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Hepcidin Mimetic in Patients With Polycythemia Vera (REVIVE)

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ClinicalTrials.gov Identifier: NCT04057040
Recruitment Status : Recruiting
First Posted : August 14, 2019
Last Update Posted : January 26, 2022
Sponsor:
Information provided by (Responsible Party):
Protagonist Therapeutics, Inc.

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.

Condition or disease Intervention/treatment Phase
Polycythemia Vera Drug: PTG-300 Drug: Placebo Phase 2

Detailed Description:
Phase 2 study in approximately sixty subjects previously diagnosed with Polycythemia Vera who require phlebotomy on a routine basis. There is a 28 week dose finding phase to identify a dose that maintains hematocrit <45%. Subjects who successfully complete the dose finding phase will be entered into a 12 week randomized withdrawal phase to confirm the response. Subsequently patients will enter into an up to 3 year open label extension to investigate long term safety.

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Study Type : Interventional  (Clinical Trial)
Estimated Enrollment : 80 participants
Allocation: Randomized
Intervention Model: Parallel Assignment
Intervention Model Description:

Part 1:

28 week open-label dose escalation phase in which each subject's dose of PTG-300 is titrated to achieve a hematocrit <45%.

Part 2:

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

Part 3:

Up to 3 year open label extension.

Masking: Triple (Participant, Investigator, Outcomes Assessor)
Masking Description: Part 1 open label, Part 2 blinded, Part 3 open label
Primary Purpose: Treatment
Official Title: A Phase 2 Study of the Hepcidin Mimetic PTG-300 in Patients With Phlebotomy-Requiring Polycythemia Vera
Actual Study Start Date : October 1, 2019
Estimated Primary Completion Date : February 17, 2022
Estimated Study Completion Date : March 1, 2025


Arm Intervention/treatment
Experimental: Dose finding PTG-300 (Part 1); PTG-300 (Part 2); Open label extension PTG-300 (Part 3) Drug: PTG-300
Active

Experimental: Dose finding PTG-300 (Part 1); Placebo (Part 2); Open label extension PTG-300 (Part 3) Drug: PTG-300
Active

Drug: Placebo
Placebo




Primary Outcome Measures :
  1. Proportion of responders during the blinded randomized withdrawal period (Week 29 to Week 41). [ Time Frame: 12 weeks ]

    A subject will be considered a responder during the blinded randomized withdrawal phase if hematocrit control is maintained without phlebotomy eligibility.

    "Phlebotomy eligibility" is defined as any one of the following criteria being met:

    • hematocrit ≥45% that was ≥3% higher than Week 29 pre-randomization hematocrit value, or
    • hematocrit >48%, or
    • an increase of ≥5% in hematocrit compared to Week 29 pre-randomization hematocrit value.


Secondary Outcome Measures :
  1. Change in rate of phlebotomy events between Week 17 through Week 29 (inclusive; 12 weeks) compared to each subject's historical rate. [ Time Frame: 12 weeks ]
  2. Change in rate of phlebotomy events between Week 1 through Week 29 (inclusive; 28 weeks) compared to each subject's historical rate. [ Time Frame: 28 weeks ]
  3. Proportion of subjects achieving a response at Week 29, with response defined as having achieved the absence of "phlebotomy eligibility" during the efficacy evaluation phase beginning at Week 17 and continuing to Week 29. [ Time Frame: 12 Weeks ]
    "Phlebotomy eligibility" in Part 1 is defined as a hematocrit ≥45% that was ≥3% higher than baseline level (defined as Part 1 pre-dose Day 1) or a hematocrit >48%.

  4. Proportion of subjects with reduction in the rate of phlebotomy events beginning at the Week 17 visit and continuing to Week 29 (12 weeks) compared to each subject's historical rate. [ Time Frame: 12 Weeks ]
    Time to "phlebotomy eligibility" from Week 29 to Week 41/End of Part 2.



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Ages Eligible for Study:   18 Years and older   (Adult, Older Adult)
Sexes Eligible for Study:   All
Accepts Healthy Volunteers:   No
Criteria

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 28 weeks (preferably up to 52 weeks) before dosing 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 have received cytoreductive therapy for at least 24 weeks and be on a stable dose or have a decreasing dose (Medical Monitor approval required) for at least 8 weeks before dosing 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.

Information from the National Library of Medicine

To learn more about this study, you or your doctor may contact the study research staff using the contact information provided by the sponsor.

Please refer to this study by its ClinicalTrials.gov identifier (NCT number): NCT04057040


Contacts
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Contact: Study Director 1-888-899-1543 ptgxclintrials@ptgx-inc.com

Locations
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Sponsors and Collaborators
Protagonist Therapeutics, Inc.
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Responsible Party: Protagonist Therapeutics, Inc.
ClinicalTrials.gov Identifier: NCT04057040    
Other Study ID Numbers: PTG-300-04
First Posted: August 14, 2019    Key Record Dates
Last Update Posted: January 26, 2022
Last Verified: January 2022

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Studies a U.S. FDA-regulated Drug Product: Yes
Studies a U.S. FDA-regulated Device Product: No
Keywords provided by Protagonist Therapeutics, Inc.:
polycythemia vera
Additional relevant MeSH terms:
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Polycythemia Vera
Polycythemia
Hematologic Diseases
Bone Marrow Neoplasms
Hematologic Neoplasms
Neoplasms by Site
Neoplasms
Bone Marrow Diseases
Myeloproliferative Disorders