Clinical Trial: Study of PRTX-100 in Adult Patients With Persistent/Chronic Immune Thrombocytopenia
Study Status: Recruiting
Recruit Status: Recruiting
Study Type: Interventional
Official Title: A Phase 1b Open-Label, Dose Escalation Study of PRTX-100 in Adult Patients With Persistent/Chronic Immune Thrombocytopenia
Brief Summary: Pre-clinical and clinical evaluations show that PRTX- 100 has biological activity that may lead to improved platelet levels where these are decreased due to immunological pathologies and that PRTX-100 has an acceptable safety profile. In vivo treatment with PRTX-100 has been shown to raise platelet counts in a mouse model of immune thrombocytopenia (ITP). The primary objective of the study is to assess the efficacy of PRTX-100 in terms of platelet response in patients with chronic/persistent ITP.
Detailed Summary:
Sponsor: Protalex, Inc.
Current Primary Outcome: Number of participants with treatment-related adverse events as assessed by Toxicity Grading Criteria based on RCTC v 2.0 and CTCAE v 4.03 [ Time Frame: 337 Days ]
Original Primary Outcome: Same as current
Current Secondary Outcome:
- Overall platelet response, change from baseline (Day 1) [ Time Frame: Days 1, 3, 8, 15, 22, 29, 36, 43, 50, 78, 106, 169, and 337 ]Defined as a platelet count ≥ 30,000/μL and at least a doubling of baseline platelet count in patients with a baseline platelet count <30,000/μL in the absence of any concomitant rescue therapy.
- Complete platelet response (number of patients) [ Time Frame: Days 3, 8, 15, 22, 29, 36, 43, 50, 78, 106, 169, and 337 ]The number of patients demonstrating a complete platelet response, defined as a platelet count ≥ 100,000/μL.
- Time to platelet response (number of days) [ Time Frame: Days 3, 8, 15, 22, 29, 36, 43, 50, 78, 106, 169, and 337 ]The mean number of days from first PRTX-100 dose (Day 1) until platelet response.
- Durability of platelet response (number of days) [ Time Frame: Days 3, 8, 15, 22, 29, 36, 43, 50, 78, 106, 169, and 337 ]The number of days from first documented platelet response to first platelet count below platelet response criteria.
- Concomitant ITP medication use (number of subjects) [ Time Frame: 337 Days ]The number of subjects considered non-responders based on concomitant ITP medication use by cohort and overall. ITP medications include thrombopoietin receptor agonists (TPO-RAs), steroid-sparing adjunctive immunosuppressive treatment (e.g. cyclosporine, azathioprine, mycophenolate), and any ITP rescue medications (e.g. IVIG) received during the study Screening and Treatment Periods.
Original Secondary Outcome: Same as current
Information By: Protalex, Inc.
Dates:
Date Received: September 30, 2015
Date Started: November 2015
Date Completion: May 2018
Last Updated: April 26, 2017
Last Verified: April 2017