Project Title

A Study of PRN1008 in Adult Patients With Immune Thrombocytopenic Purpura (ITP).

Official Title

An Adaptive, Open-Label, Dose-Finding, Phase I/II Study Investigating the Safety, Pharmacokinetics, and Clinical Activity of PRN1008, an Oral BTK Inhibitor, in Patients With Relapsed Immune Thrombocytopenic Purpura.

Project Summary

This is an adaptive, open-label, dose-finding study of PRN1008 in up to 40 patients with ITP who are refractory or relapsed with no available and approved therapeutic options, with 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. 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.

Blood Disorder

• Relapsed Immune Thrombocytopenic Purpura

Patient Recruitment Details

Patient recruitment status: Closed

Number of Patients (Globally)

40 participants.