Multiple Treatments for ALS

Recruiting at81 trial locations

Overseen by Merit Cudkowicz, MD

Age: 18+

Sex: Any

Travel: May Be Covered

Time Reimbursement: Varies

Trial Phase: Phase 2 & 3

Waitlist Available

Sponsor: Merit E. Cudkowicz, MD

No Placebo Group

Prior Safety Data

Trial Summary

What is the purpose of this trial?

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.

Research Team

Merit Cudkowicz, MD

Principal Investigator

Massachusetts General Hospital

Eligibility Criteria

This trial is for adults with ALS, diagnosed as per El Escorial criteria, who can consent and follow study procedures. They must have a vital capacity over 50%, be within 36 months of symptom onset, and able to swallow. Participants on riluzole or edaravone must be on stable doses; those not taking these drugs may join. Exclusions include significant unstable medical conditions, certain lab abnormalities, active cancer (with exceptions), pregnancy/breastfeeding without contraception use, recent investigational drug use for ALS.

Inclusion Criteria

My ALS-related weakness started less than 3 years ago.

I have not taken riluzole, or I have been on a stable dose for at least 30 days.

I have never taken edaravone or have completed at least one cycle before the screening.

Exclusion Criteria

I have no active cancer except for certain skin, cervical, or prostate cancers treated over 3 years ago.

I am a man who can father children and agree to use contraception during and after the trial as required.

Clinically significant unstable medical condition (other than ALS) that would pose a risk to the participant, according to SI's judgment (e.g., cardiovascular instability, systemic infection, untreated thyroid dysfunction, or clinically significant laboratory abnormality or EKG changes)

Treatment Details

Interventions

• ABBV-CLS-7262 (Unknown)
• CNM-Au8 (Unknown)
• Pridopidine (Unknown)
• SLS-005 Trehalose (Unknown)
• Verdiperstat (Unknown)
• Zilucoplan (Unknown)

Trial Overview The HEALEY ALS Platform Trial tests multiple potential treatments for ALS simultaneously: Zilucoplan, Verdiperstat, CNM-Au8, Pridopidine, SLS-005 Trehalose, ABBV-CLS-7262 and DNL343. It's designed to evaluate the safety and effectiveness of these investigational products in slowing down or stopping disease progression.

Participant Groups

Group I: Regimen G - DNL343 Experimental Treatment 1 Intervention

Participants are randomized to receive either active DNL343 or matching placebo.

Group II: Regimen F- ABBV-CLS-7262 Experimental Treatment 1 Intervention

Participants are randomized to receive either active ABBV-CLS-7262 or matching placebo.

Who Is Running the Clinical Trial?

Merit E. Cudkowicz, MD

Lead Sponsor

Trials

8

Recruited

2,900+

Massachusetts General Hospital

Collaborator

Trials

3,066

Recruited

13,430,000+

Dr. William Curry

Massachusetts General Hospital

Chief Medical Officer

MD from Harvard Medical School

Dr. Anne Klibanski

Massachusetts General Hospital

Chief Executive Officer since 2019

MD from Harvard Medical School