This phase I/II trial is trying to understand the safety and effectiveness of a targeted therapy (PRN1008) in people with relapsed Immune ThrombocytopeniaAn 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

Summary

This is a 2 part, adaptive, open-label dose-finding study of PRN1008 in people with Immune Thrombocytopenic Purpura (ITP). Part A enrols people with ITP who are refractory or relapsed with no available and approved therapeutic options. Eligible patients have a platelet count <30,000/μL on two counts no sooner than 7 days apart in the 15 days before treatment begins. The active treatment period is 24 weeks and the post-treatment follow-up period is 4 weeks. In the dose-finding part of the study, each patient enrolled in the study is allowed to up-titrate their dose after 28 days of PRN1008 therapy, if they do not experience a platelet response or a dose-limiting toxicity (DLT) at the last dose level. Patients who respond to PRN1008 per protocol may enter a long term-extension. Part B of the study will include people with ITP who have relapsed or have insufficient response to prior therapies. Eligible patients will have a platelet count <30,000/μL on two occasions no less than 7 days apart, within 15 days before treatment begins and a platelet count of ≤35,000/μL on Study Day 1 (SD1). The study consists of a 28-day screening period, 24-week active treatment period, and a long-term extension. After the last dose of PRN1008 there will be a 4-week safety follow-up period.

Conditions

This trial is treating patients with Immune Thrombocytopenia.

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