Phase 1/2 Factor VIII gene therapy study 19429 version 1
Research type
Research Study
Full title
A Phase 1/2 Open-Label Safety and Dose-Finding Study of BAY 2599023 (DTX201), an Adeno-Associated Virus (AAV) hu37-Mediated Gene Transfer of B-Domain Deleted Human Factor VIII, in Adults with Severe Haemophilia A
IRAS ID
232642
Contact name
Charles Hay
Contact email
Sponsor organisation
Bayer PLC
Eudract number
2017-000806-39
Duration of Study in the UK
6 years, 3 months, 19 days
Research summary
Male, 18 years old.
• Subjects with severe hemophilia A (baseline FVIII activity FVIII:C<1%)
• Previously treated with FVIII concentrate(s) (plasma derived or recombinant) for a minimum of 150 exposure days (ED)
• Are on one of the following therapies: Prophylaxis, and is able and willing to stop prophylactic treatment at specified time points throughout the study or On-demand: have had > 4 bleeding events in the last 52 weeks
• Male subjects must agree not to act as sperm donors from the time of study drug administration until notified by the investigator.
Major Exclusion criteria:
• Current evidence of inhibitor to FVIII with a titer ≥ 0.6 BU/mL
• History of inhibitor to FVIII with a titer ≥ 0.6 BU, or clinical history requiring modification of treatment, suggestive of inhibitor
• Have significant underlying liver disease as evidenced by any of the following: portal hypertension, splenomegaly, ascites, esophageal varices, hepatic encephalopathy, reduction below normal limits of serum albumin or a liver biopsy with evidence of stage 3 fibrosis
• Have significant hepatic inflammation or cirrhosis as evidenced
• Any major and/or orthopedic surgery within screening period prior to trial product administration, and at least 6 months thereafter
• History of a malignancy for which the subject has received treatment in the past 2 years except for prostate cancer being monitored without medical intervention, or surgically removed non-melanoma skin cancer
• Known or suspected autoimmune diseases
• Known prior history of hypersensitivity or anaphylaxis associated with any FVIII or immunoglobulin administration.
• Known or suspected hypersensitivity or allergic reaction to trial product(s) or related FVIII products or any component of BAY 2599023(DTX201)
• Subjects on treatment with immunomodulatory agents within the last 3 months prior to study entry or during the study
• Any individual who requires any pre-medication to tolerate FVIII treatment (e.g., antihistamines)REC name
South Central - Oxford A Research Ethics Committee
REC reference
18/SC/0432
Date of REC Opinion
24 Oct 2018
REC opinion
Further Information Favourable Opinion