NCT06389877 A Study to Evaluate the Safety and Efficacy of BEAM-302 in Adult Patients With Alpha-1 Antitrypsin Deficiency (AATD)
| NCT ID | NCT06389877 |
| Status | Recruiting |
| Phase | Phase 1, Phase 2 |
| Sponsor | Beam Therapeutics Inc. |
| Condition | Alpha 1-Antitrypsin Deficiency |
| Study Type | INTERVENTIONAL |
| Enrollment | 106 participants |
| Start Date | 2024-06-19 |
| Primary Completion | 2028-05 |
Eligibility & Interventions
Eligibility Fast-Check
Enter your details for a quick preliminary check. This does not replace medical advice.
What to Expect as a Participant
You will actively receive the study intervention — which may be a drug, biologic, device, or procedure.
Phase 1 is the earliest stage of human testing — safety and dosage are the primary focus. Visits are frequent and medical supervision is intensive. You will be among the first people to receive this treatment.
This trial targets 106 participants in total. It began in 2024-06-19 with a primary completion date of 2028-05.
⚠ This information is for research awareness only. Always consult your physician before joining any clinical trial. Participation is voluntary and you may withdraw at any time.
Brief 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).
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 \<11 μM or equivalent protein in mg/dL. * Patients receiving augmentation therapy in regions where augmentation is not SoC must be willing to washout augmentation therapy for at least 6 weeks prior to signing the ICF and for the length of the study (unless clinically indicated) * A postbronchodilator FEV1 ≥40% of predicted and an FEV1/FVC \<70% at screening. (PFTs obtained within 1 year of signing the ICF may be used for eligibility.) * Evidence of emphysema on a historic CT scan or a DLCO ≤70% of the predicted value (corrected for hemoglobin) at screening. (PFTs obtained within 1 year of signing the ICF may be used for eligibility.) Exclusion Criteria: * Body mass index \>30 * Lung or liver transplant or on waiting list for lung or liver transplant or status post lung volume reduction surgery. * Clinical evidence of severe bronchiectasis as per the discretion of the investigator (eg, excessive sputum production or recurrent infections requiring antibiotic use \[\>4x/year\]). * 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 \<1.2 would be acceptable. * Seropositive for hepatitis B (positive surface Ag). * Active hepatitis C by hepatitis C virus (HCV) antibody. If HCV antibody positive, must be HCV RNA polymerase chain reaction (PCR) negative. Part B: 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). * Evidence of METAVIR F1, F2, or F3 liver fibrosis based on a central read of a baseline liver biopsy during the screening period or a histological diagnosis made no more than 6 months before enrollment and stage confirmed by central read. * A postbronchodilator FEV1 ≥40% of predicted at screening. (PFTs obtained within 1 year of signing the ICF may be used for eligibility.) Exclusion Criteria: * Lung or liver transplant or on waiting list for lung or liver transplant or status post lung volume reduction surgery. * Clinical evidence of severe bronchiectasis as per the discretion of the investigator (eg, excessive sputum production or recurrent infections requiring antibiotic use \[\>4x/year\]) * Previous diagnosis of liver cirrhosis or complications of cirrhosis (eg, varices, ascites, hepatic encephalopathy).
Contact & Investigator
Medical Information
STUDY DIRECTOR
Beam Therapeutics
Frequently Asked Questions
Who can join the NCT06389877 clinical trial?
This trial is open to participants of all sexes, aged 18 Years or older, up to 70 Years, studying Alpha 1-Antitrypsin Deficiency. Full inclusion and exclusion criteria are listed in the Eligibility Criteria section. Always confirm your eligibility with the research team before applying.
What phase is the NCT06389877 trial and what does that mean for participants?
Phase 1 trials are the first stage of human testing. The primary goal is to assess safety and determine appropriate dosage levels. Participants are closely monitored. These trials typically involve a small number of volunteers.
Is NCT06389877 currently recruiting?
Yes, NCT06389877 is actively recruiting participants. Contact the research team at clinicalinfo@beamtx.com for enrollment information.
Where is the NCT06389877 trial being conducted?
This trial is being conducted at Birmingham, United States, Boston, United States, Charleston, United States, Adelaide, Australia and 7 additional locations.
Who is sponsoring the NCT06389877 clinical trial?
NCT06389877 is sponsored by Beam Therapeutics Inc.. The principal investigator is Medical Information at Beam Therapeutics. The trial plans to enroll 106 participants.