Trial Summary
This is a Phase 1/2, multicenter, open-label, dose-exploration (Phase 1) and dose-expansion (Phase 2) study to evaluate the safety, tolerability, PK/PD, and efficacy of BEAM-302 in adult patients with AATD-associated lung disease and/or liver disease and to determine the optimal biological dose (OBD).
Sponsor: Beam Therapeutics Inc.
Participants: ALL
Start: 2024-06-19
Completion: 2028-05
Min Age: 18 Years
Max Age: 70 Years
Eligibility Criteria
Part A: Inclusion Criteria: * Males or females 18 – 70 years of age inclusive at the time of consent. * Diagnosis of AATD and homozygous for the PiZZ mutation (confirmed by genetic testing). * Blood total AAT level 4x/year]). * Liver disease with any of the following: * FibroScan liver stiffness measurement ≥7.5 kilopascals (kPa). (For sites without access to FibroScan, APRI >0.5 can be used as a surrogate exclusion criterion [Yilmaz, 2011]. * Known history of liver cirrhosis or complications of cirrhosis (eg, varices, ascites, hepatic encephalopathy). * Presence of ≥F2 liver fibrosis if a patient has previously had a liver biopsy. * Have ALT or AST > upper limit of normal (ULN). * Total bilirubin levels > ULN; if documented Gilbert's Syndrome, total bilirubin >2 × ULN. * INR ≥1.2 at screening. If deemed appropriate by the investigator and/or prescribing physician, the patient may stop taking anticoagulants for an appropriate washout period or reversal with vitamin K and if indicated, a repeat INR within 4x/year]) * Previous diagnosis of liver cirrhosis or complications of cirrhosis (eg, varices, ascites, hepatic encephalopathy).
Data from ClinicalTrials.gov. Always verify on the primary source before clinical use.