In This Article
- A "platform trial" is a clinical trial in which multiple treatments are tested and evaluated simultaneously
- Merit Cudkowicz, MD, MSc, director of the Healey Center for ALS, said that the platform trial model will optimize chances for patients to receive active treatment
- Five treatments for amyotrophic lateral sclerosis were selected for evaluation by a group of expert scientists and members of the Healey Center Science Advisory Committee
The Sean M. Healey and AMG Center for ALS at Massachusetts General Hospital is preparing to launch the first platform trial for amyotrophic lateral sclerosis (ALS) in order to rapidly evaluate and develop treatments for patients with the disease.
The trial will simultaneously test multiple treatments for ALS, which may cut in half the time to find an effective treatment and decrease costs by a third or more.
Because several different treatments will be tested using a shared infrastructure as well as a master protocol, the platform trial model will allow for participant data to be pooled across several placebo groups, resulting in high statistical power to improve a participant's individual chance of being on active treatments in comparison with traditional trials.
Merit Cudkowicz, MD, MSc, director of the Healey Center for ALS, said that that platform trial model optimizes chances for people to receive active treatment, provides answers faster and ensures we keep learning about the disease.
She also stated that this approach to research has enthusiastic support from patients, the FDA, ALS clinicians and scientists.
The drugs being evaluated in the trial were selected by a group of expert ALS scientists and members of the Healey Center Science Advisory Committee who issued a call for submissions and selected the five therapeutics out of a pool of 30 applicants from 10 countries.
The selected treatments include: zilucoplan, a small macrocyclic peptide inhibitor of complement component 5 [C5], developed by Ra Pharmaceuticals, Inc.; Verdiperstat, an oral myeloperoxidase inhibitor, developed by Biohaven Pharmaceutical Holding Company Ltd.; Bioenergetic Nanocatalysis (CNM-Au8, nanocrystalline gold) developed by Clene Nanomedicine, Inc.; Pridopidine, a highly selective S1R agonist, developed by Prilenia Therapeutics; and IC14 immunotherapy, developed by Implicit Bioscience Ltd.
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