Polycythemia Vera
Conditions
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.
Interventions
DRUGOpen-label rusfertide
subcutaneous rusfertide
Sponsors
Protagonist Therapeutics, Inc.
Study design
Allocation
NA
Intervention model
SINGLE_GROUP
Primary purpose
TREATMENT
Masking
NONE
Intervention model description
Open-label rusfertide
Eligibility
Sex/Gender
ALL
Age
18 Years to No maximum
Healthy volunteers
No
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.
Design outcomes
Primary
| Measure | Time frame | Description |
|---|---|---|
| Hematocrit | 0-2 years | Median hematocrit |
| Phlebotomies | 0-2 years | Number of phlebotomies |
Countries
United States
Outcome results
None listed