Recruiting NCT06389877
A Study to Evaluate the Safety and Efficacy of BEAM-302 in Adult Patients With Alpha-1 Antitrypsin Deficiency (AATD)
For patients and families
In plain language
An automatic summary of structured registry data. It is an orientation aid, not a substitute for the official protocol or a physician assessment.
- What is being studied
- The protocol lists: BEAM-302.
- Who it may be relevant to
- Registry conditions: Alpha 1-Antitrypsin Deficiency. Basic parameters: 18 years — 70 years · All.
- What needs checking
- Age, condition and sex are only basic indicators. Prior treatment, laboratory values and other mandatory requirements appear in the eligibility criteria below.
- Where it takes place
- United States, Australia, Ireland, Netherlands, New Zealand +1
- Next step
- Save the trial, show it to the treating physician, and confirm current recruitment with the study center. Costs, documents and travel →
Unsure about the terms? Read our patient guide →
Official 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
Overview
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).
Interventions
- Drug BEAM-302
BEAM-302 is a lipid nanoparticle (LNP)-based therapy for the treatment of patients with AATD
Primary outcome measures
- Phase 1 Dose Exploration: Rates of treatment-emergent adverse events (TEAEs) and serious adverse events (SAEs) [Time frame: 2 years]
- Phase 2 Dose Expansion: Absolute blood levels of total AAT [Time frame: 2 Years]
Secondary outcome measures (2)
- Phase 1 Dose Exploration: Absolute blood levels of total AAT [Time frame: 2 Years]
- Phase 2 Dose Expansion: Rates of TEAEs and SAEs [Time frame: 2 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 <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).
Criteria are shown verbatim from the registry (in English). Final eligibility is always assessed by the study center.
Healthy volunteers: No
Study design
- Allocation
- N/A
- Model
- Single group
- Masking
- Open label
- Primary purpose
- Treatment
Study locations
United States · 3 centers
- Clinical Study Center — Birmingham
- Clinical Study Center — Boston
- Clinical Study Center — Charleston
Australia · 2 centers
- Clinical Study Center — Adelaide
- Clinical Study Center — Fitzroy
New Zealand · 2 centers
- Clinical Study Center — Auckland
- Clinical Study Center — Hamilton
United Kingdom · 2 centers
- Clinical Study Center — London
- Clinical Study Center — Southampton
Ireland · 1 center
- Clinical Study Center — Dublin
Netherlands · 1 center
- Clinical Study Center — Leiden
Identifiers
NCT: NCT06389877 · BTX-302-001