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Hepcidin Mimetic in Patients With Polycythemia Vera

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ClinicalTrials.gov Identifier: NCT04057040
Recruitment Status : Recruiting
First Posted : August 14, 2019
Last Update Posted : December 20, 2019
Sponsor:
Information provided by (Responsible Party):
Protagonist Therapeutics, Inc.

Tracking Information
First Submitted Date  ICMJE August 7, 2019
First Posted Date  ICMJE August 14, 2019
Last Update Posted Date December 20, 2019
Actual Study Start Date  ICMJE October 1, 2019
Estimated Primary Completion Date August 1, 2022   (Final data collection date for primary outcome measure)
Current Primary Outcome Measures  ICMJE
 (submitted: August 13, 2019)
  • Proportion of subjects with absence of phlebotomy eligibility [ Time Frame: Week 42 ]
    "Phlebotomy eligibility" is defined as a confirmed hematocrit >45% that was ≥3% higher than baseline level or a confirmed hematocrit >48%
  • Myeloproliferative Neoplasm Symptom Assessment Form Total Symptom Score (MPN-SAF TSS) [ Time Frame: 42 weeks ]
    The Myeloproliferative Neoplasm Symptom Assessment Form Total Symptom Score (MPN-SAF TSS) is a validated tool for measurement of symptoms for patients. The MPN-SAF includes 14 disease related symptoms scored from 0 (absent) to 10 (worst imaginable) and is used to calculate a total symptom score (TSS; sum of 14 individual symptom scores).
  • Number of Participants with Treatment-Emergent Adverse Events as Assessed by CTCAE v5.0 [ Time Frame: 101 weeks ]
Original Primary Outcome Measures  ICMJE Same as current
Change History
Current Secondary Outcome Measures  ICMJE
 (submitted: August 13, 2019)
  • Change from baseline in serum iron [ Time Frame: 42 weeks ]
  • Change from baseline in transferrin saturation (TSAT) [ Time Frame: 42 weeks ]
  • Change from baseline in serum transferrin [ Time Frame: 42 weeks ]
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 Hepcidin Mimetic in Patients With Polycythemia Vera
Official Title  ICMJE A Phase 2 Study of the Hepcidin Mimetic PTG-300 in Patients With Phlebotomy-Requiring Polycythemia Vera
Brief Summary This is a Phase 2 study with an open-label dose escalation phase followed by a blinded withdrawal phase and an open label extension. The study is designed to monitor the PTG-300 safety profile and to obtain preliminary evidence of efficacy of PTG-300 for the treatment of phlebotomy-requiring polycythemia vera.
Detailed Description Phase 2 study in approximately thirty subjects previously diagnosed with Polycythemia Vera who require phlebotomy on a routine basis. There is a 16 week dose finding phase followed by a dose stabilization phase. Subjects who successfully complete dose stabilization will be entered into 12 week randomized withdrawal phase to confirm the response. Subsequently patients will enter into 1 year open label extension to investigate long term safety.
Study Type  ICMJE Interventional
Study Phase  ICMJE Phase 2
Study Design  ICMJE Allocation: Randomized
Intervention Model: Sequential Assignment
Intervention Model Description:

Part 1:

28 week open-label dose escalation phase in which each subject's dose of PTG-300 is increased at 4-week intervals until the subject reaches the maximum planned dose or has a pre-specified decrease in hematocrit from baseline. After a potentially clinically active dose is found, subjects will be maintained at that dose until Week 29.

Part 2:

12-week blinded randomized withdrawal phase. Subjects are randomized 1:1 to continue PTG-300 or to receive placebo.

Part 3:

1 year open label extension.

Masking: Double (Participant, Investigator)
Masking Description:
Part 1 open label, Part 2 blinded, Part 3 open label.
Primary Purpose: Treatment
Condition  ICMJE Polycythemia Vera
Intervention  ICMJE
  • Drug: PTG-300
    Active
  • Drug: Placebo
    Placebo
Study Arms  ICMJE
  • Experimental: Experimental Dose Escalation (Part 1)
    PTG-300 Subcutaneous (SC) Weekly Each subject's dose is increased at 4 week intervals until the subject reaches the maximum planned dose or has a prespecified decrease in hematocrit from baseline.
    Intervention: Drug: PTG-300
  • Experimental: Blinded Withdrawal (Part 2) PTG-300 or placebo
    PTG-300 or placebo Subcutaneous (SC) Weekly
    Interventions:
    • Drug: PTG-300
    • Drug: Placebo
  • Experimental: Experimental Open label extension (Part 3) PTG-300
    PTG-300 Subcutaneous (SC) Weekly
    Intervention: Drug: PTG-300
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 Recruiting
Estimated Enrollment  ICMJE
 (submitted: August 13, 2019)
30
Original Estimated Enrollment  ICMJE Same as current
Estimated Study Completion Date  ICMJE November 1, 2022
Estimated Primary Completion Date August 1, 2022   (Final data collection date for primary outcome measure)
Eligibility Criteria  ICMJE

Main Inclusion Criteria: All subjects must meet ALL of the following inclusion criteria to be enrolled.

  1. Male and female subjects aged 18 years or older.
  2. Meet revised 2016 World Health Organization (WHO) criteria for the diagnosis of polycythemia vera.
  3. Records of all phlebotomies performed for at least 24 weeks (preferably up to 52 weeks) before screening are available.
  4. Subjects who are not receiving cytoreductive therapy must have been discontinued from any prior cytoreductive therapy for at least 24 weeks before screening and have recovered from any adverse events due to cytoreductive therapy.
  5. Subjects receiving cytoreductive therapy with hydroxyurea, interferon, or ruxolitinib must be on a stable dose for at least 24 weeks and be on a stable dose for at least 8 weeks before screening and with no planned change in dose.

Main Exclusion Criteria: Subjects must meet NONE of the following exclusion criteria to be enrolled:

  1. Active or chronic bleeding within 4 weeks of screening.
  2. Meets the criteria for post-PCV myelofibrosis as defined by the International Working Group-Myeloproliferative Neoplasms Research and Treatment (IWG-MRT).
  3. Known primary or secondary immunodeficiency.
  4. Any surgical procedure requiring general anesthesia within 1 month prior to screening or planned elective surgery during the study.
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: Study Director 1-888-899-1543 ptgxclintrials@ptgx-inc.com
Listed Location Countries  ICMJE United States
Removed Location Countries  
 
Administrative Information
NCT Number  ICMJE NCT04057040
Other Study ID Numbers  ICMJE PTG-300-04
Has Data Monitoring Committee Yes
U.S. FDA-regulated Product
Studies a U.S. FDA-regulated Drug Product: Yes
Studies a U.S. FDA-regulated Device Product: No
IPD Sharing Statement  ICMJE Not Provided
Responsible Party Protagonist Therapeutics, Inc.
Study Sponsor  ICMJE Protagonist Therapeutics, Inc.
Collaborators  ICMJE Not Provided
Investigators  ICMJE Not Provided
PRS Account Protagonist Therapeutics, Inc.
Verification Date December 2019

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