HEALEY ALS Platform Trial - Regimen D Pridopidine

NCT04615923 | Completed Phase 2 Results DMC FDA Drug

• 1 other identifier: 2019P003518D
• Study Type: interventional
• Target: 163
• Locations: 1 country
• Sites: 1

Brief 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.

Conditions

Amyotrophic Lateral Sclerosis

Keywords

ALS; Placebo-Controlled; Double-Blind; Regimen Specific Appendix; Lou Gehrig's Disease; Pridopidine; Prilenia Therapeutics

Outcome Measures

Primary Outcomes (2)

• 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.

  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.

  Baseline to 24 Weeks

Secondary Outcomes (7)

• Change in Bulbar Function in Participants With Bulbar Dysfunction at Baseline

  Baseline to 24 Weeks

• Bulbar Function in All Randomized Participants

  Baseline to 24 Weeks

• Respiratory Function

  Baseline to 24 Weeks

• Bulbar Function in Participants With Rapid Pre-baseline Progression

  Baseline to 24 Weeks

• Time to Bulbar Decline

  Baseline to 24 Weeks

Study Arms (2)

Pridopidine

EXPERIMENTAL

Pridopidine is administered orally twice daily for 24 weeks.

Drug: Pridopidine

Matching Placebo

PLACEBO COMPARATOR

Matching placebo is administered orally twice daily for 24 weeks.

Drug: Matching Placebo

Interventions

Pridopidine DRUG

Administration: Oral Dose: 45mg twice daily

Pridopidine

Matching Placebo DRUG

Administration: Oral Dose: one capsule twice daily

Matching Placebo

Eligibility Criteria

• Age: 18 Years+
• Sex: all
• Healthy Volunteers: No
• Age Groups: Adult (18-64), Older Adult (65+)

You may not qualify if:

• 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:
• 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).

Sponsors & Collaborators

• Merit E. Cudkowicz, MD: lead
• Prilenia: collaborator

Study Sites (1)

Healey Center for ALS at Mass General

Boston, Massachusetts, 02114, United States

Location

Related Publications (1)

• 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.

  PMID: 40067755 DERIVED

MeSH Terms

Conditions

Amyotrophic Lateral Sclerosis

Interventions

pridopidine

Condition Hierarchy (Ancestors)

Spinal Cord Diseases; Central Nervous System Diseases; Nervous System Diseases; Motor Neuron Disease; Neurodegenerative Diseases; TDP-43 Proteinopathies; Neuromuscular Diseases; Proteostasis Deficiencies; Metabolic Diseases; Nutritional and Metabolic Diseases

Results Point of Contact

• Title: Healey Center for ALS Project Management
• Organization: Healey Center for ALS at Massachusetts General Hospital

healeyalsplatform@mgh.harvard.edu

833-425-8257 (HALT ALS)

Study Officials

• Merit Cudkowicz, MD

  Massachusetts General Hospital

  PRINCIPAL INVESTIGATOR

Publication Agreements

• PI is Sponsor Employee: Yes

Study Design

• Study Type: interventional
• Phase: phase 2
• Allocation: RANDOMIZED
• Masking: QUADRUPLE
• Who Masked: PARTICIPANT, CARE PROVIDER, INVESTIGATOR, OUTCOMES ASSESSOR
• Purpose: TREATMENT
• Intervention Model: PARALLEL
• Sponsor Type: OTHER
• Responsible Party: SPONSOR INVESTIGATOR
• PI Title: Chief, Neurology Department

Study Record Dates

• First Submitted: October 29, 2020
• First Posted: November 4, 2020
• Study Start: December 18, 2020
• Primary Completion: July 14, 2022
• Study Completion: July 14, 2022
• Last Updated: August 23, 2023
• Results First Posted: August 23, 2023

Record last verified: 2023-08

Data Sharing

• IPD Sharing: Will not share

Locations

US (1)