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Hepcidin Mimetic in Patients With Polycythemia Vera

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. Know the risks and potential benefits of clinical studies and talk to your health care provider before participating. Read our disclaimer for details. Identifier: NCT04057040
Recruitment Status : Recruiting
First Posted : August 14, 2019
Last Update Posted : November 5, 2020
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 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 a 12 week randomized withdrawal phase to confirm the response. Subsequently patients will enter into a 1 year open label extension to investigate long term safety.

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Study Type : Interventional  (Clinical Trial)
Estimated Enrollment : 50 participants
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, subject 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
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 : August 1, 2022
Estimated Study Completion Date : November 1, 2022

Arm Intervention/treatment
Experimental: Experimental Dose Escalation (Part 1)
PTG-300 Subcutaneous (SC) weekly. Each subject's PTG-300 dose is increased at 4 week intervals until the subject reaches the maximum planned dose or has a prespecified decrease in hematocrit from baseline.
Drug: PTG-300

Experimental: Blinded Withdrawal (Part 2) PTG-300 or placebo
PTG-300 or placebo Subcutaneous (SC) weekly.
Drug: PTG-300

Drug: Placebo

Experimental: Experimental Open label extension (Part 3) PTG-300
PTG-300 Subcutaneous (SC) weekly
Drug: PTG-300

Primary Outcome Measures :
  1. 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%

  2. 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).

  3. Number of Participants with Treatment-Emergent Adverse Events as Assessed by CTCAE v5.0 [ Time Frame: 101 weeks ]

Secondary Outcome Measures :
  1. Change from baseline in serum iron [ Time Frame: 42 weeks ]
  2. Change from baseline in transferrin saturation (TSAT) [ Time Frame: 42 weeks ]
  3. Change from baseline in serum transferrin [ Time Frame: 42 weeks ]

Information from the National Library of Medicine

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

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.

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 identifier (NCT number): NCT04057040

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Contact: Study Director 1-888-899-1543

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United States, Arizona
Mayo Clinic - Mayo Clinic Hospital Recruiting
Phoenix, Arizona, United States, 85054
United States, California
Stanford University Recruiting
Palo Alto, California, United States, 94304
United States, Florida
Cancer Specialists of North Florida Recruiting
Jacksonville, Florida, United States, 32256
Moffitt Cancer Center Recruiting
Tampa, Florida, United States, 33612
United States, Kansas
University of Kansas Recruiting
Westwood, Kansas, United States, 66205
United States, Maryland
Center for Cancer and Blood Disorders Recruiting
Bethesda, Maryland, United States, 20817
United States, Michigan
University of Michigan Recruiting
Ann Arbor, Michigan, United States, 48109
Karmanos Cancer Center Recruiting
Detroit, Michigan, United States, 48201
United States, New York
Mount Sinai Recruiting
New York, New York, United States, 10029
New York Presbyterian Hospital - Weill Cornell Medical Center Recruiting
New York, New York, United States, 10065
United States, Texas
The University of Texas MD Anderson Cancer Center Recruiting
Houston, Texas, United States, 77030
Mazumdar Shaw Medical Center, Unit of Narayana Health Recruiting
Bangalore, Kamataka, India, 560099
Sahyadri Super Speciality Hospital Recruiting
Pune, Maharashtra, India, 411004
All India Institute of Medical Sciences Recruiting
Rishikesh, Uttarakhand, India, 249203
Sponsors and Collaborators
Protagonist Therapeutics, Inc.
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Responsible Party: Protagonist Therapeutics, Inc. Identifier: NCT04057040    
Other Study ID Numbers: PTG-300-04
First Posted: August 14, 2019    Key Record Dates
Last Update Posted: November 5, 2020
Last Verified: November 2020

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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
Hematologic Diseases
Bone Marrow Neoplasms
Hematologic Neoplasms
Neoplasms by Site
Bone Marrow Diseases
Myeloproliferative Disorders