HEALEY ALS Platform Trial - Regimen I NUZ-001

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.

Study Overview

• Status: Enrolling by invitation
• Conditions: Amyotrophic Lateral Sclerosis (ALS)
• Intervention / Treatment: Drug: NUZ-001; Drug: Matching placebo

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 I NUZ-001, the participant will complete a screening visit to assess additional Regimen I eligibility criteria. Once Regimen I eligibility criteria are confirmed, participants will complete a baseline assessment and be randomized in a 3:1 ratio to either active NUZ-001 or matching placebo.

Regimen I will enroll by invitation, as participants may not choose to enroll in Regimen I. 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 I.

For a list of enrolling sites, please see the HEALEY ALS Platform Trial Master Protocol under NCT04297683.

• Study Type: Interventional
• Enrollment (Estimated): 160
• Phase: Phase 2; Phase 3

Contacts and Locations

Study Locations

• United States
  • Massachusetts
    • Boston, Massachusetts, United States, 02114
      • Healey Center for ALS at Massachusetts General Hospital

Participation Criteria

Eligibility Criteria

• Ages Eligible for Study: Adult; Older Adult
• Accepts Healthy Volunteers: No

Description

Inclusion Criteria:

• No additional inclusion criteria beyond the inclusion criteria specified in the Master Protocol (NCT NCT04297683).

Exclusion Criteria:

• The following exclusion criteria are in addition to the exclusion criteria specified in the Master Protocol (NCT NCT04297683).

  1. Clinically significant liver disease (e.g., chronic hepatitis B, untreated hepatitis C)
  2. 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)
  3. Family history of SOD1 or VCP-associated ALS or known SOD1 or VCP mutation via genetic testing or self-report
  4. 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
  5. 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

Study Plan

How is the study designed?

Design Details

• Primary Purpose: Treatment
• Allocation: Randomized
• Interventional Model: Parallel Assignment
• Masking: Quadruple

Number of Arms

2

Arms and Interventions

Participant Group / Arm	Intervention / Treatment
Experimental: NUZ-001	Drug: NUZ-001; NUZ-001 is administered orally once daily for 36 weeks.
Placebo Comparator: Matching Placebo; Placebo Comparator: Matching Placebo	Drug: Matching placebo; Matching placebo is administered orally once daily for 36 weeks.

What is the study measuring?

Primary Outcome Measures

Outcome Measure	Measure Description	Time Frame
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 Outcome Measures

Outcome Measure	Measure Description	Time Frame
Respiratory Function	Change in respiratory function over time as measured by Slow Vital Capacity (SVC)	Baseline to 36 Weeks
Survival	Survival evaluated as time to death or permanent assisted ventilation (PAV)	Baseline to 36 Weeks

Collaborators and Investigators

• Sponsor: Merit E. Cudkowicz, MD
• Collaborators: Neurizon Therapeutics Limited
• Investigators: Principal Investigator: Merit Cudkowicz, MD, Massachusetts General Hospital

Study record dates

Study Major Dates

• Study Start (Estimated): February 16, 2026
• Primary Completion (Estimated): July 1, 2027
• Study Completion (Estimated): August 1, 2028

Study Registration Dates

• First Submitted: February 8, 2026
• First Submitted That Met QC Criteria: February 8, 2026
• First Posted (Actual): February 13, 2026

Study Record Updates

• Last Update Posted (Actual): February 17, 2026
• Last Update Submitted That Met QC Criteria: February 14, 2026
• Last Verified: February 1, 2026

More Information

Terms related to this study

• Keywords: ALS; Double-Blind; Placebo-Controlled; Lou Gehrig's Disease; Regimen Specific Appendix; NUZ-001; Neurizon
• Additional Relevant MeSH Terms: Central Nervous System Diseases; Nervous System Diseases; Neuromuscular Diseases; Metabolic Diseases; Neurodegenerative Diseases; Spinal Cord Diseases; TDP-43 Proteinopathies; Proteostasis Deficiencies; Motor Neuron Disease; Nutritional and Metabolic Diseases; Amyotrophic Lateral Sclerosis
• Other Study ID Numbers: 2019P003518I

Drug and device information, study documents

• Studies a U.S. FDA-regulated drug product: Yes
• Studies a U.S. FDA-regulated device product: No