Hepcidin Mimetic in Patients With Polycythemia Vera (REVIVE)

Polycythemia Vera Clinical Trial

Official title:

A Phase 2 Study of the Hepcidin Mimetic PTG-300 in Patients With Phlebotomy-Requiring Polycythemia Vera

Clinical Trial Details — Status: Active, not recruiting

Administrative data

• NCT number: NCT04057040
• Other study ID #: PTG-300-04
• Status: Active, not recruiting
• Phase: Phase 2
• Start date: October 1, 2019
• Est. completion date: January 1, 2026

Study information

• Verified date: November 2023
• Source: Protagonist Therapeutics, Inc.
• Contact: n/a
• Is FDA regulated: No
• Study type: Interventional

Clinical Trial 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.

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.

Recruitment information / eligibility

• Status: Active, not recruiting
• Enrollment: 80
• Est. completion date: January 1, 2026
• Est. primary completion date: October 15, 2025
• Accepts healthy volunteers: No
• Gender: All
• Age group: 18 Years and older

Eligibility

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.

Related Conditions & MeSH terms

• Polycythemia
• Polycythemia Vera

Intervention

Drug:
• PTG-300
Active
• Placebo
Placebo

Locations

Country	Name	City	State
India	Sahyadri Super Specialty Hospital	Pune	Maharashtra
India	All India Institute of Medical Sciences	Rishikesh	Uttarakhand
United States	University of Michigan	Ann Arbor	Michigan
United States	Center for Cancer and Blood Disorders	Bethesda	Maryland
United States	Cleveland Clinic - Taussig Cancer Center	Cleveland	Ohio
United States	Pontchartrain Cancer Care	Covington	Louisiana
United States	Mary Crowley Cancer Research Center	Dallas	Texas
United States	Karmanos Cancer Center	Detroit	Michigan
United States	Marin Cancer Care	Greenbrae	California
United States	The University of Texas MD Anderson Cancer Center	Houston	Texas
United States	Mount Sinai	New York	New York
United States	New York Presbyterian Hospital - Weill Cornell Medical Center	New York	New York
United States	Stanford University	Palo Alto	California
United States	Mayo Clinic - Mayo Clinic Hospital	Phoenix	Arizona
United States	Moffitt Cancer Center	Tampa	Florida
United States	University of Kansas	Westwood	Kansas

Sponsors (1)

Lead Sponsor	Collaborator
Protagonist Therapeutics, Inc.

Countries where clinical trial is conducted

United States,  India, 

Outcome

Type	Measure	Description	Time frame	Safety issue
Primary	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.	12 weeks
Secondary	Change in rate of phlebotomy events between Week 17 through Week 29 (inclusive; 12 weeks) compared to each subject's historical rate.		12 weeks
Secondary	Change in rate of phlebotomy events between Week 1 through Week 29 (inclusive; 28 weeks) compared to each subject's historical rate.		28 weeks
Secondary	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%.	12 Weeks
Secondary	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.	12 Weeks