Clinical Trial: Open-Label, Dose Escalation Study of PRTX-100 in Adults With Persistent/Chronic Immune Thrombocytopenia

Study Status: Recruiting
Recruit Status: Recruiting
Study Type: Interventional

Official Title: A Phase 1/2, 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: Platelet Response, Change from Baseline [ Time Frame: Days 3, 8, 15, 22, 29, 36, 43, 50, 78, 106, 169, and 337 ]

The primary efficacy endpoint is platelet response defined as a platelet count ≥ 30,000/μL and at least a doubling of baseline platelet count (determined on Day 1 prior to PRTX-100 administration) in patients with a baseline platelet count < 30,000/μL. In patients receiving permitted treatments for ITP with a baseline platelet count ≥ 30,000/μL and < 50,000/μL, an increase in platelet count to ≥ 50,000/μL will be considered a platelet response.


Original Primary Outcome: Platelet Response, Change from Baseline [ Time Frame: Days 3, 8, 15, 22, 29, 36, 43, 50, 78, 106, 169, and 337 ]

The primary efficacy endpoint is platelet response defined as a platelet count ≥ 30,000/μL and at least a doubling of baseline platelet count (determined on Day 1 prior to PRTX-100 administration) in patients with a baseline platelet count < 30,000/μL.


Current Secondary Outcome:

  • Complete platelet response, Change from Baseline [ Time Frame: Days 3, 8, 15, 22, 29, 36, 43, 50, 78, 106, 169, and 337 ]
    Defined as a platelet count ≥ 100,000/μL
  • Time to platelet response defined as the mean number of days from first PRTX-100 dose until platelet response [ 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 until platelet response
  • Durability of platelet response [ 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 (frequency and amount) [ Time Frame: Days 3, 8, 15, 22, 29, 36, 43, 50, 78, 106, 169, and 337 ]
    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
  • Adverse Events [ Time Frame: Days 3, 8, 15, 22, 29, 36, 43, 50, 78, 106, 169, and 337 ]
    Safety will be described by AEs, SAEs, infusion reactions, clinical laboratory tests (hematology, blood chemistry and urinalysis), vital signs, physical findings and ECGs. AE severity will be graded according to Toxicity Grading Criteria derived from published standards


Original Secondary Outcome: Same as current

Information By: Protalex, Inc.

Dates:
Date Received: March 18, 2015
Date Started: September 2015
Date Completion: June 2018
Last Updated: April 26, 2017
Last Verified: April 2017