HEALEY ALS Platform Trial - Regimen B Verdiperstat

NCT04436510 | Completed Phase 2 Results DMC FDA Drug

• 1 other identifier: 2019P003518B
• Study Type: interventional
• Target: 167
• 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 B will evaluate the safety and efficacy of a single study drug, verdiperstat, in participants with ALS.

Conditions

Amyotrophic Lateral Sclerosis

Keywords

ALS; Placebo-Controlled; Double-Blind; Regimen Specific Appendix; Lou Gehrig's Disease; Verdiperstat; Biohaven Pharmaceuticals

Outcome Measures

Primary Outcomes (2)

• Disease Progression as Assessed by the ALSFRS-R-Slope

  Change in disease severity as measured by the ALS Functional Rating Scale-Revised (ALSFRS-R) total score using a Bayesian repeated measures model that accounts for loss to follow-up due to mortality. Each of 12 questions assessing distinct functional ability 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 Even 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 (3)

• Respiratory Function

  Baseline to 24 Weeks

• Muscle Strength

  Baseline to 24 Weeks

• Number of Participants That Experienced Death or Death Equivalent

  24 Weeks

Study Arms (2)

Verdiperstat

EXPERIMENTAL

Verdiperstat is administered twice daily p.o. for 24 weeks.

Drug: Verdiperstat

Matching Placebo

PLACEBO COMPARATOR

Matching placebo is administered twice daily p.o. for 24 weeks.

Drug: Matching Placebo

Interventions

Matching Placebo DRUG

Drug: Matching Placebo Administration: Oral Dose: two tablets twice daily

Matching Placebo

Verdiperstat DRUG

Drug: Verdiperstat Administration: Oral Dose: 600mg twice daily

Verdiperstat

Eligibility Criteria

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

You may not qualify if:

• Participants who are taking strong inhibitors of CYP1A2 (i.e., ciprofloxacin, enoxacin, fluvoxamine, zafirlukast) for chronic/long-term use defined as more than two weeks.

Sponsors & Collaborators

• Merit E. Cudkowicz, MD: lead
• Biohaven Pharmaceuticals, Inc.: 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; Andrews J, Paganoni S, Macklin EA, Chibnik LB, Quintana M, Saville BR, Detry MA, Vestrucci M, Marion J, McGlothlin A, Young E, Chase M, Pothier L, Harkey B, Yu H, Sherman A, Shefner J, Hall M, Kittle G, Connolly MR, Berry JD, D'Agostino D, Tustison E, Giacomelli E, Scirocco E, Alameda G, Locatelli E, Ho D, Quick A, Heitzman D, Ajroud-Driss S, Appel SH, Shroff S, Katz J, Felice K, Maragakis NJ, Simmons Z, Goutman SA, Olney N, Miller T, Fernandes JA, Ilieva H, Jawdat O, Weiss MD, Foster L, Vu T, Ladha S, Owegi MA, Newman DS, Arcila-Londono X, Jackson CE, Swenson A, Heiman-Patterson T, Caress J, Fee D, Peltier A, Lewis R, Rosenfeld J, Walk D, Johnson K, Elliott M, Kasarskis EJ, Rutkove S, McIlduff CE, Bedlack R, Elman L, Goyal NA, Rezania K, Twydell P, Benatar M, Glass J, Cohen JA, Jones V, Zilliox L, Wymer JP, Beydoun SR, Shah J, Pattee GL, Martinez-Thompson J, Nayar S, Granit V, Donohue M, Grossman K, Campbell DJ, Qureshi IA, Cudkowicz ME, Babu S. Verdiperstat in Amyotrophic Lateral Sclerosis: Results From the Randomized HEALEY ALS Platform Trial. JAMA Neurol. 2025 Apr;82(4):333-343. doi: 10.1001/jamaneurol.2024.5249. Epub 2025 Feb 17.

  PMID: 40067754 DERIVED

MeSH Terms

Conditions

Amyotrophic Lateral Sclerosis

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: June 1, 2020
• First Posted: June 18, 2020
• Study Start: July 28, 2020
• Primary Completion: April 13, 2022
• Study Completion: December 6, 2022
• Last Updated: June 12, 2023
• Results First Posted: June 12, 2023

Record last verified: 2023-05

Data Sharing

• IPD Sharing: Will not share

Locations

US (1)