A Multi-Center, Observational Study in Males with Hemophilia A
Research type
Research Study
Full title
A Multi-Center, Observational Study in Males with Hemophilia A
IRAS ID
257217
Contact name
Gerard Dolan
Contact email
Sponsor organisation
Spark Therapeutics
Duration of Study in the UK
0 years, 11 months, 31 days
Research summary
SPK-8011-301 is a prospective, multi-centre, observational study to collect efficacy and selected safety data on frequency and type of bleeding episodes and FVIII prophylaxis replacement therapy in the usual care setting of adult males with clinically severe haemophilia A who are negative for neutralising antibody (Nab) to AAV-Spark200.
Haemophilia A is a condition in which blood is not able to clot effectively. It is caused by a missing or defective gene that makes FVIII clotting protein. People with haemophilia A have a low amount of FVIII protein or none. FVIII protein is needed to make blood clot and very low levels of FVIII protein may cause bleeding. People with haemophilia A usually take FVIII replacement therapy to allow their blood to clot.
The purpose of this observational study is to collect information on participant`s haemophilia, including the bleeding events participants experience and the Factor Eight ("FVIII") replacement therapy that they will be using. Participants will not be given any study treatment for haemophilia; instead, they will continue to use their usual FVIII replacement therapy.This study may involve 4 or more visits for approximately 7 up to 13 months. This includes a screening period of up to 8 weeks and an observation period for a minimum of 6 months.
During the Screening Period participants will provide written informed consent and undergo procedures to determine study eligibility. During the Data Collection Period participants will attend study site visits every 3 months to collect information on the protocol-defined efficacy, safety, and exploratory endpoints. Participants will be followed for a minimum of 4 weeks up to a maximum of 12 months.
The study will be conducted at approximately 40 study centers worldwide. This study is sponsored and funded by Spark Therapeutics. Approximately 50-55 participants will be enrolled on this study.REC name
London - Queen Square Research Ethics Committee
REC reference
19/LO/1671
Date of REC Opinion
12 Dec 2019
REC opinion
Further Information Favourable Opinion