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.

4 Sites

3 Participants

Recruiting

18 Years to 8 Years

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Find closest sites:

SUBMIT

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

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

Additional Studies

Additional studies can be found at ClinicalTrials.gov