OLE-Patients with Hemophilia A or B or without Inhibitory FVIII or FIX
Research type
Research Study
Full title
ATLAS-OLE: An Open-label, Long-term Safety and Efficacy Study of Fitusiran in Patients with Hemophilia A or B, with or without Inhibitory Antibodies to Factor VIII or IX
IRAS ID
259669
Contact name
John Pasi
Contact email
Sponsor organisation
Genzyme Corporation
Eudract number
2018-002880-25
Clinicaltrials.gov Identifier
Duration of Study in the UK
6 years, 3 months, 1 days
Research summary
This study targets participants who are male with haemophilia A or B with or without inhibitors, and are at least 12 years of age who have completed a Phase 3 clinical parent study of fitusiran (study drug).
Haemophilia is a rare bleeding problem in which blood does not clot normally. This means that people with haemophilia may bleed for longer periods of time after an injury or, they may develop bleeds spontaneously (bleeding episodes).
Fitusiran may make it possible to prevent or reduce the frequency of haemophilia-related bleedings in patients with haemophilia.
The purpose of the study is to evaluate if SC (subcutaneous) administration of fitusiran reduces the number of bleeds patients experience over a long period of time. The study will also monitor for potential side effects of fitusiran and any potential improvements in patients’ haemophilia. In addition, the study will examine how the body handles (distributes, breaks down, and eliminates) fitusiran by measuring the amount of fitusiran that gets into the blood after it is given to patients.
Study procedures include physical examination, vital signs, ECG, blood sampling, liver & kidney function tests and various assessments.
The study will last up to 55 months and is planned to include approximately 244 participants.
This study is being sponsored by Genzyme Corporation, a Sanofi Company.
REC name
London - Dulwich Research Ethics Committee
REC reference
19/LO/0227
Date of REC Opinion
23 May 2019
REC opinion
Further Information Favourable Opinion