Expanded Access Programs

Here you can read an article that describes Expanded Access Programs, compares them to other pathways for accessing investigational treatments, describes some of the challenges at the sponsor and site level, and lists resources which can be helpful in overcoming these challenges.

• Accessing Investigational Products Outside of a Trial: Considerations for Neuromuscular Providers

Here is a series of short videos about different aspects of EAPs

• What Are “Expanded Access Programs” and Who Qualifies for Them?
• How Does Expanded Access Differ from Other Options for PALS Who Want to Access Investigational Products?
• Barriers to Expanded Access Programs and Resources for Addressing Them

Here you can watch longer Educational Webinars containing information about EAPs

• Expanded Access to Investigational Drugs
• ALS Drug Development Webinar Series Part 3: Ethical Considerations
• ALS Drug Development Webinar Series Part 4: Site Considerations

Here you can see a roadmap and step by step guide to opening an EAP at a site

• Navigating Expanded Access

Here is a list of Expanded Access Protocols which are open to enrollment

• An Intermediate Expanded Access Protocol for Amyotrophic Lateral Sclerosis With CNM-Au8
  Location: Massachusetts General Hospital, Boston, Massachusetts, United States, 02114
  Contact: Judith Carney, RN at 617-724-8995 or jcarey8@partners.org   
• Expanded Access Protocol: Repeated Administration of Nurown® (Autologous MSC-NTF Cells) for the Treatment of ALS
  Locations:
  University of California Irvine, Irvine, California, United States, 92697
  Cedars-Sinai Medical Center, Los Angeles, California, United States, 90048
  California Pacific Medical Center, San Francisco, California, United States, 94115
  Massachusetts General Hospital, Boston, Massachusetts, United States, 02115
  UMass Medical School, Worcester, Massachusetts, United States, 01655
  Mayo Clinic, Rochester, Minnesota, United States, 55905
  Contacts: Mary Kay Turner at 201-488-0460 ext 105 or mkt@brainstorm-cell.com and Yael Gothelf, Ph.D. at 646-666-3188 ext 111 or ygothelf@brainstorm-cell.com
• Expanded Access Program for Tofersen in Participants With Superoxide Dismutase 1-Amyotropic Lateral Sclerosis
  Location: The current EAP structure does not limit patients to receive access at specific sites.
  Contact: US Biogen Clinical Trial Center at 866-633-4636 or clinicaltrials@biogen.com       

Click here for more information about the Sean M. Healey EAP Program at Massachusetts General Hospital.

Additional Resources for Clinicians

The information on Expanded Access provided on this page is sourced from public websites. Therefore, not all EAPs included here are endorsed by the Northeast Amyotrophic Lateral Sclerosis Consortium®. If you have information about an EAP not listed above, please contact Carly Allen at callen@neals.org.