The Sean M. Healey & AMG Center for ALS at Mass General, in partnership with TackleALS, is committed to establishing the first Platform Trial initiative for ALS and will support the first three treatment approaches. We work closely with the Northeast ALS Consortium (NEALS) and Berry Consultants for the design and conduct of the ALS Platform Trial. The successful partners will work closely with a Trial Design Team to tailor the arm of the Platform Trial according to the experimental treatment. This is an ongoing effort, and we fully anticipate that in addition to these first three treatments, more treatments will be added to the platform, supported by pharma, foundation partners, federal and other fundraising initiatives. We encourage the academic and industry community to engage with the Healey Center ALS Trial Design Team at any time to work together on ALS therapy development. Nominations of therapies will be on an ongoing, rolling basis after initiation of the first selected therapies.
Brief Description of Platform Trials and Goals for ALS:
Only recently have major advances in ALS science led to a rich pipeline of promising therapeutics. There is a critical need to develop innovative clinical trial approaches to efficiently and more rapidly test treatments. The Healey Center for ALS at Mass General assembled a team of experts to dramatically rethink how early phase trials in ALS are performed and to accelerate a path to effective treatments.
Borrowing from successes in cancer drug development, Healey Center faculty designed the first ALS Platform Trial. Traditionally, each trial evaluates only one drug at a time, leading to lengthy start-up and execution times. Platform trials, instead, are trials where multiple drugs are tested in multiple treatment arms in parallel, using specialized statistical tools. New regimens (drugs) can be added as they become available thereby decreasing or eliminating the gap in time from identification of a rationale therapy to testing. This infrastructure allows investigators to test more agents, faster, and using less resources. The Food and Drug Administration (FDA) is now strongly encouraging innovative clinical trial approaches. There is also great interest by patients, clinical investigators, pharma, and foundations to bring platform trials to the ALS field. Platform trials are rapidly being adopted in several fields of medicine, including the neurosciences.
For more information on platform trials
To participate in an upcoming informational webinar about the application process please contact Susie Hawkins at
Please contact Susie for queries regarding the application and review process or how to contact the ALS Trial Design Team.
Confidential Disclosure Agreements (CDAs) are executed between MGH (on behalf of the Review Committee members) and the company before the ALS Platform Trial Therapy Application Form is submitted.
- Relevance of target in human disease
- Pre-clinical data to support target and therapy
- Clinical trial readiness (availability of compound and placebo, IND)
- Availability of relevant biomarkers
Time line for Submission:
Wednesday March 20
Application Due Date:
Wednesday May 8
Notification of therapy selection:
End of May or early June 2019