Early-stage Parkinson's Disease
Conditions
Keywords
Early-stage parkinson's disease, UCB0599, Phase 2, Early-stage PD
Brief summary
The purpose of the study is to assess the safety and tolerability of UCB0599 and to demonstrate the superiority of UCB0599 over placebo with regard to clinical symptoms of disease progression over 12 and 18 months in participants diagnosed with early-stage Parkinson's Disease.
Interventions
DRUGUCB0599
Drug: UCB0599 Pharmaceutical form: Granules in capsules Route of administration: Oral use Participants will receive UCB0599 in a pre-specified sequence during the Treatment Period.
DRUGPlacebo
Drug: Placebo Pharmaceutical form: Capsules Route of administration: Oral use Participants will receive Placebo in a pre-specified sequence during the Treatment Period.
Sponsors
The Parkinson Study Group
UCB Biopharma SRL
Study design
Allocation
RANDOMIZED
Intervention model
PARALLEL
Primary purpose
TREATMENT
Masking
QUADRUPLE (Subject, Caregiver, Investigator, Outcomes Assessor)
Eligibility
Sex/Gender
ALL
Age
40 Years to 75 Years
Healthy volunteers
No
Inclusion criteria
* Study participant must be 40 to 75 years of age inclusive, at the time of signing the informed consent * Study participant has Parkinson's Disease (PD), with a diagnosis made by a neurologist according to the 2015 Movement Disorder Society criteria within 2 years of Baseline Visit (including diagnosis during Screening) * The following diagnostic criteria must be met: bradykinesia AND at least ONE of the following: muscular rigidity, or resting tremor * A Screening Dopamine Transporter Imaging with Single Photon Emission Computed Tomography (DaT-SPECT), or a historical DaT-SPECT within 3 months of the Screening Visit that has been qualified by the central reader, shows evidence of dopamine transporter deficit per study requirements and as determined by a central reader * Study participant is in the ≤2.5 modified Hoehn and Yahr stage at Screening * Study participant has never taken medications for the treatment of motor symptoms of PD and is not expected to require starting symptomatic treatment (ST) with a high likelihood in the next 6 months as far as clinical judgement allows * Study participant has never taken part in disease-modifying treatment studies directed at neurodegenerative disease (NDD) * Study participant does not take N-acetyl cysteine or other cysteine donors or glutathione precursors on a regular basis as a food supplement * Study participant is willing, competent, and able to comply with all aspects of the protocol, including follow-up schedule and biospecimen collection * Study participant has a body mass index (BMI) of 16 to 34kg/m² (inclusive) * Contraception i) A male participant must agree to use contraception during the Treatment Period and for at least 90 days after the last dose ofstudy medication and refrain from donating sperm during this period ii) A female participant is eligible to participate if she is not pregnant, not breastfeeding, andat least one of the following conditions applies: Not a woman of childbearing potential (WOCBP) OR A WOCBP who agrees to follow the contraceptive guidance during the Treatment Period and for at least 1 month after the last dose of study medication. The study participant must have a negative serum pregnancy test at Screening, which is to be confirmed negative by urine testing prior to the first dose of study medication at Baseline (Visit 3). If oral contraception is used, an additional barrier method will be required during the study as a study medication related-gastrointestinal upset or a drug interaction by CYP3A4 induction could interfere with efficacy
Exclusion criteria
* Study participant has a known hypersensitivity to any components (and/or its excipients) of the study medication or comparative drugs as stated in the protocol * Study participant has a brain magnetic resonance imaging (MRI) scan performed during Screening indicative of a clinically significant abnormality or a historical MRI scan during the 6 months before Screening Visit 1 of sufficient quality to show such abnormalities. In case of doubt, the significance is determined on a case-by-case basis in close collaboration with the Medical Monitor and should not include abnormalities like age-appropriate brain atrophy, minor white matter signals, or mild vasculopathy * Study participant has any contraindication for the brain MRI or Dopamine Transporter Imaging with Single Photon Emission Computed Tomography (DaT-SPECT) imaging * Study participant has a Montreal Cognitive Assessment (MoCA) score less than 23, indicating mild cognitive impairment or other significant cognitive impairment or clinical dementia at Screening that, in the opinion of the Investigator, would interfere with study evaluation * Study participant has abnormalities in lumbar spine previously known or determined by a Screening lumbar x-ray (if conducted) that could preclude lumbar puncture, in the opinion of the Investigator. The participant must be excluded from lumbar puncture but not from study participation * Study participant has clinically significant electrocardiogram (ECG) abnormality at Screening, in the opinion of the Investigator * Study participant has past history of use of medications for the treatment of motor symptoms of PD. Short (up to 4 weeks) past use of medications for the treatment of motor symptoms is permitted following a sufficient washout period. Medications included are: levodopa (maximum 400mg per day), dopamine agonists, monoamine oxidase B (MAO-B) inhibitors, anticholinergics, or amantadine. A sufficient washout period is at least 3 months prior to the Baseline Visit
Design outcomes
Primary
| Measure | Time frame | Description |
|---|---|---|
| Movement Disorder Society Unified Parkinson's Disease Rating Scale (MDS-UPDRS) Parts I-III Sum Score at Day 0 | Day 0 | MDS-UPDRS is a multimodal scale. Part I assessed non-motor experiences of daily living and has 2 components (0-52 possible points). Part IA contains 6 questions and is assessed by the examiner (0-24 possible points). Part IB contains 7 questions on non-motor experiences of daily living completed by participant (0-28 possible points). Part II assessed motor experiences of daily living (0-52 possible points) and includes 13 questions completed by participant. Part III assessed motor signs of PD and was administered by rater (0-52 possible points). Part III contained 33 questions based on 18 items. For all questions of each part, numeric score response options linked to accepted clinical terms: 0 to 4, 0=Normal, 1=Slight, 2=Mild, 3=Moderate, 4=Severe. Total Score equals sum of Parts I, II, and III (Score: 0-236). Higher score = more severe PD symptoms. |
| Movement Disorder Society Unified Parkinson's Disease Rating Scale (MDS-UPDRS) Parts I-III Sum Score at Month 2 | Month 2 | MDS-UPDRS is a multimodal scale. Part I assessed non-motor experiences of daily living and has 2 components (0-52 possible points). Part IA contains 6 questions and is assessed by the examiner (0-24 possible points). Part IB contains 7 questions on non-motor experiences of daily living completed by participant (0-28 possible points). Part II assessed motor experiences of daily living (0-52 possible points) and includes 13 questions completed by participant. Part III assessed motor signs of PD and was administered by rater (0-52 possible points). Part III contained 33 questions based on 18 items. For all questions of each part, numeric score response options linked to accepted clinical terms: 0 to 4, 0=Normal, 1=Slight, 2=Mild, 3=Moderate, 4=Severe. Total Score equals sum of Parts I, II, and III (Score: 0-236). Higher score = more severe PD symptoms. |
| Movement Disorder Society Unified Parkinson's Disease Rating Scale (MDS-UPDRS) Parts I-III Sum Score at Month 4 | Month 4 | MDS-UPDRS is a multimodal scale. Part I assessed non-motor experiences of daily living and has 2 components (0-52 possible points). Part IA contains 6 questions and is assessed by the examiner (0-24 possible points). Part IB contains 7 questions on non-motor experiences of daily living completed by participant (0-28 possible points). Part II assessed motor experiences of daily living (0-52 possible points) and includes 13 questions completed by participant. Part III assessed motor signs of PD and was administered by rater (0-52 possible points). Part III contained 33 questions based on 18 items. For all questions of each part, numeric score response options linked to accepted clinical terms: 0 to 4, 0=Normal, 1=Slight, 2=Mild, 3=Moderate, 4=Severe. Total Score equals sum of Parts I, II, and III (Score: 0-236). Higher score = more severe PD symptoms. |
| Movement Disorder Society Unified Parkinson's Disease Rating Scale (MDS-UPDRS) Parts I-III Sum Score at Month 6 | Month 6 | MDS-UPDRS is a multimodal scale. Part I assessed non-motor experiences of daily living and has 2 components (0-52 possible points). Part IA contains 6 questions and is assessed by the examiner (0-24 possible points). Part IB contains 7 questions on non-motor experiences of daily living completed by participant (0-28 possible points). Part II assessed motor experiences of daily living (0-52 possible points) and includes 13 questions completed by participant. Part III assessed motor signs of PD and was administered by rater (0-52 possible points). Part III contained 33 questions based on 18 items. For all questions of each part, numeric score response options linked to accepted clinical terms: 0 to 4, 0=Normal, 1=Slight, 2=Mild, 3=Moderate, 4=Severe. Total Score equals sum of Parts I, II, and III (Score: 0-236). Higher score = more severe PD symptoms. |
| Movement Disorder Society Unified Parkinson's Disease Rating Scale (MDS-UPDRS) Parts I-III Sum Score at Month 8 | Month 8 | MDS-UPDRS is a multimodal scale. Part I assessed non-motor experiences of daily living and has 2 components (0-52 possible points). Part IA contains 6 questions and is assessed by the examiner (0-24 possible points). Part IB contains 7 questions on non-motor experiences of daily living completed by participant (0-28 possible points). Part II assessed motor experiences of daily living (0-52 possible points) and includes 13 questions completed by participant. Part III assessed motor signs of PD and was administered by rater (0-52 possible points). Part III contained 33 questions based on 18 items. For all questions of each part, numeric score response options linked to accepted clinical terms: 0 to 4, 0=Normal, 1=Slight, 2=Mild, 3=Moderate, 4=Severe. Total Score equals sum of Parts I, II, and III (Score: 0-236). Higher score = more severe PD symptoms. |
| Movement Disorder Society Unified Parkinson's Disease Rating Scale (MDS-UPDRS) Parts I-III Sum Score at Month 10 | Month 10 | MDS-UPDRS is a multimodal scale. Part I assessed non-motor experiences of daily living and has 2 components (0-52 possible points). Part IA contains 6 questions and is assessed by the examiner (0-24 possible points). Part IB contains 7 questions on non-motor experiences of daily living completed by participant (0-28 possible points). Part II assessed motor experiences of daily living (0-52 possible points) and includes 13 questions completed by participant. Part III assessed motor signs of PD and was administered by rater (0-52 possible points). Part III contained 33 questions based on 18 items. For all questions of each part, numeric score response options linked to accepted clinical terms: 0 to 4, 0=Normal, 1=Slight, 2=Mild, 3=Moderate, 4=Severe. Total Score equals sum of Parts I, II, and III (Score: 0-236). Higher score = more severe PD symptoms. |
| Movement Disorder Society Unified Parkinson's Disease Rating Scale (MDS-UPDRS) Parts I-III Sum Score at Month 12 | Month 12 | MDS-UPDRS is a multimodal scale. Part I assessed non-motor experiences of daily living and has 2 components (0-52 possible points). Part IA contains 6 questions and is assessed by the examiner (0-24 possible points). Part IB contains 7 questions on non-motor experiences of daily living completed by participant (0-28 possible points). Part II assessed motor experiences of daily living (0-52 possible points) and includes 13 questions completed by participant. Part III assessed motor signs of PD and was administered by rater (0-52 possible points). Part III contained 33 questions based on 18 items. For all questions of each part, numeric score response options linked to accepted clinical terms: 0 to 4, 0=Normal, 1=Slight, 2=Mild, 3=Moderate, 4=Severe. Total Score equals sum of Parts I, II, and III (Score: 0-236). Higher score = more severe PD symptoms. |
| Movement Disorder Society Unified Parkinson's Disease Rating Scale (MDS-UPDRS) Parts I-III Sum Score at Month 14 | Month 14 | MDS-UPDRS is a multimodal scale. Part I assessed non-motor experiences of daily living and has 2 components (0-52 possible points). Part IA contains 6 questions and is assessed by the examiner (0-24 possible points). Part IB contains 7 questions on non-motor experiences of daily living completed by participant (0-28 possible points). Part II assessed motor experiences of daily living (0-52 possible points) and includes 13 questions completed by participant. Part III assessed motor signs of PD and was administered by rater (0-52 possible points). Part III contained 33 questions based on 18 items. For all questions of each part, numeric score response options linked to accepted clinical terms: 0 to 4, 0=Normal, 1=Slight, 2=Mild, 3=Moderate, 4=Severe. Total Score equals sum of Parts I, II, and III (Score: 0-236). Higher score = more severe PD symptoms. |
| Movement Disorder Society Unified Parkinson's Disease Rating Scale (MDS-UPDRS) Parts I-III Sum Score at Month 16 | Month 16 | MDS-UPDRS is a multimodal scale. Part I assessed non-motor experiences of daily living and has 2 components (0-52 possible points). Part IA contains 6 questions and is assessed by the examiner (0-24 possible points). Part IB contains 7 questions on non-motor experiences of daily living completed by participant (0-28 possible points). Part II assessed motor experiences of daily living (0-52 possible points) and includes 13 questions completed by participant. Part III assessed motor signs of PD and was administered by rater (0-52 possible points). Part III contained 33 questions based on 18 items. For all questions of each part, numeric score response options linked to accepted clinical terms: 0 to 4, 0=Normal, 1=Slight, 2=Mild, 3=Moderate, 4=Severe. Total Score equals sum of Parts I, II, and III (Score: 0-236). Higher score = more severe PD symptoms. |
| Movement Disorder Society Unified Parkinson's Disease Rating Scale (MDS-UPDRS) Parts I-III Sum Score at Month 18 | Month 18 | MDS-UPDRS is a multimodal scale. Part I assessed non-motor experiences of daily living and has 2 components (0-52 possible points). Part IA contains 6 questions and is assessed by the examiner (0-24 possible points). Part IB contains 7 questions on non-motor experiences of daily living completed by participant (0-28 possible points). Part II assessed motor experiences of daily living (0-52 possible points) and includes 13 questions completed by participant. Part III assessed motor signs of PD and was administered by rater (0-52 possible points). Part III contained 33 questions based on 18 items. For all questions of each part, numeric score response options linked to accepted clinical terms: 0 to 4, 0=Normal, 1=Slight, 2=Mild, 3=Moderate, 4=Severe. Total Score equals sum of Parts I, II, and III (Score: 0-236). Higher score = more severe PD symptoms. |
Secondary
| Measure | Time frame | Description |
|---|---|---|
| Percentage of Participants With Treatment-emergent Adverse Events (TEAEs) | From Baseline up to 30 days of safety follow-up after the last dose of the study drug (up to 19 months) | An AE is any untoward medical occurrence in a patient or clinical study participant, temporally associated with the use of study medication, whether or not considered related to the study medication. A TEAE is defined as any AE with a start date on or after the first dose of treatment or any unresolved event already present before administration of treatment that worsens in intensity following exposure to the treatment. |
| MDS-UPDRS Part III Subscale | Day 0, Months 2, 4, 6, 8, 10, 12, 14, 16, 18 | MDS-UPDRS part III includes motor items assessing speech, facial expression, rigidity, finger tapping, hand movements, pronation supination movements of hands, toe tapping, leg agility, arising from chair, gait, freezing of gait, postural stability, posture, global spontaneity of movement (body bradykinesia), postural tremor of the hands, kinetic tremor of the hands, rest tremor amplitude, and constancy of rest tremor. It included 33 scores based on 18-items, each anchored with 5 responses: 0 = normal, 1 = slight, 2 = mild, 3 = moderate, and 4 = severe. The scale range was from 0 to 132, with a lower score indicating better motor function and a higher score indicating more severe motor symptoms. |
| Percentage of Participants With TEAEs Leading to Participant Withdrawal | From Baseline up to 30 days of safety follow-up after the last dose of the study drug (up to 19 months) | An AE is any untoward medical occurrence in a patient or clinical study participant, temporally associated with the use of study medication, whether or not considered related to the study medication. A TEAE is defined as any AE with a start date on or after the first dose of treatment or any unresolved event already present before administration of treatment that worsens in intensity following exposure to the treatment. |
| Percentage of Participants With Serious TEAEs | From Baseline up to 30 days of safety follow-up after the last dose of the study drug (up to 19 months) | Serious adverse event: Death, a life-threatening adverse event, inpatient hospitalization or prolongation of existing hospitalization, a persistent or significant incapacity or substantial disruption of the ability to conduct normal life functions, or a congenital anomaly/birth defect. Important medical events that may not result in death, be life-threatening, or require hospitalization may be considered serious when, based upon appropriate medical judgment, they may jeopardize the patient or subject and may require medical or surgical intervention to prevent one of the outcomes listed in this definition. |
| MDS-UPDRS Part III Early-stage Parkinson's Disease (ePD) Subscore on Selected Items | Day 0, Months 2, 4, 6, 8, 10, 12, 14, 16, 18 | The early-stage Parkinson's disease (ePD) subscore is derived from a 15-item subset of the MDS-UPDRS Part III (Motor Examination). It includes all rigidity assessments (neck, upper limbs \[right/left\], and lower limbs \[right/left\]) and bradykinesia-related tasks: finger tapping (right/left), hand movements (right/left), pronation-supination of hands (right/left), toe tapping (right/left), and leg agility (right/left). Each item is scored on a 5-point likert scale (0 = no problem to 4 = severe), resulting in a total ePD subscore range of 0 to 60. Higher scores indicate greater motor impairment, while lower scores reflect better motor function. |
| MDS-UPDRS Part II Subscale | Day 0, Months 2, 4, 6, 8, 10, 12, 14, 16, 18 | MDS-UPDRS part II includes motor items assessing speech, saliva and drooling, chewing and swallowing, eating tasks (cutting food and handling utensils), dressing, hygiene, handwriting, doing hobbies and other activities, turning in bed, tremor, getting out of bed, a car or a deep chair, walking and balance, and freezing. Each of the items in the UPDRS is measured on a scale. It included 13-items, each anchored with 5 responses: 0 = normal, 1 = slight, 2 = mild, 3 = moderate, and 4 = severe. The scale range for Part II was 0-52, with higher scores reflecting greater severity. |
| MDS-UPDRS Part I Subscale | Day 0, Months 2, 4, 6, 8, 10, 12, 14, 16, 18 | MDS-UPDRS part I include several non-motor aspects of experiences of daily living including cognitive impairment, hallucinations and psychosis, depressed mood, anxious mood, apathy, features of dopamine dysregulation syndrome during part 1A and sleep problems, daytime sleepiness, pain and other sensation, urinary problems, constipation problems, lightheadedness on standing, and fatigue during Part 1B. Part I assessed non-motor experiences of daily living and has 2 components (Range 0-52). Part IA contained 6 questions and were assessed by examiner (Range 0-24). Part IB contained 7 questions on non-motor experiences of daily living which were completed by participant (Range 0-28). Each of items in UPDRS is measured on a scale of 0 to 4, where 0 is normal and 4 (higher score) represents severe abnormalities/worse outcome. Total Score Range 0 to 52. Total score is sum of Part IA (0-24) and Part IB (0-28) subscale scores. Higher scores indicated greater severity of non-motor symptoms. |
| Emerging Symptoms in Participants as Measured by MDS-UPDRS Part II | Baseline to Month 18 | The participant was considered to have an emerging symptom for the item, if the change from Baseline for the item is greater than 0 for 2 consecutive visits. The magnitude of change from Baseline was not considered to determine the emerging symptom. Movement Disorder Society Unified Parkinson's Disease Rating Scale (MDS-UPDRS) part II includes motor items assessing speech, saliva and drooling, chewing and swallowing, eating tasks (cutting food and handling utensils), dressing, hygiene, handwriting, doing hobbies and other activities, turning in bed, tremor, getting out of bed, a car or a deep chair, walking and balance, and freezing. This included 13-items, each anchored with 5 responses: 0 = normal, 1 = slight, 2 = mild, 3 = moderate, and 4 = severe. The scale range for Part II was 0-52, with higher scores reflecting greater severity. |
| Time to Worsening of the Disease as Measured by MDS-UPDRS Part III | Baseline to Month 18 | Time to worsening of the disease on the MDS-UPDRS III scale as defined by a 5-point increase in MDS-UPDRS III, within the 18-month period. MDS-UPDRS part III includes motor items assessing speech, facial expression, rigidity, finger tapping, hand movements, pronation supination movements of hands, toe tapping, leg agility, arising from chair, gait, freezing of gait, postural stability, posture, global spontaneity of movement (body bradykinesia), postural tremor of the hands, kinetic tremor of the hands, rest tremor amplitude, and constancy of rest tremor. It included 33 scores based on 18-items, each anchored with 5 responses: 0 = normal, 1 = slight, 2 = mild, 3 = moderate, and 4 = severe. The scale range was from 0 to 132, with a lower score indicating better motor function and a higher score indicating more severe motor symptoms. |
| Montreal Cognitive Assessment (MoCA) | Screening, Month 18 | The Montreal Cognitive Assessment (MoCA) is a standardized screening tool used to evaluate mild cognitive impairment across multiple cognitive functions i.e. visuospatial/executive function, naming, memory, attention, language, abstraction, delayed recall, and orientation. The total possible score is calculated by summing the scores across all functions ranges from: 0 to 30. A score of 26 or above is considered normal, a lower score indicates cognitive impairment. |
| Change From Screening in Dopamine Transporter Imaging With Single Photon Emission Computed Tomography (DaT-SPECT) Mean Striatum Specific Binding Ratios (SBR) | Screening, Months 12 and 18 | The change from screening in mean striatum specific binding ratios (SBR) was assessed by DaT-SPECT using 123I-Ioflupane as radiopharmaceutical. The whole striatum was calculated as the average of the SBR data values for the four following small regions: left caudate small, left putamen small, right caudate small and right putamen small. The SBR was calculated for each region with the occipital cortex as a reference region, where a lower SBR indicates worse disease. The following formula was used to calculate this: (Average \[Small region\] - Average \[Occipital region\])/ (Average \[Occipital region\]). |
| Time to Start of Symptomatic Treatment (ST) | Baseline to Month 18 | Time to start of symptomatic treatment (ST) within the 18-month period. |
| Symptomatic Treatment (ST) Intake | Month 18 | Cumulative number of participants on symptomatic treatment (ST) at 18 months are reported. |
Countries
Canada, France, Germany, Italy, Netherlands, Poland, Spain, United Kingdom, United States
Participant flow
Recruitment details
The study started to enroll participants in December 2020 and concluded in September 2024.
Pre-assignment details
The Participant Flow refers to the Randomized Set.
Participants by arm
| Arm | Count |
|---|---|
| Placebo Participants received UCB0599 matching-placebo capsules, orally, from Day 1 up to 18 months during treatment period. | 165 |
| UCB0599 180 mg/Day Participants received UCB0599 90 milligram (mg) capsules twice daily (BID), for a total daily dose of 180 mg, along with matching placebo capsules BID, administered orally from Day 1 up to 18 months during treatment period. | 166 |
| UCB0599 360 mg/Day Participants received UCB0599 180 mg capsules twice daily (BID), for a total daily dose of 360 mg, administered orally from Day 1 up to 18 months during treatment period. | 165 |
| Total | 496 |
Withdrawals & dropouts
| Period | Reason | FG000 | FG001 | FG002 |
|---|---|---|---|---|
| Overall Study | Adverse Event | 4 | 18 | 15 |
| Overall Study | Consent Withdrawn by Participant (not due to adverse event) | 5 | 6 | 3 |
| Overall Study | Dropout on the Promotor's Decision | 0 | 0 | 1 |
| Overall Study | Lack of Efficacy | 1 | 1 | 0 |
| Overall Study | Lost to Follow-up | 0 | 0 | 2 |
| Overall Study | Non-Compliance | 0 | 1 | 1 |
| Overall Study | Participant is Moving in Another Province | 0 | 1 | 0 |
| Overall Study | Participant Not Eligible. Randomized In Error | 1 | 0 | 1 |
| Overall Study | PI Decision due to Participant Safety | 0 | 0 | 1 |
| Overall Study | Protocol Violation | 0 | 0 | 1 |
| Overall Study | Site Closure; Participant declined transfer | 0 | 1 | 0 |
| Overall Study | Worsening Symptoms; Participant Withdrew | 0 | 0 | 1 |
Baseline characteristics
| Characteristic | UCB0599 180 mg/Day | UCB0599 360 mg/Day | Total | Placebo |
|---|---|---|---|---|
| Age, Continuous | 61.4 years STANDARD_DEVIATION 7.8 | 59.9 years STANDARD_DEVIATION 8.4 | 60.8 years STANDARD_DEVIATION 8.2 | 61.2 years STANDARD_DEVIATION 8.3 |
| Age, Customized 18 years to less than (<) 65 years | 105 Participants | 110 Participants | 313 Participants | 98 Participants |
| Age, Customized 65 years to <85 years | 61 Participants | 55 Participants | 183 Participants | 67 Participants |
| Race/Ethnicity, Customized Ethnicity Hispanic or Latino | 3 Participants | 5 Participants | 12 Participants | 4 Participants |
| Race/Ethnicity, Customized Ethnicity Missing | 22 Participants | 19 Participants | 66 Participants | 25 Participants |
| Race/Ethnicity, Customized Ethnicity Not Hispanic or Latino | 141 Participants | 141 Participants | 418 Participants | 136 Participants |
| Race/Ethnicity, Customized Race Asian | 1 Participants | 1 Participants | 4 Participants | 2 Participants |
| Race/Ethnicity, Customized Race Missing | 22 Participants | 19 Participants | 66 Participants | 25 Participants |
| Race/Ethnicity, Customized Race Other/Mixed | 0 Participants | 2 Participants | 6 Participants | 4 Participants |
| Race/Ethnicity, Customized Race White | 143 Participants | 143 Participants | 420 Participants | 134 Participants |
| Sex: Female, Male Female | 66 Participants | 65 Participants | 196 Participants | 65 Participants |
| Sex: Female, Male Male | 100 Participants | 100 Participants | 300 Participants | 100 Participants |
Adverse events
| Event type | EG000 affected / at risk | EG001 affected / at risk | EG002 affected / at risk |
|---|---|---|---|
| deaths Total, all-cause mortality | 0 / 164 | 1 / 165 | 1 / 164 |
| other Total, other adverse events | 102 / 164 | 97 / 165 | 89 / 164 |
| serious Total, serious adverse events | 9 / 164 | 13 / 165 | 14 / 164 |
Outcome results
Primary
Movement Disorder Society Unified Parkinson's Disease Rating Scale (MDS-UPDRS) Parts I-III Sum Score at Day 0
MDS-UPDRS is a multimodal scale. Part I assessed non-motor experiences of daily living and has 2 components (0-52 possible points). Part IA contains 6 questions and is assessed by the examiner (0-24 possible points). Part IB contains 7 questions on non-motor experiences of daily living completed by participant (0-28 possible points). Part II assessed motor experiences of daily living (0-52 possible points) and includes 13 questions completed by participant. Part III assessed motor signs of PD and was administered by rater (0-52 possible points). Part III contained 33 questions based on 18 items. For all questions of each part, numeric score response options linked to accepted clinical terms: 0 to 4, 0=Normal, 1=Slight, 2=Mild, 3=Moderate, 4=Severe. Total Score equals sum of Parts I, II, and III (Score: 0-236). Higher score = more severe PD symptoms.
Time frame: Day 0
Population: Full analysis set (FAS) included all randomized study participants who received at least a partial dose of study medication, have at least 1 post-Baseline assessment. Here, number of participants analyzed included all participants who were evaluable for this assessment.
| Arm | Measure | Value (MEAN) | Dispersion |
|---|---|---|---|
| Placebo | Movement Disorder Society Unified Parkinson's Disease Rating Scale (MDS-UPDRS) Parts I-III Sum Score at Day 0 | 34.0 score on a scale | Standard Deviation 13.2 |
| UCB0599 180 mg/Day | Movement Disorder Society Unified Parkinson's Disease Rating Scale (MDS-UPDRS) Parts I-III Sum Score at Day 0 | 33.9 score on a scale | Standard Deviation 16.4 |
| UCB0599 360 mg/Day | Movement Disorder Society Unified Parkinson's Disease Rating Scale (MDS-UPDRS) Parts I-III Sum Score at Day 0 | 30.6 score on a scale | Standard Deviation 13.4 |
Primary
Movement Disorder Society Unified Parkinson's Disease Rating Scale (MDS-UPDRS) Parts I-III Sum Score at Month 10
MDS-UPDRS is a multimodal scale. Part I assessed non-motor experiences of daily living and has 2 components (0-52 possible points). Part IA contains 6 questions and is assessed by the examiner (0-24 possible points). Part IB contains 7 questions on non-motor experiences of daily living completed by participant (0-28 possible points). Part II assessed motor experiences of daily living (0-52 possible points) and includes 13 questions completed by participant. Part III assessed motor signs of PD and was administered by rater (0-52 possible points). Part III contained 33 questions based on 18 items. For all questions of each part, numeric score response options linked to accepted clinical terms: 0 to 4, 0=Normal, 1=Slight, 2=Mild, 3=Moderate, 4=Severe. Total Score equals sum of Parts I, II, and III (Score: 0-236). Higher score = more severe PD symptoms.
Time frame: Month 10
Population: FAS included all randomized study participants who received at least a partial dose of study medication, have at least 1 post-Baseline assessment. Here, number of participants analyzed included all participants who were evaluable for this assessment.
| Arm | Measure | Value (MEAN) | Dispersion |
|---|---|---|---|
| Placebo | Movement Disorder Society Unified Parkinson's Disease Rating Scale (MDS-UPDRS) Parts I-III Sum Score at Month 10 | 35.6 score on a scale | Standard Deviation 13.6 |
| UCB0599 180 mg/Day | Movement Disorder Society Unified Parkinson's Disease Rating Scale (MDS-UPDRS) Parts I-III Sum Score at Month 10 | 33.7 score on a scale | Standard Deviation 18.4 |
| UCB0599 360 mg/Day | Movement Disorder Society Unified Parkinson's Disease Rating Scale (MDS-UPDRS) Parts I-III Sum Score at Month 10 | 33.2 score on a scale | Standard Deviation 15.6 |
Primary
Movement Disorder Society Unified Parkinson's Disease Rating Scale (MDS-UPDRS) Parts I-III Sum Score at Month 12
MDS-UPDRS is a multimodal scale. Part I assessed non-motor experiences of daily living and has 2 components (0-52 possible points). Part IA contains 6 questions and is assessed by the examiner (0-24 possible points). Part IB contains 7 questions on non-motor experiences of daily living completed by participant (0-28 possible points). Part II assessed motor experiences of daily living (0-52 possible points) and includes 13 questions completed by participant. Part III assessed motor signs of PD and was administered by rater (0-52 possible points). Part III contained 33 questions based on 18 items. For all questions of each part, numeric score response options linked to accepted clinical terms: 0 to 4, 0=Normal, 1=Slight, 2=Mild, 3=Moderate, 4=Severe. Total Score equals sum of Parts I, II, and III (Score: 0-236). Higher score = more severe PD symptoms.
Time frame: Month 12
Population: FAS included all randomized study participants who received at least a partial dose of study medication, have at least 1 post-Baseline assessment. Here, number of participants analyzed included all participants who were evaluable for this assessment.
| Arm | Measure | Value (MEAN) | Dispersion |
|---|---|---|---|
| Placebo | Movement Disorder Society Unified Parkinson's Disease Rating Scale (MDS-UPDRS) Parts I-III Sum Score at Month 12 | 37.3 score on a scale | Standard Deviation 15.3 |
| UCB0599 180 mg/Day | Movement Disorder Society Unified Parkinson's Disease Rating Scale (MDS-UPDRS) Parts I-III Sum Score at Month 12 | 36.5 score on a scale | Standard Deviation 19.2 |
| UCB0599 360 mg/Day | Movement Disorder Society Unified Parkinson's Disease Rating Scale (MDS-UPDRS) Parts I-III Sum Score at Month 12 | 34.5 score on a scale | Standard Deviation 15.4 |
Primary
Movement Disorder Society Unified Parkinson's Disease Rating Scale (MDS-UPDRS) Parts I-III Sum Score at Month 14
MDS-UPDRS is a multimodal scale. Part I assessed non-motor experiences of daily living and has 2 components (0-52 possible points). Part IA contains 6 questions and is assessed by the examiner (0-24 possible points). Part IB contains 7 questions on non-motor experiences of daily living completed by participant (0-28 possible points). Part II assessed motor experiences of daily living (0-52 possible points) and includes 13 questions completed by participant. Part III assessed motor signs of PD and was administered by rater (0-52 possible points). Part III contained 33 questions based on 18 items. For all questions of each part, numeric score response options linked to accepted clinical terms: 0 to 4, 0=Normal, 1=Slight, 2=Mild, 3=Moderate, 4=Severe. Total Score equals sum of Parts I, II, and III (Score: 0-236). Higher score = more severe PD symptoms.
Time frame: Month 14
Population: FAS included all randomized study participants who received at least a partial dose of study medication, have at least 1 post-Baseline assessment. Here, number of participants analyzed included all participants who were evaluable for this assessment.
| Arm | Measure | Value (MEAN) | Dispersion |
|---|---|---|---|
| Placebo | Movement Disorder Society Unified Parkinson's Disease Rating Scale (MDS-UPDRS) Parts I-III Sum Score at Month 14 | 37.1 score on a scale | Standard Deviation 14.6 |
| UCB0599 180 mg/Day | Movement Disorder Society Unified Parkinson's Disease Rating Scale (MDS-UPDRS) Parts I-III Sum Score at Month 14 | 34.8 score on a scale | Standard Deviation 18.8 |
| UCB0599 360 mg/Day | Movement Disorder Society Unified Parkinson's Disease Rating Scale (MDS-UPDRS) Parts I-III Sum Score at Month 14 | 33.9 score on a scale | Standard Deviation 16.9 |
Primary
Movement Disorder Society Unified Parkinson's Disease Rating Scale (MDS-UPDRS) Parts I-III Sum Score at Month 16
MDS-UPDRS is a multimodal scale. Part I assessed non-motor experiences of daily living and has 2 components (0-52 possible points). Part IA contains 6 questions and is assessed by the examiner (0-24 possible points). Part IB contains 7 questions on non-motor experiences of daily living completed by participant (0-28 possible points). Part II assessed motor experiences of daily living (0-52 possible points) and includes 13 questions completed by participant. Part III assessed motor signs of PD and was administered by rater (0-52 possible points). Part III contained 33 questions based on 18 items. For all questions of each part, numeric score response options linked to accepted clinical terms: 0 to 4, 0=Normal, 1=Slight, 2=Mild, 3=Moderate, 4=Severe. Total Score equals sum of Parts I, II, and III (Score: 0-236). Higher score = more severe PD symptoms.
Time frame: Month 16
Population: FAS included all randomized study participants who received at least a partial dose of study medication, have at least 1 post-Baseline assessment. Here, number of participants analyzed included all participants who were evaluable for this assessment.
| Arm | Measure | Value (MEAN) | Dispersion |
|---|---|---|---|
| Placebo | Movement Disorder Society Unified Parkinson's Disease Rating Scale (MDS-UPDRS) Parts I-III Sum Score at Month 16 | 37.2 score on a scale | Standard Deviation 15.6 |
| UCB0599 180 mg/Day | Movement Disorder Society Unified Parkinson's Disease Rating Scale (MDS-UPDRS) Parts I-III Sum Score at Month 16 | 35.9 score on a scale | Standard Deviation 19.8 |
| UCB0599 360 mg/Day | Movement Disorder Society Unified Parkinson's Disease Rating Scale (MDS-UPDRS) Parts I-III Sum Score at Month 16 | 33.6 score on a scale | Standard Deviation 15.4 |
Primary
Movement Disorder Society Unified Parkinson's Disease Rating Scale (MDS-UPDRS) Parts I-III Sum Score at Month 18
MDS-UPDRS is a multimodal scale. Part I assessed non-motor experiences of daily living and has 2 components (0-52 possible points). Part IA contains 6 questions and is assessed by the examiner (0-24 possible points). Part IB contains 7 questions on non-motor experiences of daily living completed by participant (0-28 possible points). Part II assessed motor experiences of daily living (0-52 possible points) and includes 13 questions completed by participant. Part III assessed motor signs of PD and was administered by rater (0-52 possible points). Part III contained 33 questions based on 18 items. For all questions of each part, numeric score response options linked to accepted clinical terms: 0 to 4, 0=Normal, 1=Slight, 2=Mild, 3=Moderate, 4=Severe. Total Score equals sum of Parts I, II, and III (Score: 0-236). Higher score = more severe PD symptoms.
Time frame: Month 18
Population: FAS included all randomized study participants who received at least a partial dose of study medication, have at least 1 post-Baseline assessment. Here, number of participants analyzed included all participants who were evaluable for this assessment.
| Arm | Measure | Value (MEAN) | Dispersion |
|---|---|---|---|
| Placebo | Movement Disorder Society Unified Parkinson's Disease Rating Scale (MDS-UPDRS) Parts I-III Sum Score at Month 18 | 37.9 score on a scale | Standard Deviation 15.5 |
| UCB0599 180 mg/Day | Movement Disorder Society Unified Parkinson's Disease Rating Scale (MDS-UPDRS) Parts I-III Sum Score at Month 18 | 35.6 score on a scale | Standard Deviation 18.1 |
| UCB0599 360 mg/Day | Movement Disorder Society Unified Parkinson's Disease Rating Scale (MDS-UPDRS) Parts I-III Sum Score at Month 18 | 34.9 score on a scale | Standard Deviation 16.4 |
Primary
Movement Disorder Society Unified Parkinson's Disease Rating Scale (MDS-UPDRS) Parts I-III Sum Score at Month 2
MDS-UPDRS is a multimodal scale. Part I assessed non-motor experiences of daily living and has 2 components (0-52 possible points). Part IA contains 6 questions and is assessed by the examiner (0-24 possible points). Part IB contains 7 questions on non-motor experiences of daily living completed by participant (0-28 possible points). Part II assessed motor experiences of daily living (0-52 possible points) and includes 13 questions completed by participant. Part III assessed motor signs of PD and was administered by rater (0-52 possible points). Part III contained 33 questions based on 18 items. For all questions of each part, numeric score response options linked to accepted clinical terms: 0 to 4, 0=Normal, 1=Slight, 2=Mild, 3=Moderate, 4=Severe. Total Score equals sum of Parts I, II, and III (Score: 0-236). Higher score = more severe PD symptoms.
Time frame: Month 2
Population: FAS included all randomized study participants who received at least a partial dose of study medication, have at least 1 post-Baseline assessment. Here, number of participants analyzed included all participants who were evaluable for this assessment.
| Arm | Measure | Value (MEAN) | Dispersion |
|---|---|---|---|
| Placebo | Movement Disorder Society Unified Parkinson's Disease Rating Scale (MDS-UPDRS) Parts I-III Sum Score at Month 2 | 34.7 score on a scale | Standard Deviation 13.9 |
| UCB0599 180 mg/Day | Movement Disorder Society Unified Parkinson's Disease Rating Scale (MDS-UPDRS) Parts I-III Sum Score at Month 2 | 34.8 score on a scale | Standard Deviation 18 |
| UCB0599 360 mg/Day | Movement Disorder Society Unified Parkinson's Disease Rating Scale (MDS-UPDRS) Parts I-III Sum Score at Month 2 | 30.3 score on a scale | Standard Deviation 14.6 |
Primary
Movement Disorder Society Unified Parkinson's Disease Rating Scale (MDS-UPDRS) Parts I-III Sum Score at Month 4
MDS-UPDRS is a multimodal scale. Part I assessed non-motor experiences of daily living and has 2 components (0-52 possible points). Part IA contains 6 questions and is assessed by the examiner (0-24 possible points). Part IB contains 7 questions on non-motor experiences of daily living completed by participant (0-28 possible points). Part II assessed motor experiences of daily living (0-52 possible points) and includes 13 questions completed by participant. Part III assessed motor signs of PD and was administered by rater (0-52 possible points). Part III contained 33 questions based on 18 items. For all questions of each part, numeric score response options linked to accepted clinical terms: 0 to 4, 0=Normal, 1=Slight, 2=Mild, 3=Moderate, 4=Severe. Total Score equals sum of Parts I, II, and III (Score: 0-236). Higher score = more severe PD symptoms.
Time frame: Month 4
Population: FAS included all randomized study participants who received at least a partial dose of study medication, have at least 1 post-Baseline assessment. Here, number of participants analyzed included all participants who were evaluable for this assessment.
| Arm | Measure | Value (MEAN) | Dispersion |
|---|---|---|---|
| Placebo | Movement Disorder Society Unified Parkinson's Disease Rating Scale (MDS-UPDRS) Parts I-III Sum Score at Month 4 | 34.7 score on a scale | Standard Deviation 14.9 |
| UCB0599 180 mg/Day | Movement Disorder Society Unified Parkinson's Disease Rating Scale (MDS-UPDRS) Parts I-III Sum Score at Month 4 | 34.3 score on a scale | Standard Deviation 18.6 |
| UCB0599 360 mg/Day | Movement Disorder Society Unified Parkinson's Disease Rating Scale (MDS-UPDRS) Parts I-III Sum Score at Month 4 | 31.4 score on a scale | Standard Deviation 15 |
Primary
Movement Disorder Society Unified Parkinson's Disease Rating Scale (MDS-UPDRS) Parts I-III Sum Score at Month 6
MDS-UPDRS is a multimodal scale. Part I assessed non-motor experiences of daily living and has 2 components (0-52 possible points). Part IA contains 6 questions and is assessed by the examiner (0-24 possible points). Part IB contains 7 questions on non-motor experiences of daily living completed by participant (0-28 possible points). Part II assessed motor experiences of daily living (0-52 possible points) and includes 13 questions completed by participant. Part III assessed motor signs of PD and was administered by rater (0-52 possible points). Part III contained 33 questions based on 18 items. For all questions of each part, numeric score response options linked to accepted clinical terms: 0 to 4, 0=Normal, 1=Slight, 2=Mild, 3=Moderate, 4=Severe. Total Score equals sum of Parts I, II, and III (Score: 0-236). Higher score = more severe PD symptoms.
Time frame: Month 6
Population: FAS included all randomized study participants who received at least a partial dose of study medication, have at least 1 post-Baseline assessment. Here, number of participants analyzed included all participants who were evaluable for this assessment.
| Arm | Measure | Value (MEAN) | Dispersion |
|---|---|---|---|
| Placebo | Movement Disorder Society Unified Parkinson's Disease Rating Scale (MDS-UPDRS) Parts I-III Sum Score at Month 6 | 37.9 score on a scale | Standard Deviation 16 |
| UCB0599 180 mg/Day | Movement Disorder Society Unified Parkinson's Disease Rating Scale (MDS-UPDRS) Parts I-III Sum Score at Month 6 | 36.6 score on a scale | Standard Deviation 20 |
| UCB0599 360 mg/Day | Movement Disorder Society Unified Parkinson's Disease Rating Scale (MDS-UPDRS) Parts I-III Sum Score at Month 6 | 32.9 score on a scale | Standard Deviation 15.7 |
Primary
Movement Disorder Society Unified Parkinson's Disease Rating Scale (MDS-UPDRS) Parts I-III Sum Score at Month 8
MDS-UPDRS is a multimodal scale. Part I assessed non-motor experiences of daily living and has 2 components (0-52 possible points). Part IA contains 6 questions and is assessed by the examiner (0-24 possible points). Part IB contains 7 questions on non-motor experiences of daily living completed by participant (0-28 possible points). Part II assessed motor experiences of daily living (0-52 possible points) and includes 13 questions completed by participant. Part III assessed motor signs of PD and was administered by rater (0-52 possible points). Part III contained 33 questions based on 18 items. For all questions of each part, numeric score response options linked to accepted clinical terms: 0 to 4, 0=Normal, 1=Slight, 2=Mild, 3=Moderate, 4=Severe. Total Score equals sum of Parts I, II, and III (Score: 0-236). Higher score = more severe PD symptoms.
Time frame: Month 8
Population: FAS included all randomized study participants who received at least a partial dose of study medication, have at least 1 post-Baseline assessment. Here, number of participants analyzed included all participants who were evaluable for this assessment.
| Arm | Measure | Value (MEAN) | Dispersion |
|---|---|---|---|
| Placebo | Movement Disorder Society Unified Parkinson's Disease Rating Scale (MDS-UPDRS) Parts I-III Sum Score at Month 8 | 35.6 score on a scale | Standard Deviation 14.3 |
| UCB0599 180 mg/Day | Movement Disorder Society Unified Parkinson's Disease Rating Scale (MDS-UPDRS) Parts I-III Sum Score at Month 8 | 34.6 score on a scale | Standard Deviation 19.7 |
| UCB0599 360 mg/Day | Movement Disorder Society Unified Parkinson's Disease Rating Scale (MDS-UPDRS) Parts I-III Sum Score at Month 8 | 32.9 score on a scale | Standard Deviation 15.5 |
Secondary
Change From Screening in Dopamine Transporter Imaging With Single Photon Emission Computed Tomography (DaT-SPECT) Mean Striatum Specific Binding Ratios (SBR)
The change from screening in mean striatum specific binding ratios (SBR) was assessed by DaT-SPECT using 123I-Ioflupane as radiopharmaceutical. The whole striatum was calculated as the average of the SBR data values for the four following small regions: left caudate small, left putamen small, right caudate small and right putamen small. The SBR was calculated for each region with the occipital cortex as a reference region, where a lower SBR indicates worse disease. The following formula was used to calculate this: (Average \[Small region\] - Average \[Occipital region\])/ (Average \[Occipital region\]).
Time frame: Screening, Months 12 and 18
Population: FAS included all randomized study participants who received at least a partial dose of study medication, have at least 1 post-Baseline assessment. Here, number of participants analyzed included all participants who were evaluable for this assessment and 'n' signifies participants who were evaluable at each specified timepoints.
| Arm | Measure | Group | Value (MEAN) | Dispersion |
|---|---|---|---|---|
| Placebo | Change From Screening in Dopamine Transporter Imaging With Single Photon Emission Computed Tomography (DaT-SPECT) Mean Striatum Specific Binding Ratios (SBR) | Contralateral caudate small: Month 12 | -0.256 specific binding ratio | Standard Deviation 0.328 |
| Placebo | Change From Screening in Dopamine Transporter Imaging With Single Photon Emission Computed Tomography (DaT-SPECT) Mean Striatum Specific Binding Ratios (SBR) | Contralateral striatum: Month 18 | -0.203 specific binding ratio | Standard Deviation 0.214 |
| Placebo | Change From Screening in Dopamine Transporter Imaging With Single Photon Emission Computed Tomography (DaT-SPECT) Mean Striatum Specific Binding Ratios (SBR) | Contralateral putamen small: Month 12 | -0.066 specific binding ratio | Standard Deviation 0.19 |
| Placebo | Change From Screening in Dopamine Transporter Imaging With Single Photon Emission Computed Tomography (DaT-SPECT) Mean Striatum Specific Binding Ratios (SBR) | Ipsilateral putamen small: Month 18 | -0.208 specific binding ratio | Standard Deviation 0.252 |
| Placebo | Change From Screening in Dopamine Transporter Imaging With Single Photon Emission Computed Tomography (DaT-SPECT) Mean Striatum Specific Binding Ratios (SBR) | Ipsilateral caudate small: Month 12 | -0.296 specific binding ratio | Standard Deviation 0.38 |
| Placebo | Change From Screening in Dopamine Transporter Imaging With Single Photon Emission Computed Tomography (DaT-SPECT) Mean Striatum Specific Binding Ratios (SBR) | Ipsilateral striatum: Month 12 | -0.237 specific binding ratio | Standard Deviation 0.262 |
| Placebo | Change From Screening in Dopamine Transporter Imaging With Single Photon Emission Computed Tomography (DaT-SPECT) Mean Striatum Specific Binding Ratios (SBR) | Ipsilateral putamen small: Month 12 | -0.178 specific binding ratio | Standard Deviation 0.26 |
| Placebo | Change From Screening in Dopamine Transporter Imaging With Single Photon Emission Computed Tomography (DaT-SPECT) Mean Striatum Specific Binding Ratios (SBR) | Ipsilateral caudate small: Month 18 | -0.312 specific binding ratio | Standard Deviation 0.383 |
| Placebo | Change From Screening in Dopamine Transporter Imaging With Single Photon Emission Computed Tomography (DaT-SPECT) Mean Striatum Specific Binding Ratios (SBR) | Contralateral putamen small: Month 18 | -0.115 specific binding ratio | Standard Deviation 0.19 |
| Placebo | Change From Screening in Dopamine Transporter Imaging With Single Photon Emission Computed Tomography (DaT-SPECT) Mean Striatum Specific Binding Ratios (SBR) | Contralateral caudate small: Month 18 | -0.291 specific binding ratio | Standard Deviation 0.33 |
| Placebo | Change From Screening in Dopamine Transporter Imaging With Single Photon Emission Computed Tomography (DaT-SPECT) Mean Striatum Specific Binding Ratios (SBR) | Ipsilateral striatum: Month 18 | -0.260 specific binding ratio | Standard Deviation 0.263 |
| Placebo | Change From Screening in Dopamine Transporter Imaging With Single Photon Emission Computed Tomography (DaT-SPECT) Mean Striatum Specific Binding Ratios (SBR) | Whole striatum: Month 18 | -0.233 specific binding ratio | Standard Deviation 0.226 |
| Placebo | Change From Screening in Dopamine Transporter Imaging With Single Photon Emission Computed Tomography (DaT-SPECT) Mean Striatum Specific Binding Ratios (SBR) | Contralateral striatum: Month 12 | -0.161 specific binding ratio | Standard Deviation 0.21 |
| Placebo | Change From Screening in Dopamine Transporter Imaging With Single Photon Emission Computed Tomography (DaT-SPECT) Mean Striatum Specific Binding Ratios (SBR) | Whole striatum: Month 12 | -0.202 specific binding ratio | Standard Deviation 0.215 |
| UCB0599 180 mg/Day | Change From Screening in Dopamine Transporter Imaging With Single Photon Emission Computed Tomography (DaT-SPECT) Mean Striatum Specific Binding Ratios (SBR) | Contralateral caudate small: Month 12 | -0.183 specific binding ratio | Standard Deviation 0.332 |
| UCB0599 180 mg/Day | Change From Screening in Dopamine Transporter Imaging With Single Photon Emission Computed Tomography (DaT-SPECT) Mean Striatum Specific Binding Ratios (SBR) | Whole striatum: Month 12 | -0.141 specific binding ratio | Standard Deviation 0.194 |
| UCB0599 180 mg/Day | Change From Screening in Dopamine Transporter Imaging With Single Photon Emission Computed Tomography (DaT-SPECT) Mean Striatum Specific Binding Ratios (SBR) | Whole striatum: Month 18 | -0.182 specific binding ratio | Standard Deviation 0.232 |
| UCB0599 180 mg/Day | Change From Screening in Dopamine Transporter Imaging With Single Photon Emission Computed Tomography (DaT-SPECT) Mean Striatum Specific Binding Ratios (SBR) | Ipsilateral striatum: Month 12 | -0.161 specific binding ratio | Standard Deviation 0.246 |
| UCB0599 180 mg/Day | Change From Screening in Dopamine Transporter Imaging With Single Photon Emission Computed Tomography (DaT-SPECT) Mean Striatum Specific Binding Ratios (SBR) | Ipsilateral striatum: Month 18 | -0.211 specific binding ratio | Standard Deviation 0.282 |
| UCB0599 180 mg/Day | Change From Screening in Dopamine Transporter Imaging With Single Photon Emission Computed Tomography (DaT-SPECT) Mean Striatum Specific Binding Ratios (SBR) | Contralateral striatum: Month 12 | -0.138 specific binding ratio | Standard Deviation 0.209 |
| UCB0599 180 mg/Day | Change From Screening in Dopamine Transporter Imaging With Single Photon Emission Computed Tomography (DaT-SPECT) Mean Striatum Specific Binding Ratios (SBR) | Contralateral striatum: Month 18 | -0.169 specific binding ratio | Standard Deviation 0.237 |
| UCB0599 180 mg/Day | Change From Screening in Dopamine Transporter Imaging With Single Photon Emission Computed Tomography (DaT-SPECT) Mean Striatum Specific Binding Ratios (SBR) | Ipsilateral caudate small: Month 12 | -0.196 specific binding ratio | Standard Deviation 0.352 |
| UCB0599 180 mg/Day | Change From Screening in Dopamine Transporter Imaging With Single Photon Emission Computed Tomography (DaT-SPECT) Mean Striatum Specific Binding Ratios (SBR) | Ipsilateral caudate small: Month 18 | -0.273 specific binding ratio | Standard Deviation 0.39 |
| UCB0599 180 mg/Day | Change From Screening in Dopamine Transporter Imaging With Single Photon Emission Computed Tomography (DaT-SPECT) Mean Striatum Specific Binding Ratios (SBR) | Ipsilateral putamen small: Month 12 | -0.125 specific binding ratio | Standard Deviation 0.255 |
| UCB0599 180 mg/Day | Change From Screening in Dopamine Transporter Imaging With Single Photon Emission Computed Tomography (DaT-SPECT) Mean Striatum Specific Binding Ratios (SBR) | Ipsilateral putamen small: Month 18 | -0.150 specific binding ratio | Standard Deviation 0.26 |
| UCB0599 180 mg/Day | Change From Screening in Dopamine Transporter Imaging With Single Photon Emission Computed Tomography (DaT-SPECT) Mean Striatum Specific Binding Ratios (SBR) | Contralateral caudate small: Month 18 | -0.263 specific binding ratio | Standard Deviation 0.37 |
| UCB0599 180 mg/Day | Change From Screening in Dopamine Transporter Imaging With Single Photon Emission Computed Tomography (DaT-SPECT) Mean Striatum Specific Binding Ratios (SBR) | Contralateral putamen small: Month 12 | -0.094 specific binding ratio | Standard Deviation 0.223 |
| UCB0599 180 mg/Day | Change From Screening in Dopamine Transporter Imaging With Single Photon Emission Computed Tomography (DaT-SPECT) Mean Striatum Specific Binding Ratios (SBR) | Contralateral putamen small: Month 18 | -0.076 specific binding ratio | Standard Deviation 0.197 |
| UCB0599 360 mg/Day | Change From Screening in Dopamine Transporter Imaging With Single Photon Emission Computed Tomography (DaT-SPECT) Mean Striatum Specific Binding Ratios (SBR) | Contralateral putamen small: Month 12 | -0.053 specific binding ratio | Standard Deviation 0.304 |
| UCB0599 360 mg/Day | Change From Screening in Dopamine Transporter Imaging With Single Photon Emission Computed Tomography (DaT-SPECT) Mean Striatum Specific Binding Ratios (SBR) | Ipsilateral putamen small: Month 18 | -0.179 specific binding ratio | Standard Deviation 0.27 |
| UCB0599 360 mg/Day | Change From Screening in Dopamine Transporter Imaging With Single Photon Emission Computed Tomography (DaT-SPECT) Mean Striatum Specific Binding Ratios (SBR) | Contralateral striatum: Month 12 | -0.099 specific binding ratio | Standard Deviation 0.278 |
| UCB0599 360 mg/Day | Change From Screening in Dopamine Transporter Imaging With Single Photon Emission Computed Tomography (DaT-SPECT) Mean Striatum Specific Binding Ratios (SBR) | Ipsilateral striatum: Month 18 | -0.194 specific binding ratio | Standard Deviation 0.318 |
| UCB0599 360 mg/Day | Change From Screening in Dopamine Transporter Imaging With Single Photon Emission Computed Tomography (DaT-SPECT) Mean Striatum Specific Binding Ratios (SBR) | Contralateral caudate small: Month 12 | -0.145 specific binding ratio | Standard Deviation 0.363 |
| UCB0599 360 mg/Day | Change From Screening in Dopamine Transporter Imaging With Single Photon Emission Computed Tomography (DaT-SPECT) Mean Striatum Specific Binding Ratios (SBR) | Ipsilateral striatum: Month 12 | -0.093 specific binding ratio | Standard Deviation 0.294 |
| UCB0599 360 mg/Day | Change From Screening in Dopamine Transporter Imaging With Single Photon Emission Computed Tomography (DaT-SPECT) Mean Striatum Specific Binding Ratios (SBR) | Whole striatum: Month 12 | -0.102 specific binding ratio | Standard Deviation 0.254 |
| UCB0599 360 mg/Day | Change From Screening in Dopamine Transporter Imaging With Single Photon Emission Computed Tomography (DaT-SPECT) Mean Striatum Specific Binding Ratios (SBR) | Contralateral caudate small: Month 18 | -0.248 specific binding ratio | Standard Deviation 0.398 |
| UCB0599 360 mg/Day | Change From Screening in Dopamine Transporter Imaging With Single Photon Emission Computed Tomography (DaT-SPECT) Mean Striatum Specific Binding Ratios (SBR) | Whole striatum: Month 18 | -0.193 specific binding ratio | Standard Deviation 0.254 |
| UCB0599 360 mg/Day | Change From Screening in Dopamine Transporter Imaging With Single Photon Emission Computed Tomography (DaT-SPECT) Mean Striatum Specific Binding Ratios (SBR) | Ipsilateral caudate small: Month 18 | -0.210 specific binding ratio | Standard Deviation 0.439 |
| UCB0599 360 mg/Day | Change From Screening in Dopamine Transporter Imaging With Single Photon Emission Computed Tomography (DaT-SPECT) Mean Striatum Specific Binding Ratios (SBR) | Ipsilateral caudate small: Month 12 | -0.080 specific binding ratio | Standard Deviation 0.485 |
| UCB0599 360 mg/Day | Change From Screening in Dopamine Transporter Imaging With Single Photon Emission Computed Tomography (DaT-SPECT) Mean Striatum Specific Binding Ratios (SBR) | Contralateral putamen small: Month 18 | -0.128 specific binding ratio | Standard Deviation 0.209 |
| UCB0599 360 mg/Day | Change From Screening in Dopamine Transporter Imaging With Single Photon Emission Computed Tomography (DaT-SPECT) Mean Striatum Specific Binding Ratios (SBR) | Ipsilateral putamen small: Month 12 | -0.106 specific binding ratio | Standard Deviation 0.219 |
| UCB0599 360 mg/Day | Change From Screening in Dopamine Transporter Imaging With Single Photon Emission Computed Tomography (DaT-SPECT) Mean Striatum Specific Binding Ratios (SBR) | Contralateral striatum: Month 18 | -0.188 specific binding ratio | Standard Deviation 0.259 |
Secondary
Emerging Symptoms in Participants as Measured by MDS-UPDRS Part II
The participant was considered to have an emerging symptom for the item, if the change from Baseline for the item is greater than 0 for 2 consecutive visits. The magnitude of change from Baseline was not considered to determine the emerging symptom. Movement Disorder Society Unified Parkinson's Disease Rating Scale (MDS-UPDRS) part II includes motor items assessing speech, saliva and drooling, chewing and swallowing, eating tasks (cutting food and handling utensils), dressing, hygiene, handwriting, doing hobbies and other activities, turning in bed, tremor, getting out of bed, a car or a deep chair, walking and balance, and freezing. This included 13-items, each anchored with 5 responses: 0 = normal, 1 = slight, 2 = mild, 3 = moderate, and 4 = severe. The scale range for Part II was 0-52, with higher scores reflecting greater severity.
Time frame: Baseline to Month 18
Population: FAS included all randomized study participants who received at least a partial dose of study medication, have at least 1 post-Baseline assessment.
| Arm | Measure | Group | Value (COUNT_OF_PARTICIPANTS) |
|---|---|---|---|
| Placebo | Emerging Symptoms in Participants as Measured by MDS-UPDRS Part II | Number of Emerging symptoms: 10 | 1 Participants |
| Placebo | Emerging Symptoms in Participants as Measured by MDS-UPDRS Part II | Number of Emerging symptoms: 5 | 15 Participants |
| Placebo | Emerging Symptoms in Participants as Measured by MDS-UPDRS Part II | Number of Emerging symptoms: 2 | 35 Participants |
| Placebo | Emerging Symptoms in Participants as Measured by MDS-UPDRS Part II | Number of Emerging symptoms: 9 | 2 Participants |
| Placebo | Emerging Symptoms in Participants as Measured by MDS-UPDRS Part II | Number of Emerging symptoms: 6 | 4 Participants |
| Placebo | Emerging Symptoms in Participants as Measured by MDS-UPDRS Part II | Number of Emerging symptoms: 1 | 18 Participants |
| Placebo | Emerging Symptoms in Participants as Measured by MDS-UPDRS Part II | Number of Emerging symptoms: 8 | 4 Participants |
| Placebo | Emerging Symptoms in Participants as Measured by MDS-UPDRS Part II | Number of Emerging symptoms: 7 | 4 Participants |
| Placebo | Emerging Symptoms in Participants as Measured by MDS-UPDRS Part II | Number of Emerging symptoms: 11 | 0 Participants |
| Placebo | Emerging Symptoms in Participants as Measured by MDS-UPDRS Part II | Number of Emerging symptoms: 3 | 37 Participants |
| Placebo | Emerging Symptoms in Participants as Measured by MDS-UPDRS Part II | Number of Emerging symptoms: 13 | 0 Participants |
| Placebo | Emerging Symptoms in Participants as Measured by MDS-UPDRS Part II | Number of Emerging symptoms: 0 | 28 Participants |
| Placebo | Emerging Symptoms in Participants as Measured by MDS-UPDRS Part II | Number of Emerging symptoms: 4 | 15 Participants |
| Placebo | Emerging Symptoms in Participants as Measured by MDS-UPDRS Part II | Number of Emerging symptoms: 12 | 0 Participants |
| UCB0599 180 mg/Day | Emerging Symptoms in Participants as Measured by MDS-UPDRS Part II | Number of Emerging symptoms: 10 | 0 Participants |
| UCB0599 180 mg/Day | Emerging Symptoms in Participants as Measured by MDS-UPDRS Part II | Number of Emerging symptoms: 0 | 36 Participants |
| UCB0599 180 mg/Day | Emerging Symptoms in Participants as Measured by MDS-UPDRS Part II | Number of Emerging symptoms: 1 | 41 Participants |
| UCB0599 180 mg/Day | Emerging Symptoms in Participants as Measured by MDS-UPDRS Part II | Number of Emerging symptoms: 2 | 33 Participants |
| UCB0599 180 mg/Day | Emerging Symptoms in Participants as Measured by MDS-UPDRS Part II | Number of Emerging symptoms: 3 | 15 Participants |
| UCB0599 180 mg/Day | Emerging Symptoms in Participants as Measured by MDS-UPDRS Part II | Number of Emerging symptoms: 4 | 13 Participants |
| UCB0599 180 mg/Day | Emerging Symptoms in Participants as Measured by MDS-UPDRS Part II | Number of Emerging symptoms: 5 | 13 Participants |
| UCB0599 180 mg/Day | Emerging Symptoms in Participants as Measured by MDS-UPDRS Part II | Number of Emerging symptoms: 6 | 7 Participants |
| UCB0599 180 mg/Day | Emerging Symptoms in Participants as Measured by MDS-UPDRS Part II | Number of Emerging symptoms: 7 | 4 Participants |
| UCB0599 180 mg/Day | Emerging Symptoms in Participants as Measured by MDS-UPDRS Part II | Number of Emerging symptoms: 8 | 1 Participants |
| UCB0599 180 mg/Day | Emerging Symptoms in Participants as Measured by MDS-UPDRS Part II | Number of Emerging symptoms: 9 | 1 Participants |
| UCB0599 180 mg/Day | Emerging Symptoms in Participants as Measured by MDS-UPDRS Part II | Number of Emerging symptoms: 11 | 0 Participants |
| UCB0599 180 mg/Day | Emerging Symptoms in Participants as Measured by MDS-UPDRS Part II | Number of Emerging symptoms: 12 | 0 Participants |
| UCB0599 180 mg/Day | Emerging Symptoms in Participants as Measured by MDS-UPDRS Part II | Number of Emerging symptoms: 13 | 0 Participants |
| UCB0599 360 mg/Day | Emerging Symptoms in Participants as Measured by MDS-UPDRS Part II | Number of Emerging symptoms: 12 | 0 Participants |
| UCB0599 360 mg/Day | Emerging Symptoms in Participants as Measured by MDS-UPDRS Part II | Number of Emerging symptoms: 9 | 0 Participants |
| UCB0599 360 mg/Day | Emerging Symptoms in Participants as Measured by MDS-UPDRS Part II | Number of Emerging symptoms: 4 | 15 Participants |
| UCB0599 360 mg/Day | Emerging Symptoms in Participants as Measured by MDS-UPDRS Part II | Number of Emerging symptoms: 3 | 26 Participants |
| UCB0599 360 mg/Day | Emerging Symptoms in Participants as Measured by MDS-UPDRS Part II | Number of Emerging symptoms: 10 | 0 Participants |
| UCB0599 360 mg/Day | Emerging Symptoms in Participants as Measured by MDS-UPDRS Part II | Number of Emerging symptoms: 2 | 23 Participants |
| UCB0599 360 mg/Day | Emerging Symptoms in Participants as Measured by MDS-UPDRS Part II | Number of Emerging symptoms: 0 | 38 Participants |
| UCB0599 360 mg/Day | Emerging Symptoms in Participants as Measured by MDS-UPDRS Part II | Number of Emerging symptoms: 11 | 0 Participants |
| UCB0599 360 mg/Day | Emerging Symptoms in Participants as Measured by MDS-UPDRS Part II | Number of Emerging symptoms: 1 | 31 Participants |
| UCB0599 360 mg/Day | Emerging Symptoms in Participants as Measured by MDS-UPDRS Part II | Number of Emerging symptoms: 7 | 4 Participants |
| UCB0599 360 mg/Day | Emerging Symptoms in Participants as Measured by MDS-UPDRS Part II | Number of Emerging symptoms: 6 | 10 Participants |
| UCB0599 360 mg/Day | Emerging Symptoms in Participants as Measured by MDS-UPDRS Part II | Number of Emerging symptoms: 13 | 0 Participants |
| UCB0599 360 mg/Day | Emerging Symptoms in Participants as Measured by MDS-UPDRS Part II | Number of Emerging symptoms: 8 | 3 Participants |
| UCB0599 360 mg/Day | Emerging Symptoms in Participants as Measured by MDS-UPDRS Part II | Number of Emerging symptoms: 5 | 14 Participants |
Secondary
MDS-UPDRS Part III Early-stage Parkinson's Disease (ePD) Subscore on Selected Items
The early-stage Parkinson's disease (ePD) subscore is derived from a 15-item subset of the MDS-UPDRS Part III (Motor Examination). It includes all rigidity assessments (neck, upper limbs \[right/left\], and lower limbs \[right/left\]) and bradykinesia-related tasks: finger tapping (right/left), hand movements (right/left), pronation-supination of hands (right/left), toe tapping (right/left), and leg agility (right/left). Each item is scored on a 5-point likert scale (0 = no problem to 4 = severe), resulting in a total ePD subscore range of 0 to 60. Higher scores indicate greater motor impairment, while lower scores reflect better motor function.
Time frame: Day 0, Months 2, 4, 6, 8, 10, 12, 14, 16, 18
Population: FAS included all randomized study participants who received at least a partial dose of study medication, have at least 1 post-Baseline assessment. Here, number of participants analyzed included all participants who were evaluable for this assessment and 'n' signifies participants who were evaluable at specified timepoints.
| Arm | Measure | Group | Value (MEAN) | Dispersion |
|---|---|---|---|---|
| Placebo | MDS-UPDRS Part III Early-stage Parkinson's Disease (ePD) Subscore on Selected Items | Month 12 | 13.7 score on a scale | Standard Deviation 6.98 |
| Placebo | MDS-UPDRS Part III Early-stage Parkinson's Disease (ePD) Subscore on Selected Items | Month 6 | 13.9 score on a scale | Standard Deviation 7.39 |
| Placebo | MDS-UPDRS Part III Early-stage Parkinson's Disease (ePD) Subscore on Selected Items | Month 2 | 13.2 score on a scale | Standard Deviation 7.33 |
| Placebo | MDS-UPDRS Part III Early-stage Parkinson's Disease (ePD) Subscore on Selected Items | Month 10 | 13.4 score on a scale | Standard Deviation 6.46 |
| Placebo | MDS-UPDRS Part III Early-stage Parkinson's Disease (ePD) Subscore on Selected Items | Month 8 | 13.4 score on a scale | Standard Deviation 6.82 |
| Placebo | MDS-UPDRS Part III Early-stage Parkinson's Disease (ePD) Subscore on Selected Items | Day 0 | 13.3 score on a scale | Standard Deviation 7.11 |
| Placebo | MDS-UPDRS Part III Early-stage Parkinson's Disease (ePD) Subscore on Selected Items | Month 18 | 13.8 score on a scale | Standard Deviation 7.34 |
| Placebo | MDS-UPDRS Part III Early-stage Parkinson's Disease (ePD) Subscore on Selected Items | Month 14 | 13.8 score on a scale | Standard Deviation 7.14 |
| Placebo | MDS-UPDRS Part III Early-stage Parkinson's Disease (ePD) Subscore on Selected Items | Month 4 | 13.4 score on a scale | Standard Deviation 7.5 |
| Placebo | MDS-UPDRS Part III Early-stage Parkinson's Disease (ePD) Subscore on Selected Items | Month 16 | 13.8 score on a scale | Standard Deviation 7.24 |
| UCB0599 180 mg/Day | MDS-UPDRS Part III Early-stage Parkinson's Disease (ePD) Subscore on Selected Items | Month 8 | 12.7 score on a scale | Standard Deviation 8.6 |
| UCB0599 180 mg/Day | MDS-UPDRS Part III Early-stage Parkinson's Disease (ePD) Subscore on Selected Items | Month 16 | 12.9 score on a scale | Standard Deviation 8.26 |
| UCB0599 180 mg/Day | MDS-UPDRS Part III Early-stage Parkinson's Disease (ePD) Subscore on Selected Items | Day 0 | 12.6 score on a scale | Standard Deviation 7.96 |
| UCB0599 180 mg/Day | MDS-UPDRS Part III Early-stage Parkinson's Disease (ePD) Subscore on Selected Items | Month 2 | 13.2 score on a scale | Standard Deviation 8.48 |
| UCB0599 180 mg/Day | MDS-UPDRS Part III Early-stage Parkinson's Disease (ePD) Subscore on Selected Items | Month 4 | 13.1 score on a scale | Standard Deviation 8.76 |
| UCB0599 180 mg/Day | MDS-UPDRS Part III Early-stage Parkinson's Disease (ePD) Subscore on Selected Items | Month 6 | 13.4 score on a scale | Standard Deviation 8.42 |
| UCB0599 180 mg/Day | MDS-UPDRS Part III Early-stage Parkinson's Disease (ePD) Subscore on Selected Items | Month 10 | 12.1 score on a scale | Standard Deviation 8.14 |
| UCB0599 180 mg/Day | MDS-UPDRS Part III Early-stage Parkinson's Disease (ePD) Subscore on Selected Items | Month 12 | 12.7 score on a scale | Standard Deviation 8.2 |
| UCB0599 180 mg/Day | MDS-UPDRS Part III Early-stage Parkinson's Disease (ePD) Subscore on Selected Items | Month 14 | 12.7 score on a scale | Standard Deviation 8.35 |
| UCB0599 180 mg/Day | MDS-UPDRS Part III Early-stage Parkinson's Disease (ePD) Subscore on Selected Items | Month 18 | 12.9 score on a scale | Standard Deviation 7.9 |
| UCB0599 360 mg/Day | MDS-UPDRS Part III Early-stage Parkinson's Disease (ePD) Subscore on Selected Items | Month 4 | 11.8 score on a scale | Standard Deviation 7.64 |
| UCB0599 360 mg/Day | MDS-UPDRS Part III Early-stage Parkinson's Disease (ePD) Subscore on Selected Items | Month 16 | 12.4 score on a scale | Standard Deviation 7.83 |
| UCB0599 360 mg/Day | MDS-UPDRS Part III Early-stage Parkinson's Disease (ePD) Subscore on Selected Items | Month 12 | 12.9 score on a scale | Standard Deviation 7.61 |
| UCB0599 360 mg/Day | MDS-UPDRS Part III Early-stage Parkinson's Disease (ePD) Subscore on Selected Items | Month 2 | 11.6 score on a scale | Standard Deviation 7.57 |
| UCB0599 360 mg/Day | MDS-UPDRS Part III Early-stage Parkinson's Disease (ePD) Subscore on Selected Items | Month 18 | 12.6 score on a scale | Standard Deviation 7.61 |
| UCB0599 360 mg/Day | MDS-UPDRS Part III Early-stage Parkinson's Disease (ePD) Subscore on Selected Items | Month 14 | 12.5 score on a scale | Standard Deviation 7.88 |
| UCB0599 360 mg/Day | MDS-UPDRS Part III Early-stage Parkinson's Disease (ePD) Subscore on Selected Items | Month 8 | 11.8 score on a scale | Standard Deviation 7.49 |
| UCB0599 360 mg/Day | MDS-UPDRS Part III Early-stage Parkinson's Disease (ePD) Subscore on Selected Items | Month 6 | 12.4 score on a scale | Standard Deviation 7.58 |
| UCB0599 360 mg/Day | MDS-UPDRS Part III Early-stage Parkinson's Disease (ePD) Subscore on Selected Items | Day 0 | 11.8 score on a scale | Standard Deviation 6.85 |
| UCB0599 360 mg/Day | MDS-UPDRS Part III Early-stage Parkinson's Disease (ePD) Subscore on Selected Items | Month 10 | 11.9 score on a scale | Standard Deviation 7.98 |
Secondary
MDS-UPDRS Part III Subscale
MDS-UPDRS part III includes motor items assessing speech, facial expression, rigidity, finger tapping, hand movements, pronation supination movements of hands, toe tapping, leg agility, arising from chair, gait, freezing of gait, postural stability, posture, global spontaneity of movement (body bradykinesia), postural tremor of the hands, kinetic tremor of the hands, rest tremor amplitude, and constancy of rest tremor. It included 33 scores based on 18-items, each anchored with 5 responses: 0 = normal, 1 = slight, 2 = mild, 3 = moderate, and 4 = severe. The scale range was from 0 to 132, with a lower score indicating better motor function and a higher score indicating more severe motor symptoms.
Time frame: Day 0, Months 2, 4, 6, 8, 10, 12, 14, 16, 18
Population: FAS included all randomized study participants who received at least a partial dose of study medication, have at least 1 post-Baseline assessment. Here, number of participants analyzed included all participants who were evaluable for this assessment and number analyzed (n) signifies participants who were evaluable at specified timepoints.
| Arm | Measure | Group | Value (MEAN) | Dispersion |
|---|---|---|---|---|
| Placebo | MDS-UPDRS Part III Subscale | Month 6 | 24.3 score on a scale | Standard Deviation 10.9 |
| Placebo | MDS-UPDRS Part III Subscale | Month 14 | 24.1 score on a scale | Standard Deviation 10.1 |
| Placebo | MDS-UPDRS Part III Subscale | Month 8 | 23.3 score on a scale | Standard Deviation 10 |
| Placebo | MDS-UPDRS Part III Subscale | Month 4 | 23.1 score on a scale | Standard Deviation 10.5 |
| Placebo | MDS-UPDRS Part III Subscale | Month 2 | 22.9 score on a scale | Standard Deviation 10.2 |
| Placebo | MDS-UPDRS Part III Subscale | Month 10 | 23.3 score on a scale | Standard Deviation 9.2 |
| Placebo | MDS-UPDRS Part III Subscale | Month 18 | 24.2 score on a scale | Standard Deviation 10.1 |
| Placebo | MDS-UPDRS Part III Subscale | Month 16 | 24.3 score on a scale | Standard Deviation 10.1 |
| Placebo | MDS-UPDRS Part III Subscale | Month 12 | 24.1 score on a scale | Standard Deviation 10 |
| Placebo | MDS-UPDRS Part III Subscale | Day 0 | 22.6 score on a scale | Standard Deviation 9.8 |
| UCB0599 180 mg/Day | MDS-UPDRS Part III Subscale | Month 12 | 22.8 score on a scale | Standard Deviation 12 |
| UCB0599 180 mg/Day | MDS-UPDRS Part III Subscale | Month 14 | 22.2 score on a scale | Standard Deviation 11.9 |
| UCB0599 180 mg/Day | MDS-UPDRS Part III Subscale | Month 16 | 22.8 score on a scale | Standard Deviation 12.3 |
| UCB0599 180 mg/Day | MDS-UPDRS Part III Subscale | Month 4 | 22.5 score on a scale | Standard Deviation 12.7 |
| UCB0599 180 mg/Day | MDS-UPDRS Part III Subscale | Month 18 | 23.0 score on a scale | Standard Deviation 11.7 |
| UCB0599 180 mg/Day | MDS-UPDRS Part III Subscale | Month 6 | 23.2 score on a scale | Standard Deviation 12.4 |
| UCB0599 180 mg/Day | MDS-UPDRS Part III Subscale | Month 8 | 22.2 score on a scale | Standard Deviation 12.6 |
| UCB0599 180 mg/Day | MDS-UPDRS Part III Subscale | Month 10 | 21.6 score on a scale | Standard Deviation 12.1 |
| UCB0599 180 mg/Day | MDS-UPDRS Part III Subscale | Month 2 | 22.7 score on a scale | Standard Deviation 12.4 |
| UCB0599 180 mg/Day | MDS-UPDRS Part III Subscale | Day 0 | 21.9 score on a scale | Standard Deviation 11.3 |
| UCB0599 360 mg/Day | MDS-UPDRS Part III Subscale | Month 18 | 22.2 score on a scale | Standard Deviation 10.9 |
| UCB0599 360 mg/Day | MDS-UPDRS Part III Subscale | Day 0 | 20.4 score on a scale | Standard Deviation 9.5 |
| UCB0599 360 mg/Day | MDS-UPDRS Part III Subscale | Month 2 | 20.0 score on a scale | Standard Deviation 10.3 |
| UCB0599 360 mg/Day | MDS-UPDRS Part III Subscale | Month 4 | 20.7 score on a scale | Standard Deviation 10.6 |
| UCB0599 360 mg/Day | MDS-UPDRS Part III Subscale | Month 8 | 21.0 score on a scale | Standard Deviation 10.7 |
| UCB0599 360 mg/Day | MDS-UPDRS Part III Subscale | Month 10 | 21.0 score on a scale | Standard Deviation 11 |
| UCB0599 360 mg/Day | MDS-UPDRS Part III Subscale | Month 12 | 21.8 score on a scale | Standard Deviation 10.5 |
| UCB0599 360 mg/Day | MDS-UPDRS Part III Subscale | Month 14 | 21.8 score on a scale | Standard Deviation 11.4 |
| UCB0599 360 mg/Day | MDS-UPDRS Part III Subscale | Month 16 | 21.7 score on a scale | Standard Deviation 10.7 |
| UCB0599 360 mg/Day | MDS-UPDRS Part III Subscale | Month 6 | 21.3 score on a scale | Standard Deviation 10.8 |
Secondary
MDS-UPDRS Part II Subscale
MDS-UPDRS part II includes motor items assessing speech, saliva and drooling, chewing and swallowing, eating tasks (cutting food and handling utensils), dressing, hygiene, handwriting, doing hobbies and other activities, turning in bed, tremor, getting out of bed, a car or a deep chair, walking and balance, and freezing. Each of the items in the UPDRS is measured on a scale. It included 13-items, each anchored with 5 responses: 0 = normal, 1 = slight, 2 = mild, 3 = moderate, and 4 = severe. The scale range for Part II was 0-52, with higher scores reflecting greater severity.
Time frame: Day 0, Months 2, 4, 6, 8, 10, 12, 14, 16, 18
Population: FAS included all randomized study participants who received at least a partial dose of study medication, have at least 1 post-Baseline assessment. Here, number of participants analyzed included all participants who were evaluable for this assessment and 'n' signifies participants who were evaluable at specified timepoints.
| Arm | Measure | Group | Value (MEAN) | Dispersion |
|---|---|---|---|---|
| Placebo | MDS-UPDRS Part II Subscale | Day 0 | 6.0 score on a scale | Standard Deviation 4 |
| Placebo | MDS-UPDRS Part II Subscale | Month 2 | 6.5 score on a scale | Standard Deviation 4.3 |
| Placebo | MDS-UPDRS Part II Subscale | Month 4 | 6.8 score on a scale | Standard Deviation 4.6 |
| Placebo | MDS-UPDRS Part II Subscale | Month 6 | 7.7 score on a scale | Standard Deviation 5.1 |
| Placebo | MDS-UPDRS Part II Subscale | Month 8 | 7.0 score on a scale | Standard Deviation 4.6 |
| Placebo | MDS-UPDRS Part II Subscale | Month 10 | 6.9 score on a scale | Standard Deviation 4.5 |
| Placebo | MDS-UPDRS Part II Subscale | Month 12 | 6.9 score on a scale | Standard Deviation 4.9 |
| Placebo | MDS-UPDRS Part II Subscale | Month 14 | 7.1 score on a scale | Standard Deviation 4.8 |
| Placebo | MDS-UPDRS Part II Subscale | Month 16 | 7.1 score on a scale | Standard Deviation 5.1 |
| Placebo | MDS-UPDRS Part II Subscale | Month 18 | 7.3 score on a scale | Standard Deviation 5 |
| UCB0599 180 mg/Day | MDS-UPDRS Part II Subscale | Month 16 | 7.2 score on a scale | Standard Deviation 5.7 |
| UCB0599 180 mg/Day | MDS-UPDRS Part II Subscale | Day 0 | 6.3 score on a scale | Standard Deviation 4.6 |
| UCB0599 180 mg/Day | MDS-UPDRS Part II Subscale | Month 10 | 6.7 score on a scale | Standard Deviation 5.4 |
| UCB0599 180 mg/Day | MDS-UPDRS Part II Subscale | Month 8 | 7.1 score on a scale | Standard Deviation 5.7 |
| UCB0599 180 mg/Day | MDS-UPDRS Part II Subscale | Month 2 | 6.9 score on a scale | Standard Deviation 5.2 |
| UCB0599 180 mg/Day | MDS-UPDRS Part II Subscale | Month 18 | 6.8 score on a scale | Standard Deviation 5.2 |
| UCB0599 180 mg/Day | MDS-UPDRS Part II Subscale | Month 14 | 7.2 score on a scale | Standard Deviation 5.8 |
| UCB0599 180 mg/Day | MDS-UPDRS Part II Subscale | Month 4 | 7.1 score on a scale | Standard Deviation 5.4 |
| UCB0599 180 mg/Day | MDS-UPDRS Part II Subscale | Month 12 | 7.3 score on a scale | Standard Deviation 5.5 |
| UCB0599 180 mg/Day | MDS-UPDRS Part II Subscale | Month 6 | 7.7 score on a scale | Standard Deviation 6.2 |
| UCB0599 360 mg/Day | MDS-UPDRS Part II Subscale | Month 14 | 7.0 score on a scale | Standard Deviation 5.1 |
| UCB0599 360 mg/Day | MDS-UPDRS Part II Subscale | Month 6 | 6.7 score on a scale | Standard Deviation 5 |
| UCB0599 360 mg/Day | MDS-UPDRS Part II Subscale | Month 8 | 6.9 score on a scale | Standard Deviation 4.7 |
| UCB0599 360 mg/Day | MDS-UPDRS Part II Subscale | Month 10 | 7.1 score on a scale | Standard Deviation 4.7 |
| UCB0599 360 mg/Day | MDS-UPDRS Part II Subscale | Month 16 | 6.7 score on a scale | Standard Deviation 4.5 |
| UCB0599 360 mg/Day | MDS-UPDRS Part II Subscale | Month 12 | 7.3 score on a scale | Standard Deviation 5.1 |
| UCB0599 360 mg/Day | MDS-UPDRS Part II Subscale | Day 0 | 5.7 score on a scale | Standard Deviation 4.3 |
| UCB0599 360 mg/Day | MDS-UPDRS Part II Subscale | Month 18 | 7.3 score on a scale | Standard Deviation 5.3 |
| UCB0599 360 mg/Day | MDS-UPDRS Part II Subscale | Month 2 | 6.1 score on a scale | Standard Deviation 4.1 |
| UCB0599 360 mg/Day | MDS-UPDRS Part II Subscale | Month 4 | 6.4 score on a scale | Standard Deviation 4.5 |
Secondary
MDS-UPDRS Part I Subscale
MDS-UPDRS part I include several non-motor aspects of experiences of daily living including cognitive impairment, hallucinations and psychosis, depressed mood, anxious mood, apathy, features of dopamine dysregulation syndrome during part 1A and sleep problems, daytime sleepiness, pain and other sensation, urinary problems, constipation problems, lightheadedness on standing, and fatigue during Part 1B. Part I assessed non-motor experiences of daily living and has 2 components (Range 0-52). Part IA contained 6 questions and were assessed by examiner (Range 0-24). Part IB contained 7 questions on non-motor experiences of daily living which were completed by participant (Range 0-28). Each of items in UPDRS is measured on a scale of 0 to 4, where 0 is normal and 4 (higher score) represents severe abnormalities/worse outcome. Total Score Range 0 to 52. Total score is sum of Part IA (0-24) and Part IB (0-28) subscale scores. Higher scores indicated greater severity of non-motor symptoms.
Time frame: Day 0, Months 2, 4, 6, 8, 10, 12, 14, 16, 18
Population: FAS included all randomized study participants who received at least a partial dose of study medication, have at least 1 post-Baseline assessment. Here, number of participants analyzed included all participants who were evaluable for this assessment and 'n' signifies participants who were evaluable at specified timepoints.
| Arm | Measure | Group | Value (MEAN) | Dispersion |
|---|---|---|---|---|
| Placebo | MDS-UPDRS Part I Subscale | Day 0 | 5.4 score on a scale | Standard Deviation 3.9 |
| Placebo | MDS-UPDRS Part I Subscale | Month 2 | 5.2 score on a scale | Standard Deviation 4.2 |
| Placebo | MDS-UPDRS Part I Subscale | Month 4 | 5.1 score on a scale | Standard Deviation 4.4 |
| Placebo | MDS-UPDRS Part I Subscale | Month 6 | 5.9 score on a scale | Standard Deviation 4.8 |
| Placebo | MDS-UPDRS Part I Subscale | Month 8 | 5.5 score on a scale | Standard Deviation 4.4 |
| Placebo | MDS-UPDRS Part I Subscale | Month 10 | 5.4 score on a scale | Standard Deviation 4.2 |
| Placebo | MDS-UPDRS Part I Subscale | Month 12 | 6.2 score on a scale | Standard Deviation 5.1 |
| Placebo | MDS-UPDRS Part I Subscale | Month 14 | 5.9 score on a scale | Standard Deviation 4.6 |
| Placebo | MDS-UPDRS Part I Subscale | Month 16 | 5.9 score on a scale | Standard Deviation 4.8 |
| Placebo | MDS-UPDRS Part I Subscale | Month 18 | 6.4 score on a scale | Standard Deviation 5.1 |
| UCB0599 180 mg/Day | MDS-UPDRS Part I Subscale | Month 16 | 5.9 score on a scale | Standard Deviation 5.1 |
| UCB0599 180 mg/Day | MDS-UPDRS Part I Subscale | Day 0 | 5.5 score on a scale | Standard Deviation 4.4 |
| UCB0599 180 mg/Day | MDS-UPDRS Part I Subscale | Month 10 | 5.4 score on a scale | Standard Deviation 4.1 |
| UCB0599 180 mg/Day | MDS-UPDRS Part I Subscale | Month 8 | 5.3 score on a scale | Standard Deviation 4.2 |
| UCB0599 180 mg/Day | MDS-UPDRS Part I Subscale | Month 2 | 5.2 score on a scale | Standard Deviation 3.8 |
| UCB0599 180 mg/Day | MDS-UPDRS Part I Subscale | Month 18 | 5.9 score on a scale | Standard Deviation 4.6 |
| UCB0599 180 mg/Day | MDS-UPDRS Part I Subscale | Month 14 | 5.8 score on a scale | Standard Deviation 5 |
| UCB0599 180 mg/Day | MDS-UPDRS Part I Subscale | Month 4 | 5.0 score on a scale | Standard Deviation 4 |
| UCB0599 180 mg/Day | MDS-UPDRS Part I Subscale | Month 12 | 6.2 score on a scale | Standard Deviation 4.8 |
| UCB0599 180 mg/Day | MDS-UPDRS Part I Subscale | Month 6 | 5.7 score on a scale | Standard Deviation 4.5 |
| UCB0599 360 mg/Day | MDS-UPDRS Part I Subscale | Month 14 | 5.0 score on a scale | Standard Deviation 4.2 |
| UCB0599 360 mg/Day | MDS-UPDRS Part I Subscale | Month 6 | 5.1 score on a scale | Standard Deviation 3.9 |
| UCB0599 360 mg/Day | MDS-UPDRS Part I Subscale | Month 8 | 5.0 score on a scale | Standard Deviation 3.9 |
| UCB0599 360 mg/Day | MDS-UPDRS Part I Subscale | Month 10 | 4.9 score on a scale | Standard Deviation 4.1 |
| UCB0599 360 mg/Day | MDS-UPDRS Part I Subscale | Month 16 | 5.1 score on a scale | Standard Deviation 4.1 |
| UCB0599 360 mg/Day | MDS-UPDRS Part I Subscale | Month 12 | 5.3 score on a scale | Standard Deviation 4.4 |
| UCB0599 360 mg/Day | MDS-UPDRS Part I Subscale | Day 0 | 4.7 score on a scale | Standard Deviation 3.7 |
| UCB0599 360 mg/Day | MDS-UPDRS Part I Subscale | Month 18 | 5.3 score on a scale | Standard Deviation 4.5 |
| UCB0599 360 mg/Day | MDS-UPDRS Part I Subscale | Month 2 | 4.3 score on a scale | Standard Deviation 3.5 |
| UCB0599 360 mg/Day | MDS-UPDRS Part I Subscale | Month 4 | 4.5 score on a scale | Standard Deviation 3.8 |
Secondary
Montreal Cognitive Assessment (MoCA)
The Montreal Cognitive Assessment (MoCA) is a standardized screening tool used to evaluate mild cognitive impairment across multiple cognitive functions i.e. visuospatial/executive function, naming, memory, attention, language, abstraction, delayed recall, and orientation. The total possible score is calculated by summing the scores across all functions ranges from: 0 to 30. A score of 26 or above is considered normal, a lower score indicates cognitive impairment.
Time frame: Screening, Month 18
Population: FAS included all randomized study participants who received at least a partial dose of study medication, have at least 1 post-Baseline assessment. Here, 'n' signifies participants who were evaluable at each specified timepoints.
| Arm | Measure | Group | Value (MEAN) | Dispersion |
|---|---|---|---|---|
| Placebo | Montreal Cognitive Assessment (MoCA) | Screening | 27.8 score on a scale | Standard Deviation 1.8 |
| Placebo | Montreal Cognitive Assessment (MoCA) | Month 18 | 27.4 score on a scale | Standard Deviation 2.2 |
| UCB0599 180 mg/Day | Montreal Cognitive Assessment (MoCA) | Screening | 27.6 score on a scale | Standard Deviation 2 |
| UCB0599 180 mg/Day | Montreal Cognitive Assessment (MoCA) | Month 18 | 27.3 score on a scale | Standard Deviation 2.4 |
| UCB0599 360 mg/Day | Montreal Cognitive Assessment (MoCA) | Screening | 27.8 score on a scale | Standard Deviation 1.7 |
| UCB0599 360 mg/Day | Montreal Cognitive Assessment (MoCA) | Month 18 | 27.3 score on a scale | Standard Deviation 2.4 |
Secondary
Percentage of Participants With Serious TEAEs
Serious adverse event: Death, a life-threatening adverse event, inpatient hospitalization or prolongation of existing hospitalization, a persistent or significant incapacity or substantial disruption of the ability to conduct normal life functions, or a congenital anomaly/birth defect. Important medical events that may not result in death, be life-threatening, or require hospitalization may be considered serious when, based upon appropriate medical judgment, they may jeopardize the patient or subject and may require medical or surgical intervention to prevent one of the outcomes listed in this definition.
Time frame: From Baseline up to 30 days of safety follow-up after the last dose of the study drug (up to 19 months)
Population: Safety set included all randomized study participants who receive at least a partial dose of study medication.
| Arm | Measure | Value (NUMBER) |
|---|---|---|
| Placebo | Percentage of Participants With Serious TEAEs | 5.5 percentage of participants |
| UCB0599 180 mg/Day | Percentage of Participants With Serious TEAEs | 7.9 percentage of participants |
| UCB0599 360 mg/Day | Percentage of Participants With Serious TEAEs | 8.5 percentage of participants |
Secondary
Percentage of Participants With TEAEs Leading to Participant Withdrawal
An AE is any untoward medical occurrence in a patient or clinical study participant, temporally associated with the use of study medication, whether or not considered related to the study medication. A TEAE is defined as any AE with a start date on or after the first dose of treatment or any unresolved event already present before administration of treatment that worsens in intensity following exposure to the treatment.
Time frame: From Baseline up to 30 days of safety follow-up after the last dose of the study drug (up to 19 months)
Population: Safety set included all randomized study participants who receive at least a partial dose of study medication.
| Arm | Measure | Value (NUMBER) |
|---|---|---|
| Placebo | Percentage of Participants With TEAEs Leading to Participant Withdrawal | 2.4 percentage of participants |
| UCB0599 180 mg/Day | Percentage of Participants With TEAEs Leading to Participant Withdrawal | 10.9 percentage of participants |
| UCB0599 360 mg/Day | Percentage of Participants With TEAEs Leading to Participant Withdrawal | 9.8 percentage of participants |
Secondary
Percentage of Participants With Treatment-emergent Adverse Events (TEAEs)
An AE is any untoward medical occurrence in a patient or clinical study participant, temporally associated with the use of study medication, whether or not considered related to the study medication. A TEAE is defined as any AE with a start date on or after the first dose of treatment or any unresolved event already present before administration of treatment that worsens in intensity following exposure to the treatment.
Time frame: From Baseline up to 30 days of safety follow-up after the last dose of the study drug (up to 19 months)
Population: Safety set included all randomized study participants who receive at least a partial dose of study medication.
| Arm | Measure | Value (NUMBER) |
|---|---|---|
| Placebo | Percentage of Participants With Treatment-emergent Adverse Events (TEAEs) | 87.8 percentage of participants |
| UCB0599 180 mg/Day | Percentage of Participants With Treatment-emergent Adverse Events (TEAEs) | 86.7 percentage of participants |
| UCB0599 360 mg/Day | Percentage of Participants With Treatment-emergent Adverse Events (TEAEs) | 87.2 percentage of participants |
Secondary
Symptomatic Treatment (ST) Intake
Cumulative number of participants on symptomatic treatment (ST) at 18 months are reported.
Time frame: Month 18
Population: FAS included all randomized study participants who received at least a partial dose of study medication, have at least 1 post-Baseline assessment. Here, number of participants analyzed included all participants who were evaluable for this assessment.
| Arm | Measure | Value (COUNT_OF_PARTICIPANTS) |
|---|---|---|
| Placebo | Symptomatic Treatment (ST) Intake | 111 Participants |
| UCB0599 180 mg/Day | Symptomatic Treatment (ST) Intake | 89 Participants |
| UCB0599 360 mg/Day | Symptomatic Treatment (ST) Intake | 88 Participants |
Secondary
Time to Start of Symptomatic Treatment (ST)
Time to start of symptomatic treatment (ST) within the 18-month period.
Time frame: Baseline to Month 18
Population: FAS included all randomized study participants who received at least a partial dose of study medication, have at least 1 post-Baseline assessment.
| Arm | Measure | Value (MEAN) |
|---|---|---|
| Placebo | Time to Start of Symptomatic Treatment (ST) | 10.58 months |
| UCB0599 180 mg/Day | Time to Start of Symptomatic Treatment (ST) | 11.59 months |
| UCB0599 360 mg/Day | Time to Start of Symptomatic Treatment (ST) | 11.80 months |
p-value: 0.12495% CI: [-0.27, 2.27]Wald Chi-square
p-value: 0.06495% CI: [-0.07, 2.51]Wald Chi-sqaure
Secondary
Time to Worsening of the Disease as Measured by MDS-UPDRS Part III
Time to worsening of the disease on the MDS-UPDRS III scale as defined by a 5-point increase in MDS-UPDRS III, within the 18-month period. MDS-UPDRS part III includes motor items assessing speech, facial expression, rigidity, finger tapping, hand movements, pronation supination movements of hands, toe tapping, leg agility, arising from chair, gait, freezing of gait, postural stability, posture, global spontaneity of movement (body bradykinesia), postural tremor of the hands, kinetic tremor of the hands, rest tremor amplitude, and constancy of rest tremor. It included 33 scores based on 18-items, each anchored with 5 responses: 0 = normal, 1 = slight, 2 = mild, 3 = moderate, and 4 = severe. The scale range was from 0 to 132, with a lower score indicating better motor function and a higher score indicating more severe motor symptoms.
Time frame: Baseline to Month 18
Population: FAS included all randomized study participants who received at least a partial dose of study medication, have at least 1 post-Baseline assessment.
| Arm | Measure | Value (MEAN) |
|---|---|---|
| Placebo | Time to Worsening of the Disease as Measured by MDS-UPDRS Part III | 8.64 months |
| UCB0599 180 mg/Day | Time to Worsening of the Disease as Measured by MDS-UPDRS Part III | 8.95 months |
| UCB0599 360 mg/Day | Time to Worsening of the Disease as Measured by MDS-UPDRS Part III | 9.45 months |
p-value: 0.64795% CI: [-1, 1.61]Wald Chi-square
p-value: 0.21495% CI: [-0.47, 2.09]Wald Chi-square