Rilzabrutinib in Adults and Adolescents with Immune Thrombocytopenia
Research type
Research Study
Full title
A Phase 3, Multicenter, Randomized, Double-blind, Placebo-Controlled, Parallel-Group Study with an Open-Label Extension to Evaluate the Efficacy and Safety of Oral Rilzabrutinib (PRN1008) in Adults and Adolescents with Persistent or Chronic Immune Thrombocytopenia (ITP)
IRAS ID
287737
Contact name
John Grainger
Contact email
Sponsor organisation
Principia Biopharma, Inc.
Eudract number
2020-002063-60
Duration of Study in the UK
4 years, 0 months, 0 days
Research summary
Immune Thrombocytopenia (ITP) is characterized by autoantibody-mediated destruction of platelets and impaired platelet production, which result in thrombocytopenia and a predisposition to bleeding associated with morbidity and mortality. The purpose of this study is to explore the efficacy and safety of rilzabrutinib treatment in ITP patients who still have low platelet counts despite having used multiple other therapies.
Rilzabrutinib is an investigational medicine developed by the Sponsor to treat autoimmune and inflammatory diseases, such as ITP. Rilzabrutinib inhibits Bruton’s tyrosine kinase (BTK), an enzyme inside cells that plays an important role in inflammation and autoimmunity.
This is a global, randomized, parallel-group, double-blind, multicenter clinical study in patients with primary ITP who had a response to either intravenous immunoglobulin (IVIg) or corticosteroid (CS) that was not sustained.
Principia Biopharma Inc., is sponsoring and funding this clinical research study. This study will be conducted at approximately 125 sites around the world and will involve approximately 194 male and female patients with ITP, aged 12 and older.
REC name
North West - Haydock Research Ethics Committee
REC reference
20/NW/0438
Date of REC Opinion
8 Dec 2020
REC opinion
Further Information Favourable Opinion