A Study to Evaluate the Safety and Efficacy of BEAM-302 in Adult Patients With Alpha-1 Antitrypsin Deficiency (AATD)
Condition(s) studied
Investigational drug(s) / intervention(s)
BEAM-302: BEAM-302 is a lipid nanoparticle (LNP)-based therapy for the treatment of patients with AATD
Study summary
This is a Phase 1/2, multicenter, open-label study conducted in 3 parts (Part A, Part B, and Part C) to evaluate the safety, tolerability, PK/PD, and efficacy of BEAM-302 in adult patients with AATD-associated lung disease and/or liver disease. Parts A and B are designed to evaluate safety and tolerability and identify the optimal biological dose (OBD) in patients with AATD-associated lung disease (Part A) and/or liver disease (Part B), with optional expansion cohorts to further evaluate a potential OBD. Part C is designed to demonstrate the efficacy and safety of BEAM-302 at the OBD in patients with AATD-associated lung disease with or without liver disease in the pivotal cohort.
Eligibility
Primary outcome measure(s)
- Rates of treatment-emergent adverse events (TEAEs) and serious adverse events (SAEs) — 3 years
- Absolute blood levels of total AAT — 3 Years
- Mean total and functional AAT in blood — 1 year
Trial sites (12)
| Facility | City | Region | Status |
|---|---|---|---|
| Clinical Study Center | Birmingham | Alabama | Recruiting |
| Clinical Study Center | Boston | Massachusetts | Recruiting |
| Clinical Study Center | Charleston | South Carolina | Recruiting |
| Clinical Study Center | Adelaide | Australia | Recruiting |
| Clinical Study Center | Fitzroy | Australia | Recruiting |
| Clinical Study Center | Dublin | Ireland | Recruiting |
| Clinical Study Center | Leiden | Netherlands | Recruiting |
| Clinical Study Center | Auckland | New Zealand | Recruiting |
| Clinical Study Center | Hamilton | New Zealand | Recruiting |
| Clinical Study Center | Birmingham | United Kingdom | Recruiting |
| Clinical Study Center | London | United Kingdom | Recruiting |
| Clinical Study Center | Southampton | United Kingdom | Recruiting |
Other trials for the same condition
This page summarises publicly available registry data for informational purposes — not medical advice. Eligibility is determined by each study team; patients should discuss participation with their clinician.
View NCT06389877 on ClinicalTrials.gov ↗ ← All trials in Australia