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Recruiting Phase 1/2

A Study to Evaluate the Safety and Efficacy of BEAM-302 in Adult Patients With Alpha-1 Antitrypsin Deficiency (AATD)

NCT06389877 · tracked via the Priya Life Science Australia tracker
Phase
Phase 1/2
Started
2024-06-19
Last updated
2026-10-05

Condition(s) studied

Alpha 1-Antitrypsin Deficiency

Investigational drug(s) / intervention(s)

BEAM-302

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

Sex
ALL
Min age
18 Years
Max age
75 Years
Healthy volunteers
No
Part A: Key Inclusion Criteria: * Age 18-70 years. * Confirmed PiZZ AATD diagnosis. * Blood total AAT level \<11 μM. * Willing to discontinue augmentation therapy as required by protocol. * A postbronchodilator FEV1 ≥40% (Ph 1) of predicted or ≥35% (Ph 2) and an FEV1/FVC \<70% at screening (Ph 1 only). * Evidence of emphysema on chest imaging. Key Exclusion Criteria: * BMI \>30. * Lung or liver transplant or on waiting list for lung or liver transplant or status post lung volume reduction. * Clinical evidence of severe bronchiectasis. * Significant liver disease as defined in the protocol. * Active hepatitis A, B or C infection Part B: Key Inclusion Criteria: * Age 18-70 years. * Confirmed PiZZ AATD diagnosis. * Clinical diagnosis of AATD-related liver disease along with evidence of METAVIR F1, F2, or F3 liver fibrosis. * A postbronchodilator FEV1 ≥40% (Phase 1) or ≥35% (Phase 2) of predicted at screening. * FibroScan ≤12 kPa. Key Exclusion Criteria: * Lung or liver transplant or on waiting list for lung or liver transplant or status post lung volume reduction. * Clinical evidence of severe bronchiectasis. * Previous diagnosis of liver cirrhosis or complications of cirrhosis. Part C: Key Inclusion Criteria: * Age 18-75 years. * Confirmed PiZZ AATD diagnosis. * Willing to discontinue augmentation therapy as required by protocol. * A postbronchodilator FEV1 ≥30% of predicted. * Evidence of emphysema chest imaging. * Liver stiffness \<12 kPa. Key Exclusion Criteria: * Lung or liver transplant or on waiting list for lung or liver transplant or status post lung volume reduction. * Clinical evidence of severe bronchiectasis. * BMI \>40. * Known history of liver cirrhosis or complications of cirrhosis.

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)

FacilityCityRegionStatus
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
Official registry record

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