Yükleniyor... / Loading...
HOPE-B is an open-label, single-dose, multi-center, multinational Phase 3 trial designed to demonstrate the efficacy of AMT-061 (etranacogene dezaparvovec) and further describe its safety profile. AMT-061 is a recombinant AAV5 vector containing the Padua variant of a codon-optimized human FIX cDNA under a liver-specific promoter, administered as an intravenous infusion at 2 x 10^13 gc/kg. Eligible participants are males aged 18 or older with severe or moderately severe congenital hemophilia B who are currently on factor IX prophylaxis and have more than 150 previous exposure days to factor IX protein.
The summary above is a plain-language rendering of the official record. The original English title is shown for reference.
Criteria are reproduced from the ClinicalTrials.gov record in the original English. Only the trial team can determine eligibility.
Inclusion Criteria: 1. Male 2. Age ≥18 years 3. Subjects with congenital hemophilia B, classified as severe or moderately severe, and are currently on factor IX prophylaxis 4. \>150 previous exposure days of treatment with factor IX protein Exclusion Criteria: 1. History of factor IX inhibitors 2. Positive factor IX inhibitor test at screening 3. Select screening laboratory value \>2 times upper limit of normal 4. Positive human immunodeficiency virus (HIV) test at screening, not controlled with anti-viral therapy 5. Active infection with hepatitis B or C virus at screening 6. History of Hepatitis B or C exposure, currently controlled by antiviral therapy at the end of the lead-in phase 7. Previous gene therapy treatment 8. Receipt of an experimental agent within 60 days prior to screening 9. Current participation or anticipated participation within one year after study drug administration in this trial in any other interventional clinical trial involving drugs or devices