A Phase 2/3 interventional study of NUZ-001 and Matching placebo in Amyotrophic Lateral Sclerosis (ALS), sponsored by Merit E. Cudkowicz, MD. Active, not recruiting at 1 site in United States. Open to participants aged 18 Years and older. Per ClinicalTrials.gov, last updated 2026-07-20.
Sponsored by Merit E. Cudkowicz, MD · Phase 2/3, Interventional, and Treatment
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.
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.
981 studies on the registry are indexed under Amyotrophic Lateral Sclerosis; 283 are open to participants now.
This study's planned enrollment of 240 is above the median of 36 across 667 interventional studies indexed under Amyotrophic Lateral Sclerosis.
Browse Amyotrophic Lateral Sclerosis studies →Merit E. Cudkowicz, MD is the lead sponsor of 9 studies on the registry; none are open to participants now.
Of its 7 completed or terminated interventional studies of FDA-regulated products, 7 (100%) have results posted.
Counted across the registry records on this site, refreshed daily.
Exclusion Criteria:
The following exclusion criteria are in addition to the exclusion criteria specified in the Master Protocol (NCT NCT04297683).
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)
a. Note: Elevated total bilirubin attributable to documented Gilbert's syndrome is not considered exclusionary when there is no other evidence of clinically significant hepatic dysfunction.
NUZ-001
Drug: NUZ-001
Placebo Comparator: Matching Placebo
Drug: Matching placebo
NUZ-001 is administered orally once daily for 36 weeks.
Matching placebo is administered orally once daily for 36 weeks.
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.
Time frame: Baseline to 36 Weeks
Respiratory Function
Change in respiratory function over time as measured by Slow Vital Capacity (SVC)
Time frame: Baseline to 36 Weeks
Survival
Survival evaluated as time to death or permanent assisted ventilation (PAV)
Time frame: Baseline to 36 Weeks
This study is active, not recruiting, as verified in Jul 2026. You cannot join it, but the record below documents what was studied.
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Amyotrophic Lateral Sclerosis→
Merit E. Cudkowicz, MD