What is this study studying for Amyotrophic Lateral Sclerosis?

This study is evaluating the safety and effectiveness of multiple investigational drugs for treating ALS. Researchers are testing these treatments in a controlled setting to better understand their effects on disease progression. The trial is led by Merit E. Cudkowicz, MD, and aims to find new options for people with ALS.

Who may be eligible for this Amyotrophic Lateral Sclerosis clinical trial?

Adults aged 18 years or older diagnosed with possible, probable, or definite ALS may qualify. Participants must have muscle weakness onset within 24 months and lung function at or above 50% predicted capacity. Both men and women can participate. Please review eligibility criteria for more details.

Is there any cost to participate in this Amyotrophic Lateral Sclerosis clinical trial?

Study-related care, including the study drug or procedure and all trial visits, is provided at no cost. However, any personal travel or accommodation expenses are not guaranteed to be covered and may vary. Participants should plan accordingly for these potential costs.

Actively Recruiting

Phase 2

Phase 3

Age: 18Years +

All Genders

ID04297683

HEALEY ALS Platform Trial Master Protocol Evaluating Multiple Investigational Drugs for Amyotrophic Lateral Sclerosis

Led by Merit E. Cudkowicz, MD · Updated on 2026-06-04

1500

Participants Needed

Research Sites

56 weeks

Total Duration

AI-Summary

What this Trial Is About

The HEALEY ALS Platform Trial is an ongoing multi-center study designed to evaluate the safety and effectiveness of multiple investigational treatments for Amyotrophic Lateral Sclerosis ALS. This trial uses a single Master Protocol to test several investigational drugs either simultaneously or one after another. Each treatment regimen includes a placebo-controlled comparison, with new regimens added over time as new investigational products become available. Participants are randomly assigned to one of the active treatment regimens available at screening. Within each regimen, they receive either the study drug or a matching placebo in a 31 ratio. The study includes various drugs such as Zilucoplan, Verdiperstat, CNM-Au8, Pridopidine, SLS-005 Trehalose, ABBV-CLS-7262, DNL343, and NUZ-001, administered through oral doses, subcutaneous injections, or infusions at specified doses and schedules. During the trial, participants will undergo assessments including measurements of disease progression over 36 weeks as the primary outcome. Secondary outcomes include respiratory function and survival. The trial employs quadruple masking and randomized allocation to ensure unbiased results. Participants are monitored throughout the study for safety and compliance, with ongoing enrollment as new treatment regimens are introduced. The total study duration varies per participant depending on the regimen and ongoing trial additions.

CONDITIONS

Brief Title

HEALEY ALS Platform Trial - Master Protocol

Who Can Participate

Age: 18Years +

All Genders

Eligibility Criteria

You may qualify if you...

Sporadic or familial ALS diagnosed as possible, probable, lab-supported probable, or definite ALS by revised El Escorial criteria
Age 18 years or older
Ability to provide informed consent and comply with study procedures
Onset of ALS-related weakness within 24 months at screening
Vital Capacity at or above 50% of predicted capacity measured by Slow Vital Capacity or Forced Vital Capacity
Either not taking riluzole or on a stable riluzole dose for at least 30 days before screening
Either not taking edaravone or completed at least one cycle before screening
Ability to swallow pills and liquids at screening and expected to continue during the study
Geographically accessible to the study site

You will not qualify if you...

Clinically significant unstable medical conditions or abnormal lab values including liver enzymes above 3 times normal or kidney function below specified threshold
Unstable psychiatric disease, cognitive impairment, dementia, or substance abuse impairing informed consent
Active cancer or recent history of cancer except certain skin or in situ carcinomas treated without recurrence for 3 years
Use of investigational ALS treatments within 5 half-lives or 30 days before screening
Prior exposure to investigational gene therapies for ALS
Pregnancy, breastfeeding, or unwillingness to use effective contraception during and after the trial
Any condition increasing risk or preventing full study compliance
Unresolved disqualifying conditions or insufficient wash-out period if rescreened

Research Team

HEALEY Center for ALS at Massachusetts General Hospital

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