Phase 3 paediatric study of Avatrombopag in Immune Thrombocytopenia
Research type
Research Study
Full title
A Phase 3b, Multi-center, Randomized, Double-blind, Placebo-controlled, Parallel-group Trial with an Open-label Extension Phase to Evaluate the Efficacy and Safety of Avatrombopag for the Treatment of Thrombocytopenia in Pediatric Subjects with Immune Thrombocytopenia for ≥6 Months
IRAS ID
1003418
Contact name
John Grainger
Contact email
Sponsor organisation
Dova Pharmaceuticals, Inc.
Eudract number
2020-003232-24
Clinicaltrials.gov Identifier
Research summary
Immune thrombocytopenia is a condition where there is a shortage of blood cells called
platelets due to the body’s immune system attacking and destroying these cells. Platelets
are needed for normal blood clotting. Avatrombopag is a medication that is approved in
Europe and the USA to treat certain types of thrombocytopenia in adults, but in this study
we would like to test the medicine in children and young adults to see how well it works
and how safe it is. There remains an unmet medical need for new treatment options for
paediatric patients with this condition.
The study is planned to involve 72 children and young adults who have immune
thrombocytopenia.
Depending on the participant’s age the study medication will be given as either a tablet
or a powder to be added to food or drink. The study will be conducted in 2 parts. In the
first part of the study (called the Core Phase) participants will receive either
avatrombopag or placebo in a 3:1 ratio. In the second part of the study (called the
Extension Phase) all participants will receive avatrombopag.
Participation in the study will last for a total of up to 29 months (i.e. 2 years and 5
months).
The Core Phase lasts for up to 5 months. In this part of the study participants will be
asked to visit the study site twice in the first week, and then every week for 12 weeks.
Participants will be asked to take the study medicine during this 12 week period.
If participants enter the Extension Phase, after they complete treatment in the Core
Phase they will immediately begin the Extension Phase. Participants will be asked to visit
the study site every month for 2 years. Participants will be asked to take the study
medicine during this period.REC name
London - Westminster Research Ethics Committee
REC reference
20/LO/1195
Date of REC Opinion
30 Dec 2020
REC opinion
Further Information Favourable Opinion