Trial · NCT04615923
HEALEY ALS Platform Trial - Regimen D Pridopidine
NCT04615923, Phase 2, Phase 3, Completed, Merit E. Cudkowicz, MD; results first posted 2023-08-23
3 sourcestrials-2026-11-12-0.5.0
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ConductScience Library. HEALEY ALS Platform Trial - Regimen D Pridopidine. Trials, record NCT04615923. Release trials-2026-11-12-0.5.0, 12 Nov 2026. https://conductscience.org/library/trials/NCT04615923
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Trial
As registered on ClinicalTrials.gov, in the graph's capture of the registry.
- Title
- HEALEY ALS Platform Trial - Regimen D Pridopidine
- NCT number
- NCT04615923
- Status
- Completed
- Phases
- Phase 2, Phase 3
- Enrollment
- 163 participants, actual
- Lead sponsor
- Merit E. Cudkowicz, MD
- Conditions
- Amyotrophic Lateral Sclerosis
- Interventions
- Pridopidine, Matching Placebo
- First posted
- 2020-11-04
- Last updated
- 2023-08-23
- Results first posted
- 2023-08-23
Source ClinicalTrials.gov
Registered outcomes9
Each outcome the trial registered, primary first, in ClinicalTrials.gov's words.
| List | Outcome | Time frame | Description |
|---|---|---|---|
| Primary | Disease Progression as Assessed by the ALSFRS-R Total Score | Baseline to 24 Weeks | 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. |
| Primary | Mortality Event Rate | Baseline to 24 Weeks | 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. |
| Secondary | Change in Bulbar Function in Participants With Bulbar Dysfunction at Baseline | Baseline to 24 Weeks | 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. |
| Secondary | Bulbar Function in All Randomized Participants | Baseline to 24 Weeks | 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. |
| Secondary | Respiratory Function | Baseline to 24 Weeks | Change in respiratory function over time as measured by Slow Vital Capacity (SVC). |
| Secondary | Bulbar Function in Participants With Rapid Pre-baseline Progression | Baseline to 24 Weeks | 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. |
| Secondary | Time to Bulbar Decline | Baseline to 24 Weeks | 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. |
| Secondary | Muscle Strength | Baseline to 24 Weeks | Change in muscle strength over time as measured isometrically using hand-held dynamometry (HHD). |
| Secondary | Number of Participants That Experienced Death or Death Equivalent | Baseline to 24 Weeks | 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. |
Source ClinicalTrials.gov
Outcomes9
Each outcome the trial posted results for, in ClinicalTrials.gov's words.
| Type | Outcome | Time frame | Population | Units | Measured as |
|---|---|---|---|---|---|
| PRIMARY | Disease Progression as Assessed by the ALSFRS-R Total Score | Baseline to 24 Weeks | 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. | points per month | MEAN |
| PRIMARY | Mortality Event Rate | Baseline to 24 Weeks | 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. | events per month | MEAN |
| SECONDARY | Change in Bulbar Function in Participants With Bulbar Dysfunction at Baseline | Baseline to 24 Weeks | 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. | point change from baseline | LEAST_SQUARES_MEAN |
| SECONDARY | Bulbar Function in All Randomized Participants | Baseline to 24 Weeks | 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. | point change from baseline | LEAST_SQUARES_MEAN |
| SECONDARY | Respiratory Function | Baseline to 24 Weeks | 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. | percent change | LEAST_SQUARES_MEAN |
| SECONDARY | Bulbar Function in Participants With Rapid Pre-baseline Progression | Baseline to 24 Weeks | 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. | points change from baseline | LEAST_SQUARES_MEAN |
| SECONDARY | Time to Bulbar Decline | Baseline to 24 Weeks | 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. | days | MEDIAN |
| SECONDARY | Muscle Strength | Baseline to 24 Weeks | 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. | percent change | LEAST_SQUARES_MEAN |
| SECONDARY | Number of Participants That Experienced Death or Death Equivalent | Baseline to 24 Weeks | 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. | Participants | COUNT_OF_PARTICIPANTS |
Source ClinicalTrials.gov
Results by group20
Each measurement the trial posted, group by group, in the words and numbers it posted.
| Outcome | Group | Category | Value | Spread | Units |
|---|---|---|---|---|---|
| Disease Progression as Assessed by the ALSFRS-R Total Score | Pridopidine | -0.99 | Standard Deviation 0.077 | points per month | |
| Disease Progression as Assessed by the ALSFRS-R Total Score | Matching Placebo | -1.00 | Standard Deviation 0.070 | points per month | |
| Mortality Event Rate | Pridopidine | 0.012 | Standard Deviation 0.0029 | events per month | |
| Mortality Event Rate | Matching Placebo | 0.012 | Standard Deviation 0.0029 | events per month | |
| Change in Bulbar Function in Participants With Bulbar Dysfunction at Baseline | Pridopidine | -1.78 | Standard Error 0.261 | point change from baseline | |
| Change in Bulbar Function in Participants With Bulbar Dysfunction at Baseline | Matching Placebo | -1.69 | Standard Error 0.210 | point change from baseline | |
| Bulbar Function in All Randomized Participants | Pridopidine | -1.16 | Standard Error 0.169 | point change from baseline | |
| Bulbar Function in All Randomized Participants | Matching Placebo | -1.25 | Standard Error 0.141 | point change from baseline | |
| Respiratory Function | Pridopidine | -8.81 | Standard Error 1.351 | percent change | |
| Respiratory Function | Matching Placebo | -8.35 | Standard Error 1.132 | percent change |
1 to 10 of 20Source ClinicalTrials.gov
See all 20 results by groupParticipant flow6
How many started, completed or left each group, period by period.
| Period | Milestone | Group | Participants | Units counted |
|---|---|---|---|---|
| Overall Study | STARTED | Pridopidine | 120 | |
| Overall Study | STARTED | Matching Placebo | 42 | |
| Overall Study | COMPLETED | Pridopidine | 96 | |
| Overall Study | COMPLETED | Matching Placebo | 38 | |
| Overall Study | NOT COMPLETED | Pridopidine | 24 | |
| Overall Study | NOT COMPLETED | Matching Placebo | 4 |
Source ClinicalTrials.gov
Adverse events90
Each adverse event the trial posted, group by group: the participants affected out of those at risk.
| Type | Event | Organ system | Group | Affected | At risk | Events |
|---|---|---|---|---|---|---|
| other | Muscular weakness | Nervous system disorders | Pridopidine | 29 | 121 | 33 |
| other | Muscular weakness | Nervous system disorders | Matching Placebo | 13 | 41 | 21 |
| other | Neuromyopathy | Nervous system disorders | Pridopidine | 24 | 121 | 37 |
| other | Neuromyopathy | Nervous system disorders | Matching Placebo | 3 | 41 | 3 |
| other | Dysarthria | Nervous system disorders | Pridopidine | 10 | 121 | 10 |
| other | Dysarthria | Nervous system disorders | Matching Placebo | 4 | 41 | 5 |
| other | Dizziness | Nervous system disorders | Pridopidine | 9 | 121 | 10 |
| other | Dizziness | Nervous system disorders | Matching Placebo | 4 | 41 | 5 |
| other | Headache | Nervous system disorders | Pridopidine | 6 | 121 | 7 |
| other | Headache | Nervous system disorders | Matching Placebo | 1 | 41 | 1 |
1 to 10 of 90Source ClinicalTrials.gov
See all 90 adverse eventsAdverse event totals6
The totals the trial posted for each group.
| Total | Group | Affected | At risk |
|---|---|---|---|
| Total, all-cause mortality | Pridopidine | 2 | 120 |
| Total, all-cause mortality | Matching Placebo | 0 | 42 |
| Total, other adverse events | Pridopidine | 106 | 121 |
| Total, other adverse events | Matching Placebo | 37 | 41 |
| Total, serious adverse events | Pridopidine | 16 | 121 |
| Total, serious adverse events | Matching Placebo | 3 | 41 |
Source ClinicalTrials.gov
Papers2
The papers the trial lists on ClinicalTrials.gov.
Grants0
The grants NIH RePORTER links to the trial, newest funded first: NIH's, and some of AHRQ's, CDC's, FDA's and VA's. Each shows its title, principal investigators, organization and funder in its newest year.
None in this release.
Trial comparisons3
Comparisons of the trial's two groups, calculated by ConductScience from the counts it posted.
| Comparison | Source |
|---|---|
| Pridopidine vs Matching Placebo: deaths from any cause | Trial comparisons |
| Pridopidine vs Matching Placebo: Number of Participants That Experienced Death or Death Equivalent | Trial comparisons |
| Pridopidine vs Matching Placebo: serious adverse events | Trial comparisons |
Tests0
The ConductCognition tests this trial's registered outcomes name, as ScienceGraph matched them in trial-tests-2026-11-12-0.2.1.
None in this release.
Data dates
The day each source was copied for this release.
Sources 3
- ClinicalTrials.gov's posted results, through CTTI's AACTFreely available. Acknowledgement requested
- ClinicalTrials.govNo license stated
- NIH RePORTER's clinical studies and project filesPublic domain
Each source's terms
- ClinicalTrials.gov's posted results, through CTTI's AACTClinicalTrials.gov data via Aggregate Analysis of ClinicalTrials.gov (AACT), CTTI. CTTI makes its materials freely available and requests acknowledgement. https://aact.ctti-clinicaltrials.org/documentation/299
- ClinicalTrials.govnone stated
- NIH RePORTER's clinical studies and project filesPublic domain (NIH RePORTER)
ClinicalTrials.gov's data carry an international copyright outside the United States: https://clinicaltrials.gov/about-site/terms-conditions.
For software
Read this trial from software.
The record and each of its lists are JSON, twenty rows a page. No key.
GET https://conductscience.org/api/trials/v1/NCT04615923
GET https://conductscience.org/api/trials/v1/NCT04615923/events?page=2