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

Stopped early · Phase 1/Phase 2

Conditions studied: Hemophilia B

In brief

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.

Key facts

Study ID

NCT02618915

Run by

Ultragenyx Pharmaceutical Inc

People needed

6

Starts

2015-12-16

Expected to finish

2017-10-18

Last updated by the study team

2018-11-14

Who can join

Age: 18 and older. Sex: male. Healthy volunteers: not accepted.

You may qualify if…

• Male ≥ 18 years of age.
• Moderate/severe or severe hemophilia B (baseline FIX activity ≤ 2% of normal or documented history of FIX activity ≤2%).
• At least 3 bleeding episodes per year that require on-demand treatment with FIX OR are treated with a prophylactic regimen of FIX.
• At least 100 days exposure history to FIX.
• No documented history of inhibitors (neutralizing antibodies) to exogenous FIX.
• No known allergic reaction to exogenous FIX or any component of DTX101.
• Willing to stop prophylactic treatment with recombinant FIX at specified time points during the study.

You may not qualify if…

• History of significant liver disease (ie, portal hypertension).
• Significant hepatic inflammation or cirrhosis.
• Evidence of active hepatitis B virus (HBV) or hepatitis C virus (HCV) infection.
• History of human immunodeficiency virus (HIV) infection AND any of the following: CD4+ cell count < 350 cells/mm\^3, 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 polymerase chain reaction.
• Anti-AAVrh10 neutralizing antibody titer > 1:5.
• Participation (current or previous) in another gene therapy study.
• Participation in another investigational medicine study within 3 months before screening.
• NOTE: Other protocol defined inclusion/exclusion criteria may apply.

Where it is running

• Arkansas Children's Hospital — Little Rock, Arkansas, United States
• Orthopaedic Institute for Children — Los Angeles, California, United States
• University of Florida — Gainesville, Florida, United States
• Boston Children's Hospital — Boston, Massachusetts, United States
• University of Michigan Hospital and Health Systems, Michigan Clinical Research Unit — Ann Arbor, Michigan, United States
• Vanderbilt Hemostasis-Thrombosis Clinic — Nashville, Tennessee, United States
• Specialized Hospital for Active Treatment for Hematological Disease — Sofia, Bulgaria
• Basingstoke and North Hampshire Hospital, Haemophilia, Haemostasis and Thrombosis Centre — Basingstoke, Hampshire, United Kingdom
• The Christie NHS Foundation Trust — Manchester, United Kingdom

Full record on ClinicalTrials.gov

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