Clinical trial attitudes among individuals with Alpha-1 antitrypsin deficiency.

Purpose: This study examined characteristics of clinical trials that influence interest in participation among individuals with alpha-1 antitrypsin deficiency (AATD).

Procedures: A cross-sectional survey was completed by individuals with AATD. Thirty-four items described characteristics of clinical trials, which were rated from 1 (would not participate) to 5 (highly interested in participating).

Augmentation Therapy for Alpha-1 Antitrypsin Deficiency: Patient Experiences With Self-Infusion, Home Providers, and Clinics.

Background: Currently approved therapies for individuals with alpha-1 antitrypsin deficiency (AATD) are intravenously infused products. The burdens and demographics of infusion practices in the United States are not well-characterized.

Research Question: What is the prevalence of different infusion practices in the United States?

Study Design And Methods: AlphaNet disease management participants completed a survey that captured current and past infusion practices.