BEAM-302 BTX-302-001
Research type
Research Study
Full title
A Phase 1/2 Dose-Exploration and Dose-Expansion Study to Evaluate the Safety and Efficacy of BEAM-302 in Adult Patients with Alpha-1 Antitrypsin Deficiency (AATD)-Associated Lung Disease and/or Liver Disease
IRAS ID
1009231
Contact name
Anne Marie Woodland
Contact email
Sponsor organisation
Beam Therapeutics Inc.
Research summary
The BTX-302-001 study is a study that is being conducted to see if BEAM-302 is safe and may improve the symptoms and quality of life in people with alpha-1 antitrypsin deficiency (AATD) associated lung disease and/or liver disease. Having two copies of the PiZ mutation in the SERAPINA1 gene is the most common cause of AATD. BEAM-302 is designed to correct the mutation and restore normal function of the gene. BEAM-302 is given intravenously once during the study period.
The two year study consists of 16 visits over 4 study periods. The first period is the Screening Period and this begins after the patient signs consent and decides if they want to take part in the study. During this period, physicians will look at the patient’s medical records, conduct some additional tests and determine if the patient meets study requirements. If the patient meets study requirements, the patient will move on to the Dosing Period. The Dosing Period is when the patient will receive an infusion of BEAM-302. During this period the patient will stay in the hospital overnight for monitoring for at least 48 hours after they receive the infusion. Once the patient is discharged from the hospital, the patient will enter the Dose Limiting Toxicity Period. The Dose Limiting Toxicity Period will last 28 days after the patient receives the BEAM-302 infusion and will require the patient to visit the study site about once a week to monitor their health during this time. After the Dose Limiting Toxicity Period the patient will enter the Follow-Up Period. The Follow-Up Period will last approximately 22 Months and will require 8 visits to the study site to monitor the patient’s health and status.
REC name
North East - York Research Ethics Committee
REC reference
24/NE/0013
Date of REC Opinion
1 Mar 2024
REC opinion
Further Information Favourable Opinion