Open-label Extension Study of ALN-AT3SC in Haemophilia Patients
Research type
Research Study
Full title
An Open-label Extension Study of Subcutaneously Administered ALN-AT3SC in Patients with Moderate or Severe Hemophilia A or B who have Participated in a Previous Clinical Study with ALN-AT3SC
IRAS ID
184400
Contact name
Catherine Bagot
Contact email
Sponsor organisation
Alnylam Pharmaceuticals, Inc.
Eudract number
2015-001395-21
Duration of Study in the UK
3 years, 1 months, 1 days
Research summary
Haemophilia is an inherited bleeding disorder in which blood does not clot normally because the body is unable to produce or has a defective blood factor protein (for example factor VIII or IX), which is required for blood clotting. Patients with haemophilia will typically bleed after injury, but also can have frequent spontaneous bleeds, most commonly into muscles and joints. Recurrent bleeding into a joint can result in a chronic and disabling arthropathy that is painful and disfiguring, and can significantly impair a patient’s mobility and quality of life.
ALN-AT3SC is an investigational medicine being developed by Alnylam Pharmaceuticals, Inc (the Sponsor) for haemophilia A or B treatment. ALN-AT3SC works by reducing the amount of a protein called antithrombin (AT) produced in the liver which has a role in preventing blood from clotting. In patients with haemophilia, researchers want to test whether lowering AT protein may help them clot more easily leading to improved haemostasis. Data from a prior study with ALN-AT3SC suggest that a reduction in AT levels is sustained for up to 60 days following a single dose of ALN-AT3SC.
This is a multicenter, multinational, open label extension study to evaluate the long term safety, tolerability and effectiveness of ALN-AT3SC in male patients with moderate or severe haemophilia A or B, who have previously tolerated dosing in a clinical study with ALN-AT3SC. ALN-AT3SC will be administered by subcutaneous injection (under the skin of abdomen) once every 30 days and treatment will last for approximately 2 years. Up to 30 patients will be enrolled in this study, 8 of which are planned to be enrolled from the UK.REC name
HSC REC A
REC reference
15/NI/0127
Date of REC Opinion
22 Jul 2015
REC opinion
Further Information Favourable Opinion