ALN-AT3SC-003
Research type
Research Study
Full title
ATLAS-INH: A Phase 3 Study to Evaluate the Efficacy and Safety of Fitusiran in Patients with Hemophilia A or B, with inhibitory antibodies to Factor VIII or IX.
IRAS ID
228319
Contact name
John Pasi
Contact email
Sponsor organisation
Genzyme Corporation
Eudract number
2016-001463-36
Duration of Study in the UK
1 years, 6 months, 2 days
Research summary
Research Summary
This study targets participants who are male with severe haemophilia A or B, with inhibitors, and are at least 12 years of age and are already using BPAs as on-demand (i.e use as needed for episodic bleeding) 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 makes it function by reducing 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 fifty-four (54) male patients with haemophilia with inhibitors are expected to participate. It is expected that about five (5) patients will have haemophilia B, and about five (5) patients will be adolescents.
The study will last up to 11 months, from the time of participant signing the informed consent to the last planned study visit (“Month 9”). Patients who complete the study may be eligible for an open-label extension study. Patients who received fitusiran and do not enrol in the extension study will have antithrombin follow-up for up to 7 months following their last dose.
The Study is being sponsored by Alnylam Pharmaceuticals Incorporated.Summary of results
As per Sanofi Trial Transparency commitment, this study is not eligible for Lay summary
REC name
London - Chelsea Research Ethics Committee
REC reference
18/LO/0124
Date of REC Opinion
16 Feb 2018
REC opinion
Favourable Opinion