Bone Marrow Disease, Essential Thrombocytosis, Hematological Disease, Leukemia, Leukemia, Myelocytic, Acute, Myelodysplastic/Myeloproliferative Neoplasm, Myelodysplastic Syndrome (MDS), Myelofibrosis, Myeloproliferative Disorders, Neoplasms, Neoplasms by Histologic Type, Precancerous Conditions, Preleukemia, Primary Myelofibrosis
Conditions
Keywords
Phase 1, Phase 2, Oncology, BET Inhibitor, Ruxolitinib, Pelabresib (CPI-0610)
Brief summary
Phase 1 Part: This was an open-label, sequential dose escalation study of pelabresib (CPI-0610) in patients who had previously been treated for Acute Leukemia, Myelodysplastic/Myeloproliferative Neoplasms. Phase 2 Part: This was an open-label study of pelabresib (CPI-0610), administered with and without Ruxolitinib, in patients diagnosed with Myeloproliferative Neoplasms (Myelofibrosis and Essential Thrombocythemia). Pelabresib (CPI-0610) was a small molecule inhibitor of bromodomain and extra-terminal (BET) proteins.
Interventions
DRUGPelabresib
CPI-0610 was administered orally once daily for 14 consecutive days, followed by a 7-day break (1 cycle = 21 days)
DRUGRuxolitinib
Ruxolitinib was given orally, twice daily (BID), on a continuous basis for 21 consecutive days of each 21-day cycle.
Sponsors
Constellation Pharmaceuticals
The Leukemia and Lymphoma Society
Study design
Allocation
NON_RANDOMIZED
Intervention model
PARALLEL
Primary purpose
TREATMENT
Masking
NONE
Eligibility
Sex/Gender
ALL
Age
18 Years to No maximum
Healthy volunteers
No
Inclusion criteria
Phase I (Dose Escalation) - Inclusion and
Exclusion criteria
1. Inclusion Criteria (Phase I): * Age: Adults ≥18 years. * Diagnosis: Histologically or cytologically confirmed diagnosis of one of the following hematologic malignancies: * Acute myelogenous leukemia (AML) * Acute lymphocytic leukemia (ALL) * Acute undifferentiated or biphenotypic leukemia * Chronic myeloid leukemia (CML) in blast crisis * Myelodysplastic syndrome (MDS) * Myelodysplastic/myeloproliferative neoplasms (MDS/MPN) * Myelofibrosis (MF) * Performance Status: ECOG ≤2. * Organ Function: * Serum total bilirubin ≤1.5 × ULN * AST/ALT ≤2.5 × ULN (up to 5 × ULN if due to leukemic infiltration) * Serum creatinine ≤2.0 × ULN or CrCl ≥30 mL/min * Hematology (MF only): * Platelet count ≥50 × 10⁹/L and ANC ≥1 × 10⁹/L (MF not on ruxolitinib) * Platelet count ≥75 × 10⁹/L and ANC ≥1 × 10⁹/L (MF on ruxolitinib) * Other: * DIPSS-plus risk category of intermediate-2 or high (MF only) * Serum glucose ≤160 mg/dL (or HbA1C ≤7%) * Fully recovered from major surgery and acute toxic effects of prior therapy * Negative pregnancy test for women of childbearing potential * Agreement to use appropriate contraception * Written informed consent 2.
Design outcomes
Primary
| Measure | Time frame | Description |
|---|---|---|
| Phase 1: Frequency of Dose-limiting Toxicities (DLTs) | Up to 21 days | A dose-limiting toxicity (DLT) was defined as an adverse event or abnormal laboratory value assessed by the Investigator as unrelated to disease progression, intercurrent illness, or concomitant medications that occurred within the first cycle of treatment (21-day cycle) with pelabresib (CPI-0610), and that met any of the criteria specified in the protocol. |
| Phase 2 (Cohorts 1B, 2B, and Arm 3): Number of Participants With Splenic Response Rate (SVR35) at Week 24 | Week 24 (Cycle 9 Day 1) | Splenic Response Rate (SVR35) at Week 24 was defined as the proportion of participants who demonstrated a reduction of at least 35% in spleen size from baseline, as measured by imaging techniques (MRI or CT), following 24 weeks of treatment. |
| Phase 2 (Cohorts 1A and 2A): Number of Participants Enrolled as Transfusion Dependent (TD) With Conversion Rate From Red Blood Cell (RBC) Transfusion Dependence (TD) to Transfusion Independence (TI) | Any 12 consecutive weeks (rolling window) during active treatment with pelabresib (CPI-0610), from first dose through treatment discontinuation (up to approximately 8 years for Phase II) | Conversion rate was defined as the proportion of participants who converted from transfusion dependence (TD) to transfusion independence (TI). TD was characterized by receiving an average of at least 2 units of red blood cell (RBC) transfusions per month-amounting to a minimum of 6 units over the 12 weeks prior to enrollment-while TI was defined as the absence of RBC transfusions during any consecutive 12-week period. |
| Phase 2 (Arm 4): Number of Participants With Complete Hematological Response (CHR) Rate | Over 2 consecutive cycles (rolling window) (1 cycle = 21 days) | Complete Hematological Response (CHR) Rate was defined as the proportion of participants who fulfilled the criteria for CHR, based on the modified European LeukemiaNet (ELN) guidelines (Barosi et al 2009): platelet count ≤400 × 10⁹/L, white blood cell (WBC) count ≤10 × 10⁹/L, confirmation of laboratory values after one treatment cycle, and normal spleen size determined by palpation or imaging. |
Secondary
| Measure | Time frame | Description |
|---|---|---|
| Phase 1: Number of Adverse Events and Serious Adverse Events as Assessed by CTCAE Criteria | Up to approximately 6 months | The distribution of adverse events was performed through the analysis of frequencies for treatment-emergent adverse events (TEAEs) and serious adverse events (TESAEs), based on the monitoring of relevant clinical and laboratory safety parameters. Treatment-emergent adverse events (TEAEs) in this study were defined as events that began after the first dose of study treatment and continued until 30 days after the last dose, or events that were present prior to the first dose and increased in severity based on preferred term within 30 days following the last dose of pelabresib (CPI-0610). |
| Phase 2 (All Arms): Number of Adverse Events and Serious Adverse Events as Assessed by CTCAE Criteria | Up to approximately 387 weeks | The distribution of adverse events was performed through the analysis of frequencies for treatment-emergent adverse events (TEAEs) and serious adverse events (TESAEs), based on the monitoring of relevant clinical and laboratory safety parameters. Treatment-emergent adverse events (TEAEs) in this study were defined as events that began after the first dose of study treatment and continued until 30 days after the last dose, or events that were present prior to the first dose and increased in severity based on preferred term within 30 days following the last dose of pelabresib (CPI-0610). |
| Phase 2 (All Arms): Symptom Improvement From the Patient Global Impression of Change (PGIC) at 12 and 24 Weeks | Week 12 (Cycle 5 Day 1), Week 24 weeks (Cycle 9 Day 1) | The Patient Global Impression of Change (PGIC) was a single-question, patient-reported assessment that asked individuals to rate their overall change in myeloproliferative neoplasm (MPN) symptoms since starting study treatment. The participants selected one of seven options, ranging from 'Very much improved' to 'Very much worse'. 'No Change from Baseline' indicated stable symptoms (no improvement or worsening), negative change from Baseline indicated a reduction in symptom severity (improvement), and positive change from Baseline indicated an increase in symptom severity (worsening). |
| Phase 2 (Arms 1, 2 and 3): Percent Change From Baseline in Total Symptom Score (TSS) From the Myelofibrosis Symptom Assessment Form (MFSAF v4.0) at 12 and 24 Weeks | Baseline, Week 12 (Cycle 5 Day 1), Week 24 (Cycle 9 Day 1) | The MFSAF (Myelofibrosis Symptom Assessment Form) was completed by participants every day for 7 days before Day 1 of each treatment cycle, including the 7 days before starting Cycle 1. It used a 24-hour recall format, asking participants to rate the worst severity of seven symptoms (fatigue, night sweats, pruritus, abdominal discomfort, pain under the ribs on the left side, early satiety, and bone pain) during the past 24 hours. Each symptom was rated on a scale from 0 (Absent) to 10 (Worst Imaginable). The Total Symptom Score (TSS) was the sum of 7 symptoms (range: 0-70). 'No Change from Baseline' indicated stable symptoms (no improvement or worsening), negative change from Baseline indicated a reduction in symptom severity (improvement), and positive change from Baseline indicated an increase in symptom severity (worsening). |
| Phase 2 (Arms 1, 2 and 3): Number of Participants Who Achieved a ≥ 50% Reduction in Total Symptom Score (TSS) at 12 and 24 Weeks | Week 12 (Cycle 5 Day 1), Week 24 (Cycle 9 Day 1) | The proportion of study participants who experienced a reduction of at least 50% in their Total Symptom Score (TSS), as assessed using the Myelofibrosis Symptom Assessment Form (MFSAF v4.0), was evaluated at both 12 and 24 weeks relative to their baseline score. |
| Phase 2 (Arms 1, 2 and 3): Number of Participants With Overall Splenic Response Rate (Overall SVR35) | Through Phase II completion, an average of 6 years | Overall Splenic Response Rate (SVR35) was defined as the proportion of participants who achieved a reduction of at least 35% in spleen size from baseline, as measured by imaging (MRI or CT), at any point between Cycle 1 Day 1 and the End of Study Visit, whichever occurred first. |
| Phase 2 (Arms 1, 2 and 3): Duration of Overall Splenic Response (Overall SVR35) | From first onset of splenic response until loss of response, assessed up to approximately 6 years | Duration of Overall Splenic Response (overall SVR35) was defined as the time from the first occurrence of a at least 35% reduction in spleen volume from baseline until the earliest of the following: a reduction of less than 35% from baseline combined with an increase of more than 25% from the nadir in spleen volume (as measured by MRI or CT), or death. The nadir was defined as the lowest spleen volume recorded after baseline and up to the evaluation point at which the initial splenic response was achieved. |
| Phase 2 (Cohorts 1A and 2A): Number of Participants With Splenic Response Rate (SVR35) at 12 and 24 Weeks | Week 12 (Cycle 5 Day 1), Week 24 weeks (Cycle 9 Day 1) | Splenic Response Rate (SVR35) at 12 and 24 weeks was defined as the proportion of participants who achieved a reduction of at least 35% in spleen size from baseline, as determined by imaging (MRI or CT), following 12 and 24 weeks of treatment, respectively. |
| Phase 2 (Cohorts 1A and 2A): Duration of Red Blood Cell (RBC) Transfusion Independence (TI) in Participants Who Enroll as Transfusion Dependent (TD) | From first onset of TI to earliest onset of loss of TI, assessed up to approximately 6 years | Duration of Red Blood Cell (RBC) Transfusion Independence (TI) was defined as the longest continuous period during which participants, having achieved at least 12 weeks of transfusion independence, remained free from RBC transfusions. |
| Phase 2 (Cohorts 1A and 2A): Early Anemic Response Rate in Participants Who Enroll as Transfusion Dependent (TD) | Through Phase II completion, an average of 6 years | Early anemic response rate was defined as the proportion of participants who achieved an average increase of at least 1 g/dL in hemoglobin concentration over any rolling 8-week (56-day) period following baseline. This calculation was performed after applying the 14/3 day rule, which stipulates that hemoglobin measurements must be spaced at least 14 days apart, and that at least 3 such measurements are required within the 8-week window to ensure a reliable average. Importantly, this increase had to occur without any red blood cell (RBC) transfusions during the treatment period and up to the time of the latest hemoglobin assessment for each patient. |
| Phase 2 (Cohorts 1B, 2B, and Arm 3): Number of Participants With Splenic Response Rate (SVR35) at 12 Weeks | Week 12 (Cycle 5 Day 1) | Splenic Response Rate (SVR35) at Week 12 was defined as the proportion of participants who achieved a reduction of at least 35% in spleen size from baseline, as measured by imaging (MRI or CT), following 12 weeks of treatment. |
| Phase 2 (Cohorts 1B, 2B, and Arm 3): Anemic Response Rate in Participants Who Enroll as Non-transfusion-dependent (Non-TD) | Through Phase II completion, an average of 6 years | Anemic response was defined as a sustained average increase in hemoglobin concentration of at least 1.5 g/dL over any rolling 12-week (84-day) period following baseline. This calculation excluded periods involving red blood cell (RBC) transfusions and was performed after applying the 14/3-day rule for valid hemoglobin assessments. The response had to be maintained through to the most recent available hemoglobin measurement for each patient. |
| Phase 2 (Arm 4): Percentage of Participants Who Achieve a ≥50% Reduction From Baseline in the Myeloproliferative Neoplasm Symptom Assessment Form (MPN-SAF) Total Score | Baseline, 12 weeks (Cycle 5, Day 1), 24 weeks (Cycle 9, Day 1) | The Myeloproliferative Neoplasm Symptom Assessment Form (MPN-SAF) is a patient-reported questionnaire designed to measure symptom burden in participants with myeloproliferative neoplasms (MPNs). Participants rate the severity of several symptoms (such as fatigue, night sweats, itching, abdominal discomfort, bone pain, early satiety, and others) over the past 24 hours. Each symptom is scored on a scale from 0 (absent/as good as it can be) to 10 (worst imaginable/as bad as it can be). The total score is the sum of all individual symptom scores, providing an overall measure of symptom burden. A 50% reduction in the MPN-SAF total score at 12 or 24 weeks means the patient's overall symptom burden has improved by half compared to their baseline (pre-treatment) score. |
| Phase 2 (Arm 4): Partial Hematological Response Rate (PHR) | Over 2 consecutive cycles (rolling window) (1 cycle = 21 days) | Partial Hematological Response Rate (PHR) was defined as the proportion of participants who met the following criteria over two consecutive cycles up to the last administration of study treatment: * Platelet count \> 400-600 x 10\^9/L * WBC count within normal range (i.e., ≤ 10 x 10\^9/L) * Laboratory results confirmed after 1 cycle (after 3weeks) |
| Phase 2 (Arm 4): Overall Hematological Response Rate (OHR) | Through Phase II completion, an average of 6 years | Confirmed Overall Hematological Response (OHR) Rate was defined as the proportion of participants with either a confirmed complete or a partial hematological response at any time. |
| Phase 2 (Arm 4): Duration of Overall Hematological Response Rate (OHR) | Through Phase II completion, an average of 6 years | Duration of Overall Hematological Response (OHR) Rate was defined as the time from when the overall hematological response was first met until the time at which the overall hematological response was lost, i.e., the criteria for an overall hematological response (CHR or PHR) were not observed or death occurred, whichever came first. |
| Phase 2 (Arm 4): Rate of Hemorrhagic and Thromboembolic (TE) Events | Through Phase II completion, an average of 6 years | Rate of hemorrhagic and thromboembolic (TE) events was defined as the proportion of participants with hemorrhagic or thromboembolic events throughout the study. |
| Phase 2 (All Arms): Maximum Observed Plasma Concentration (Cmax) of Pelabresib | Cycle 1 Day 14 (30 minutes, 1 hour, 2 hours, 4 hours, 6 hours, and 8 hours post-dose). 1 cycle = 21 days. | Pharmacokinetic (PK) parameters were calculated based on Pelabresib plasma concentrations and actual sampling time points. Cmax was listed and summarized using descriptive statistics. |
| Phase 2 (All Arms): Time to Reach Maximum Concentration (Tmax) of Pelabresib | Cycle 1 Day 14 (predose, 30 minutes, 1 hour, 2 hours, 4 hours, 6 hours, and 8 hours post-dose). 1 cycle = 21 days. | Pharmacokinetic (PK) parameters were calculated based on Pelabresib plasma concentrations and actual sampling time points. Tmax was listed and summarized using descriptive statistics. |
| Phase 2 (All Arms): Predose (Trough) Concentration at the End of a Dosing Interval (Ctrough) of Pelabresib | Cycle 1 Day 14 (predose, 30 minutes, 1 hour, 2 hours, 4 hours, 6 hours, and 8 hours post-dose). 1 cycle = 21 days. | Pharmacokinetic (PK) parameters were calculated based on Pelabresib plasma concentrations and actual sampling time points. Ctrough was listed and summarized using descriptive statistics. |
| Phase 2 (All Arms): Area Under the Concentration-Time Curve From Time Zero to the Last Observed Concentration (AUClast) of Pelabresib | Cycle 1 Day 14 (30 minutes, 1 hour, 2 hours, 4 hours, 6 hours, and 8 hours post-dose). 1 cycle = 21 days. | Pharmacokinetic (PK) parameters were calculated based on Pelabresib plasma concentrations and actual sampling time points. AUClast was listed and summarized using descriptive statistics. |
| Phase 2 (All Arms): Area Under the Concentration-Time Curve From Time Zero to 8 Hours Post-dose at Steady State (AUC0-8,ss) of Pelabresib | Cycle 1 Day 14 (30 minutes, 1 hour, 2 hours, 4 hours, 6 hours, and 8 hours post-dose). 1 cycle = 21 days. | Pharmacokinetic (PK) parameters were calculated based on Pelabresib plasma concentrations and actual sampling time points. AUC0-8,ss was listed and summarized using descriptive statistics. |
| Phase 2 (All Arms): Time of Last Measurable Concentration (Tlast) of Pelabresib | Cycle 1 Day 14 (30 minutes, 1 hour, 2 hours, 4 hours, 6 hours, and 8 hours post-dose). 1 cycle = 21 days. | Pharmacokinetic (PK) parameters were calculated based on Pelabresib plasma concentrations and actual sampling time points. Tlast was listed and summarized using descriptive statistics. |
| Phase 2 (Arm 2 - Cohort 2A): Maximum Observed Plasma Concentration (Cmax) of Ruxolitinib | Cycle 1 Day 14 (30 minutes, 1 hour, 2 hours, 4 hours, 6 hours, and 8 hours post-dose). 1 cycle = 21 days. | Pharmacokinetic (PK) parameters were calculated based on ruxolitinib plasma concentrations and actual sampling time points. Cmax was listed and summarized using descriptive statistics. |
| Phase 2 (Arm 2 - Cohort 2A): Time to Reach Maximum Concentration (Tmax) of Ruxolitinib | Cycle 1 Day 14 (30 minutes, 1 hour, 2 hours, 4 hours, 6 hours, and 8 hours post-dose). 1 cycle = 21 days. | Pharmacokinetic (PK) parameters were calculated based on ruxolitinib plasma concentrations and actual sampling time points. Tmax was listed and summarized using descriptive statistics. |
| Phase 2 (Arm 2 - Cohort 2A): Predose (Trough) Concentration at the End of a Dosing Interval (Ctrough) of Ruxolitinib | Cycle 1 Day 14 (predose, 30 minutes, 1 hour, 2 hours, 4 hours, 6 hours, and 8 hours post-dose). 1 cycle = 21 days. | Pharmacokinetic (PK) parameters were calculated based on ruxolitinib plasma concentrations and actual sampling time points. Ctrough was listed and summarized using descriptive statistics. |
| Phase 2 (Arm 2 - Cohort 2A): Area Under the Concentration-Time Curve From Time Zero to the Last Observed Concentration (AUClast) of Ruxolitinib | Cycle 1 Day 14 (30 minutes, 1 hour, 2 hours, 4 hours, 6 hours, and 8 hours post-dose). 1 cycle = 21 days. | Pharmacokinetic (PK) parameters were calculated based on ruxolitinib plasma concentrations and actual sampling time points. AUClast was listed and summarized using descriptive statistics. |
| Phase 2 (Arm 2 - Cohort 2A): Area Under the Concentration-Time Curve From Time Zero to 8 Hours Post-dose at Steady State (AUC0-8,ss) of Ruxolitinib | Cycle 1 Day 14 (30 minutes, 1 hour, 2 hours, 4 hours, 6 hours, and 8 hours post-dose). 1 cycle = 21 days. | Pharmacokinetic (PK) parameters were calculated based on ruxolitinib plasma concentrations and actual sampling time points. AUC0-8,ss was listed and summarized using descriptive statistics. |
| Phase 2 (Arm 2 - Cohort 2B): Maximum Observed Plasma Concentration (Cmax) of Ruxolitinib | Cycle 1 Day 14 (30 minutes, 1 hour, 2 hours, 4 hours, 6 hours, and 8 hours post-dose). 1 cycle = 21 days. | Pharmacokinetic (PK) parameters were calculated based on ruxolitinib plasma concentrations and actual sampling time points. Cmax was listed and summarized using descriptive statistics. |
| Phase 2 (Arm 2 - Cohort 2B): Time to Reach Maximum Concentration (Tmax) of Ruxolitinib | Cycle 1 Day 14 (30 minutes, 1 hour, 2 hours, 4 hours, 6 hours, and 8 hours post-dose). 1 cycle = 21 days. | Pharmacokinetic (PK) parameters were calculated based on ruxolitinib plasma concentrations and actual sampling time points. Tmax was listed and summarized using descriptive statistics. |
| Phase 2 (Arm 2 - Cohort 2B): Predose (Trough) Concentration at the End of a Dosing Interval (Ctrough) of Ruxolitinib | Cycle 1 Day 14 (30 minutes, 1 hour, 2 hours, 4 hours, 6 hours, and 8 hours post-dose). 1 cycle = 21 days. | Pharmacokinetic (PK) parameters were calculated based on ruxolitinib plasma concentrations and actual sampling time points. Ctrough was listed and summarized using descriptive statistics. |
| Phase 2 (Arm 2 - Cohort 2B): Area Under the Concentration-Time Curve From Time Zero to the Last Observed Concentration (AUClast) of Ruxolitinib | Cycle 1 Day 14 (30 minutes, 1 hour, 2 hours, 4 hours, 6 hours, and 8 hours post-dose). 1 cycle = 21 days. | Pharmacokinetic (PK) parameters were calculated based on ruxolitinib plasma concentrations and actual sampling time points. AUClast was listed and summarized using descriptive statistics. |
| Phase 2 (Arm 2 - Cohort 2B): Area Under the Concentration-Time Curve From Time Zero to 8 Hours Post-dose at Steady State (AUC0-8,ss) of Ruxolitinib | Cycle 1 Day 14 (30 minutes, 1 hour, 2 hours, 4 hours, 6 hours, and 8 hours post-dose). 1 cycle = 21 days. | Pharmacokinetic (PK) parameters were calculated based on ruxolitinib plasma concentrations and actual sampling time points. AUC0-8,ss was listed and summarized using descriptive statistics. |
| Phase 2 (Arm 3): Maximum Observed Plasma Concentration (Cmax) of Ruxolitinib | Cycle 1 Day 14 (30 minutes, 1 hour, 2 hours, 4 hours, 6 hours, and 8 hours post-dose). 1 cycle = 21 days. | Pharmacokinetic (PK) parameters were calculated based on ruxolitinib plasma concentrations and actual sampling time points. Cmax was listed and summarized using descriptive statistics. |
| Phase 2 (Arm 3): Time to Reach Maximum Concentration (Tmax) of Ruxolitinib | Cycle 1 Day 14 (30 minutes, 1 hour, 2 hours, 4 hours, 6 hours, and 8 hours post-dose). 1 cycle = 21 days. | Pharmacokinetic (PK) parameters were calculated based on ruxolitinib plasma concentrations and actual sampling time points. Tmax was listed and summarized using descriptive statistics. |
| Phase 2 (Arm 3): Predose (Trough) Concentration at the End of a Dosing Interval (Ctrough) of Ruxolitinib | Cycle 1 Day 14 (predose, 30 minutes, 1 hour, 2 hours, 4 hours, 6 hours, and 8 hours post-dose). 1 cycle = 21 days. | Pharmacokinetic (PK) parameters were calculated based on ruxolitinib plasma concentrations and actual sampling time points. Ctrough was listed and summarized using descriptive statistics. |
| Phase 2 (Arm 3): Area Under the Concentration-Time Curve From Time Zero to the Last Observed Concentration (AUClast) of Ruxolitinib | Cycle 1 Day 14 (30 minutes, 1 hour, 2 hours, 4 hours, 6 hours, and 8 hours post-dose). 1 cycle = 21 days. | Pharmacokinetic (PK) parameters were calculated based on ruxolitinib plasma concentrations and actual sampling time points. AUClast was listed and summarized using descriptive statistics. |
| Phase 2 (Arm 3): Area Under the Concentration-Time Curve From Time Zero to 8 Hours Post-dose at Steady State (AUC0-8,ss) of Ruxolitinib | Cycle 1 Day 14 (30 minutes, 1 hour, 2 hours, 4 hours, 6 hours, and 8 hours post-dose). 1 cycle = 21 days. | Pharmacokinetic (PK) parameters were calculated based on ruxolitinib plasma concentrations and actual sampling time points. AUC0-8,ss was listed and summarized using descriptive statistics. |
Countries
Belgium, Canada, France, Germany, Italy, Netherlands, Poland, United Kingdom, United States
Contacts
STUDY_DIRECTORNovartis Pharmaceuticals
Novartis Pharmaceuticals
Participant flow
Recruitment details
This study was conducted at 54 centers across 9 countries (Belgium, Canada, France, Germany, Italy, Netherlands, Poland, United Kingdom, United States)
Pre-assignment details
Not completed = Participants who discontinued study treatment
Baseline characteristics
| Characteristic | — |
|---|---|
| Age, Continuous | 65.0 Years |
| Race (NIH/OMB) American Indian or Alaska Native | 0 Participants |
| Race (NIH/OMB) Asian | 8 Participants |
| Race (NIH/OMB) Black or African American | 0 Participants |
| Race (NIH/OMB) More than one race | 12 Participants |
| Race (NIH/OMB) Native Hawaiian or Other Pacific Islander | 0 Participants |
| Race (NIH/OMB) Unknown or Not Reported | 0 Participants |
| Race (NIH/OMB) White | 295 Participants |
| Sex: Female, Male Female | 3 Participants |
| Sex: Female, Male Male | 4 Participants |
Adverse events
| Event type | EG000 affected / at risk | EG001 affected / at risk | EG002 affected / at risk | EG003 affected / at risk | EG004 affected / at risk | EG005 affected / at risk | EG006 affected / at risk | EG007 affected / at risk | EG008 affected / at risk | EG009 affected / at risk | EG010 affected / at risk | EG011 affected / at risk | EG012 affected / at risk | EG013 affected / at risk | EG014 affected / at risk | EG015 affected / at risk | EG016 affected / at risk |
|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|
| deaths Total, all-cause mortality | 0 / 3 | 1 / 5 | 2 / 5 | 0 / 3 | 1 / 3 | 1 / 4 | 1 / 7 | 1 / 8 | 1 / 6 | 8 / 44 | 6 / 48 | 3 / 52 | 3 / 59 | 3 / 28 | 7 / 84 | 0 / 21 | 22 / 292 |
| other Total, other adverse events | 3 / 3 | 5 / 5 | 5 / 5 | 3 / 3 | 3 / 3 | 4 / 4 | 7 / 7 | 8 / 8 | 6 / 6 | 44 / 44 | 48 / 48 | 50 / 52 | 58 / 59 | 28 / 28 | 84 / 84 | 21 / 21 | 289 / 292 |
| serious Total, serious adverse events | 1 / 3 | 4 / 5 | 3 / 5 | 1 / 3 | 2 / 3 | 4 / 4 | 6 / 7 | 6 / 8 | 6 / 6 | 33 / 44 | 18 / 48 | 23 / 52 | 29 / 59 | 18 / 28 | 40 / 84 | 5 / 21 | 133 / 292 |
Outcome results
None listed