This open-label, single-dose, single-arm, multi-center Phase 2 trial evaluates the gene therapy AMT-061 (AAV5-hFIXco-Padua), with screening, treatment plus post-treatment follow-up, and long-term follow-up phases. AMT-061 is a recombinant AAV5 vector containing the Padua variant of a codon-optimized human FIX cDNA under a liver-specific promoter, given as an intravenous infusion at a dose of 2 x 10^13 gc/kg. Eligible participants are males aged 18 or older with severe or moderately severe congenital hemophilia B and more than 20 previous exposure days to FIX protein; those with a history of FIX inhibitors are excluded.
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 4. \>20 previous exposure days of treatment with FIX protein Exclusion Criteria: 1. History of FIX inhibitors 2. Positive FIX inhibitor test at screening 3. Select screening laboratory values \> 2 times upper normal limit: 4. Positive human immunodeficiency virus (HIV) 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