Pragmatic Solutions to Clinical Trial Endpoints for Lung Diseases in AATD, and AAT Variant Nomenclature
The Alpha-1 Foundation was excited to host the 26th Gordon L. Snider Critical Issues Workshop on Thursday, March 21 and Friday, March 22, 2024. The goal of the GLS topical workshops is to provide new information that speeds the journey to a cure for Alpha-1 Antitrypsin Deficiency (Alpha-1). The twin goals of this workshop were to 1) optimize a single phase 3 clinical trial outcome measure from the scientific community that would be advanced to regulators to balance feasibility and science and allow new therapies for AATD to advance; and 2) to contemplate a simplified, rational approach to classifying and naming variants in the AAT gene, SERPINA1. With these goals in mind, the meeting discussed and envisioned potential alternative approaches to current nomenclature. This GLS workshop also explored functional activity, targeted proteases, dosing, acute phase reactivity, and next steps to advance a pragmatic outcome measure for Alpha-1 lung disease.
Co-chaired by Dr. Andrew Wilson (Boston University Medical Center), Dr. Charlie Strange (Medical University of South Carolina) and Dr. Monica Goldklang (Columbia University Irvine Medical Center) the program engaged the participation over 80 experts in the Alpha-1 field.
“This two-day workshop was a culmination of two important discussions: shared data collection amongst key scientific and industry stakeholders to help validate endpoints for clinical assessment and eventual novel therapy approval to benefit patients, and simplifying nomenclature associated AATD gene variants to better classify patient conditions,” said President & CEO Scott Santarella.
The Alpha-1 Foundation acknowledges the following sponsors for their ongoing commitment to the advancement of research: AlphaNet, Beam Therapeutics, CSL Behring, Grifols and Takeda.
Thank you to our workshop presenters:
Johanna Rommens, PHD (The Hospital for Sick Children)
Mark Brantly, MD (University of Florida)
Tomas Caroll, PhD (Royal College of Surgeons in Ireland)
Andrew Wilson, MD (Boston University)
Charlie Strange (Medical University of South Carolina)
Monica Goldklang, MD (Columbia University)
Jeanine D’Armiento, MD, PhD (Columbia University)
Carson Veldstra (Inhibrx)
Jeffrey Siegel, MD (Food and Drug Administration)
Prateek Shukla, MD (Food and Drug Administration)
Christine Wendt, MD (University of Minnesota)
Mark Forshag, MD, MHA (Medical Affairs and Clinical Development Consultant)
Alice Turner, PhD (University of Birmingham)
Gerry McElvaney, MD (Royal College of Surgeons in Ireland)
