Collaboration in ALS Research: Sabrina Paganoni, MD, PhD


The investigator at the Healey & AMG Center for ALS at Massachusetts General Hospital also discussed further research that needs to be done in ALS.

“This is a great model of how research projects and clinical trials should be designed and launched. We need to continue to establish these large-scale collaborations among international experts, and continue to include the voice of people living with ALS, and patient organizations so that they can have a voice into the trial design. We all need to learn from each other and leverage each other's perspectives and skills. We know that we can accomplish so much more when we work together.”

The upcoming phase 3 study A35-004 PHOENIX will evaluate the investigational agent AMX0035 for the treatment of amyotrophic lateral sclerosis (ALS). Amylyx Pharmaceuticals announced the trial design at the European Network to Cure ALS (ENCALS) Meeting 2021, May 12-14.

The trial will run for 48 weeks and will be randomized and placebo-controlled. Its primary outcomes include ALS Functional Rating Scale–Revised and survival over 48 weeks. It will also assess change in slow vital capacity measured both at home with a self-administered spirometer to support virtual data collection and at clinic sites, serial assessments of patient-reported outcomes, ventilation-free survival rates, and others as secondary efficacy outcomes.

NeurologyLive spoke with principal investigator of the previous study, CENTAUR (NCT03127514), and investigator of the PHOENIX study, Sabrina Paganoni, MD, PhD, investigator, Healey & AMG Center for ALS, Mass General and member, Executive Committee of Northeast ALS Consortium, to learn more about the first-of-its-kind collaboration between ENCALS and the Northeast ALS Consortium and the potential of such collaborations in ALS research.

1. Amylyx Pharmaceuticals presents trial design of global phase 3 PHOENIX study of AMX0035 in ALS at the European Network to Cure ALS (ENCALS) meeting 2021. News release. Amylyx Pharmaceuticals. Published online May 12, 2021.
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