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

Phase 3

• Conditions: Polycythemia Vera
• Interventions: Drug: Open-label rusfertide

• Registration Number: NCT06033586
• Lead Sponsor: Protagonist Therapeutics, Inc.

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.

Detailed Description

This is an open-label study designed to assess the long-term safety and efficacy of rusfertide. This study aims to provide long-term rusfertide treatment to subjects who complete dosing until the end-of-treatment of a previous Phase 2 rusfertide study and are likely to continue benefiting from treatment with rusfertide based on their improved control of hematocrit levels \<45% and reduced need for therapeutic phlebotomies. This study will provide long-term rusfertide treatment to these subjects and obtain data on safety and efficacy of long-term treatment with rusfertide.

Study Timeline

• Study First Submitted: 2023-09-05
• Study First Submitted QC: 2023-09-05
• Study First Posted: 2023-09-13
• Study Start: 2024-01-22
• Status Verified: 2024-07
• Last Update Submitted: 2024-07-15
• Last Update Posted: 2024-07-17
• Primary Completion: 2026-04-01
• Study Completion: 2027-04-01

Recruitment & Eligibility

• Status: ENROLLING_BY_INVITATION
• Sex: All
• Target Recruitment: 50

Inclusion Criteria

• 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.

Key

Read More

Exclusion Criteria

• 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.

Read More

Study & Design

• Study Type: INTERVENTIONAL
• Study Design: SINGLE_GROUP

Arm && Interventions

Group	Intervention	Description
Open-label rusfertide	Open-label rusfertide	Open-label rusfertide

Primary Outcome Measures

Name	Time	Method
Hematocrit	0-2 years	Median hematocrit
Phlebotomies	0-2 years	Number of phlebotomies

Trial Locations

Locations (1)

Pontchartrain Cancer Care; 🇺🇸 Covington, Louisiana, United States