A Phase 1 Study of AIR-001 in Adults with Alpha-1 Antitrypsin Deficiency

The primary objective of this Phase 1 study is to evaluate the safety and tolerability of AIR-001 in adult patients diagnosed with alpha-1 antitrypsin deficiency. Determining the safety profile and the maximum tolerated dose is essential for establishing the clinical feasibility of this therapeutic intervention in this specific patient population.

This clinical trial involves a total of 26 patients. The study population includes both male and female participants. The age range of the cohort corresponds to age codes 3 and 4. The subjects are diagnosed with alpha-1 antitrypsin deficiency, which is a rare disease. The population is characterized as vulnerable.

This is a Phase 1 study evaluating the administration of AIR-001 in adults diagnosed with Alpha-1 Antitrypsin Deficiency. The research methodology involves a sequence of study visits starting with a screening visit to assess eligibility. Subsequent follow-up visits and an end-of-study visit are scheduled to monitor safety and clinical parameters. The total duration of participant involvement is determined by the protocol-defined intervals between these visits. Early termination from the study may occur based on predefined clinical criteria or safety considerations.

Efficacy assessment in this Phase 1 clinical trial for alpha-1 antitrypsin deficiency will be conducted according to the established study protocol. The specific parameters, measurement methods, and schedules for evaluating therapeutic effectiveness are not defined in the current documentation.