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Hemophilia B

Dose Confirmation Trial of AAV5-hFIXco-Padua
NCT03489291 | PHASE 2 | INTERVENTIONAL

This is an open-label, single-dose, single-arm, multi-center trial, with a screening, a treatment + post-treatment follow-up phase, and a long-term follow-up phase. The IMP AMT-061 is a recombinant adeno-associated viral vector of serotype 5 (AAV5) containing the Padua variant of a codon-optimized human FIX complementary deoxyribonucleic acid (cDNA) under the control of a liver-specific promoter. The IMP is identified as AAV5-hFIXco-Padua (AMT- 061). The pharmaceutical form of AMT-061 is a solution for intravenous infusion. The administered dose of AMT-061 will be 2 x 10\^13 gc/kg.

Trial Information
4 Sites
3 Participants
Recruiting
18 Years to 75 Years

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Phoenix Childrens Hospital
Phoenix,Arizona,United States,85016
University of California, Davis
Sacramento,California,United States,95817
University of California, San Diego
San Diego,California,United States,92122
University of Michigan
Ann Arbor,Michigan,United States,48109

Study Eligibility Criteria

Additional Studies

Additional studies can be found at ClinicalTrials.gov