Evaluating BEAM-302 for treating Alpha-1 Antitrypsin Deficiency in adults

Is this trial a fit for you?

A plain-English reading of the study's rules. Only the study team can confirm whether you qualify.

What this trial studies

This Phase 1/2 clinical trial is designed to assess the safety, tolerability, pharmacokinetics, pharmacodynamics, and efficacy of BEAM-302 in adult patients suffering from Alpha-1 Antitrypsin Deficiency (AATD) with associated lung and/or liver disease. The study will involve a multicenter approach, starting with a dose-exploration phase followed by a dose-expansion phase to determine the optimal biological dose. Participants will be closely monitored for their response to the treatment and any adverse effects throughout the trial.

Why it matters

Who can join: the full rules

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.

Where it's running

• Clinical Study Center
  Birmingham, Alabama, United States Recruiting
• Clinical Study Center
  Boston, Massachusetts, United States Recruiting
• Clinical Study Center
  Charleston, South Carolina, United States Recruiting
• Clinical Study Center
  Adelaide, Australia Recruiting
• Clinical Study Center
  Fitzroy, Australia Recruiting
• Clinical Study Center
  Dublin, Ireland Recruiting

Study contacts

• Study coordinator: Medical Information
• Email: clinicalinfo@beamtx.com
• Phone: 857-327-8641

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How to take part

1. Review the eligibility criteria above with your treating physician.
2. Check the official listing on ClinicalTrials.gov for the latest contact details and recruitment status.
3. Contact the study coordinator or principal investigator to ask for pre-screening. Pre-screening is free and never obligates you to enroll.