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Hepcidin Mimetic in Patients With Polycythemia Vera (REVIVE)

Primary Purpose

Polycythemia Vera

Status
Active
Phase
Phase 2
Locations
International
Study Type
Interventional
Intervention
PTG-300
Placebo
Sponsored by
Protagonist Therapeutics, Inc.
About
Eligibility
Locations
Arms
Outcomes
Full info

About this trial

This is an interventional treatment trial for Polycythemia Vera focused on measuring polycythemia vera

Eligibility Criteria

18 Years - undefined (Adult, Older Adult)All SexesDoes not accept healthy volunteers

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.

Sites / Locations

  • Mayo Clinic - Mayo Clinic Hospital
  • Marin Cancer Care
  • Stanford University
  • Moffitt Cancer Center
  • University of Kansas
  • Pontchartrain Cancer Care
  • Center for Cancer and Blood Disorders
  • University of Michigan
  • Karmanos Cancer Center
  • Mount Sinai
  • New York Presbyterian Hospital - Weill Cornell Medical Center
  • Cleveland Clinic - Taussig Cancer Center
  • Mary Crowley Cancer Research Center
  • The University of Texas MD Anderson Cancer Center
  • Sahyadri Super Specialty Hospital
  • All India Institute of Medical Sciences

Arms of the Study

Arm 1

Arm 2

Arm Type

Experimental

Experimental

Arm Label

Dose finding PTG-300 (Part 1); PTG-300 (Part 2); Open label extension PTG-300 (Part 3)

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

Arm Description

Outcomes

Primary Outcome Measures

Proportion of responders during the blinded randomized withdrawal period (Week 29 to Week 41).
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

Change in rate of phlebotomy events between Week 17 through Week 29 (inclusive; 12 weeks) compared to each subject's historical rate.
Change in rate of phlebotomy events between Week 1 through Week 29 (inclusive; 28 weeks) compared to each subject's historical rate.
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.
"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%.
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 to "phlebotomy eligibility" from Week 29 to Week 41/End of Part 2.

Full Information

First Posted
August 7, 2019
Last Updated
April 12, 2022
Sponsor
Protagonist Therapeutics, Inc.
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1. Study Identification

Unique Protocol Identification Number
NCT04057040
Brief Title
Hepcidin Mimetic in Patients With Polycythemia Vera (REVIVE)
Official Title
A Phase 2 Study of the Hepcidin Mimetic PTG-300 in Patients With Phlebotomy-Requiring Polycythemia Vera
Study Type
Interventional

2. Study Status

Record Verification Date
April 2022
Overall Recruitment Status
Active, not recruiting
Study Start Date
October 1, 2019 (Actual)
Primary Completion Date
October 15, 2025 (Anticipated)
Study Completion Date
January 1, 2026 (Anticipated)

3. Sponsor/Collaborators

Responsible Party, by Official Title
Sponsor
Name of the Sponsor
Protagonist Therapeutics, Inc.

4. Oversight

Studies a U.S. FDA-regulated Drug Product
Yes
Studies a U.S. FDA-regulated Device Product
No
Data Monitoring Committee
Yes

5. Study Description

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 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.

6. Conditions and Keywords

Primary Disease or Condition Being Studied in the Trial, or the Focus of the Study
Polycythemia Vera
Keywords
polycythemia vera

7. Study Design

Primary Purpose
Treatment
Study Phase
Phase 2
Interventional Study Model
Parallel Assignment
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
ParticipantInvestigatorOutcomes Assessor
Masking Description
Part 1 open label, Part 2 blinded, Part 3 open label
Allocation
Randomized
Enrollment
80 (Anticipated)

8. Arms, Groups, and Interventions

Arm Title
Dose finding PTG-300 (Part 1); PTG-300 (Part 2); Open label extension PTG-300 (Part 3)
Arm Type
Experimental
Arm Title
Dose finding PTG-300 (Part 1); Placebo (Part 2); Open label extension PTG-300 (Part 3)
Arm Type
Experimental
Intervention Type
Drug
Intervention Name(s)
PTG-300
Intervention Description
Active
Intervention Type
Drug
Intervention Name(s)
Placebo
Intervention Description
Placebo
Primary Outcome Measure Information:
Title
Proportion of responders during the blinded randomized withdrawal period (Week 29 to Week 41).
Description
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.
Time Frame
12 weeks
Secondary Outcome Measure Information:
Title
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
Title
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
Title
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.
Description
"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%.
Time Frame
12 Weeks
Title
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.
Description
Time to "phlebotomy eligibility" from Week 29 to Week 41/End of Part 2.
Time Frame
12 Weeks

10. Eligibility

Sex
All
Minimum Age & Unit of Time
18 Years
Accepts Healthy Volunteers
No
Eligibility Criteria
Main Inclusion Criteria: All subjects must meet ALL of the following inclusion criteria to be enrolled. Male and female subjects aged 18 years or older. Meet revised 2016 World Health Organization (WHO) criteria for the diagnosis of polycythemia vera. Records of all phlebotomies performed for at least 28 weeks (preferably up to 52 weeks) before dosing are available. 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. 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: Active or chronic bleeding within 4 weeks of screening. Meets the criteria for post-PCV myelofibrosis as defined by the International Working Group-Myeloproliferative Neoplasms Research and Treatment (IWG-MRT). Known primary or secondary immunodeficiency. Any surgical procedure requiring general anesthesia within 1 month prior to screening or planned elective surgery during the study.
Facility Information:
Facility Name
Mayo Clinic - Mayo Clinic Hospital
City
Phoenix
State/Province
Arizona
ZIP/Postal Code
85054
Country
United States
Facility Name
Marin Cancer Care
City
Greenbrae
State/Province
California
ZIP/Postal Code
94904
Country
United States
Facility Name
Stanford University
City
Palo Alto
State/Province
California
ZIP/Postal Code
94304
Country
United States
Facility Name
Moffitt Cancer Center
City
Tampa
State/Province
Florida
ZIP/Postal Code
33612
Country
United States
Facility Name
University of Kansas
City
Westwood
State/Province
Kansas
ZIP/Postal Code
66205
Country
United States
Facility Name
Pontchartrain Cancer Care
City
Covington
State/Province
Louisiana
ZIP/Postal Code
70433
Country
United States
Facility Name
Center for Cancer and Blood Disorders
City
Bethesda
State/Province
Maryland
ZIP/Postal Code
20817
Country
United States
Facility Name
University of Michigan
City
Ann Arbor
State/Province
Michigan
ZIP/Postal Code
48109
Country
United States
Facility Name
Karmanos Cancer Center
City
Detroit
State/Province
Michigan
ZIP/Postal Code
48201
Country
United States
Facility Name
Mount Sinai
City
New York
State/Province
New York
ZIP/Postal Code
10029
Country
United States
Facility Name
New York Presbyterian Hospital - Weill Cornell Medical Center
City
New York
State/Province
New York
ZIP/Postal Code
10065
Country
United States
Facility Name
Cleveland Clinic - Taussig Cancer Center
City
Cleveland
State/Province
Ohio
ZIP/Postal Code
44106
Country
United States
Facility Name
Mary Crowley Cancer Research Center
City
Dallas
State/Province
Texas
ZIP/Postal Code
75230
Country
United States
Facility Name
The University of Texas MD Anderson Cancer Center
City
Houston
State/Province
Texas
ZIP/Postal Code
77030
Country
United States
Facility Name
Sahyadri Super Specialty Hospital
City
Pune
State/Province
Maharashtra
ZIP/Postal Code
411004
Country
India
Facility Name
All India Institute of Medical Sciences
City
Rishikesh
State/Province
Uttarakhand
ZIP/Postal Code
249203
Country
India

12. IPD Sharing Statement

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Hepcidin Mimetic in Patients With Polycythemia Vera (REVIVE)

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