HEALEY ALS Platform Trial - Regimen I NUZ-001

Merit E. Cudkowicz, MD

Status and phase: Invitation-only; Phase 3; Phase 2

Conditions: Amyotrophic Lateral Sclerosis (ALS)

Treatments: Drug: Matching placebo; Drug: NUZ-001

Study type: Interventional

Funder types: Other; Industry

Identifiers

NCT07410806

2019P003518I

Details and patient eligibility

About

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.

Full 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.

Enrollment: 160 estimated patients

Sex: All

Ages: 18+ years old

Volunteers: No Healthy Volunteers

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

Trial design

Primary purpose: Treatment

Allocation: Randomized

Interventional model: Parallel Assignment

Masking: Quadruple Blind

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160 participants in 2 patient groups, including a placebo group

NUZ-001

Experimental group

Description:

NUZ-001

Treatment:

Drug: NUZ-001

Matching Placebo

Placebo Comparator group

Description:

Placebo Comparator: Matching Placebo

Treatment:

Drug: Matching placebo