=10 mmHg | 0 Participants |
| Part 1: Venglustat 15 mg (ROW) | Part 1: Number of Participants With Potentially Clinically Significant Vital Signs Abnormalities | Weight >=5% DFB | 0 Participants |
| Part 1: Venglustat 15 mg (ROW) | Part 1: Number of Participants With Potentially Clinically Significant Vital Signs Abnormalities | SBP (supine) >=160 mmHg and IFB >=20 mmHg | 2 Participants |
| Part 1: Venglustat 15 mg (ROW) | Part 1: Number of Participants With Potentially Clinically Significant Vital Signs Abnormalities | DBP (Orthostatic): <=-10 mmHg | 0 Participants |
| Part 1: Venglustat 15 mg (ROW) | Part 1: Number of Participants With Potentially Clinically Significant Vital Signs Abnormalities | SBP (Orthostatic): <=-20 mmHg | 0 Participants |
| Part 1: Venglustat 15 mg (ROW) | Part 1: Number of Participants With Potentially Clinically Significant Vital Signs Abnormalities | Weight >=5% IFB | 0 Participants |
| Part 1: Venglustat 15 mg (ROW) | Part 1: Number of Participants With Potentially Clinically Significant Vital Signs Abnormalities | DBP (supine) >=110 mmHg and IFB >=10 mmHg | 0 Participants |
| Part 1: Venglustat 15 mg (ROW) | Part 1: Number of Participants With Potentially Clinically Significant Vital Signs Abnormalities | SBP (supine) <=95 mmHg and DFB >=20 mmHg | 0 Participants |
| Part 1: Placebo (Japan Only) | Part 1: Number of Participants With Potentially Clinically Significant Vital Signs Abnormalities | Weight >=5% DFB | 0 Participants |
| Part 1: Placebo (Japan Only) | Part 1: Number of Participants With Potentially Clinically Significant Vital Signs Abnormalities | SBP (supine) <=95 mmHg and DFB >=20 mmHg | 0 Participants |
| Part 1: Placebo (Japan Only) | Part 1: Number of Participants With Potentially Clinically Significant Vital Signs Abnormalities | SBP (supine) >=160 mmHg and IFB >=20 mmHg | 0 Participants |
| Part 1: Placebo (Japan Only) | Part 1: Number of Participants With Potentially Clinically Significant Vital Signs Abnormalities | DBP (supine) <=45 mmHg and DFB >=10 mmHg | 0 Participants |
| Part 1: Placebo (Japan Only) | Part 1: Number of Participants With Potentially Clinically Significant Vital Signs Abnormalities | DBP (supine) >=110 mmHg and IFB >=10 mmHg | 0 Participants |
| Part 1: Placebo (Japan Only) | Part 1: Number of Participants With Potentially Clinically Significant Vital Signs Abnormalities | SBP (Orthostatic): <=-20 mmHg | 0 Participants |
| Part 1: Placebo (Japan Only) | Part 1: Number of Participants With Potentially Clinically Significant Vital Signs Abnormalities | DBP (Orthostatic): <=-10 mmHg | 0 Participants |
| Part 1: Placebo (Japan Only) | Part 1: Number of Participants With Potentially Clinically Significant Vital Signs Abnormalities | HR (supine) <=50 bpm and DFB >= 20 bpm | 0 Participants |
| Part 1: Placebo (Japan Only) | Part 1: Number of Participants With Potentially Clinically Significant Vital Signs Abnormalities | HR (supine) >=120 bpm and IFB >=20 bpm | 0 Participants |
| Part 1: Placebo (Japan Only) | Part 1: Number of Participants With Potentially Clinically Significant Vital Signs Abnormalities | Weight >=5% IFB | 0 Participants |
| Part 1: Venglustat 4 mg (Japan Only) | Part 1: Number of Participants With Potentially Clinically Significant Vital Signs Abnormalities | Weight >=5% DFB | 0 Participants |
| Part 1: Venglustat 4 mg (Japan Only) | Part 1: Number of Participants With Potentially Clinically Significant Vital Signs Abnormalities | HR (supine) <=50 bpm and DFB >= 20 bpm | 0 Participants |
| Part 1: Venglustat 4 mg (Japan Only) | Part 1: Number of Participants With Potentially Clinically Significant Vital Signs Abnormalities | HR (supine) >=120 bpm and IFB >=20 bpm | 0 Participants |
| Part 1: Venglustat 4 mg (Japan Only) | Part 1: Number of Participants With Potentially Clinically Significant Vital Signs Abnormalities | SBP (Orthostatic): <=-20 mmHg | 0 Participants |
| Part 1: Venglustat 4 mg (Japan Only) | Part 1: Number of Participants With Potentially Clinically Significant Vital Signs Abnormalities | Weight >=5% IFB | 0 Participants |
| Part 1: Venglustat 4 mg (Japan Only) | Part 1: Number of Participants With Potentially Clinically Significant Vital Signs Abnormalities | DBP (supine) <=45 mmHg and DFB >=10 mmHg | 0 Participants |
| Part 1: Venglustat 4 mg (Japan Only) | Part 1: Number of Participants With Potentially Clinically Significant Vital Signs Abnormalities | SBP (supine) <=95 mmHg and DFB >=20 mmHg | 0 Participants |
| Part 1: Venglustat 4 mg (Japan Only) | Part 1: Number of Participants With Potentially Clinically Significant Vital Signs Abnormalities | SBP (supine) >=160 mmHg and IFB >=20 mmHg | 0 Participants |
| Part 1: Venglustat 4 mg (Japan Only) | Part 1: Number of Participants With Potentially Clinically Significant Vital Signs Abnormalities | DBP (Orthostatic): <=-10 mmHg | 0 Participants |
| Part 1: Venglustat 4 mg (Japan Only) | Part 1: Number of Participants With Potentially Clinically Significant Vital Signs Abnormalities | DBP (supine) >=110 mmHg and IFB >=10 mmHg | 0 Participants |
| Part 1: Venglustat 8 mg (Japan Only) | Part 1: Number of Participants With Potentially Clinically Significant Vital Signs Abnormalities | DBP (supine) <=45 mmHg and DFB >=10 mmHg | 0 Participants |
| Part 1: Venglustat 8 mg (Japan Only) | Part 1: Number of Participants With Potentially Clinically Significant Vital Signs Abnormalities | SBP (Orthostatic): <=-20 mmHg | 0 Participants |
| Part 1: Venglustat 8 mg (Japan Only) | Part 1: Number of Participants With Potentially Clinically Significant Vital Signs Abnormalities | HR (supine) <=50 bpm and DFB >= 20 bpm | 0 Participants |
| Part 1: Venglustat 8 mg (Japan Only) | Part 1: Number of Participants With Potentially Clinically Significant Vital Signs Abnormalities | DBP (supine) >=110 mmHg and IFB >=10 mmHg | 0 Participants |
| Part 1: Venglustat 8 mg (Japan Only) | Part 1: Number of Participants With Potentially Clinically Significant Vital Signs Abnormalities | Weight >=5% IFB | 0 Participants |
| Part 1: Venglustat 8 mg (Japan Only) | Part 1: Number of Participants With Potentially Clinically Significant Vital Signs Abnormalities | HR (supine) >=120 bpm and IFB >=20 bpm | 0 Participants |
| Part 1: Venglustat 8 mg (Japan Only) | Part 1: Number of Participants With Potentially Clinically Significant Vital Signs Abnormalities | SBP (supine) >=160 mmHg and IFB >=20 mmHg | 0 Participants |
| Part 1: Venglustat 8 mg (Japan Only) | Part 1: Number of Participants With Potentially Clinically Significant Vital Signs Abnormalities | Weight >=5% DFB | 0 Participants |
| Part 1: Venglustat 8 mg (Japan Only) | Part 1: Number of Participants With Potentially Clinically Significant Vital Signs Abnormalities | SBP (supine) <=95 mmHg and DFB >=20 mmHg | 2 Participants |
| Part 1: Venglustat 8 mg (Japan Only) | Part 1: Number of Participants With Potentially Clinically Significant Vital Signs Abnormalities | DBP (Orthostatic): <=-10 mmHg | 0 Participants |
| Part 1: Venglustat 15 mg (Japan Only) | Part 1: Number of Participants With Potentially Clinically Significant Vital Signs Abnormalities | DBP (supine) <=45 mmHg and DFB >=10 mmHg | 0 Participants |
| Part 1: Venglustat 15 mg (Japan Only) | Part 1: Number of Participants With Potentially Clinically Significant Vital Signs Abnormalities | Weight >=5% IFB | 0 Participants |
| Part 1: Venglustat 15 mg (Japan Only) | Part 1: Number of Participants With Potentially Clinically Significant Vital Signs Abnormalities | Weight >=5% DFB | 0 Participants |
| Part 1: Venglustat 15 mg (Japan Only) | Part 1: Number of Participants With Potentially Clinically Significant Vital Signs Abnormalities | DBP (supine) >=110 mmHg and IFB >=10 mmHg | 0 Participants |
| Part 1: Venglustat 15 mg (Japan Only) | Part 1: Number of Participants With Potentially Clinically Significant Vital Signs Abnormalities | SBP (supine) <=95 mmHg and DFB >=20 mmHg | 0 Participants |
| Part 1: Venglustat 15 mg (Japan Only) | Part 1: Number of Participants With Potentially Clinically Significant Vital Signs Abnormalities | DBP (Orthostatic): <=-10 mmHg | 0 Participants |
| Part 1: Venglustat 15 mg (Japan Only) | Part 1: Number of Participants With Potentially Clinically Significant Vital Signs Abnormalities | SBP (Orthostatic): <=-20 mmHg | 0 Participants |
| Part 1: Venglustat 15 mg (Japan Only) | Part 1: Number of Participants With Potentially Clinically Significant Vital Signs Abnormalities | SBP (supine) >=160 mmHg and IFB >=20 mmHg | 0 Participants |
| Part 1: Venglustat 15 mg (Japan Only) | Part 1: Number of Participants With Potentially Clinically Significant Vital Signs Abnormalities | HR (supine) <=50 bpm and DFB >= 20 bpm | 0 Participants |
| Part 1: Venglustat 15 mg (Japan Only) | Part 1: Number of Participants With Potentially Clinically Significant Vital Signs Abnormalities | HR (supine) >=120 bpm and IFB >=20 bpm | 0 Participants |
Primary
Part 1: Number of Participants With Treatment-Emergent Adverse Events (TEAEs), and Treatment-Emergent Serious Adverse Events (TESAEs)
An adverse event (AE) was defined as any untoward medical occurrence in a participant who received study drug and does not necessarily had to have a causal relationship with the treatment. Serious AEs (SAEs) were any untoward medical occurrence that resulted in any of the following outcomes: death, life-threatening, required initial or prolonged in-patient hospitalization, persistent or significant disability/incapacity, congenital anomaly/birth defect, or considered as medically important event. TEAEs were the AEs that developed or worsened or became serious during the TEAE period (defined as the period from the time of first investigational medicinal product \[IMP\] administration up of 6 weeks after the last administration of the IMP).
Time frame: From the first IMP administration up to 6 weeks after the last administration of the IMP (i.e., up to 42 weeks for ROW and up to 58 weeks for Japanese participants)
Population: Analysis was performed on safety population which included both non-Japanese (ROW) and Japanese participants who received at least 1 dose of study medication in Part 1 of the study. This outcome measure (OM) was planned to be analyzed and reported for Part 1 only and not for Part 2, as pre-specified in protocol.
| Arm | Measure | Group | Value (COUNT_OF_PARTICIPANTS) |
|---|---|---|---|
| Part 1: Placebo (ROW) | Part 1: Number of Participants With Treatment-Emergent Adverse Events (TEAEs), and Treatment-Emergent Serious Adverse Events (TESAEs) | Any TEAE | 4 Participants |
| Part 1: Placebo (ROW) | Part 1: Number of Participants With Treatment-Emergent Adverse Events (TEAEs), and Treatment-Emergent Serious Adverse Events (TESAEs) | Any TESAE | 0 Participants |
| Part 1: Venglustat 4 mg (ROW) | Part 1: Number of Participants With Treatment-Emergent Adverse Events (TEAEs), and Treatment-Emergent Serious Adverse Events (TESAEs) | Any TEAE | 4 Participants |
| Part 1: Venglustat 4 mg (ROW) | Part 1: Number of Participants With Treatment-Emergent Adverse Events (TEAEs), and Treatment-Emergent Serious Adverse Events (TESAEs) | Any TESAE | 0 Participants |
| Part 1: Venglustat 8 mg (ROW) | Part 1: Number of Participants With Treatment-Emergent Adverse Events (TEAEs), and Treatment-Emergent Serious Adverse Events (TESAEs) | Any TEAE | 4 Participants |
| Part 1: Venglustat 8 mg (ROW) | Part 1: Number of Participants With Treatment-Emergent Adverse Events (TEAEs), and Treatment-Emergent Serious Adverse Events (TESAEs) | Any TESAE | 0 Participants |
| Part 1: Venglustat 15 mg (ROW) | Part 1: Number of Participants With Treatment-Emergent Adverse Events (TEAEs), and Treatment-Emergent Serious Adverse Events (TESAEs) | Any TEAE | 4 Participants |
| Part 1: Venglustat 15 mg (ROW) | Part 1: Number of Participants With Treatment-Emergent Adverse Events (TEAEs), and Treatment-Emergent Serious Adverse Events (TESAEs) | Any TESAE | 0 Participants |
| Part 1: Placebo (Japan Only) | Part 1: Number of Participants With Treatment-Emergent Adverse Events (TEAEs), and Treatment-Emergent Serious Adverse Events (TESAEs) | Any TEAE | 2 Participants |
| Part 1: Placebo (Japan Only) | Part 1: Number of Participants With Treatment-Emergent Adverse Events (TEAEs), and Treatment-Emergent Serious Adverse Events (TESAEs) | Any TESAE | 0 Participants |
| Part 1: Venglustat 4 mg (Japan Only) | Part 1: Number of Participants With Treatment-Emergent Adverse Events (TEAEs), and Treatment-Emergent Serious Adverse Events (TESAEs) | Any TEAE | 3 Participants |
| Part 1: Venglustat 4 mg (Japan Only) | Part 1: Number of Participants With Treatment-Emergent Adverse Events (TEAEs), and Treatment-Emergent Serious Adverse Events (TESAEs) | Any TESAE | 0 Participants |
| Part 1: Venglustat 8 mg (Japan Only) | Part 1: Number of Participants With Treatment-Emergent Adverse Events (TEAEs), and Treatment-Emergent Serious Adverse Events (TESAEs) | Any TESAE | 0 Participants |
| Part 1: Venglustat 8 mg (Japan Only) | Part 1: Number of Participants With Treatment-Emergent Adverse Events (TEAEs), and Treatment-Emergent Serious Adverse Events (TESAEs) | Any TEAE | 3 Participants |
| Part 1: Venglustat 15 mg (Japan Only) | Part 1: Number of Participants With Treatment-Emergent Adverse Events (TEAEs), and Treatment-Emergent Serious Adverse Events (TESAEs) | Any TEAE | 2 Participants |
| Part 1: Venglustat 15 mg (Japan Only) | Part 1: Number of Participants With Treatment-Emergent Adverse Events (TEAEs), and Treatment-Emergent Serious Adverse Events (TESAEs) | Any TESAE | 0 Participants |
Primary
Part 2: Change From Baseline to Week 52 in Movement Disorder Society-Unified Parkinson's Disease Rating Scale (MDS-UPDRS) Part II+III Total Score
MDS-UPDRS is a multimodal scale consisting of 4 parts. Part II assessed motor experiences of daily living (total score range: 0 to 52). It contained 13 questions completed by the participant. Part III assessed the motor signs of PD and was administered by the rater (total score range: 0 to 132). Part III contained 33 scores based on 18 items. In both parts, higher score indicated more severe symptoms. For each question in both parts, numeric score was assigned between 0 to 4, where 0=Normal, 1=Slight, 2=Mild, 3=Moderate, 4=Severe. MDS-UPDRS Total Part II and III score = sum of Part II and III scores with score ranged from 0 (no symptom) to 184 (severe symptoms), where higher scores reflected more severe symptoms of PD. Data for this OM was not planned to be collected and analyzed for Part 1, Part 2: DB period re-randomized participants and Part 2 LTFU period, as pre-specified in protocol.
Time frame: Baseline to Week 52
Population: Analyzed on intent-to-treat (ITT) population which included all randomized participants of Part 2 and analyzed in treatment group to which they were randomized. Here, overall number of participants analyzed = participants evaluable for this OM.
| Arm | Measure | Value (LEAST_SQUARES_MEAN) | Dispersion |
|---|---|---|---|
| Part 1: Placebo (ROW) | Part 2: Change From Baseline to Week 52 in Movement Disorder Society-Unified Parkinson's Disease Rating Scale (MDS-UPDRS) Part II+III Total Score | 4.71 units on a scale | Standard Error 1.27 |
| Part 1: Venglustat 4 mg (ROW) | Part 2: Change From Baseline to Week 52 in Movement Disorder Society-Unified Parkinson's Disease Rating Scale (MDS-UPDRS) Part II+III Total Score | 7.29 units on a scale | Standard Error 1.36 |
Comparison: Least-squares (LS) mean, standard errors (SE) and p-value were estimated from mixed-effect model with repeated measures (MMRM) analysis which included fixed categorical effects of treatment group, randomization strata, time point, treatment-by-time point and strata-by-time point interaction and continuous fixed covariates Baseline value and Baseline value-by-time point interaction.p-value: =0.167995% CI: [-1.1, 6.27]MMRM
Secondary
Part 2: Change From Baseline to Week 52 in Hoehn and Yahr (H and Y) Score
H and Y scale measured how Parkinson's symptoms progress and the level of disability. Scale allocated stage scores were from 0 to 5 to indicate relative level of disability as: Stage 0: no symptoms; Stage 1: symptoms on one side of the body only; Stage 2: symptoms on both sides of the body, without impairment of balance; Stage 3: Mild to moderate bilateral disease; some postural instability; physically independent; Stage 4: Severe disability; still able to walk or stand unassisted and Stage 5: Wheelchair bound or bedridden unless aided, where higher stage score described an increased severity of disease.
Time frame: Baseline to Week 52
Population: Analysis was performed on ITT population. Here, overall number of participants analyzed = participants evaluable for this OM. Data for this OM was not planned to be collected and analyzed for Part 1, Part 2: DB period re-randomized participants and Part 2 LTFU period, as pre-specified in protocol.
| Arm | Measure | Value (LEAST_SQUARES_MEAN) | Dispersion |
|---|---|---|---|
| Part 1: Placebo (ROW) | Part 2: Change From Baseline to Week 52 in Hoehn and Yahr (H and Y) Score | 0.18 units on a scale | Standard Error 0.05 |
| Part 1: Venglustat 4 mg (ROW) | Part 2: Change From Baseline to Week 52 in Hoehn and Yahr (H and Y) Score | 0.20 units on a scale | Standard Error 0.06 |
Comparison: LS mean, SE and P-value: estimated from MMRM analysis which included fixed categorical effects of treatment group, randomization strata, time point, treatment-by-time point and strata-by-time point interaction and continuous fixed covariates Baseline value and Baseline value-by-time-interaction.p-value: =0.7495% CI: [-0.12, 0.17]MMRM
Secondary
Part 2: Change From Baseline to Week 52 in Movement Disorder Society-Unified Parkinson's Disease Rating Scale (MDS-UPDRS) Part I+ II+III Score
MDS-UPDRS is a multimodal scale consisting of 4 parts. Part I assessed non-motor experiences of daily living and has 2 components (total score range: 0 to 52): Part IA contained 6 questions and was assessed by the examiner (total score range: 0 to 24). Part IB contained 7 questions on non-motor experiences of daily living which was completed by the participant (total score range: 0 to 28). Part II (13 questions completed by the participant) assessed motor experiences of daily living (total score range: 0 to 52). Part III assessed motor signs of PD and was administered by the rater (total score range: 0 to 132). Part III contained 33 scores based on 18 items. In all parts, higher score indicated more symptoms. For each question, numeric score was assigned between 0 to 4, where 0=Normal, 1=Slight, 2=Mild, 3=Moderate, 4=Severe. MDS-UPDRS total score = sum of Parts I, II, and III (Range: 0 to 236). Higher score = more severe symptoms of PD.
Time frame: Baseline to Week 52
Population: Analysis was performed on ITT population. Here, overall number of participants analyzed = participants evaluable for this OM. Data for this OM was not planned to be collected and analyzed for Part 1, Part 2: DB period re-randomized participants and Part 2 LTFU period, as pre-specified in protocol.
| Arm | Measure | Value (LEAST_SQUARES_MEAN) | Dispersion |
|---|---|---|---|
| Part 1: Placebo (ROW) | Part 2: Change From Baseline to Week 52 in Movement Disorder Society-Unified Parkinson's Disease Rating Scale (MDS-UPDRS) Part I+ II+III Score | 5.87 units on a scale | Standard Error 1.45 |
| Part 1: Venglustat 4 mg (ROW) | Part 2: Change From Baseline to Week 52 in Movement Disorder Society-Unified Parkinson's Disease Rating Scale (MDS-UPDRS) Part I+ II+III Score | 9.99 units on a scale | Standard Error 1.55 |
Comparison: LS mean, SE and P-value were estimated from MMRM analysis which included fixed categorical effects of treatment group, randomization strata, time point, treatment-by-time point and strata-by-time point interaction and continuous fixed covariates Baseline value and Baseline value-by-time-interactionp-value: =0.053595% CI: [-0.06, 8.32]MMRM
Secondary
Part 2: Change From Baseline to Week 52 in Parkinson's Disease Cognitive Rating Scale (PD-CRS) Total Score
The PD-CRS detects early cognitive impairment in Parkinson's disease. It is composed of 2 scales, the fronto-subcortical scale (items: sustained attention, working memory, alternating and action verbal fluency, clock drawing, immediate, and delayed free recall verbal memory) and the posterior-cortical scale (items: confrontation naming and clock copying). The total score of the fronto-subcortical scale (sum of all items) ranged from 0 (worst) to 104 (maximum score indicates better) and the total score of the posterior-cortical scale (sum of all items) ranged from 0 (worst) to 30 (maximum score indicates better). The PD-CRS Total score = the sum of PD-CRS fronto-subcortical score and the PD-CRS posterior-cortical score, which ranged from 0 to 134, where higher score = less impairment.
Time frame: Baseline to Week 52
Population: Analysis was performed on ITT population. Here, overall number of participants analyzed = participants evaluable for this OM. Data for this OM was not planned to be collected and analyzed for Part 1, Part 2: DB period re-randomized participants and Part 2 LTFU period, as pre-specified in protocol.
| Arm | Measure | Value (LEAST_SQUARES_MEAN) | Dispersion |
|---|---|---|---|
| Part 1: Placebo (ROW) | Part 2: Change From Baseline to Week 52 in Parkinson's Disease Cognitive Rating Scale (PD-CRS) Total Score | 0.32 units on a scale | Standard Error 1.27 |
| Part 1: Venglustat 4 mg (ROW) | Part 2: Change From Baseline to Week 52 in Parkinson's Disease Cognitive Rating Scale (PD-CRS) Total Score | -0.65 units on a scale | Standard Error 1.35 |
Comparison: LS mean, SE and P-value were estimated from MMRM analysis which included fixed categorical effects of treatment group, randomization strata, time point, treatment-by-time point and strata-by-time point interaction and continuous fixed covariates Baseline value and Baseline value-by-time point interaction.p-value: =0.599695% CI: [-4.63, 2.68]MMRM