Long term follow-up of DTX101 in adults with Haemophilia B
Research type
Research Study
Full title
A Long-Term Follow-up Study to Evaluate the Safety, Tolerability, and Efficacy of Adeno-Associated Virus (AAV) rh10-Mediated Gene Transfer of Human Factor IX in Adults With Moderate/Severe to Severe Hemophilia B
IRAS ID
224520
Contact name
Charles Hay
Contact email
Sponsor organisation
Dimension Therapeutics, Inc.
Eudract number
2016-003430-25
Clinicaltrials.gov Identifier
Clinicaltrials.gov Identifier
16543, IND Number
Duration of Study in the UK
4 years, 3 months, 28 days
Research summary
Research Summary
Haemophilia B is genetic bleeding disorder that affects approximately 1 in 20-25,000 male births. Mutations in the factor IX gene prevent normal blood clotting. The disease is characterized by frequent, spontaneous internal bleeding that can lead to chronic joint damage, intracranial haemorrhage and even death. In patients with moderate/severe to severe haemophilia B, 90% of bleeding episodes occur in the joints and if not treated properly, this can lead to debilitating damage and a decreased quality of life.
The current standard of care for haemophilia B is intravenous injections of FIX concentrates at the time of a bleed or prophylactically to prevent bleeding. This treatment does not completely prevent the chronic and debilitating damage to joints and soft tissues that follow a bleeding episode.
Study 101HEMB02 is sponsored by Dimension Therapeutics, Inc. The primary reason for the study is to determine the long-term safety and efficacy of a single intravenous (IV) dose of DTX101 administered to adults with moderate/severe to severe haemophilia B during Study 101HEMB01.
Study 101HEMB01 was the first time DTX101 was given to humans. DTX101 is experimental and has not been shown to be safe or effective. DTX101 contains an inactive, non-disease causing virus paired with working copies of the human factor IX gene. It is hoped that DTX101 will be safe and more effective at preventing bleeds than current treatment options and would also avoid the risks and inconvenience of regular IV infusions.
All participants who received a dose of DTX101 and completed Study 101HEMB01 will be asked to participate in Study 101HEMB02. No study drug will be administered during Study 101HEMB02. Clinical visits will be approximately every 13 weeks for 4 years. Visits will include physical examinations, ECGs, completion of quality of life questionnaires and diaries, and collection of blood and urine samples.
Summary of Results
A PDF of the lay summary can be found on https://eur03.safelinks.protection.outlook.com/?url=http%3A%2F%2Fwww.ultragenyx.com%2Fclinical-trials%2F&data=05%7C01%7Capprovals%40hra.nhs.uk%7Ccb9f27124f2f43fda1de08dacd55c4ae%7C8e1f0acad87d4f20939e36243d574267%7C0%7C0%7C638048065495048196%7CUnknown%7CTWFpbGZsb3d8eyJWIjoiMC4wLjAwMDAiLCJQIjoiV2luMzIiLCJBTiI6Ik1haWwiLCJXVCI6Mn0%3D%7C3000%7C%7C%7C&sdata=Z%2BDjdY0%2BjvJf7vsswSMf6TR%2Bf8k2v3%2BW%2BhEmcjfQ6BU%3D&reserved=0
REC name
North East - York Research Ethics Committee
REC reference
17/NE/0136
Date of REC Opinion
11 May 2017
REC opinion
Favourable Opinion