Clinical trial NCT06033586: Study to Evaluate the Long-term Safety of Rusfertide (PTG-300) in Subjects With Polycythemia Vera
Official title: An Extension Study to Evaluate the Long-term Safety of Rusfertide (PTG-300) in Subjects With Polycythemia Vera (THRIVE)
Study type: INTERVENTIONAL
Overall status: ENROLLING_BY_INVITATION
Phase: PHASE3
Conditions: Polycythemia Vera
Lead sponsor: Protagonist Therapeutics, Inc.
Sponsor class: INDUSTRY
First posted: 2023-09-13
Enrollment: 50
Sex: ALL
Minimum age (years): 18
Standard ages: ADULT
OLDER_ADULT
Accepts healthy volunteers: no
Allocation: NA
Intervention model: SINGLE_GROUP
Intervention model description: Open-label rusfertide
Primary purpose: TREATMENT

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.

Eligibility criteria: 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 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.

Primary outcomes:
measure: Hematocrit
timeFrame: 0-2 years
measure: Phlebotomies
timeFrame: 0-2 years
Secondary outcomes:
Other outcomes:
Locations:
city: Covington
country: United States
facility: Pontchartrain Cancer Care
state: Louisiana
Study officials:
References: