Study to Evaluate the Long-term Safety of Rusfertide (PTG-300) in Subjects With Polycythemia Vera

NCT06033586 | Active Not Recruiting Phase 3 DMC FDA Drug

• 1 other identifier: PTG-300-21
• Study Type: interventional
• Target: 46
• Locations: 1 country
• Sites: 1

Brief Summary

The goal of this clinical trial is to assess long-term safety and efficacy of rusfertide in subjects with polycythemia vera. Subjects who complete dosing with rusfertide until the end-of-treatment visit of a Phase 2 rusfertide study and meet the inclusion/exclusion criteria for this study, are eligible to participate in this open-label study and continue treatment with rusfertide.

Conditions

Polycythemia Vera

Outcome Measures

Primary Outcomes (2)

• Hematocrit

  Median hematocrit

  0-2 years

• Phlebotomies

  Number of phlebotomies

  0-2 years

Study Arms (1)

Open-label rusfertide

EXPERIMENTAL

Open-label rusfertide

Drug: Open-label rusfertide

Interventions

Open-label rusfertide DRUG

subcutaneous rusfertide

Open-label rusfertide

Eligibility Criteria

• Age: 18 Years+
• Sex: all
• Healthy Volunteers: No
• Age Groups: Adult (18-64), Older Adult (65+)

You may qualify if:

• Subject who has completed at least 12 months of dosing with rusfertide and successfully completed the end of treatment visit of a previous Phase 2 study of rusfertide.
• Subject understands the study procedures, is willing and able to adhere to study requirements and agrees to participate in the study by giving written informed consent.

You may not qualify if:

• Subject who, in the opinion of the investigator, should not participate in the study.
• Subject who discontinue early from a previous rusfertide study for reasons other than enrolling in this study.
• Pregnant or lactating females.
• Women of childbearing potential (WOCBP) who do not agree to use medically acceptable contraception (\<1% annual failure rate) during the study and for 30 days after the last dose of study drug.
• Men with partners of childbearing potential who do not agree to use medically acceptable contraception (\<1% annual failure rate) during the study and for 90 days after the last dose of study drug.
• Men who do not agree to use a condom during the study and for 90 days after the last dose of study drug regardless of the partner's childbearing potential.
• A female subject intends to donate eggs (ova, oocytes) for the purposes of assisted reproduction during the study and for a period of 30 days after receiving the last dose of study drug.
• A male subject intends to donate sperm for the purpose of reproduction during the study and for a minimum of 90 days after receiving the last dose of study drug.
• Plan to use investigational treatment other than rusfertide during the course of the study or within 28 days after last rusfertide dose.
• Subject with hypersensitivity to rusfertide or to any of the excipients.
• In the investigator's opinion the subject has progressive disease that cannot be managed by adjusting concurrent cytoreductive therapy.

Sponsors & Collaborators

• Protagonist Therapeutics, Inc.: lead

Study Sites (1)

Pontchartrain Cancer Care

Covington, Louisiana, 70433, United States

Location

MeSH Terms

Conditions

Polycythemia Vera

Condition Hierarchy (Ancestors)

Bone Marrow Neoplasms; Hematologic Neoplasms; Neoplasms by Site; Neoplasms; Bone Marrow Diseases; Hematologic Diseases; Hemic and Lymphatic Diseases; Myeloproliferative Disorders

Study Design

• Study Type: interventional
• Phase: phase 3
• Allocation: NA
• Masking: NONE
• Purpose: TREATMENT
• Intervention Model: SINGLE GROUP
• Sponsor Type: INDUSTRY
• Responsible Party: SPONSOR

Model Details: Open-label rusfertide

Study Record Dates

• First Submitted: September 5, 2023
• First Posted: September 13, 2023
• Study Start: January 22, 2024
• Primary Completion: April 1, 2026
• Study Completion (Estimated): April 1, 2027
• Last Updated: August 6, 2025

Record last verified: 2025-08

Data Sharing

• IPD Sharing: Will not share

Locations

US (1)