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 ]

Adverse events from AEs, SAEs, infusion reactions, clinical laboratory tests (hematology, blood chemistry and urinalysis), vital signs, physical findings and ECGs over the course of the study. AE severity will be graded according to Toxicity Grading Criteria derived from published standards.


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