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

Study Results

Results pending

The study team has not published outcome measurements, participant flow, or safety data for this trial yet. Check back later for updates.

Basic Information

Get a concise snapshot of the trial, including recruitment status, study phase, enrollment targets, and key timeline milestones.

Recruitment Status

RECRUITING

Clinical Phase

PHASE1/PHASE2

Total Enrollment

106 participants

Study Classification

INTERVENTIONAL

Study Start Date

2024-06-19

Study Completion Date

2027-08-31

Brief Summary

Review the sponsor-provided synopsis that highlights what the study is about and why it is being conducted.

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).

Related Clinical Trials

Explore similar clinical trials based on study characteristics and research focus.

A Study of Belcesiran in Patients With AATLD

NCT04764448

Alpha 1-Antitrypsin Deficiency

TERMINATED PHASE2

S-Adenosylmethionine Therapy for Non-Alcoholic Steatohepatitis

NCT00108589

Hepatitis

COMPLETED PHASE3

The Efficacy of S-adenosyl Methionine (SAMe) Versus Pentoxiphylline in Patients With Non-alcoholic Steatohepatitis With Fibrosis

NCT02231333

Non-alcoholic Steatohepatitis

COMPLETED NA

Assessment of Endothelial Function in Patients With Non Alcoholic Fatty Liver Disease and the Impact of Statin Treatment

NCT01987310

Non Alcoholic Fatty Liver Disease

WITHDRAWN NA

Ursodeoxycholic Acid Combined With Total Glucosides of Paeony in the Treatment of PBC With AIH Features 1

NCT04618575

Hepatitis Primary Biliary Cholangitis Autoimmune

UNKNOWN PHASE4

Detailed Description

Dive into the extended narrative that explains the scientific background, objectives, and procedures in greater depth.

Conditions

See the medical conditions and disease areas that this research is targeting or investigating.

Alpha 1-Antitrypsin Deficiency

Study Design

Understand how the trial is structured, including allocation methods, masking strategies, primary purpose, and other design elements.

Allocation Method

NA

Intervention Model

SINGLE_GROUP

Primary Study Purpose

TREATMENT

Blinding Strategy

NONE

Study Groups

Review each arm or cohort in the study, along with the interventions and objectives associated with them.

BEAM-302 Drug Product

Group Type EXPERIMENTAL

BEAM-302

Intervention Type DRUG

BEAM-302 is a lipid nanoparticle (LNP)-based therapy for the treatment of patients with AATD

Interventions

Learn about the drugs, procedures, or behavioral strategies being tested and how they are applied within this trial.

BEAM-302

BEAM-302 is a lipid nanoparticle (LNP)-based therapy for the treatment of patients with AATD

Intervention Type DRUG

Eligibility Criteria

Check the participation requirements, including inclusion and exclusion rules, age limits, and whether healthy volunteers are accepted.

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.)

* 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

* 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:

* 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).

Minimum Eligible Age

18 Years

Maximum Eligible Age

70 Years

Eligible Sex

ALL

Accepts Healthy Volunteers

No