By InvitationPhase 2/3

HEALEY ALS Platform Trial - Regimen I NUZ-001

United States160 participantsStarted 2026-02-16

Plain-language 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.

Who can participate

Age range18 Years

SexALL

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Exclusion criteria

✕. 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

What they're measuring

Disease Progression

Timeframe: Baseline to 36 Weeks

Trial details

NCT IDNCT07410806

SponsorMerit E. Cudkowicz, MD

Sponsor typeOTHER

Study typeINTERVENTIONAL

Primary completion2027-07-01

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