Multicenter expanded access program for access to investigational products for amyotrophic lateral sclerosis

Area of Science:

• Neurology
• Clinical Trials
• Drug Development

Background:

• Expanded Access (EA) provides FDA-regulated access to investigational products (IPs) for serious diseases.
• Patients ineligible for clinical trials often lack access to novel therapeutics.
• Amyotrophic Lateral Sclerosis (ALS) is a progressive neurodegenerative disease with limited treatment options.

Purpose of the Study:

• To describe the design and operations of a multicenter, multidrug Expanded Access Program (EAP) for ALS.
• To assess the feasibility of enrolling patients ineligible for clinical trials into EAPs.
• To provide access to investigational products (IPs) for ALS patients through a coordinated program.

Main Methods:

• Established a central coordination center for program design and execution.
• Developed templated documents and processes for streamlined study design and regulatory submissions.
• Implemented three Expanded Access Protocols (EAPs) linked to the HEALEY ALS Platform Trial, enrolling patients with advanced or long-standing ALS.

Main Results:

• Screened 85 participants across three EAPs with a low screen failure rate (3.5%).
• Successfully enrolled participants, informing the duration of the platform trial regimens.
• The verdiperstat EAP concluded with a mean participation of 5.8 months; CNM-Au8 and pridopidine EAPs are ongoing.

Conclusions:

• Multicenter Expanded Access Programs (EAPs) can be successfully implemented in parallel with clinical trials.
• These programs effectively enroll patients with amyotrophic lateral sclerosis (ALS) who do not meet clinical trial eligibility criteria.
• EA programs offer a crucial pathway for accessing investigational treatments for ALS patients outside of traditional clinical trials.