The Healey Center Platform Trial for ALS

Leveraging Innovative Trial Designs to Accelerate Path to Cures

Overview

Amyotrophic lateral sclerosis (ALS, Lou Gehrig’s disease), is a neurological disease with limited treatment options. 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 more rapidly test treatments, in the right people and at the right time, and improve access to experimental treatments for people with ALS. The Healey Center for ALS at Mass General has 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 and NEALS faculty are leading the first ALS Platform Trial. Traditionally, each trial evaluates only one drug at a time, and requires lengthy start-up and execution times. Platform trials, instead, are trials where multiple drugs are tested at the same time, 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. Thus, the focus is on the disease, rather than any individual experimental agent, and the platform remains open long-term until successful cures are found. The Healy Center Platform Trial for ALS will test multiple promising experimental therapeutics using an efficient and informative early-phase design with increased access for people with ALS.

Funding Source

Healy Center Logo

Request for Proposals of Therapies for the ALS Platform Trial

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 CLICK HERE.

To participate in an upcoming informational webinar about the application process please contact Susie Hawkins at slhawkins@mgh.harvard.edu or 617-643-1807.

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 Massachusetts General Hospital (on behalf of the Review Committee members) and the company before the ALS Platform Trial Therapy Application Form is submitted.

Review Criteria

  • 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

Release Date: Wednesday March 20th, 2019
Application Due Date: Wednesday May 8th, 2019
Notification of therapy selection: End of May or early June 2019

Click here for CDA template
Submit completed CDA form to healeycenterforals@mgh.harvard.edu

Click here for ALS Platform Trial Therapy Application Form
Submit completed application form to healeycenterforals@mgh.harvard.edu

Learn more about Healey Center, NEALS Consortium and Tackle ALS

https://www.massgeneral.org/als/healeycenter/
https://www.neals.org/
https://www.tackleals.com/

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