PRN1008 – an Oral BTK Inhibitor in Patients with Relapsed ITP
Research type
Research Study
Full title
An Adaptive, Open-Label, Dose-Finding, Phase 1/2 Study Investigating the Safety, Pharmacokinetics, and Clinical Activity of PRN1008, an Oral BTK Inhibitor, in Patients with Relapsed Immune Thrombocytopenia
IRAS ID
241907
Contact name
Nichola Cooper
Contact email
Sponsor organisation
Principia Biopharma Inc.
Eudract number
2017-004012-19
Clinicaltrials.gov Identifier
Duration of Study in the UK
1 years, 1 months, 28 days
Research summary
Immune thrombocytopenic purpura (ITP) is an autoimmune disease which causes a shortage of platelets, the clotting cells in the blood. According to the ITP Support Association approximately 3000–4000 people are affected by ITP at any one time in the UK.
A majority of patients respond initially to first line treatments such as corticosteroids, the rate of continued response to treatment and reduction of symptoms is low. Novel, safe and effective, oral treatments to maintain platelet counts in patients with ITP would be a significant therapeutic advantage.
PRN1008 binds strongly to, and reduces the activity of Bruton’s tyrosine kinase (BTK). BTK is an enzyme which is crucial to the development of certain white blood cells. There is preliminary evidence to support the role of drugs which target BTK in patients with autoimmune cytopenias, the group of disorders to which ITP belongs.
This study will be conducted at 4 hospitals in the UK and consists of a treatment period and a follow up period, each 12 weeks long. The study will recruit patients with ITP who are unresponsive to treatment and with a platelet count of less than 30,000/μL. The study aims to evaluate the safety and effectiveness of PRN1008 at four dose levels: 200mg, 400mg each once a day, then 600mg and 800mg each twice a day.
Each participant enrolled on to the study will increase their dose level after 28 days of treatment if they do not experience an increase in their platelet count or dose limiting toxicity (too many side effects to increase the dose). The first 3 or 6 eligible participants at each dose level will be evaluated by an Independent Data Safety Monitor to choose the starting dose for additional participants. Participants will have weekly visits/laboratory tests throughout the study.
REC name
London - West London & GTAC Research Ethics Committee
REC reference
18/LO/0430
Date of REC Opinion
11 Jun 2018
REC opinion
Further Information Favourable Opinion