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PRTX-100-203 Open-Label, Dose Escalation Study in Adult Patients With ITP

The safety and scientific validity of this study is the responsibility of the study sponsor and investigators. Listing a study does not mean it has been evaluated by the U.S. Federal Government. Read our disclaimer for details.
 
ClinicalTrials.gov Identifier: NCT02566603
Recruitment Status : Terminated (Enough data has been collected to allow analysis of the safety profile and risk-benefit.)
First Posted : October 2, 2015
Last Update Posted : March 25, 2019
Sponsor:
Information provided by (Responsible Party):
Protalex, Inc.

Tracking Information
First Submitted Date  ICMJE September 30, 2015
First Posted Date  ICMJE October 2, 2015
Last Update Posted Date March 25, 2019
Study Start Date  ICMJE November 2015
Actual Primary Completion Date May 17, 2018   (Final data collection date for primary outcome measure)
Current Primary Outcome Measures  ICMJE
 (submitted: October 1, 2015)
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 Measures  ICMJE Same as current
Change History
Current Secondary Outcome Measures  ICMJE
 (submitted: October 1, 2015)
  • 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 Measures  ICMJE Same as current
Current Other Pre-specified Outcome Measures Not Provided
Original Other Pre-specified Outcome Measures Not Provided
 
Descriptive Information
Brief Title  ICMJE PRTX-100-203 Open-Label, Dose Escalation Study in Adult Patients With ITP
Official Title  ICMJE 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 Description Not Provided
Study Type  ICMJE Interventional
Study Phase  ICMJE Phase 1
Phase 2
Study Design  ICMJE Allocation: N/A
Intervention Model: Single Group Assignment
Masking: None (Open Label)
Primary Purpose: Treatment
Condition  ICMJE Immune Thrombocytopenia
Intervention  ICMJE
  • Drug: PRTX-100
    Four weekly infusions of PRTX-100 at a level of 3 micrograms of PRTX-100 per kilogram of patient weight, infused over 30 minutes, followed by four hours of observation after start of infusion.
    Other Names:
    • SpA
    • Staphylococcal Protein A
  • Drug: PRTX-100
    Four weekly infusions of PRTX-100 at a level of 6 micrograms of PRTX-100 per kilogram of patient weight, infused over 30 to 60 minutes, followed by four hours of observation after start of infusion.
    Other Names:
    • SpA
    • Staphylococcal Protein A
  • Drug: PRTX-100
    Four weekly infusions of PRTX-100 at a level of 12 micrograms of PRTX-100 per kilogram of patient weight, infused over 60 minutes, followed by four hours of observation after start of infusion.
    Other Names:
    • SpA
    • Staphylococcal Protein A
  • Drug: PRTX-100
    Four weekly infusions of PRTX-100 at a level of 18 micrograms of PRTX-100 per kilogram of patient weight, infused over 60 minutes, followed by four hours of observation after start of infusion.
    Other Names:
    • SpA
    • Staphylococcal Protein A
  • Drug: PRTX-100
    Four weekly infusions of PRTX-100 at a level of 24 micrograms of PRTX-100 per kilogram of patient weight, infused over 60 minutes, followed by four hours of observation after start of infusion.
    Other Names:
    • SpA
    • Staphylococcal Protein A
Study Arms  ICMJE Experimental: PRTX-100
Patients will be assigned to consecutive PRTX-100 interventions as they are enrolled into the study. Between three and six patients will be enrolled per intervention level. Intervention levels range from 3 to 24 micrograms of PRTX-100 per kilogram of patient weight. Patients may receive up to four weekly infusions of PRTX-100 over the study treatment period. PRTX-100 doses ≤ 500 μg will be infused intravenously over 30 minutes. PRTX-100 doses > 500 μg will be infused over 60 minutes. Patients will remain under observation for 4 hours after initiation of PRTX-100 dosing for safety management.
Interventions:
  • Drug: PRTX-100
  • Drug: PRTX-100
  • Drug: PRTX-100
  • Drug: PRTX-100
  • Drug: PRTX-100
Publications * Not Provided

*   Includes publications given by the data provider as well as publications identified by ClinicalTrials.gov Identifier (NCT Number) in Medline.
 
Recruitment Information
Recruitment Status  ICMJE Terminated
Actual Enrollment  ICMJE
 (submitted: March 21, 2019)
15
Original Estimated Enrollment  ICMJE
 (submitted: October 1, 2015)
30
Actual Study Completion Date  ICMJE May 17, 2018
Actual Primary Completion Date May 17, 2018   (Final data collection date for primary outcome measure)
Eligibility Criteria  ICMJE

Inclusion Criteria:

  1. Willing and able to provide written informed consent prior to initiation of any study-related procedures
  2. Male or female ≥ 18 years of age
  3. ITP that has persisted for ≥ 3 months. ITP must be diagnosed in accordance The American Society of Hematology 2011 Evidence-based Practice Guideline for Immune Thrombocytopenia (Neunert et al. 2011) or the International Consensus Report on The Investigation and Management of Primary Immune Thrombocytopenia (Provan et al. 2010), as locally applicable.
  4. Received ≥ 1 typical regimen for the treatment of ITP. Splenectomy is considered one regimen.
  5. A mean platelet count of < 30,000/μL with no individual platelet count > 55,000/μL. The mean platelet count must be determined based on 2 platelet counts including one obtained within ≤ 7 days of first PRTX-100 dose and the other within ≤ 30 days of the first dose of PRTX-100.
  6. If on corticosteroids, a dose of < 1 mg/kg prednisone per day or equivalent that has been stable for ≥ 21 days prior to the first dose of PRTX-100. High-dose pulse steroid therapy is NOT allowed within 14 days prior to the first dose of PRTX-100.
  7. If on steroid-sparing adjunctive immunosuppression with cyclosporine, azathioprine, mycophenolate, or 6-mercaptopurine, the dose must have been stable for ≥ 30 days prior to the first dose of PRTX-100 and must be expected to remain stable through study Day 29, unless dose reduction is required due to toxicities. Treatment with other cytotoxic agents (e.g. cyclophosphamide, vincristine) are not allowed within three months prior to the first dose of PRTX- 100.
  8. Any prior treatment with rituximab or any other anti-CD20 agent must have been > 6 months prior to the first dose of PRTX-100
  9. If female, must not be pregnant (as indicated by screening negative pregnancy test), must not be nursing and must be one of the following:

    • Surgically sterile (bilateral tubal ligation, hysterectomy)
    • Postmenopausal with last natural menses > 24 months prior
    • Premenopausal and using an acceptable form of birth control. Acceptable forms of birth control include: hormonal contraceptives (implantable, oral, patch) used for ≥ 2 months prior to screening or double barrier methods (any combination of two of the following: intrauterine device [IUD], male or female condom with spermicidal gel, diaphragm, sponge, cervical cap). All premenopausal females must have a negative urine or serum pregnancy test at screening and on Day 1 prior to first PRTX-100 treatment.

Exclusion Criteria:

  1. Splenectomy ≤ 90 days prior to the first dose of PRTX-100
  2. Exposure to TPO-RA within 2 weeks before inclusion
  3. Previous treatment with rituximab within <6 months prior to the first dose of PRTX-100
  4. Bleeding score ≥ 8 (Khellaf M et al. Haematologica 2005)
  5. Unstable coronary artery disease or other medical condition (such as type 1 diabetes) that, in the investigator's opinion, might increase the risk to the patient
  6. Evidence of active infection requiring antibiotic therapy ≤ 14 days prior to the first dose of PRTX-100
  7. Myelodysplastic syndrome. If clinically significant anemia or pancytopenia exists, documentation of a bone marrow aspirate within 24 months prior to the first dose of PRTX- 100 showing no evidence of myelodysplasia is required.
  8. Medical history systemic lupus erythematosus or any cause of secondary ITP
  9. History of any treatment for cancer within the past two years other than basal cell or squamous cell carcinoma of the skin that has been treated with curative intent
  10. Seropositive for human immunodeficiency virus (HIV)
  11. History of acute/chronic hepatitis B or C and/or carriers of hepatitis B or C (positive for hepatitis B surface antigen or positive anti-hepatitis C antibody test)
  12. History suggestive of substance abuse
  13. Clinically significant abnormalities in screening laboratory tests, including:

    • Absolute neutrophil count < 1.0 x109/L
    • Hemoglobin < 10 g/dL
    • Absolute lymphocyte count < 0.8 x109/L
    • Alanine transaminase (ALT) or aspartate transaminase (AST) > 2 x upper limit of normal (ULN)
    • Lactate dehydrogenase > 3 x ULN
    • Total bilirubin level >1.5 x ULN
    • Serum creatinine level > 0.14 mmol/L (1.6 mg/dL) in males or 0.12 mmol/L (1.4 mg/dL) in females
  14. Treatment with IVIG ≤ 14 days prior to the first dose of PRTX-100
  15. Treatment with an anti-Rh D antigen agent (e.g. WinRho®) ≤ 14 days prior to the first dose of PRTX-100
  16. Use of any investigational drug ≤ 30 days or 5 half-lives of the investigational drug (whichever is longer) prior to the first dose of PRTX-100
Sex/Gender  ICMJE
Sexes Eligible for Study: All
Ages  ICMJE 18 Years and older   (Adult, Older Adult)
Accepts Healthy Volunteers  ICMJE No
Contacts  ICMJE Contact information is only displayed when the study is recruiting subjects
Listed Location Countries  ICMJE France,   United Kingdom
Removed Location Countries  
 
Administrative Information
NCT Number  ICMJE NCT02566603
Other Study ID Numbers  ICMJE PRTX-100-203
2014-005626-35 ( EudraCT Number )
Has Data Monitoring Committee Yes
U.S. FDA-regulated Product Not Provided
IPD Sharing Statement  ICMJE Not Provided
Responsible Party Protalex, Inc.
Study Sponsor  ICMJE Protalex, Inc.
Collaborators  ICMJE Not Provided
Investigators  ICMJE
Study Director: William E Gannon, MD Protalex, Inc.
PRS Account Protalex, Inc.
Verification Date March 2019

ICMJE     Data element required by the International Committee of Medical Journal Editors and the World Health Organization ICTRP