Safety and Dose Finding Study of DTX101 (AAVrh10FIX) in Adults With Moderate/Severe to Severe Hemophilia B

Status: Recruiting
Phase:
DFCI Protocol ID:

A Phase 1/2, open-label, dose-finding safety study of single ascending doses of DTX101 in adult males with moderate/severe to severe hemophilia B.

Conducting Institutions:

Children's Hospital Boston, Dana-Farber Cancer Institute

Overall PI:

Stacy Croteau, MD, Children's Hospital Boston

Site-responsible Investigators:

Contacts:

Dana-Farber Cancer Institute: Childrens Hospital Pediatric Clinical Translation Investigation Program CTIP, ctip@partners.org

Eligibility Criteria

Inclusion Criteria:

  1. Male ≥18 years of age.

  2. Moderate/severe or severe hemophilia B (baseline FIX activity ≤2% of normal or
     documented history of FIX activity ≤2%).

  3. At least 3 bleeding episodes per year that require on-demand treatment with FIX OR
     are treated with a prophylactic regimen of FIX.

  4. At least 100 days exposure history to FIX.

  5. No documented history of inhibitors (neutralizing antibodies) to exogenous FIX.

  6. No known allergic reaction to exogenous FIX or any component of DTX101.

  7. Willing to stop prophylactic treatment with recombinant FIX at specified time points
     during the study.

Exclusion Criteria:

  1. History of significant liver disease (ie;portal hypertension).

  2. Significant hepatic inflammation or cirrhosis.

  3. Evidence of active hepatitis B virus (HBV) or hepatitis C virus (HCV) infection.

  4. History of human immunodeficiency virus (HIV) infection AND any of the following:
     CD4+ cell count <350 cells/mm3, change in antiretroviral therapy regimen within 6
     months prior to Day 0, or plasma viral load >200 copies/mL, on 2 separate occasions,
     as measured by PCR.

  5. Anti-AAVrh10 neutralizing antibody titer >1:5.

  6. Participation (current or previous) in another gene therapy study.

  7. Participation in another investigational medicine study within 3 months before
     screening.

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