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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 : September 2, 2019
Sponsor:
Information provided by (Responsible Party):
Protagonist Therapeutics

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 12 week randomized withdrawal phase to confirm the response. Subsequently patients will enter into 1 year open label extension to investigate long term safety.

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Study Type : Interventional  (Clinical Trial)
Estimated Enrollment : 30 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, 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
Official Title: A Phase 2 Study of the Hepcidin Mimetic PTG-300 in Patients With Phlebotomy-Requiring Polycythemia Vera
Estimated Study Start Date : August 2019
Estimated Primary Completion Date : February 2021
Estimated Study Completion Date : March 2021


Arm Intervention/treatment
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.
Drug: PTG-300
Active
Other Name: PTG-300 10 mg to 80 mg

Experimental: Blinded Withdrawal (Part 2) PTG-300
PTG-300 Subcutaneous (SC) Weekly
Drug: PTG-300
Active
Other Name: PTG-300 10 mg to 80 mg

Placebo Comparator: Blinded Withdrawal (Part 2) Placebo
Placebo (SC) weekly
Drug: Placebo
Placebo




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 ]


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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. Receiving aspirin therapy for polycythemia vera (subjects for whom aspirin is not contraindicated).
  4. Records of all phlebotomies performed for at least 24 weeks before screening are available.
  5. 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.
  6. Subjects receiving cytoreductive therapy with hydroxyurea, interferon, or ruxolitinib must be on a stable dose for at least 24 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 ClinicalTrials.gov identifier (NCT number): NCT04057040


Contacts
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Contact: Suneel Gupta 510-474-0978 s.gupta@ptgx-inc.com
Contact: Frank Valone f.valone@ptgx.inc.com

Locations
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United States, Florida
Moffitt Recruiting
Tampa, Florida, United States, 33612
United States, Maryland
Center for Cancer and Blood Disorders Recruiting
Bethesda, Maryland, United States, 20817
United States, Michigan
Karmanos Cancer Center Recruiting
Detroit, Michigan, United States, 48201
United States, New York
Mount Sinai Recruiting
New York, New York, United States, 10029
United States, Texas
The University of Texas MD Anderson Cancer Center Recruiting
Houston, Texas, United States, 77030
The University of Texas at San Antonio Recruiting
San Antonio, Texas, United States, 78229
Sponsors and Collaborators
Protagonist Therapeutics

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Responsible Party: Protagonist Therapeutics
ClinicalTrials.gov Identifier: NCT04057040     History of Changes
Other Study ID Numbers: PTG-300-04
First Posted: August 14, 2019    Key Record Dates
Last Update Posted: September 2, 2019
Last Verified: August 2019

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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:
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
Hepcidins
Anti-Infective Agents