Romiplostim in Thrombocytopenic Paediatric Patients with ITP
Research type
Research Study
Full title
A Single Arm, Open-label, Long-term Efficacy and Safety Study of Romiplostim in Thrombocytopenic Pediatric Subjects With Immune Thrombocytopenia (ITP)
IRAS ID
156734
Contact name
Veena Dhir
Contact email
Sponsor organisation
Amgen Inc
Eudract number
2011-005019-96
Duration of Study in the UK
5 years, 6 months, 1 days
Research summary
The purpose of this study is to find out more about romiplostim in people with low platelet counts, a condition called thrombocytopenia. Platelets are necessary to help the blood clot. People with low platelet counts are at risk for bleeding and bruising. In children the most common cause is immune thrombocytopenia or “ITP”.
This study will see if romiplostim is safe in children with ITP and whether it causes any side effects.
This study will also look to see if romiplostim can raise the platelet counts in children with ITP and to evaluate changes in bone marrow morphology (structure) after long-term exposure to romiplostim.
Romiplostim is still experimental in children with ITP, but is approved by regulatory health agencies (like the Food and Drug Administration [FDA] or European Medicines Agency [EMA]) for adults with ITP.The route of administration, schedule, starting dose, and subsequent dose adjustments of romiplostim have been well established in previous ITP studies in both children and adults and will be used for dosing and administration in this study. At first, Romiplostim will be administered in the clinic (by qualified health care provider at NHS sites) as a subcutaneous injection. Once a stable dose is established the patient will be able to self-administer at home. Eligible patients will be between 1 to 18 years old. The maximum time on study (including a max 3 year treatment period) is 3 years & 2 mths.
REC name
North West - Haydock Research Ethics Committee
REC reference
14/NW/1354
Date of REC Opinion
20 Nov 2014
REC opinion
Further Information Favourable Opinion