Trial Outcomes & Findings for HEALEY ALS Platform Trial - Regimen D Pridopidine (NCT NCT04615923)
NCT ID: NCT04615923
Last Updated: 2023-08-23
Results Overview
Change in disease severity over time as measured by the ALS Functional Rating Scale-Revised (ALSFRS-R). Each type of function is scored from 4 (normal) to 0 (no ability), with a maximum total score of 48 and a minimum total score of 0. Patients with higher scores have more physical function.
Recruitment status
COMPLETED
Study phase
PHASE2/PHASE3
Target enrollment
163 participants
Primary outcome timeframe
Baseline to 24 Weeks
Results posted on
2023-08-23
Participant Flow
Participant milestones
| Measure |
Pridopidine
Pridopidine is administered orally twice daily for 24 weeks.
Pridopidine: Administration: Oral
Dose: 45mg twice daily
|
Matching Placebo
Matching placebo is administered orally twice daily for 24 weeks.
Matching Placebo: Administration: Oral
Dose: one capsule twice daily
|
|---|---|---|
|
Overall Study
STARTED
|
120
|
42
|
|
Overall Study
COMPLETED
|
96
|
38
|
|
Overall Study
NOT COMPLETED
|
24
|
4
|
Reasons for withdrawal
Withdrawal data not reported
Baseline Characteristics
Number analyzed in row differs from overall number due to missing data.
Baseline characteristics by cohort
| Measure |
Pridopidine
n=120 Participants
Pridopidine is administered orally twice daily for 24 weeks.
Pridopidine: Administration: Oral
Dose: 45mg twice daily
|
Matching Placebo
n=42 Participants
Matching placebo is administered orally twice daily for 24 weeks.
Matching Placebo: Administration: Oral
Dose: one capsule twice daily
|
Total
n=162 Participants
Total of all reporting groups
|
|---|---|---|---|
|
Age, Continuous
|
57.9 years
STANDARD_DEVIATION 10.10 • n=120 Participants
|
57.1 years
STANDARD_DEVIATION 9.86 • n=42 Participants
|
57.7 years
STANDARD_DEVIATION 10.01 • n=162 Participants
|
|
Sex: Female, Male
Female
|
48 Participants
n=120 Participants
|
9 Participants
n=42 Participants
|
57 Participants
n=162 Participants
|
|
Sex: Female, Male
Male
|
72 Participants
n=120 Participants
|
33 Participants
n=42 Participants
|
105 Participants
n=162 Participants
|
|
Ethnicity (NIH/OMB)
Hispanic or Latino
|
11 Participants
n=120 Participants
|
1 Participants
n=42 Participants
|
12 Participants
n=162 Participants
|
|
Ethnicity (NIH/OMB)
Not Hispanic or Latino
|
107 Participants
n=120 Participants
|
41 Participants
n=42 Participants
|
148 Participants
n=162 Participants
|
|
Ethnicity (NIH/OMB)
Unknown or Not Reported
|
2 Participants
n=120 Participants
|
0 Participants
n=42 Participants
|
2 Participants
n=162 Participants
|
|
Race (NIH/OMB)
American Indian or Alaska Native
|
0 Participants
n=120 Participants
|
0 Participants
n=42 Participants
|
0 Participants
n=162 Participants
|
|
Race (NIH/OMB)
Asian
|
5 Participants
n=120 Participants
|
1 Participants
n=42 Participants
|
6 Participants
n=162 Participants
|
|
Race (NIH/OMB)
Native Hawaiian or Other Pacific Islander
|
0 Participants
n=120 Participants
|
0 Participants
n=42 Participants
|
0 Participants
n=162 Participants
|
|
Race (NIH/OMB)
Black or African American
|
7 Participants
n=120 Participants
|
2 Participants
n=42 Participants
|
9 Participants
n=162 Participants
|
|
Race (NIH/OMB)
White
|
105 Participants
n=120 Participants
|
39 Participants
n=42 Participants
|
144 Participants
n=162 Participants
|
|
Race (NIH/OMB)
More than one race
|
1 Participants
n=120 Participants
|
0 Participants
n=42 Participants
|
1 Participants
n=162 Participants
|
|
Race (NIH/OMB)
Unknown or Not Reported
|
2 Participants
n=120 Participants
|
0 Participants
n=42 Participants
|
2 Participants
n=162 Participants
|
|
El Escorial Diagnosis
Clinically Definite ALS
|
50 Participants
n=120 Participants
|
18 Participants
n=42 Participants
|
68 Participants
n=162 Participants
|
|
El Escorial Diagnosis
Clinically Probable ALS
|
40 Participants
n=120 Participants
|
12 Participants
n=42 Participants
|
52 Participants
n=162 Participants
|
|
El Escorial Diagnosis
Clinically Probable ALS - Laboratory Supported
|
22 Participants
n=120 Participants
|
5 Participants
n=42 Participants
|
27 Participants
n=162 Participants
|
|
El Escorial Diagnosis
Clinically Possible ALS
|
8 Participants
n=120 Participants
|
7 Participants
n=42 Participants
|
15 Participants
n=162 Participants
|
|
ALS Onset Location
Axial
|
1 Participants
n=120 Participants
|
2 Participants
n=42 Participants
|
3 Participants
n=162 Participants
|
|
ALS Onset Location
Bulbar
|
23 Participants
n=120 Participants
|
10 Participants
n=42 Participants
|
33 Participants
n=162 Participants
|
|
ALS Onset Location
Generalized
|
2 Participants
n=120 Participants
|
0 Participants
n=42 Participants
|
2 Participants
n=162 Participants
|
|
ALS Onset Location
Limb
|
93 Participants
n=120 Participants
|
30 Participants
n=42 Participants
|
123 Participants
n=162 Participants
|
|
ALS Onset Location
Multiple
|
1 Participants
n=120 Participants
|
0 Participants
n=42 Participants
|
1 Participants
n=162 Participants
|
|
Baseline Edaravone Use Flag
No
|
92 Participants
n=120 Participants
|
32 Participants
n=42 Participants
|
124 Participants
n=162 Participants
|
|
Baseline Edaravone Use Flag
Yes
|
28 Participants
n=120 Participants
|
10 Participants
n=42 Participants
|
38 Participants
n=162 Participants
|
|
Baseline Riluzole Use Flag
No
|
30 Participants
n=120 Participants
|
9 Participants
n=42 Participants
|
39 Participants
n=162 Participants
|
|
Baseline Riluzole Use Flag
Yes
|
90 Participants
n=120 Participants
|
33 Participants
n=42 Participants
|
123 Participants
n=162 Participants
|
|
Kings Stage
1 Region with Neuromuscular Dysfunction
|
21 Participants
n=120 Participants
|
7 Participants
n=42 Participants
|
28 Participants
n=162 Participants
|
|
Kings Stage
2 Regions with Neuromuscular Dysfunction
|
36 Participants
n=120 Participants
|
7 Participants
n=42 Participants
|
43 Participants
n=162 Participants
|
|
Kings Stage
3 Regions with Neuromuscular Dysfunction
|
31 Participants
n=120 Participants
|
18 Participants
n=42 Participants
|
49 Participants
n=162 Participants
|
|
Kings Stage
4a/4b Nutritional/Respiratory Failure
|
32 Participants
n=120 Participants
|
10 Participants
n=42 Participants
|
42 Participants
n=162 Participants
|
|
ALSFRS-R Total Score
|
34.3 points
STANDARD_DEVIATION 6.71 • n=120 Participants
|
35.0 points
STANDARD_DEVIATION 7.07 • n=42 Participants
|
34.5 points
STANDARD_DEVIATION 6.79 • n=162 Participants
|
|
Baseline Decline in ALSFRS-R
|
0.77 points per month
STANDARD_DEVIATION 0.5 • n=120 Participants
|
0.71 points per month
STANDARD_DEVIATION 0.455 • n=42 Participants
|
0.75 points per month
STANDARD_DEVIATION 0.488 • n=162 Participants
|
|
Body Mass Index
|
26.1 kg/m^2
STANDARD_DEVIATION 4.63 • n=120 Participants
|
25.9 kg/m^2
STANDARD_DEVIATION 4.07 • n=42 Participants
|
26.1 kg/m^2
STANDARD_DEVIATION 4.48 • n=162 Participants
|
|
Delay in ALS Symptom Onset and Diagnosis
|
10.5 months
STANDARD_DEVIATION 6.34 • n=120 Participants
|
10.4 months
STANDARD_DEVIATION 6.50 • n=42 Participants
|
10.5 months
STANDARD_DEVIATION 6.36 • n=162 Participants
|
|
SVC
|
78.3 percent predicted
STANDARD_DEVIATION 17.35 • n=116 Participants • Number analyzed in row differs from overall number due to missing data.
|
77.5 percent predicted
STANDARD_DEVIATION 16.72 • n=42 Participants • Number analyzed in row differs from overall number due to missing data.
|
78.1 percent predicted
STANDARD_DEVIATION 17.13 • n=158 Participants • Number analyzed in row differs from overall number due to missing data.
|
|
Serum Cereatinine Concentration
|
0.7 mg/dL
STANDARD_DEVIATION 0.19 • n=120 Participants
|
0.8 mg/dL
STANDARD_DEVIATION 0.18 • n=42 Participants
|
0.7 mg/dL
STANDARD_DEVIATION 0.19 • n=162 Participants
|
|
Time Since Symptom Onsent at Baseline (Months since ALS symptom onset)
|
20.6 months
STANDARD_DEVIATION 8.16 • n=120 Participants
|
21.6 months
STANDARD_DEVIATION 10.0 • n=42 Participants
|
20.8 months
STANDARD_DEVIATION 8.65 • n=162 Participants
|
|
Weight
|
76.6 kg
STANDARD_DEVIATION 15.92 • n=120 Participants
|
79.3 kg
STANDARD_DEVIATION 15.63 • n=42 Participants
|
77.3 kg
STANDARD_DEVIATION 15.84 • n=162 Participants
|
|
Serum NfL Concentration
|
92.7 ng/L
STANDARD_DEVIATION 61.24 • n=119 Participants • Number analyzed in row differs from overall number due to missing data.
|
114.6 ng/L
STANDARD_DEVIATION 73.25 • n=40 Participants • Number analyzed in row differs from overall number due to missing data.
|
98.2 ng/L
STANDARD_DEVIATION 64.93 • n=159 Participants • Number analyzed in row differs from overall number due to missing data.
|
PRIMARY outcome
Timeframe: Baseline to 24 WeeksPopulation: Outcome measure data was analyzed using the Full Analysis Set, which included shared placebo from other regimens, as pre-specified in the Regimen Statistical Analysis Plan. Regimen A (NCT04436497), Regimen B (NCT04436510) and Regimen C (NCT04414345) contributed placebo participants into the shared placebo cohort used for analysis.
Change in disease severity over time as measured by the ALS Functional Rating Scale-Revised (ALSFRS-R). Each type of function is scored from 4 (normal) to 0 (no ability), with a maximum total score of 48 and a minimum total score of 0. Patients with higher scores have more physical function.
Outcome measures
| Measure |
Pridopidine
n=120 Participants
Pridopidine is administered orally twice daily for 24 weeks.
Pridopidine: Administration: Oral
Dose: 45mg twice daily
|
Matching Placebo
n=162 Participants
Matching placebo is administered orally twice daily for 24 weeks.
Matching Placebo: Administration: Oral
Dose: one capsule twice daily
|
|---|---|---|
|
Disease Progression as Assessed by the ALSFRS-R Total Score
|
-0.99 points per month
Standard Deviation 0.077
|
-1.00 points per month
Standard Deviation 0.070
|
PRIMARY outcome
Timeframe: Baseline to 24 WeeksPopulation: Outcome measure data was analyzed using the Full Analysis Set, which included shared placebo from other regimens, as pre-specified in the Regimen Statistical Analysis Plan. Regimen A (NCT04436497), Regimen B (NCT04436510) and Regimen C (NCT04414345) contributed placebo participants into the shared placebo cohort used for analysis.
Mortality is defined as death or death equivalent. A participant is determined to meet the criteria of death equivalent if permanent assisted ventilation (PAV) is used for more than 22 hours per day for more than seven days in a row. The rate of mortality was estimated from a Bayesian shared-parametric model that assumed exponentially distributed survival times.
Outcome measures
| Measure |
Pridopidine
n=120 Participants
Pridopidine is administered orally twice daily for 24 weeks.
Pridopidine: Administration: Oral
Dose: 45mg twice daily
|
Matching Placebo
n=162 Participants
Matching placebo is administered orally twice daily for 24 weeks.
Matching Placebo: Administration: Oral
Dose: one capsule twice daily
|
|---|---|---|
|
Mortality Event Rate
|
0.012 events per month
Standard Deviation 0.0029
|
0.012 events per month
Standard Deviation 0.0029
|
SECONDARY outcome
Timeframe: Baseline to 24 WeeksPopulation: Outcome measure data was analyzed using the Full Analysis Set, which included shared placebo from other regimens, as pre-specified in the Regimen Statistical Analysis Plan. Regimen A (NCT04436497), Regimen B (NCT04436510) and Regimen C (NCT04414345) contributed placebo participants into the shared placebo cohort used for analysis.
Change in bulbar function over time in participants with bulbar dysfunction at baseline as measured by the ALS Functional Rating Scale-Revised (ALSFRS-R) bulbar subdomain. Each question is scored from 4 (normal) to 0 (no ability), with a maximum total score of 12 and a minimum total score of 0 for the bulbar subdomain. Patients with higher scores have more bulbar function. Participants were classified as having bulbar dysfunction at baseline if their score on the ALSFRS-R bulbar domain (Q1-Q3) score was less than 12.
Outcome measures
| Measure |
Pridopidine
n=75 Participants
Pridopidine is administered orally twice daily for 24 weeks.
Pridopidine: Administration: Oral
Dose: 45mg twice daily
|
Matching Placebo
n=103 Participants
Matching placebo is administered orally twice daily for 24 weeks.
Matching Placebo: Administration: Oral
Dose: one capsule twice daily
|
|---|---|---|
|
Change in Bulbar Function in Participants With Bulbar Dysfunction at Baseline
|
-1.78 point change from baseline
Standard Error 0.261
|
-1.69 point change from baseline
Standard Error 0.210
|
SECONDARY outcome
Timeframe: Baseline to 24 WeeksPopulation: Outcome measure data was analyzed using the Full Analysis Set, which included shared placebo from other regimens, as pre-specified in the Regimen Statistical Analysis Plan. Regimen A (NCT04436497), Regimen B (NCT04436510) and Regimen C (NCT04414345) contributed placebo participants into the shared placebo cohort used for analysis.
Change in bulbar function over time in all randomized participants as measured by the ALS Functional Rating Scale-Revised (ALSFRS-R) bulbar subdomain. Each question is scored from 4 (normal) to 0 (no ability), with a maximum total score of 12 and a minimum total score of 0 for the bulbar subdomain. Patients with higher scores have more bulbar function.
Outcome measures
| Measure |
Pridopidine
n=120 Participants
Pridopidine is administered orally twice daily for 24 weeks.
Pridopidine: Administration: Oral
Dose: 45mg twice daily
|
Matching Placebo
n=162 Participants
Matching placebo is administered orally twice daily for 24 weeks.
Matching Placebo: Administration: Oral
Dose: one capsule twice daily
|
|---|---|---|
|
Bulbar Function in All Randomized Participants
|
-1.16 point change from baseline
Standard Error 0.169
|
-1.25 point change from baseline
Standard Error 0.141
|
SECONDARY outcome
Timeframe: Baseline to 24 WeeksPopulation: Outcome measure data was analyzed using the Full Analysis Set, which included shared placebo from other regimens, as pre-specified in the Regimen Statistical Analysis Plan. Regimen A (NCT04436497), Regimen B (NCT04436510) and Regimen C (NCT04414345) contributed placebo participants into the shared placebo cohort used for analysis.
Change in respiratory function over time as measured by Slow Vital Capacity (SVC).
Outcome measures
| Measure |
Pridopidine
n=120 Participants
Pridopidine is administered orally twice daily for 24 weeks.
Pridopidine: Administration: Oral
Dose: 45mg twice daily
|
Matching Placebo
n=162 Participants
Matching placebo is administered orally twice daily for 24 weeks.
Matching Placebo: Administration: Oral
Dose: one capsule twice daily
|
|---|---|---|
|
Respiratory Function
|
-8.81 percent change
Standard Error 1.351
|
-8.35 percent change
Standard Error 1.132
|
SECONDARY outcome
Timeframe: Baseline to 24 WeeksPopulation: Outcome measure data was analyzed using the Full Analysis Set, which included shared placebo from other regimens, as pre-specified in the Regimen Statistical Analysis Plan. Regimen A (NCT04436497), Regimen B (NCT04436510) and Regimen C (NCT04414345) contributed placebo participants into the shared placebo cohort used for analysis.
Change in bulbar function over time in participants with rapid pre-baseline progression as measured by the ALS Functional Rating Scale-Revised (ALSFRS-R) bulbar subdomain. Each question is scored from 4 (normal) to 0 (no ability), with a maximum total score of 12 and a minimum total score of 0 for the bulbar subdomain. Patients with higher scores have more bulbar function. Participants were classified with rapid pre-baseline progression if their change in ALSFRS-R total score between Master Protocol Screening and Regimen Baseline was greater than or equal or 0.75 points per month.
Outcome measures
| Measure |
Pridopidine
n=51 Participants
Pridopidine is administered orally twice daily for 24 weeks.
Pridopidine: Administration: Oral
Dose: 45mg twice daily
|
Matching Placebo
n=48 Participants
Matching placebo is administered orally twice daily for 24 weeks.
Matching Placebo: Administration: Oral
Dose: one capsule twice daily
|
|---|---|---|
|
Bulbar Function in Participants With Rapid Pre-baseline Progression
|
-1.54 points change from baseline
Standard Error 0.297
|
-1.54 points change from baseline
Standard Error 0.302
|
SECONDARY outcome
Timeframe: Baseline to 24 WeeksPopulation: Outcome measure data was analyzed using the Full Analysis Set, which included shared placebo from other regimens, as pre-specified in the Regimen Statistical Analysis Plan. Regimen A (NCT04436497), Regimen B (NCT04436510) and Regimen C (NCT04414345) contributed placebo participants into the shared placebo cohort used for analysis.
Time to first decline of 1-point or greater post baseline in the ALSFRS-R bulbar domain score among all participants. Analysis performed using interval-censored survival analysis. Results presented as median interval (upper \& lower bounded) time to event.
Outcome measures
| Measure |
Pridopidine
n=120 Participants
Pridopidine is administered orally twice daily for 24 weeks.
Pridopidine: Administration: Oral
Dose: 45mg twice daily
|
Matching Placebo
n=162 Participants
Matching placebo is administered orally twice daily for 24 weeks.
Matching Placebo: Administration: Oral
Dose: one capsule twice daily
|
|---|---|---|
|
Time to Bulbar Decline
Lower Bound of Interval
|
84 days
Interval 53.0 to 141.0
|
66 days
Interval 56.0 to 105.0
|
|
Time to Bulbar Decline
Upper Bound of Interval
|
86 days
Interval 83.0 to 141.0
|
77 days
Interval 68.0 to 115.0
|
SECONDARY outcome
Timeframe: Baseline to 24 WeeksPopulation: Outcome measure data was analyzed using the Full Analysis Set, which included shared placebo from other regimens, as pre-specified in the Regimen Statistical Analysis Plan. Regimen A (NCT04436497), Regimen B (NCT04436510) and Regimen C (NCT04414345) contributed placebo participants into the shared placebo cohort used for analysis.
Change in muscle strength over time as measured isometrically using hand-held dynamometry (HHD).
Outcome measures
| Measure |
Pridopidine
n=120 Participants
Pridopidine is administered orally twice daily for 24 weeks.
Pridopidine: Administration: Oral
Dose: 45mg twice daily
|
Matching Placebo
n=162 Participants
Matching placebo is administered orally twice daily for 24 weeks.
Matching Placebo: Administration: Oral
Dose: one capsule twice daily
|
|---|---|---|
|
Muscle Strength
|
-26.74 percent change
Standard Error 2.556
|
-24.97 percent change
Standard Error 2.123
|
SECONDARY outcome
Timeframe: Baseline to 24 WeeksPopulation: Outcome measure data was analyzed using the Full Analysis Set, which included shared placebo from other regimens, as pre-specified in the Regimen Statistical Analysis Plan. Regimen A (NCT04436497), Regimen B (NCT04436510) and Regimen C (NCT04414345) contributed placebo participants into the shared placebo cohort used for analysis.
The number of participants who died or met the criterion for a death equivalent from the date of their baseline visit to the end of the Week 24visit window (generally 175 days after baseline). The death equivalent criterion is use of permanent assisted ventilation (PAV) for more than 22 hours per day for more than 7 days in a row.
Outcome measures
| Measure |
Pridopidine
n=120 Participants
Pridopidine is administered orally twice daily for 24 weeks.
Pridopidine: Administration: Oral
Dose: 45mg twice daily
|
Matching Placebo
n=162 Participants
Matching placebo is administered orally twice daily for 24 weeks.
Matching Placebo: Administration: Oral
Dose: one capsule twice daily
|
|---|---|---|
|
Number of Participants That Experienced Death or Death Equivalent
|
5 Participants
|
6 Participants
|
Adverse Events
Pridopidine
Serious events: 16 serious events
Other events: 106 other events
Deaths: 2 deaths
Matching Placebo
Serious events: 3 serious events
Other events: 37 other events
Deaths: 0 deaths
Serious adverse events
| Measure |
Pridopidine
n=121 participants at risk
Pridopidine is administered orally twice daily for 24 weeks.
Pridopidine: Administration: Oral
Dose: 45mg twice daily
|
Matching Placebo
n=41 participants at risk
Matching placebo is administered orally twice daily for 24 weeks.
Matching Placebo: Administration: Oral
Dose: one capsule twice daily
|
|---|---|---|
|
Nervous system disorders
Amyotrophic lateral sclerosis
|
1.7%
2/121 • Number of events 2 • Up to 35 weeks after participant signed Master Protocol consent.
The total at risk number for serious adverse events and other adverse events differs from the overall numbers due to a participant excluded from full analysis set for efficacy analyses and baseline characteristics due to a diagnosis other than ALS in one participant and due to participant included in the full analysis set for efficacy analyses and baseline characteristics due to being randomized but who never initiated study drug and thus was not considered as risk for safety outcomes.
|
0.00%
0/41 • Up to 35 weeks after participant signed Master Protocol consent.
The total at risk number for serious adverse events and other adverse events differs from the overall numbers due to a participant excluded from full analysis set for efficacy analyses and baseline characteristics due to a diagnosis other than ALS in one participant and due to participant included in the full analysis set for efficacy analyses and baseline characteristics due to being randomized but who never initiated study drug and thus was not considered as risk for safety outcomes.
|
|
Nervous system disorders
Syncope
|
1.7%
2/121 • Number of events 2 • Up to 35 weeks after participant signed Master Protocol consent.
The total at risk number for serious adverse events and other adverse events differs from the overall numbers due to a participant excluded from full analysis set for efficacy analyses and baseline characteristics due to a diagnosis other than ALS in one participant and due to participant included in the full analysis set for efficacy analyses and baseline characteristics due to being randomized but who never initiated study drug and thus was not considered as risk for safety outcomes.
|
0.00%
0/41 • Up to 35 weeks after participant signed Master Protocol consent.
The total at risk number for serious adverse events and other adverse events differs from the overall numbers due to a participant excluded from full analysis set for efficacy analyses and baseline characteristics due to a diagnosis other than ALS in one participant and due to participant included in the full analysis set for efficacy analyses and baseline characteristics due to being randomized but who never initiated study drug and thus was not considered as risk for safety outcomes.
|
|
Nervous system disorders
Cerebellar stroke
|
0.83%
1/121 • Number of events 1 • Up to 35 weeks after participant signed Master Protocol consent.
The total at risk number for serious adverse events and other adverse events differs from the overall numbers due to a participant excluded from full analysis set for efficacy analyses and baseline characteristics due to a diagnosis other than ALS in one participant and due to participant included in the full analysis set for efficacy analyses and baseline characteristics due to being randomized but who never initiated study drug and thus was not considered as risk for safety outcomes.
|
0.00%
0/41 • Up to 35 weeks after participant signed Master Protocol consent.
The total at risk number for serious adverse events and other adverse events differs from the overall numbers due to a participant excluded from full analysis set for efficacy analyses and baseline characteristics due to a diagnosis other than ALS in one participant and due to participant included in the full analysis set for efficacy analyses and baseline characteristics due to being randomized but who never initiated study drug and thus was not considered as risk for safety outcomes.
|
|
Nervous system disorders
Ischaemic stroke
|
0.83%
1/121 • Number of events 1 • Up to 35 weeks after participant signed Master Protocol consent.
The total at risk number for serious adverse events and other adverse events differs from the overall numbers due to a participant excluded from full analysis set for efficacy analyses and baseline characteristics due to a diagnosis other than ALS in one participant and due to participant included in the full analysis set for efficacy analyses and baseline characteristics due to being randomized but who never initiated study drug and thus was not considered as risk for safety outcomes.
|
0.00%
0/41 • Up to 35 weeks after participant signed Master Protocol consent.
The total at risk number for serious adverse events and other adverse events differs from the overall numbers due to a participant excluded from full analysis set for efficacy analyses and baseline characteristics due to a diagnosis other than ALS in one participant and due to participant included in the full analysis set for efficacy analyses and baseline characteristics due to being randomized but who never initiated study drug and thus was not considered as risk for safety outcomes.
|
|
Nervous system disorders
Presyncope
|
0.83%
1/121 • Number of events 1 • Up to 35 weeks after participant signed Master Protocol consent.
The total at risk number for serious adverse events and other adverse events differs from the overall numbers due to a participant excluded from full analysis set for efficacy analyses and baseline characteristics due to a diagnosis other than ALS in one participant and due to participant included in the full analysis set for efficacy analyses and baseline characteristics due to being randomized but who never initiated study drug and thus was not considered as risk for safety outcomes.
|
0.00%
0/41 • Up to 35 weeks after participant signed Master Protocol consent.
The total at risk number for serious adverse events and other adverse events differs from the overall numbers due to a participant excluded from full analysis set for efficacy analyses and baseline characteristics due to a diagnosis other than ALS in one participant and due to participant included in the full analysis set for efficacy analyses and baseline characteristics due to being randomized but who never initiated study drug and thus was not considered as risk for safety outcomes.
|
|
Respiratory, thoracic and mediastinal disorders
Respiratory failure
|
2.5%
3/121 • Number of events 3 • Up to 35 weeks after participant signed Master Protocol consent.
The total at risk number for serious adverse events and other adverse events differs from the overall numbers due to a participant excluded from full analysis set for efficacy analyses and baseline characteristics due to a diagnosis other than ALS in one participant and due to participant included in the full analysis set for efficacy analyses and baseline characteristics due to being randomized but who never initiated study drug and thus was not considered as risk for safety outcomes.
|
2.4%
1/41 • Number of events 1 • Up to 35 weeks after participant signed Master Protocol consent.
The total at risk number for serious adverse events and other adverse events differs from the overall numbers due to a participant excluded from full analysis set for efficacy analyses and baseline characteristics due to a diagnosis other than ALS in one participant and due to participant included in the full analysis set for efficacy analyses and baseline characteristics due to being randomized but who never initiated study drug and thus was not considered as risk for safety outcomes.
|
|
Respiratory, thoracic and mediastinal disorders
Pulmonary embolism
|
0.83%
1/121 • Number of events 1 • Up to 35 weeks after participant signed Master Protocol consent.
The total at risk number for serious adverse events and other adverse events differs from the overall numbers due to a participant excluded from full analysis set for efficacy analyses and baseline characteristics due to a diagnosis other than ALS in one participant and due to participant included in the full analysis set for efficacy analyses and baseline characteristics due to being randomized but who never initiated study drug and thus was not considered as risk for safety outcomes.
|
0.00%
0/41 • Up to 35 weeks after participant signed Master Protocol consent.
The total at risk number for serious adverse events and other adverse events differs from the overall numbers due to a participant excluded from full analysis set for efficacy analyses and baseline characteristics due to a diagnosis other than ALS in one participant and due to participant included in the full analysis set for efficacy analyses and baseline characteristics due to being randomized but who never initiated study drug and thus was not considered as risk for safety outcomes.
|
|
Injury, poisoning and procedural complications
Ankle fracture
|
0.00%
0/121 • Up to 35 weeks after participant signed Master Protocol consent.
The total at risk number for serious adverse events and other adverse events differs from the overall numbers due to a participant excluded from full analysis set for efficacy analyses and baseline characteristics due to a diagnosis other than ALS in one participant and due to participant included in the full analysis set for efficacy analyses and baseline characteristics due to being randomized but who never initiated study drug and thus was not considered as risk for safety outcomes.
|
2.4%
1/41 • Number of events 1 • Up to 35 weeks after participant signed Master Protocol consent.
The total at risk number for serious adverse events and other adverse events differs from the overall numbers due to a participant excluded from full analysis set for efficacy analyses and baseline characteristics due to a diagnosis other than ALS in one participant and due to participant included in the full analysis set for efficacy analyses and baseline characteristics due to being randomized but who never initiated study drug and thus was not considered as risk for safety outcomes.
|
|
Injury, poisoning and procedural complications
Hip fracture
|
0.83%
1/121 • Number of events 1 • Up to 35 weeks after participant signed Master Protocol consent.
The total at risk number for serious adverse events and other adverse events differs from the overall numbers due to a participant excluded from full analysis set for efficacy analyses and baseline characteristics due to a diagnosis other than ALS in one participant and due to participant included in the full analysis set for efficacy analyses and baseline characteristics due to being randomized but who never initiated study drug and thus was not considered as risk for safety outcomes.
|
0.00%
0/41 • Up to 35 weeks after participant signed Master Protocol consent.
The total at risk number for serious adverse events and other adverse events differs from the overall numbers due to a participant excluded from full analysis set for efficacy analyses and baseline characteristics due to a diagnosis other than ALS in one participant and due to participant included in the full analysis set for efficacy analyses and baseline characteristics due to being randomized but who never initiated study drug and thus was not considered as risk for safety outcomes.
|
|
Injury, poisoning and procedural complications
Post lumbar puncture syndrome
|
0.83%
1/121 • Number of events 1 • Up to 35 weeks after participant signed Master Protocol consent.
The total at risk number for serious adverse events and other adverse events differs from the overall numbers due to a participant excluded from full analysis set for efficacy analyses and baseline characteristics due to a diagnosis other than ALS in one participant and due to participant included in the full analysis set for efficacy analyses and baseline characteristics due to being randomized but who never initiated study drug and thus was not considered as risk for safety outcomes.
|
0.00%
0/41 • Up to 35 weeks after participant signed Master Protocol consent.
The total at risk number for serious adverse events and other adverse events differs from the overall numbers due to a participant excluded from full analysis set for efficacy analyses and baseline characteristics due to a diagnosis other than ALS in one participant and due to participant included in the full analysis set for efficacy analyses and baseline characteristics due to being randomized but who never initiated study drug and thus was not considered as risk for safety outcomes.
|
|
Injury, poisoning and procedural complications
Traumatic intracranial haemorrhage
|
0.83%
1/121 • Number of events 1 • Up to 35 weeks after participant signed Master Protocol consent.
The total at risk number for serious adverse events and other adverse events differs from the overall numbers due to a participant excluded from full analysis set for efficacy analyses and baseline characteristics due to a diagnosis other than ALS in one participant and due to participant included in the full analysis set for efficacy analyses and baseline characteristics due to being randomized but who never initiated study drug and thus was not considered as risk for safety outcomes.
|
0.00%
0/41 • Up to 35 weeks after participant signed Master Protocol consent.
The total at risk number for serious adverse events and other adverse events differs from the overall numbers due to a participant excluded from full analysis set for efficacy analyses and baseline characteristics due to a diagnosis other than ALS in one participant and due to participant included in the full analysis set for efficacy analyses and baseline characteristics due to being randomized but who never initiated study drug and thus was not considered as risk for safety outcomes.
|
|
Cardiac disorders
Atrial fibrillation
|
1.7%
2/121 • Number of events 2 • Up to 35 weeks after participant signed Master Protocol consent.
The total at risk number for serious adverse events and other adverse events differs from the overall numbers due to a participant excluded from full analysis set for efficacy analyses and baseline characteristics due to a diagnosis other than ALS in one participant and due to participant included in the full analysis set for efficacy analyses and baseline characteristics due to being randomized but who never initiated study drug and thus was not considered as risk for safety outcomes.
|
0.00%
0/41 • Up to 35 weeks after participant signed Master Protocol consent.
The total at risk number for serious adverse events and other adverse events differs from the overall numbers due to a participant excluded from full analysis set for efficacy analyses and baseline characteristics due to a diagnosis other than ALS in one participant and due to participant included in the full analysis set for efficacy analyses and baseline characteristics due to being randomized but who never initiated study drug and thus was not considered as risk for safety outcomes.
|
|
Infections and infestations
COVID-19 pneumonia
|
0.83%
1/121 • Number of events 1 • Up to 35 weeks after participant signed Master Protocol consent.
The total at risk number for serious adverse events and other adverse events differs from the overall numbers due to a participant excluded from full analysis set for efficacy analyses and baseline characteristics due to a diagnosis other than ALS in one participant and due to participant included in the full analysis set for efficacy analyses and baseline characteristics due to being randomized but who never initiated study drug and thus was not considered as risk for safety outcomes.
|
0.00%
0/41 • Up to 35 weeks after participant signed Master Protocol consent.
The total at risk number for serious adverse events and other adverse events differs from the overall numbers due to a participant excluded from full analysis set for efficacy analyses and baseline characteristics due to a diagnosis other than ALS in one participant and due to participant included in the full analysis set for efficacy analyses and baseline characteristics due to being randomized but who never initiated study drug and thus was not considered as risk for safety outcomes.
|
|
Infections and infestations
Sepsis
|
0.00%
0/121 • Up to 35 weeks after participant signed Master Protocol consent.
The total at risk number for serious adverse events and other adverse events differs from the overall numbers due to a participant excluded from full analysis set for efficacy analyses and baseline characteristics due to a diagnosis other than ALS in one participant and due to participant included in the full analysis set for efficacy analyses and baseline characteristics due to being randomized but who never initiated study drug and thus was not considered as risk for safety outcomes.
|
2.4%
1/41 • Number of events 1 • Up to 35 weeks after participant signed Master Protocol consent.
The total at risk number for serious adverse events and other adverse events differs from the overall numbers due to a participant excluded from full analysis set for efficacy analyses and baseline characteristics due to a diagnosis other than ALS in one participant and due to participant included in the full analysis set for efficacy analyses and baseline characteristics due to being randomized but who never initiated study drug and thus was not considered as risk for safety outcomes.
|
|
Gastrointestinal disorders
Pneumoperitoneum
|
0.83%
1/121 • Number of events 1 • Up to 35 weeks after participant signed Master Protocol consent.
The total at risk number for serious adverse events and other adverse events differs from the overall numbers due to a participant excluded from full analysis set for efficacy analyses and baseline characteristics due to a diagnosis other than ALS in one participant and due to participant included in the full analysis set for efficacy analyses and baseline characteristics due to being randomized but who never initiated study drug and thus was not considered as risk for safety outcomes.
|
0.00%
0/41 • Up to 35 weeks after participant signed Master Protocol consent.
The total at risk number for serious adverse events and other adverse events differs from the overall numbers due to a participant excluded from full analysis set for efficacy analyses and baseline characteristics due to a diagnosis other than ALS in one participant and due to participant included in the full analysis set for efficacy analyses and baseline characteristics due to being randomized but who never initiated study drug and thus was not considered as risk for safety outcomes.
|
|
Metabolism and nutrition disorders
Failure to thrive
|
0.83%
1/121 • Number of events 1 • Up to 35 weeks after participant signed Master Protocol consent.
The total at risk number for serious adverse events and other adverse events differs from the overall numbers due to a participant excluded from full analysis set for efficacy analyses and baseline characteristics due to a diagnosis other than ALS in one participant and due to participant included in the full analysis set for efficacy analyses and baseline characteristics due to being randomized but who never initiated study drug and thus was not considered as risk for safety outcomes.
|
0.00%
0/41 • Up to 35 weeks after participant signed Master Protocol consent.
The total at risk number for serious adverse events and other adverse events differs from the overall numbers due to a participant excluded from full analysis set for efficacy analyses and baseline characteristics due to a diagnosis other than ALS in one participant and due to participant included in the full analysis set for efficacy analyses and baseline characteristics due to being randomized but who never initiated study drug and thus was not considered as risk for safety outcomes.
|
Other adverse events
| Measure |
Pridopidine
n=121 participants at risk
Pridopidine is administered orally twice daily for 24 weeks.
Pridopidine: Administration: Oral
Dose: 45mg twice daily
|
Matching Placebo
n=41 participants at risk
Matching placebo is administered orally twice daily for 24 weeks.
Matching Placebo: Administration: Oral
Dose: one capsule twice daily
|
|---|---|---|
|
Nervous system disorders
Muscular weakness
|
24.0%
29/121 • Number of events 33 • Up to 35 weeks after participant signed Master Protocol consent.
The total at risk number for serious adverse events and other adverse events differs from the overall numbers due to a participant excluded from full analysis set for efficacy analyses and baseline characteristics due to a diagnosis other than ALS in one participant and due to participant included in the full analysis set for efficacy analyses and baseline characteristics due to being randomized but who never initiated study drug and thus was not considered as risk for safety outcomes.
|
31.7%
13/41 • Number of events 21 • Up to 35 weeks after participant signed Master Protocol consent.
The total at risk number for serious adverse events and other adverse events differs from the overall numbers due to a participant excluded from full analysis set for efficacy analyses and baseline characteristics due to a diagnosis other than ALS in one participant and due to participant included in the full analysis set for efficacy analyses and baseline characteristics due to being randomized but who never initiated study drug and thus was not considered as risk for safety outcomes.
|
|
Nervous system disorders
Neuromyopathy
|
19.8%
24/121 • Number of events 37 • Up to 35 weeks after participant signed Master Protocol consent.
The total at risk number for serious adverse events and other adverse events differs from the overall numbers due to a participant excluded from full analysis set for efficacy analyses and baseline characteristics due to a diagnosis other than ALS in one participant and due to participant included in the full analysis set for efficacy analyses and baseline characteristics due to being randomized but who never initiated study drug and thus was not considered as risk for safety outcomes.
|
7.3%
3/41 • Number of events 3 • Up to 35 weeks after participant signed Master Protocol consent.
The total at risk number for serious adverse events and other adverse events differs from the overall numbers due to a participant excluded from full analysis set for efficacy analyses and baseline characteristics due to a diagnosis other than ALS in one participant and due to participant included in the full analysis set for efficacy analyses and baseline characteristics due to being randomized but who never initiated study drug and thus was not considered as risk for safety outcomes.
|
|
Nervous system disorders
Dysarthria
|
8.3%
10/121 • Number of events 10 • Up to 35 weeks after participant signed Master Protocol consent.
The total at risk number for serious adverse events and other adverse events differs from the overall numbers due to a participant excluded from full analysis set for efficacy analyses and baseline characteristics due to a diagnosis other than ALS in one participant and due to participant included in the full analysis set for efficacy analyses and baseline characteristics due to being randomized but who never initiated study drug and thus was not considered as risk for safety outcomes.
|
9.8%
4/41 • Number of events 5 • Up to 35 weeks after participant signed Master Protocol consent.
The total at risk number for serious adverse events and other adverse events differs from the overall numbers due to a participant excluded from full analysis set for efficacy analyses and baseline characteristics due to a diagnosis other than ALS in one participant and due to participant included in the full analysis set for efficacy analyses and baseline characteristics due to being randomized but who never initiated study drug and thus was not considered as risk for safety outcomes.
|
|
Nervous system disorders
Dizziness
|
7.4%
9/121 • Number of events 10 • Up to 35 weeks after participant signed Master Protocol consent.
The total at risk number for serious adverse events and other adverse events differs from the overall numbers due to a participant excluded from full analysis set for efficacy analyses and baseline characteristics due to a diagnosis other than ALS in one participant and due to participant included in the full analysis set for efficacy analyses and baseline characteristics due to being randomized but who never initiated study drug and thus was not considered as risk for safety outcomes.
|
9.8%
4/41 • Number of events 5 • Up to 35 weeks after participant signed Master Protocol consent.
The total at risk number for serious adverse events and other adverse events differs from the overall numbers due to a participant excluded from full analysis set for efficacy analyses and baseline characteristics due to a diagnosis other than ALS in one participant and due to participant included in the full analysis set for efficacy analyses and baseline characteristics due to being randomized but who never initiated study drug and thus was not considered as risk for safety outcomes.
|
|
Nervous system disorders
Headache
|
5.0%
6/121 • Number of events 7 • Up to 35 weeks after participant signed Master Protocol consent.
The total at risk number for serious adverse events and other adverse events differs from the overall numbers due to a participant excluded from full analysis set for efficacy analyses and baseline characteristics due to a diagnosis other than ALS in one participant and due to participant included in the full analysis set for efficacy analyses and baseline characteristics due to being randomized but who never initiated study drug and thus was not considered as risk for safety outcomes.
|
2.4%
1/41 • Number of events 1 • Up to 35 weeks after participant signed Master Protocol consent.
The total at risk number for serious adverse events and other adverse events differs from the overall numbers due to a participant excluded from full analysis set for efficacy analyses and baseline characteristics due to a diagnosis other than ALS in one participant and due to participant included in the full analysis set for efficacy analyses and baseline characteristics due to being randomized but who never initiated study drug and thus was not considered as risk for safety outcomes.
|
|
Nervous system disorders
Muscle contractions involuntary
|
3.3%
4/121 • Number of events 4 • Up to 35 weeks after participant signed Master Protocol consent.
The total at risk number for serious adverse events and other adverse events differs from the overall numbers due to a participant excluded from full analysis set for efficacy analyses and baseline characteristics due to a diagnosis other than ALS in one participant and due to participant included in the full analysis set for efficacy analyses and baseline characteristics due to being randomized but who never initiated study drug and thus was not considered as risk for safety outcomes.
|
7.3%
3/41 • Number of events 4 • Up to 35 weeks after participant signed Master Protocol consent.
The total at risk number for serious adverse events and other adverse events differs from the overall numbers due to a participant excluded from full analysis set for efficacy analyses and baseline characteristics due to a diagnosis other than ALS in one participant and due to participant included in the full analysis set for efficacy analyses and baseline characteristics due to being randomized but who never initiated study drug and thus was not considered as risk for safety outcomes.
|
|
Nervous system disorders
Cognitive disorder
|
2.5%
3/121 • Number of events 3 • Up to 35 weeks after participant signed Master Protocol consent.
The total at risk number for serious adverse events and other adverse events differs from the overall numbers due to a participant excluded from full analysis set for efficacy analyses and baseline characteristics due to a diagnosis other than ALS in one participant and due to participant included in the full analysis set for efficacy analyses and baseline characteristics due to being randomized but who never initiated study drug and thus was not considered as risk for safety outcomes.
|
7.3%
3/41 • Number of events 3 • Up to 35 weeks after participant signed Master Protocol consent.
The total at risk number for serious adverse events and other adverse events differs from the overall numbers due to a participant excluded from full analysis set for efficacy analyses and baseline characteristics due to a diagnosis other than ALS in one participant and due to participant included in the full analysis set for efficacy analyses and baseline characteristics due to being randomized but who never initiated study drug and thus was not considered as risk for safety outcomes.
|
|
Gastrointestinal disorders
Constipation
|
11.6%
14/121 • Number of events 18 • Up to 35 weeks after participant signed Master Protocol consent.
The total at risk number for serious adverse events and other adverse events differs from the overall numbers due to a participant excluded from full analysis set for efficacy analyses and baseline characteristics due to a diagnosis other than ALS in one participant and due to participant included in the full analysis set for efficacy analyses and baseline characteristics due to being randomized but who never initiated study drug and thus was not considered as risk for safety outcomes.
|
19.5%
8/41 • Number of events 9 • Up to 35 weeks after participant signed Master Protocol consent.
The total at risk number for serious adverse events and other adverse events differs from the overall numbers due to a participant excluded from full analysis set for efficacy analyses and baseline characteristics due to a diagnosis other than ALS in one participant and due to participant included in the full analysis set for efficacy analyses and baseline characteristics due to being randomized but who never initiated study drug and thus was not considered as risk for safety outcomes.
|
|
Gastrointestinal disorders
Diarrhoea
|
12.4%
15/121 • Number of events 22 • Up to 35 weeks after participant signed Master Protocol consent.
The total at risk number for serious adverse events and other adverse events differs from the overall numbers due to a participant excluded from full analysis set for efficacy analyses and baseline characteristics due to a diagnosis other than ALS in one participant and due to participant included in the full analysis set for efficacy analyses and baseline characteristics due to being randomized but who never initiated study drug and thus was not considered as risk for safety outcomes.
|
9.8%
4/41 • Number of events 5 • Up to 35 weeks after participant signed Master Protocol consent.
The total at risk number for serious adverse events and other adverse events differs from the overall numbers due to a participant excluded from full analysis set for efficacy analyses and baseline characteristics due to a diagnosis other than ALS in one participant and due to participant included in the full analysis set for efficacy analyses and baseline characteristics due to being randomized but who never initiated study drug and thus was not considered as risk for safety outcomes.
|
|
Gastrointestinal disorders
Nausea
|
12.4%
15/121 • Number of events 18 • Up to 35 weeks after participant signed Master Protocol consent.
The total at risk number for serious adverse events and other adverse events differs from the overall numbers due to a participant excluded from full analysis set for efficacy analyses and baseline characteristics due to a diagnosis other than ALS in one participant and due to participant included in the full analysis set for efficacy analyses and baseline characteristics due to being randomized but who never initiated study drug and thus was not considered as risk for safety outcomes.
|
7.3%
3/41 • Number of events 3 • Up to 35 weeks after participant signed Master Protocol consent.
The total at risk number for serious adverse events and other adverse events differs from the overall numbers due to a participant excluded from full analysis set for efficacy analyses and baseline characteristics due to a diagnosis other than ALS in one participant and due to participant included in the full analysis set for efficacy analyses and baseline characteristics due to being randomized but who never initiated study drug and thus was not considered as risk for safety outcomes.
|
|
Gastrointestinal disorders
Dysphagia
|
10.7%
13/121 • Number of events 13 • Up to 35 weeks after participant signed Master Protocol consent.
The total at risk number for serious adverse events and other adverse events differs from the overall numbers due to a participant excluded from full analysis set for efficacy analyses and baseline characteristics due to a diagnosis other than ALS in one participant and due to participant included in the full analysis set for efficacy analyses and baseline characteristics due to being randomized but who never initiated study drug and thus was not considered as risk for safety outcomes.
|
12.2%
5/41 • Number of events 6 • Up to 35 weeks after participant signed Master Protocol consent.
The total at risk number for serious adverse events and other adverse events differs from the overall numbers due to a participant excluded from full analysis set for efficacy analyses and baseline characteristics due to a diagnosis other than ALS in one participant and due to participant included in the full analysis set for efficacy analyses and baseline characteristics due to being randomized but who never initiated study drug and thus was not considered as risk for safety outcomes.
|
|
Gastrointestinal disorders
Dry mouth
|
8.3%
10/121 • Number of events 10 • Up to 35 weeks after participant signed Master Protocol consent.
The total at risk number for serious adverse events and other adverse events differs from the overall numbers due to a participant excluded from full analysis set for efficacy analyses and baseline characteristics due to a diagnosis other than ALS in one participant and due to participant included in the full analysis set for efficacy analyses and baseline characteristics due to being randomized but who never initiated study drug and thus was not considered as risk for safety outcomes.
|
7.3%
3/41 • Number of events 3 • Up to 35 weeks after participant signed Master Protocol consent.
The total at risk number for serious adverse events and other adverse events differs from the overall numbers due to a participant excluded from full analysis set for efficacy analyses and baseline characteristics due to a diagnosis other than ALS in one participant and due to participant included in the full analysis set for efficacy analyses and baseline characteristics due to being randomized but who never initiated study drug and thus was not considered as risk for safety outcomes.
|
|
Gastrointestinal disorders
Salivary hypersecretion
|
8.3%
10/121 • Number of events 10 • Up to 35 weeks after participant signed Master Protocol consent.
The total at risk number for serious adverse events and other adverse events differs from the overall numbers due to a participant excluded from full analysis set for efficacy analyses and baseline characteristics due to a diagnosis other than ALS in one participant and due to participant included in the full analysis set for efficacy analyses and baseline characteristics due to being randomized but who never initiated study drug and thus was not considered as risk for safety outcomes.
|
4.9%
2/41 • Number of events 2 • Up to 35 weeks after participant signed Master Protocol consent.
The total at risk number for serious adverse events and other adverse events differs from the overall numbers due to a participant excluded from full analysis set for efficacy analyses and baseline characteristics due to a diagnosis other than ALS in one participant and due to participant included in the full analysis set for efficacy analyses and baseline characteristics due to being randomized but who never initiated study drug and thus was not considered as risk for safety outcomes.
|
|
Gastrointestinal disorders
Gastroesophageal reflux disease
|
3.3%
4/121 • Number of events 4 • Up to 35 weeks after participant signed Master Protocol consent.
The total at risk number for serious adverse events and other adverse events differs from the overall numbers due to a participant excluded from full analysis set for efficacy analyses and baseline characteristics due to a diagnosis other than ALS in one participant and due to participant included in the full analysis set for efficacy analyses and baseline characteristics due to being randomized but who never initiated study drug and thus was not considered as risk for safety outcomes.
|
9.8%
4/41 • Number of events 4 • Up to 35 weeks after participant signed Master Protocol consent.
The total at risk number for serious adverse events and other adverse events differs from the overall numbers due to a participant excluded from full analysis set for efficacy analyses and baseline characteristics due to a diagnosis other than ALS in one participant and due to participant included in the full analysis set for efficacy analyses and baseline characteristics due to being randomized but who never initiated study drug and thus was not considered as risk for safety outcomes.
|
|
Injury, poisoning and procedural complications
Fall
|
28.1%
34/121 • Number of events 65 • Up to 35 weeks after participant signed Master Protocol consent.
The total at risk number for serious adverse events and other adverse events differs from the overall numbers due to a participant excluded from full analysis set for efficacy analyses and baseline characteristics due to a diagnosis other than ALS in one participant and due to participant included in the full analysis set for efficacy analyses and baseline characteristics due to being randomized but who never initiated study drug and thus was not considered as risk for safety outcomes.
|
29.3%
12/41 • Number of events 28 • Up to 35 weeks after participant signed Master Protocol consent.
The total at risk number for serious adverse events and other adverse events differs from the overall numbers due to a participant excluded from full analysis set for efficacy analyses and baseline characteristics due to a diagnosis other than ALS in one participant and due to participant included in the full analysis set for efficacy analyses and baseline characteristics due to being randomized but who never initiated study drug and thus was not considered as risk for safety outcomes.
|
|
Injury, poisoning and procedural complications
Contusion
|
6.6%
8/121 • Number of events 11 • Up to 35 weeks after participant signed Master Protocol consent.
The total at risk number for serious adverse events and other adverse events differs from the overall numbers due to a participant excluded from full analysis set for efficacy analyses and baseline characteristics due to a diagnosis other than ALS in one participant and due to participant included in the full analysis set for efficacy analyses and baseline characteristics due to being randomized but who never initiated study drug and thus was not considered as risk for safety outcomes.
|
7.3%
3/41 • Number of events 6 • Up to 35 weeks after participant signed Master Protocol consent.
The total at risk number for serious adverse events and other adverse events differs from the overall numbers due to a participant excluded from full analysis set for efficacy analyses and baseline characteristics due to a diagnosis other than ALS in one participant and due to participant included in the full analysis set for efficacy analyses and baseline characteristics due to being randomized but who never initiated study drug and thus was not considered as risk for safety outcomes.
|
|
Injury, poisoning and procedural complications
Post lumbar puncture syndrome
|
3.3%
4/121 • Number of events 4 • Up to 35 weeks after participant signed Master Protocol consent.
The total at risk number for serious adverse events and other adverse events differs from the overall numbers due to a participant excluded from full analysis set for efficacy analyses and baseline characteristics due to a diagnosis other than ALS in one participant and due to participant included in the full analysis set for efficacy analyses and baseline characteristics due to being randomized but who never initiated study drug and thus was not considered as risk for safety outcomes.
|
9.8%
4/41 • Number of events 4 • Up to 35 weeks after participant signed Master Protocol consent.
The total at risk number for serious adverse events and other adverse events differs from the overall numbers due to a participant excluded from full analysis set for efficacy analyses and baseline characteristics due to a diagnosis other than ALS in one participant and due to participant included in the full analysis set for efficacy analyses and baseline characteristics due to being randomized but who never initiated study drug and thus was not considered as risk for safety outcomes.
|
|
Injury, poisoning and procedural complications
Skin abrasion
|
1.7%
2/121 • Number of events 2 • Up to 35 weeks after participant signed Master Protocol consent.
The total at risk number for serious adverse events and other adverse events differs from the overall numbers due to a participant excluded from full analysis set for efficacy analyses and baseline characteristics due to a diagnosis other than ALS in one participant and due to participant included in the full analysis set for efficacy analyses and baseline characteristics due to being randomized but who never initiated study drug and thus was not considered as risk for safety outcomes.
|
9.8%
4/41 • Number of events 6 • Up to 35 weeks after participant signed Master Protocol consent.
The total at risk number for serious adverse events and other adverse events differs from the overall numbers due to a participant excluded from full analysis set for efficacy analyses and baseline characteristics due to a diagnosis other than ALS in one participant and due to participant included in the full analysis set for efficacy analyses and baseline characteristics due to being randomized but who never initiated study drug and thus was not considered as risk for safety outcomes.
|
|
General disorders
Fatigue
|
9.9%
12/121 • Number of events 14 • Up to 35 weeks after participant signed Master Protocol consent.
The total at risk number for serious adverse events and other adverse events differs from the overall numbers due to a participant excluded from full analysis set for efficacy analyses and baseline characteristics due to a diagnosis other than ALS in one participant and due to participant included in the full analysis set for efficacy analyses and baseline characteristics due to being randomized but who never initiated study drug and thus was not considered as risk for safety outcomes.
|
12.2%
5/41 • Number of events 5 • Up to 35 weeks after participant signed Master Protocol consent.
The total at risk number for serious adverse events and other adverse events differs from the overall numbers due to a participant excluded from full analysis set for efficacy analyses and baseline characteristics due to a diagnosis other than ALS in one participant and due to participant included in the full analysis set for efficacy analyses and baseline characteristics due to being randomized but who never initiated study drug and thus was not considered as risk for safety outcomes.
|
|
General disorders
Oedema peripheral
|
7.4%
9/121 • Number of events 10 • Up to 35 weeks after participant signed Master Protocol consent.
The total at risk number for serious adverse events and other adverse events differs from the overall numbers due to a participant excluded from full analysis set for efficacy analyses and baseline characteristics due to a diagnosis other than ALS in one participant and due to participant included in the full analysis set for efficacy analyses and baseline characteristics due to being randomized but who never initiated study drug and thus was not considered as risk for safety outcomes.
|
7.3%
3/41 • Number of events 4 • Up to 35 weeks after participant signed Master Protocol consent.
The total at risk number for serious adverse events and other adverse events differs from the overall numbers due to a participant excluded from full analysis set for efficacy analyses and baseline characteristics due to a diagnosis other than ALS in one participant and due to participant included in the full analysis set for efficacy analyses and baseline characteristics due to being randomized but who never initiated study drug and thus was not considered as risk for safety outcomes.
|
|
General disorders
Complication associated with device
|
5.8%
7/121 • Number of events 8 • Up to 35 weeks after participant signed Master Protocol consent.
The total at risk number for serious adverse events and other adverse events differs from the overall numbers due to a participant excluded from full analysis set for efficacy analyses and baseline characteristics due to a diagnosis other than ALS in one participant and due to participant included in the full analysis set for efficacy analyses and baseline characteristics due to being randomized but who never initiated study drug and thus was not considered as risk for safety outcomes.
|
4.9%
2/41 • Number of events 2 • Up to 35 weeks after participant signed Master Protocol consent.
The total at risk number for serious adverse events and other adverse events differs from the overall numbers due to a participant excluded from full analysis set for efficacy analyses and baseline characteristics due to a diagnosis other than ALS in one participant and due to participant included in the full analysis set for efficacy analyses and baseline characteristics due to being randomized but who never initiated study drug and thus was not considered as risk for safety outcomes.
|
|
Respiratory, thoracic and mediastinal disorders
Dyspnoea
|
5.8%
7/121 • Number of events 7 • Up to 35 weeks after participant signed Master Protocol consent.
The total at risk number for serious adverse events and other adverse events differs from the overall numbers due to a participant excluded from full analysis set for efficacy analyses and baseline characteristics due to a diagnosis other than ALS in one participant and due to participant included in the full analysis set for efficacy analyses and baseline characteristics due to being randomized but who never initiated study drug and thus was not considered as risk for safety outcomes.
|
4.9%
2/41 • Number of events 2 • Up to 35 weeks after participant signed Master Protocol consent.
The total at risk number for serious adverse events and other adverse events differs from the overall numbers due to a participant excluded from full analysis set for efficacy analyses and baseline characteristics due to a diagnosis other than ALS in one participant and due to participant included in the full analysis set for efficacy analyses and baseline characteristics due to being randomized but who never initiated study drug and thus was not considered as risk for safety outcomes.
|
|
Infections and infestations
COVID-19
|
9.9%
12/121 • Number of events 12 • Up to 35 weeks after participant signed Master Protocol consent.
The total at risk number for serious adverse events and other adverse events differs from the overall numbers due to a participant excluded from full analysis set for efficacy analyses and baseline characteristics due to a diagnosis other than ALS in one participant and due to participant included in the full analysis set for efficacy analyses and baseline characteristics due to being randomized but who never initiated study drug and thus was not considered as risk for safety outcomes.
|
9.8%
4/41 • Number of events 4 • Up to 35 weeks after participant signed Master Protocol consent.
The total at risk number for serious adverse events and other adverse events differs from the overall numbers due to a participant excluded from full analysis set for efficacy analyses and baseline characteristics due to a diagnosis other than ALS in one participant and due to participant included in the full analysis set for efficacy analyses and baseline characteristics due to being randomized but who never initiated study drug and thus was not considered as risk for safety outcomes.
|
|
Musculoskeletal and connective tissue disorders
Musculoskeletal pain
|
7.4%
9/121 • Number of events 12 • Up to 35 weeks after participant signed Master Protocol consent.
The total at risk number for serious adverse events and other adverse events differs from the overall numbers due to a participant excluded from full analysis set for efficacy analyses and baseline characteristics due to a diagnosis other than ALS in one participant and due to participant included in the full analysis set for efficacy analyses and baseline characteristics due to being randomized but who never initiated study drug and thus was not considered as risk for safety outcomes.
|
4.9%
2/41 • Number of events 2 • Up to 35 weeks after participant signed Master Protocol consent.
The total at risk number for serious adverse events and other adverse events differs from the overall numbers due to a participant excluded from full analysis set for efficacy analyses and baseline characteristics due to a diagnosis other than ALS in one participant and due to participant included in the full analysis set for efficacy analyses and baseline characteristics due to being randomized but who never initiated study drug and thus was not considered as risk for safety outcomes.
|
|
Musculoskeletal and connective tissue disorders
Arthralgia
|
7.4%
9/121 • Number of events 13 • Up to 35 weeks after participant signed Master Protocol consent.
The total at risk number for serious adverse events and other adverse events differs from the overall numbers due to a participant excluded from full analysis set for efficacy analyses and baseline characteristics due to a diagnosis other than ALS in one participant and due to participant included in the full analysis set for efficacy analyses and baseline characteristics due to being randomized but who never initiated study drug and thus was not considered as risk for safety outcomes.
|
0.00%
0/41 • Up to 35 weeks after participant signed Master Protocol consent.
The total at risk number for serious adverse events and other adverse events differs from the overall numbers due to a participant excluded from full analysis set for efficacy analyses and baseline characteristics due to a diagnosis other than ALS in one participant and due to participant included in the full analysis set for efficacy analyses and baseline characteristics due to being randomized but who never initiated study drug and thus was not considered as risk for safety outcomes.
|
|
Musculoskeletal and connective tissue disorders
Pain in extremity
|
2.5%
3/121 • Number of events 3 • Up to 35 weeks after participant signed Master Protocol consent.
The total at risk number for serious adverse events and other adverse events differs from the overall numbers due to a participant excluded from full analysis set for efficacy analyses and baseline characteristics due to a diagnosis other than ALS in one participant and due to participant included in the full analysis set for efficacy analyses and baseline characteristics due to being randomized but who never initiated study drug and thus was not considered as risk for safety outcomes.
|
7.3%
3/41 • Number of events 3 • Up to 35 weeks after participant signed Master Protocol consent.
The total at risk number for serious adverse events and other adverse events differs from the overall numbers due to a participant excluded from full analysis set for efficacy analyses and baseline characteristics due to a diagnosis other than ALS in one participant and due to participant included in the full analysis set for efficacy analyses and baseline characteristics due to being randomized but who never initiated study drug and thus was not considered as risk for safety outcomes.
|
|
Psychiatric disorders
Anxiety
|
6.6%
8/121 • Number of events 8 • Up to 35 weeks after participant signed Master Protocol consent.
The total at risk number for serious adverse events and other adverse events differs from the overall numbers due to a participant excluded from full analysis set for efficacy analyses and baseline characteristics due to a diagnosis other than ALS in one participant and due to participant included in the full analysis set for efficacy analyses and baseline characteristics due to being randomized but who never initiated study drug and thus was not considered as risk for safety outcomes.
|
9.8%
4/41 • Number of events 4 • Up to 35 weeks after participant signed Master Protocol consent.
The total at risk number for serious adverse events and other adverse events differs from the overall numbers due to a participant excluded from full analysis set for efficacy analyses and baseline characteristics due to a diagnosis other than ALS in one participant and due to participant included in the full analysis set for efficacy analyses and baseline characteristics due to being randomized but who never initiated study drug and thus was not considered as risk for safety outcomes.
|
|
Psychiatric disorders
Insomnia
|
7.4%
9/121 • Number of events 10 • Up to 35 weeks after participant signed Master Protocol consent.
The total at risk number for serious adverse events and other adverse events differs from the overall numbers due to a participant excluded from full analysis set for efficacy analyses and baseline characteristics due to a diagnosis other than ALS in one participant and due to participant included in the full analysis set for efficacy analyses and baseline characteristics due to being randomized but who never initiated study drug and thus was not considered as risk for safety outcomes.
|
2.4%
1/41 • Number of events 1 • Up to 35 weeks after participant signed Master Protocol consent.
The total at risk number for serious adverse events and other adverse events differs from the overall numbers due to a participant excluded from full analysis set for efficacy analyses and baseline characteristics due to a diagnosis other than ALS in one participant and due to participant included in the full analysis set for efficacy analyses and baseline characteristics due to being randomized but who never initiated study drug and thus was not considered as risk for safety outcomes.
|
|
Psychiatric disorders
Depression
|
5.0%
6/121 • Number of events 6 • Up to 35 weeks after participant signed Master Protocol consent.
The total at risk number for serious adverse events and other adverse events differs from the overall numbers due to a participant excluded from full analysis set for efficacy analyses and baseline characteristics due to a diagnosis other than ALS in one participant and due to participant included in the full analysis set for efficacy analyses and baseline characteristics due to being randomized but who never initiated study drug and thus was not considered as risk for safety outcomes.
|
2.4%
1/41 • Number of events 1 • Up to 35 weeks after participant signed Master Protocol consent.
The total at risk number for serious adverse events and other adverse events differs from the overall numbers due to a participant excluded from full analysis set for efficacy analyses and baseline characteristics due to a diagnosis other than ALS in one participant and due to participant included in the full analysis set for efficacy analyses and baseline characteristics due to being randomized but who never initiated study drug and thus was not considered as risk for safety outcomes.
|
Additional Information
Healey Center for ALS Project Management
Healey Center for ALS at Massachusetts General Hospital
Phone: 833-425-8257 (HALT ALS)
Email: [email protected]
Results disclosure agreements
- Principal investigator is a sponsor employee
- Publication restrictions are in place