HEALEY ALS Platform Trial

What is the HEALEY ALS Platform Trial?

This platform trial aims to accelerate the process of drug development and the path for effective treatment for ALS/MND by testing multiple drugs simultaneously and adaptively. This design has been seen to be successful in cancer trials. Compared to traditional drug development, the platform trial is estimated to find an effective therapy more quickly (average 3.4 vs. 8.5 years), with fewer total participants (average 880 vs. 1400), and fewer participants on placebo (average 220 vs. 700).

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. 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.

Current regimens active in the trial:

  • Verdiperstat (Phase 3)
  • CNM-Au8
  • Pridopidine
  • Trehalose/SLS-005 

Regimens that have been stopped early due to lack of benefit:

  • Zilucoplan (Phase 2)

What do the drugs do?

This trial is recruiting in the US only.

Latest News

Sept 2022 - Results from the Verdiperstat arm were announced. Unfortunately the primary endpoint was not met. You can read more in the press release

March 2022 - Zilucoplan arm stopped early for futility. You can read more here

Feb 2022 - 5th arm SLS-005 (Trehalose) enrolled its first participants. You can read more here.

Jan 2022 - The trial completes enrollment for 4th arm Pridopipine. You can read more here.

Nov 2021 - The trial completes enrollment in the first 3 arms. You can read more here.


Want to find out more about the Healey ALS Platform Trial? Check out the resources below:


Improving clinical trial design


HEALEY ALS Platform Trial

Massachusetts General Weekly Webinar:

Latest News & Updates from the HEALEY ALS Platform Trial

For further information and support contact our MND Connect helpline

mndconnect 0808 802 6262