Expanded Access to Sodium Phenylbutyrate/Taurursodiol Coformulation in Amyotrophic Lateral Sclerosis: Learnings From an ALS Community Partnership


Clinical Trials

Poster Number: 38


Machelle Manuel, PhD, Amylyx Pharmaceuticals, Inc., Cambridge, Massachusetts, Sandy Morris, BA, BS, MBA, I AM ALS, Washington, DC, Philip Green, BS, I AM ALS, Washington, DC, Robert Hebron, JD, I AM ALS, Washington, DC, Cali Orsulak, BscPharm, BCPS, CDE, I AM ALS, Washington, DC, Jamie Timmons, MD, Amylyx Pharmaceuticals, Inc., Cambridge, MA, Patrick Yeramian, MD, MBA, Amylyx Pharmaceuticals, Inc., Cambridge, MA, Erin Whitney, BS, MBA, Amylyx Pharmaceuticals, Inc., Cambridge, MA, Josh Cohen, BSE, Amylyx Pharmaceuticals, Justin Klee, ScB, Amylyx Pharmaceuticals, Sabrina Paganoni, MD, PhD, Neurological Clinical Research Institute, Massachusetts General Hospital, Boston, MA, Merit Cudkowitz, MD, Harvard Medical School

Background: Amyotrophic lateral sclerosis (ALS) is a rapidly progressive, fatal disorder for which many investigational therapies are currently under evaluation in clinical trials. Despite the breadth of studies, recent analyses suggest that 50-95% of people living with ALS may be ineligible for ALS trials based on inclusion criteria (van Eijk RPA, et al. Neurology. 2019;92(5):e451-e460, Healey platform trial; numbers vary depending on trial enrollment criteria). Modification of criteria for clinical trials to be more inclusive and implementation of expanded access programs (EAPs) can provide access to investigational therapies. EAPs in particular provide access by making therapies available outside of clinical trial settings to people who are currently ineligible for such trials. EAPs have become a focus of ALS advocacy efforts, clinicians, and pharmaceutical companies. In recent years, a number of EAPs for ALS have been implemented in parallel with clinical trials; however, to date, the reach of EAPs to people living with ALS has been constrained by enrollment of only small numbers of participants.
Objective: Describe the development and design of an intermediate-sized EAP to occur in parallel with a large phase 3 ALS trial.
Methods: PHOENIX (NCT05021536) is an ongoing phase 3 trial evaluating the safety and efficacy of coformulated sodium phenylbutyrate/taurursodiol (PB/TURSO) in ALS. The concurrent EAP will enroll approximately 250 adults with ALS from approximately 25 US sites. Enrollment criteria include diagnosis by a physician experienced in ALS management and >36 months from symptom onset. Exclusion criteria include current eligibility for or enrollment in a therapeutic study currently offered at the site. In addition to expanding PB/TURSO access for people living with ALS, another objective of the program is to collect safety data in a broader population of people with ALS.
Conclusions: EAPs can be successfully launched in parallel with pivotal trials. Critical input and feedback were provided by people living with ALS and their caregivers in the development of this EAP, fulfilling the values of ALS advocate-driven and people-centric research espoused in the Morris ALS Principles (morrisalsprinciples.org). By sharing our learnings, we aim to assist future programs to maximize engagement of ALS centers and people living with ALS and to provide unique and valuable information that supplements the data obtained in concurrent clinical trials.