NCT04297683

HEALEY ALS Platform Trial - Master Protocol

HEALEY ALS Platform Trial

• Status: Recruiting
• Phase: Phase 2 / Phase 3
• Study type: Interventional
• Enrollment: 1,500 (estimated)
• Sponsor: Merit E. Cudkowicz, MD · Academic / Other
• Sex: All
• Age: 18 Years
• Healthy volunteers: Not accepted

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.

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. In this trial, multiple investigational products for ALS will be tested simultaneously or sequentially. Each investigational product will be tested in a regimen. Each regimen consists of a placebo-controlled trial, meaning that the active investigational product and matching placebo will be tested in each regimen. The additional details that govern the testing of each investigational product will be summarized in separate regimen-specific appendices (RSAs). Each regimen will have a separate ClinicalTrials.gov posting, which will include specific information about the regimen. All regimen-specific outcome measures will be detailed in each regimen posting. Participants will have an equal chance to be randomized to all regimens that are active at the time of screening. Once randomized to a regimen, participants will be randomized in a 3:1 ratio to either study drug or placebo. The following regimens are active in the trial: Regimen I - NUZ-001 New regimens will be continuously added as new investigational products become available. The HEALEY ALS Platform Trial will enroll additional participants as each new regimen is available.

Conditions

• Amyotrophic Lateral Sclerosis

Interventions

Type	Name	Description
DRUG	Zilucoplan	Drug: Zilucoplan Administration: Subcutaneous injection Dose: Minimum of .0.22 mg/kg daily to a maximum dose of 0.42 mg/kg daily, dependent on weight
DRUG	Verdiperstat	Drug: Verdiperstat Administration: Oral Dose: 600mg twice daily
DRUG	CNM-Au8	Drug: CNM-Au8 Administration: Oral Dose: 30 mg or 60 mg daily
DRUG	Pridopidine	Drug: Pridopidine Administration: Oral Dose: 45mg twice daily
DRUG	SLS-005 Trehalose	Drug: SLS-005 Trehalose Administration: Infusion Dose: 0.75 g/kg weekly
DRUG	ABBV-CLS-7262	Drug: ABBV-CLS-7162 Administration: Oral Dose: Dose 1 or Dose 2
DRUG	DNL343	Drug: DNL343 Administration: Oral Dose: Once per day
DRUG	NUZ-001	Drug: NUZ-001. Administration: Oral. Dose: Once per day.

Timeline

• Start date: 2020-06-14
• Primary completion: 2027-07-01
• Completion: 2028-08-01
• First posted: 2020-03-05
• Last updated: 2026-04-16

Locations

75 sites across 1 country: United States

Regulatory

• FDA-regulated drug study