Amyotrophic Lateral SclerosisPhase 2/3NCT04615923
HEALEY ALS Platform Trial - Regimen D Pridopidine
A Phase 2/3 study testing Pridopidine in people with Amyotrophic Lateral Sclerosis, run by Merit E. Cudkowicz, MD at 1 site in United States.
Trial summary
The HEALEY ALS Platform Trial is a perpetual multi-center, multi-regimen clinical trial evaluating the safety and efficacy of investigational products for the treatment of ALS. Regimen D will evaluate the safety and efficacy of a single study drug, pridopidine, in participants with ALS. Read more about this study
- Who is it for?
- Ages 18 years and older
- Conditions the study is for
- Amyotrophic Lateral Sclerosis
- What is being tested?
- Pridopidine
- Type of treatment
- Pill or oral medicine
- Could I receive a placebo?
- Yes, some participants may receive a placebo
- How many people
- 163 participants
- Where
- 1 siteUnited States
- Study dates
- 18 Dec 2020to 14 Jul 2022
- Phase
- Phase 2/3
- Are there results?
- Yes, posted in the registrySummary in the doctors' view
Key requirements
Who can take part, as listed by the sponsor. Only the study team can confirm whether you are eligible, so take this list to your doctor.
- Ages 18 years and older
- All sexes
- No healthy volunteers
- Adult, Older adult
Who may be able to join
- No additional inclusion criteria beyond the inclusion criteria specified in the Master Protocol (NCT NCT04297683).
Who cannot join
- The following exclusion criteria are in addition to the exclusion criteria specified in the Master Protocol (NCT NCT04297683).
- Participants with a confirmed prolonged Fridericia-corrected QT (QTcF) interval (defined as a QTcF interval of >450 ms for men and >470 ms for women).
- Participants with clinically significant heart disease, clinically significant history of arrhythmia, symptomatic or uncontrolled atrial fibrillation despite treatment, or asymptomatic sustained ventricular tachycardia, or presence of left bundle branch block.
- Participants with known history of long QT syndrome or a first degree relative with this condition.
- Participants using prohibited medications within the 4 weeks prior to the Regimen Specific Screening Visit, as detailed in section 5.9.
- Participants using the following medications at the time of the Regimen Specific Screening Visit:
Show all 10Show fewer
- Nuedexta - at a dosage higher than 20 mg dextromethorphan + 10 mg quinidine BID
- Citalopram - at a dosage higher than 20 mg/day
- Escitalopram - at a dosage higher than 10 mg/day
- Participants with a known allergy to any ingredient of the study intervention (pridopidine, silicified microcrystalline cellulose, and magnesium stearate).
Trial locations and contacts
1 site in 1 country.
United States1 site
Healey Center for ALS at Mass General
Boston, Massachusetts, 02114
About this study
The HEALEY ALS Platform Trial is a perpetual multi-center, multi-regimen clinical trial evaluating the safety and efficacy of investigational products for the treatment of ALS.
Regimen D will evaluate the safety and efficacy of a single study drug, pridopidine, in participants with ALS.
Read the detailed description
The HEALEY ALS Platform Trial is a perpetual multi-center, multi-regimen clinical trial evaluating the safety and efficacy of investigational products for the treatment of ALS. This trial is designed as a perpetual platform trial. This means that there is a single Master Protocol dictating the conduct of the trial. The HEALEY ALS Platform Trial Master Protocol is registered as NCT04297683.
Once a participant enrolls into the Master Protocol and meets all eligibility criteria, the participant will be eligible to be randomized into any currently enrolling regimen. All participants will have an equal chance of being randomized to any currently enrolling regimen.
If a participant is randomized to Regimen D Pridopidine, the participant will complete a screening visit to assess additional Regimen D eligibility criteria. Once Regimen D eligibility criteria are confirmed, participants will complete a baseline assessment and be randomized in a 3:1 ratio to either active pridopidine or matching placebo.
Regimen D will enroll by invitation, as participants may not choose to enroll in Regimen D. Participants must first enroll into the Master Protocol and be eligible to participate in the Master Protocol before being able to be randomly assigned to Regimen D.
For a list of enrolling sites, please see the HEALEY ALS Platform Trial Master Protocol under NCT04297683.
- Conditions
- Amyotrophic Lateral Sclerosis
- Being tested
- Pridopidine, Matching Placebo
- Keywords
- ALS, Placebo-Controlled, Double-Blind, Regimen Specific Appendix, Lou Gehrig's Disease, Pridopidine, Prilenia Therapeutics
- Medical subject headings
- Amyotrophic Lateral Sclerosis
What participants receive
What participants receive. In a randomised study each person is assigned to one group by chance.
PridopidineDrug
Administration: Oral
Dose: 45mg twice daily
Matching PlaceboDrug
Administration: Oral
Dose: one capsule twice daily
| Study group | Role | What they receive |
|---|---|---|
| Pridopidine | Experimental | Pridopidine is administered orally twice daily for 24 weeks. Receives: Drug: Pridopidine |
| Matching Placebo | Placebo comparator | Matching placebo is administered orally twice daily for 24 weeks. Receives: Drug: Matching Placebo |
What is being measured
The primary outcome is the main question the study is designed to answer.
Disease Progression as Assessed by the ALSFRS-R Total Score
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.
Measured: Baseline to 24 Weeks
Mortality Event Rate
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.
Measured: Baseline to 24 Weeks
Secondary outcomes (7)
Change in Bulbar Function in Participants With Bulbar Dysfunction at Baseline
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.
Measured: Baseline to 24 Weeks
Bulbar Function in All Randomized Participants
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.
Measured: Baseline to 24 Weeks
Respiratory Function
Change in respiratory function over time as measured by Slow Vital Capacity (SVC).
Measured: Baseline to 24 Weeks
Bulbar Function in Participants With Rapid Pre-baseline Progression
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.
Measured: Baseline to 24 Weeks
Time to Bulbar Decline
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.
Measured: Baseline to 24 Weeks
Muscle Strength
Change in muscle strength over time as measured isometrically using hand-held dynamometry (HHD).
Measured: Baseline to 24 Weeks
Number of Participants That Experienced Death or Death Equivalent
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.
Measured: Baseline to 24 Weeks
Timeline
From the start of the study to its completion; the mark is today. Estimated dates are the sponsor’s plan.
- First posted4 Nov 2020
- Study start18 Dec 2020
- Primary completion14 Jul 2022
- Study completion14 Jul 2022
Resources
- Official record on ClinicalTrials.govThe registry entry this page is built from, with its full change history.
- Posted resultsParticipant flow, outcome measures and adverse events reported by the sponsor.
- Publications (2)Papers the sponsor has linked to this study.
- Other trials for Amyotrophic Lateral SclerosisStudies of the same condition, open and under way.
- What the trial phases meanHow a treatment moves from first tests in people to approval.
More details
For doctors and researchers: how the study is designed, who runs it, and its registry IDs.
Study design
- Study type
- Interventional
- Phase
- Phase 2/3
- Primary purpose
- Treatment
- Allocation
- Randomised
- Intervention model
- Parallel groups
- Masking
- Quadruple blind
- Who is masked
- Participant, Care provider, Investigator, Outcomes assessor
- Enrolment
- 163 participants (actual)
Sponsor and investigators
- Lead sponsor
- Merit E. Cudkowicz, MD
- Sponsor type
- University, hospital or other
- Collaborators
- Prilenia
- Responsible party
- Sponsor-investigator, Merit E. Cudkowicz, MD, Massachusetts General Hospital
- Investigators
- Merit Cudkowicz, MD, Principal investigator, Massachusetts General Hospital
IDs, dates and publications
- ClinicalTrials.gov ID
- NCT04615923
- Sponsor's protocol ID
- 2019P003518D
- Status last verified
- Aug 2023
- First submitted
- 29 Oct 2020
- Last updated
- 23 Aug 2023
- Results posted
- Yes, on ClinicalTrials.gov
- Data monitoring committee
- Yes
- FDA-regulated drug
- Yes
- FDA-regulated device
- No
- Individual data sharing
- No
Publications
Geva M, Goldberg YP, Leitner ML, Cruz-Herranz A, Hand R, Chen K, Gershoni Emek N, Tan AM, Paganoni S, Berry JD, Macklin EA, Shefner JM, Cudkowicz ME, Hayden MR. Pridopidine treatment in ALS: subgroup analyses from the HEALEY ALS Platform trial. Amyotroph Lateral Scler Frontotemporal Degener. 2026 May;27(3-4):432-444. doi: 10.1080/21678421.2025.2597935. Epub 2025 Dec 17.
PubMed 41406304Writing Committee for the HEALEY ALS Platform Trial; Shefner JM, Oskarsson B, Macklin EA, Chibnik LB, Quintana M, Saville BR, Detry MA, Vestrucci M, Marion J, McGlothlin A, Heiman-Patterson T, Chase M, Pothier L, Harkey BA, Yu H, Sherman AV, Hall M, Kittle G, Berry JD, Babu S, Andrews J, D'Agostino D, Tustison E, Scirocco E, Giacomelli E, Alameda G, Locatelli E, Ho D, Quick A, Ajroud-Driss S, Katz J, Heitzman D, Appel SH, Shroff S, Felice K, Maragakis NJ, Simmons Z, Miller TM, Olney N, Weiss MD, Goutman SA, Fernandes JA, Jawdat O, Owegi MA, Foster LA, Vu T, Ilieva H, Newman DS, Arcila-Londono X, Jackson CE, Ladha S, Caress JB, Swenson A, Peltier A, Lewis RA, Fee D, Elliott M, Bedlack R, Kasarskis EJ, Elman L, Rosenfeld J, Walk D, McIlduff C, Twydell P, Young E, Johnson K, Rezania K, Goyal NA, Cohen JA, Benatar M, Jones V, Shah J, Beydoun SR, Wymer JP, Zilliox L, Nayar S, Pattee GL, Martinez-Thompson J, Leitner ML, Chen K, Goldberg YP, Cohen Y, Geva M, Hayden MR, Paganoni S, Cudkowicz ME; HEALEY ALS Platform Trial Study Group. Pridopidine in Amyotrophic Lateral Sclerosis: The HEALEY ALS Platform Trial. JAMA. 2025 Feb 17;333(13):1128-37. doi: 10.1001/jama.2024.26429. Online ahead of print.
PubMed 40067755
Study at a glance
From the registry record, last updated by the sponsor on 23 Aug 2023. Recruitment status is as the sponsor reported it: confirm with the study team.
- Official title
- HEALEY ALS Platform Trial - Regimen D Pridopidine
- ClinicalTrials.gov ID
- NCT04615923
- Acronym / sponsor ID
- 2019P003518D
- Conditions
- Amyotrophic Lateral Sclerosis
- Keywords
- ALS, Placebo-Controlled, Double-Blind, Regimen Specific Appendix, Lou Gehrig's Disease, Pridopidine, Prilenia Therapeutics
- Phase
- Phase 2/3
- Study type
- Interventional
- Recruitment status
- Completed
- Enrolment
- 163 participants (actual)
- Lead sponsor
- Merit E. Cudkowicz, MDUniversity, hospital or other
- Collaborators
- Prilenia
- Study start
- 18 Dec 2020 (actual)
- Primary completion
- 14 Jul 2022 (actual)
- Study completion
- 14 Jul 2022 (actual)
- First posted
- 4 Nov 2020 (actual)
- Results
- Posted 23 Aug 2023
Clinical summary
The research question as the sponsor registered it. This describes what the study plans to test, not what it found.
The HEALEY ALS Platform Trial is a perpetual multi-center, multi-regimen clinical trial evaluating the safety and efficacy of investigational products for the treatment of ALS.
Regimen D will evaluate the safety and efficacy of a single study drug, pridopidine, in participants with ALS.
Detailed description
The HEALEY ALS Platform Trial is a perpetual multi-center, multi-regimen clinical trial evaluating the safety and efficacy of investigational products for the treatment of ALS. This trial is designed as a perpetual platform trial. This means that there is a single Master Protocol dictating the conduct of the trial. The HEALEY ALS Platform Trial Master Protocol is registered as NCT04297683.
Once a participant enrolls into the Master Protocol and meets all eligibility criteria, the participant will be eligible to be randomized into any currently enrolling regimen. All participants will have an equal chance of being randomized to any currently enrolling regimen.
If a participant is randomized to Regimen D Pridopidine, the participant will complete a screening visit to assess additional Regimen D eligibility criteria. Once Regimen D eligibility criteria are confirmed, participants will complete a baseline assessment and be randomized in a 3:1 ratio to either active pridopidine or matching placebo.
Regimen D will enroll by invitation, as participants may not choose to enroll in Regimen D. Participants must first enroll into the Master Protocol and be eligible to participate in the Master Protocol before being able to be randomly assigned to Regimen D.
For a list of enrolling sites, please see the HEALEY ALS Platform Trial Master Protocol under NCT04297683.
Study design
- Primary purpose
- Treatment
- Allocation
- Randomised
- Intervention model
- Parallel groups
- Masking
- Quadruple blind (participant, care provider, investigator, outcomes assessor)
- Number of arms
- 2
| Arm | Type | Receives |
|---|---|---|
| Pridopidine | Experimental | Drug: PridopidinePridopidine is administered orally twice daily for 24 weeks. |
| Matching Placebo | Placebo comparator | Drug: Matching PlaceboMatching placebo is administered orally twice daily for 24 weeks. |
Who may qualify
- Ages
- 18 years and older
- Sex
- All
- Healthy volunteers
- Not accepted
- Age groups
- Adult, Older adult
Key inclusion criteria (1 listed)
- No additional inclusion criteria beyond the inclusion criteria specified in the Master Protocol (NCT NCT04297683).
Key exclusion criteria (10 listed)
- The following exclusion criteria are in addition to the exclusion criteria specified in the Master Protocol (NCT NCT04297683).
Eligibility is decided by the study team after screening; this overview does not establish that a patient qualifies. Full inclusion and exclusion criteria.
Interventions
| Intervention | Type | Arms |
|---|---|---|
| PridopidineAdministration: Oral Dose: 45mg twice daily | Drug | Pridopidine |
| Matching PlaceboAdministration: Oral Dose: one capsule twice daily | Drug | Matching Placebo |
Primary outcome
Disease Progression as Assessed by the ALSFRS-R Total Score
Time frame: 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.
Mortality Event Rate
Time frame: 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.
7 secondary outcomes
Change in Bulbar Function in Participants With Bulbar Dysfunction at Baseline
Time frame: Baseline to 24 Weeks
Bulbar Function in All Randomized Participants
Time frame: Baseline to 24 Weeks
Respiratory Function
Time frame: Baseline to 24 Weeks
Bulbar Function in Participants With Rapid Pre-baseline Progression
Time frame: Baseline to 24 Weeks
Time to Bulbar Decline
Time frame: Baseline to 24 Weeks
Muscle Strength
Time frame: Baseline to 24 Weeks
Number of Participants That Experienced Death or Death Equivalent
Time frame: Baseline to 24 Weeks
Results and safety
Loading the posted results from ClinicalTrials.gov…
Research links
- Official record on ClinicalTrials.govLast updated by the sponsor 23 Aug 2023.
- Posted resultsParticipant flow, baseline, all outcome measures and adverse events.
- Derived from this study: PubMed 41406304Geva M, Goldberg YP, Leitner ML, Cruz-Herranz A, Hand R, Chen K, Gershoni Emek N, Tan AM, Paganoni S, Berry JD, Macklin EA, Shefner JM, Cudkowicz ME, Hayden MR. Pridopidine treatment in ALS: subgroup analyses from the HEALEY ALS Platform trial. Amyotroph Lateral Scler Frontotemporal Degener. 2026 May;27(3-4):432-444. doi: 10.1080/21678421.2025.2597935. Epub 2025 Dec 17.
- Derived from this study: PubMed 40067755Writing Committee for the HEALEY ALS Platform Trial; Shefner JM, Oskarsson B, Macklin EA, Chibnik LB, Quintana M, Saville BR, Detry MA, Vestrucci M, Marion J, McGlothlin A, Heiman-Patterson T, Chase M, Pothier L, Harkey BA, Yu H, Sherman AV, Hall M, Kittle G, Berry JD, Babu S, Andrews J, D'Agostino D, Tustison E, Scirocco E, Giacomelli E, Alameda G, Locatelli E, Ho D, Quick A, Ajroud-Driss S, Katz J, Heitzman D, Appel SH, Shroff S, Felice K, Maragakis NJ, Simmons Z, Miller TM, Olney N, Weiss MD, Goutman SA, Fernandes JA, Jawdat O, Owegi MA, Foster LA, Vu T, Ilieva H, Newman DS, Arcila-Londono X, Jackson CE, Ladha S, Caress JB, Swenson A, Peltier A, Lewis RA, Fee D, Elliott M, Bedlack R, Kasarskis EJ, Elman L, Rosenfeld J, Walk D, McIlduff C, Twydell P, Young E, Johnson K, Rezania K, Goyal NA, Cohen JA, Benatar M, Jones V, Shah J, Beydoun SR, Wymer JP, Zilliox L, Nayar S, Pattee GL, Martinez-Thompson J, Leitner ML, Chen K, Goldberg YP, Cohen Y, Geva M, Hayden MR, Paganoni S, Cudkowicz ME; HEALEY ALS Platform Trial Study Group. Pridopidine in Amyotrophic Lateral Sclerosis: The HEALEY ALS Platform Trial. JAMA. 2025 Feb 17;333(13):1128-37. doi: 10.1001/jama.2024.26429. Online ahead of print.
- Individual participant dataSharing planned: No
Related trials for Amyotrophic Lateral Sclerosis
Enrolling by invitationPhase 2
The BANYAN Trial, an ALS MyMatch Trial Evaluating Safety, Biomarker Activity, and Microglial Activation of Nasal Foralumab
ALS (Amyotrophic Lateral Sclerosis), ALS - Amyotrophic Lateral Sclerosis
RecruitingObservational study
Imaging Assessment of Swallowing and Speech Muscles in Inclusion Body Myositis
IBM, Inclusion Body Myositis
RecruitingObservational study
Register for Medical Data, Functional Scales, and Care Outcomes in ALS, SMA, and Other Motor System Disorders
ALS (Amyotrophic Lateral Sclerosis), SMA - Spinal Muscular Atrophy
RecruitingPhase 3
Pridopidine Phase 3 Study to Evaluate Efficacy and Safety in ALS
Amyotrophic Lateral Sclerosis
RecruitingObservational study
Investigating Complex Neurodegenerative Disorders Related to Amyotrophic Lateral Sclerosis and Frontotemporal Dementia
Frontotemporal Dementia, Amyotrophic Lateral Sclerosis
Referral and contact
AzTrials does not enrol participants or take referrals. Contact the study team or a site listed below; they decide who can take part. This study is not recruiting at the moment.
Merit Cudkowicz, MD principal investigator
Massachusetts General Hospital
1 site in 1 country: see every site and its status.
Questions about this trial
What is this clinical trial studying?
A Phase 2/3 study testing Pridopidine in people with Amyotrophic Lateral Sclerosis, run by Merit E. Cudkowicz, MD at 1 site in United States.
The full registered description is in the About this study section and the official record.
Who may be eligible to take part?
The sponsor lists 1 inclusion and 10 exclusion criteria. Eligibility is decided by the study team after screening, so read the full criteria and contact a listed site.
Is this clinical trial currently recruiting?
The record says: finished, as of the sponsor's last update on 23 Aug 2023. Status can change between updates, so confirm with the study contact before making plans.
Where is the study taking place?
The record lists 1 site in United States. Whether a site is taking part right now should be confirmed with that site. See every site.
What are the possible benefits and risks?
They depend on the study and the treatment, and no study can promise a benefit. Some participants in this study may receive a placebo. The study team explains the known risks during informed consent, before anyone joins; the record itself does not list them.
What does this combined phase mean?
Phase 2 studies give the treatment to a larger group to see whether it has the intended effect and to keep checking safety.
Phase 3 studies compare the treatment with the current standard treatment or a placebo in a large group, to confirm how well it works and watch for side effects.
Not every study follows the same path, and some studies (observational studies, device studies) have no phase.
Are results available for this study?
Yes. Results are posted in the registry (first posted 23 Aug 2023). A summary is in the For doctors view, and the full results are on ClinicalTrials.gov.
How can a doctor refer a patient or contact the research team?
The record lists no contact. The official record names the sponsor, who can be asked directly. AzTrials does not enrol participants or take referrals.
What does the primary outcome measure?
Disease Progression as Assessed by the ALSFRS-R Total Score (time frame: Baseline to 24 Weeks). The study lists 2 primary outcomes in all. It is what the study was designed to measure, not a finding.
Where can I read the original study information?
On the ClinicalTrials.gov record for NCT04615923, which also lists 2 linked publications.
Does a listing mean the treatment is approved or proven to work?
No. A listing means the study is registered. It does not show that the treatment is safe, works, or is approved by a regulator.
How often is this information updated?
AzTrials copies changed records from ClinicalTrials.gov every night and reloads the whole registry every week. The sponsor last updated this record on 23 Aug 2023; AzTrials last refreshed its copy on 29 Oct 2026.