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Tests treatment safety and results for Alpha 1-Antitrypsin Deficiency
Official title A Study to Evaluate the Safety and Efficacy of BEAM-302 in Adult Patients With Alpha-1 Antitrypsin Deficiency (AATD)
ClinicalTrials.gov ID: NCT06389877
What this study is testing
What is BEAM-302?
BEAM-302 is an investigational medicine, being studied as a potential treatment for alpha 1-antitrypsin deficiency.
Plain-language explanation of the investigational treatment - it is being studied and is not an approved or proven therapy. The study team can confirm the details.
- What it's testing
- 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).
- Phase 2: a mid-size study of how well it works
A plain-language read of the study's public ClinicalTrials.gov listing. The study team confirms the details.
Who can take part
Ages 18 to 70
You may be able to join if
- Part A:
- 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...
You likely can't join if
- 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...
- 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...
- Known history of liver cirrhosis or complications of cirrhosis (eg, varices, ascites, hepatic encephalopathy).
See the full eligibility criteria
- Part A:
- 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...
- 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.)
- 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...
- 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...
- 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).
The study team makes the final eligibility decision.
Where it's taking place
- Birmingham, Alabama, United States
- Boston, Massachusetts, United States
- Charleston, South Carolina, United States
- Adelaide, Australia
- Fitzroy, Australia
- Dublin, Ireland
- Leiden, Netherlands
- Auckland, New Zealand
- Hamilton, New Zealand
- London, United Kingdom
- Southampton, United Kingdom
Questions & answers
Do participants get paid in this trial?
This listing doesn't specify compensation. Many trials still reimburse travel or offer a stipend, so it's worth asking the study team when you connect.
Is it free to join, and do I need insurance?
Searching and applying through BridgeMD is free. In clinical trials the study-related treatment and visits are generally provided at no cost to you. You usually don't need insurance to take part - confirm specifics with the study team.
How long does this study last?
The listing doesn't state an exact length. The study team walks you through the schedule and number of visits before you decide to enroll.
Who can join this trial?
This study is enrolling all sexes, 18 years to 70 years. The study team makes the final eligibility decision.
Where is this trial taking place?
Study sites include Birmingham, Alabama, United States; Boston, Massachusetts, United States; Charleston, South Carolina, United States; Adelaide, Australia; Fitzroy, Australia; Dublin, Ireland and 5 more location(s). Enter your location above to see the nearest site and check your eligibility.
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BridgeMD is an information and trial-matching tool - not medical advice, and not the study sponsor. Details come from ClinicalTrials.gov; the study team decides eligibility.