ALN-AT3SC-009
Research type
Research Study
Full title
ATLAS- PPX: an open-label, multi-national, switching study to describe the efficacy and safety of fitusiran prophylaxis in patients with haemophilia A and B with inhibitory antibodies to factor VIII or IX previously receiving bypassing agent prophylaxis.
IRAS ID
228704
Contact name
Catherine Bagot
Contact email
Sponsor organisation
Alnylam Pharmaceuticals, Inc.
Eudract number
2016-004087-19
Duration of Study in the UK
2 years, 10 months, 0 days
Research summary
This study target are participants who are male with severe haemophilia A or B, with or without inhibitors, are at least 12 years of age and are already using bypassing agents (BPA) as prophylactic treatment for their haemophilia.
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 (study drug) may make it possible to reduce or prevent haemophilia-related bleeding. The technology behind fitusiran reduces the amount of a substance called ‘antithrombin’ (AT). Reducing AT may reduce the number of bleeds a person experiences, and data from ongoing small trials in haemophilia patients suggests this.
The study’s goal is to determine the frequency of bleeding episodes in patients receiving fitusiran as prophylactic treatment of haemophilia. The study will also monitor for potential side effects of fitusiran and measure how the body handles (distributes, breaks down, and eliminates) fitusiran. Participants’ disease state and safety will be carefully monitored by their study doctors through various tests and observations.
About thirty (30) male patients with haemophilia will have inhibitors, seven(7) patients will have haemophilia B, and about three (3) patients will be adolescents.
The study will last up to 15 months, from the time of participant signing the informed consent to the last planned study visit (“End of Study Visit”). Patients who complete the study may be eligible for participation in an open-label extension study. For patients who do not enroll in the extension study, antithrombin activity level will be monitored for up to 7 months following their last dose.
The Study is being sponsored by Alnylam Pharmaceuticals Incorporated.REC name
London - Central Research Ethics Committee
REC reference
18/LO/0438
Date of REC Opinion
9 May 2018
REC opinion
Further Information Favourable Opinion