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

Study Overview
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Brief Summary
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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 or Phase 3 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
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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 or Phase 3 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.

Official Title
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An Extension Study to Evaluate the Long-term Safety of Rusfertide (PTG-300) in Subjects With Polycythemia Vera (THRIVE)

Conditions
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Polycythemia Vera

Intervention / Treatment
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* Drug: Open-label rusfertide


Participation Criteria
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Eligibility Criteria
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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 or Phase 3 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 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.

Ages Eligible for Study
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Minimum Age: 18 Years

Sexes Eligible for Study
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All

Accepts Healthy Volunteers
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No

Study Plan
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How is the study designed?
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Design Details

Primary Purpose: Treatment
Intervention Model: Single Group Assignment
Interventional Model Description: Open-label rusfertide
Masking: None (Open Label)


Arms and Interventions

| Participant Group/Arm | Intervention/Treatment |
| --- | --- |
| Experimental: Open-label rusfertide<br>Open-label rusfertide | Drug: Open-label rusfertide<br>* subcutaneous rusfertide<br>|

What is the study measuring?
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Primary Outcome Measures

| Outcome Measure | Measure Description | Time Frame |
| --- | --- | --- |
| Hematocrit | Median hematocrit | 0-2 years | 
| Phlebotomies | Number of phlebotomies | 0-2 years |