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U.S. Department of Health and Human Services

Vaccines, Blood & Biologics

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Improving Endpoints, Improving Care: Alpha-1 Antitrypsin Augmentation Therapy and Clinical Trials; Public Workshop

Transcripts

March 23-24, 2009 (PDF 1.4 MB)

Workshop Goals and Objectives

The Food and Drug Administration (FDA) is announcing a public workshop entitled: Improving Endpoints, Improving Care: Alpha-1 Antitrypsin Augmentation Therapy and Clinical Trials. The purpose of the public workshop is to identify the most useful clinical trial endpoints and surrogate markers for alpha-1 antitrypsin (AAT) augmentation therapy. FDA, Alpha-1 Foundation and the Department of Health and Human Services, Office of Public Health and Science are convening this workshop to facilitate the design of future clinical trials intended to establish clinical efficacy of AAT products. The public workshop will feature presentations and panel discussions led by experts from academic institutions, government and industry.

Alpha-1 antitrypsin deficiency is a genetic condition that leads to decreased levels of alpha-1 antitrypsin in the blood and significantly increases the risk of serious lung disease in adults and liver disease in infants, children and adults. Intravenous augmentation therapy with FDA-licensed, plasma-derived AAT products has become the standard of care for treatment in the subset of patients with alpha-1 antitrypsin deficiency who have moderate pulmonary disease. Since the original product approvals, additional data collection and advances in understanding of AAT deficiency suggest the need to revisit and improve clinical trial efficacy endpoints.

The public workshop will facilitate scientific discussions to identify the most relevant and feasible, currently available and future clinical trial efficacy endpoints for AAT augmentation therapy and further evaluate its usefulness to a broader patient population. Topics to be discussed include: (1) alpha-1 antitrypsin deficiency disease characteristics, progression and pulmonary pathophysiology; (2) patient selection for clinical trials; 3) current challenges to the development of endpoints for clinical trials; and 4) currently available and future clinical trial endpoints, including functional markers of disease progression, and radiological and biochemical endpoints.

 

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