HEALEY ALS Platform Trial - Regimen D Pridopidine
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.
Timeline
- Start
- 2020-12-18
- Primary completion
- 2022-07-14
- Completion
- 2022-07-14
Outcome
Missed primary endpoint
paper Although the primary endpoint (ALS Functional Rating Scale-Revised (ALSFRS-R) total score accounting for survival at 24 weeks) was not met PMID 41406304 ↗
paper there was no significant difference between pridopidine and placebo in the primary end point PMID 40067755 ↗
release “While pridopidine did not meet the primary endpoint of change from baseline to week 24 in the Revised Amyotrophic Lateral Sclerosis Functional Rating Scale (ALSFRS-R)” prilenia.com ↗
release “pridopidine did not meet the primary outcome measure, data from the Phase 2 HEALEY ALS Platform Trial” prilenia.com ↗
release “The HEALEY trial did not meet its primary or secondary endpoints in the full population” prilenia.com ↗
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.
- Writing 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.
Drugs
| Evaluation | Drug | Modality | Dose | Route |
|---|---|---|---|---|
| Subject | Pridopidine | Small molecule | 45 mg | Oral |