HEALEY ALS Platform Trial - Regimen I NUZ-001

NCT07410806 | Enrolling By Invitation Phase 2 DMC FDA Drug

• 1 other identifier: 2019P003518I
• Study Type: interventional
• Target: 160
• 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 I will evaluate the safety and efficacy of a single study drug, NUZ-001, in participants with ALS.

Conditions

Amyotrophic Lateral Sclerosis (ALS)

Keywords

ALS; Placebo-Controlled; Double-Blind; Regimen Specific Appendix; Lou Gehrig's Disease; NUZ-001; Neurizon

Outcome Measures

Primary Outcomes (1)

• Disease Progression

  Change in disease severity over time as measured by the Amyotrophic Lateral Sclerosis Functional Rating Scale-Revised (ALSFRS-R) total score and survival. 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 36 Weeks

Secondary Outcomes (2)

• Respiratory Function

  Baseline to 36 Weeks

• Survival

  Baseline to 36 Weeks

Study Arms (2)

NUZ-001

EXPERIMENTAL

NUZ-001

Drug: NUZ-001

Matching Placebo

PLACEBO COMPARATOR

Placebo Comparator: Matching Placebo

Drug: Matching placebo

Interventions

NUZ-001 DRUG

NUZ-001 is administered orally once daily for 36 weeks.

NUZ-001

Matching placebo DRUG

Matching placebo is administered orally once daily for 36 weeks.

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:

• Clinically significant liver disease (e.g., chronic hepatitis B, untreated hepatitis C)
• Prior history of drug-induced liver injury (DILI) and/or laboratory results at Master Protocol Screening that indicate inadequate liver function (ALT, AST or alkaline phosphatase \>3 times the upper limit of normal \[x ULN\] and/or total bilirubin level \>2 x ULN)
• Family history of SOD1 or VCP-associated ALS or known SOD1 or VCP mutation via genetic testing or self-report
• Use of any prohibited medications as outlined in the Regimen-specific Appendix (RSA) within 30 days prior to Baseline or anticipated use during the study treatment period
• Participants who are taking Nuedexta® and have a prolonged Fridericia-corrected QT (QTcF) interval (QTcF \> 450 ms (males) or \> 470 ms (females)) at Master Protocol Screening

Sponsors & Collaborators

• Merit E. Cudkowicz, MD: lead
• Neurizon Therapeutics Limited: collaborator

Study Sites (1)

Healey Center for ALS at Massachusetts General Hospital

Boston, Massachusetts, 02114, United States

Location

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

Study Officials

• Merit Cudkowicz, MD

  Massachusetts General Hospital

  PRINCIPAL INVESTIGATOR

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: Executive Director, Neurology Department

Study Record Dates

• First Submitted: February 8, 2026
• First Posted: February 13, 2026
• Study Start: February 16, 2026
• Primary Completion (Estimated): July 1, 2027
• Study Completion (Estimated): August 1, 2028
• Last Updated: February 17, 2026

Record last verified: 2026-02

Locations

US (1)