Hepcidin Mimetic for Polycythemia Vera
What You Need to Know Before You Apply
What is the purpose of this trial?
This trial tests a treatment called PTG-300 (a hepcidin mimetic) for individuals with polycythemia vera, a condition where the body produces too many red blood cells, leading to frequent phlebotomies (blood removal procedures). The study aims to determine if PTG-300 is safe and effective in reducing the need for these phlebotomies. Participants will initially receive different doses of PTG-300, then either continue with PTG-300 or switch to a placebo, and finally return to PTG-300. This trial may suit adults diagnosed with polycythemia vera who require regular phlebotomies and are either not on cytoreductive therapy or are on a stable dose. As a Phase 2 trial, this research focuses on measuring the treatment's effectiveness in an initial, smaller group of people.
Will I have to stop taking my current medications?
If you are on cytoreductive therapy with hydroxyurea, interferon, or ruxolitinib, you can continue as long as your dose is stable or decreasing for at least 8 weeks before starting the trial. If you are not on these therapies, you must have stopped any prior cytoreductive therapy at least 24 weeks before joining the trial.
Is there any evidence suggesting that PTG-300 is likely to be safe for humans?
Research has shown that PTG-300, also known as rusfertide, has been tested in other studies to assess its safety in humans. In one study, patients with polycythemia vera, a blood disorder, experienced mostly mild side effects from rusfertide that did not cause major issues.
Another study found that rusfertide reduced the need for phlebotomy, a procedure to remove blood, and helped manage red blood cell levels without causing serious side effects.
While these results are encouraging, it is important to note that this trial remains in the middle stages of testing. More information is needed about its safety in larger groups. However, the results so far suggest that rusfertide is generally well-tolerated in people with polycythemia vera.12345Why do researchers think this study treatment might be promising for polycythemia vera?
Unlike the standard treatments for polycythemia vera, such as phlebotomy, hydroxyurea, and interferon, PTG-300 offers a novel approach by mimicking the hormone hepcidin. Hepcidin plays a key role in regulating iron levels, which can help control red blood cell production without the need for frequent blood removal or cytotoxic agents. Researchers are excited because PTG-300 could offer a more targeted and potentially safer way to manage the condition, minimizing side effects and improving patients' quality of life. Additionally, the open-label extension in the trial suggests a commitment to understanding long-term benefits and safety of PTG-300.
What evidence suggests that PTG-300 might be an effective treatment for polycythemia vera?
Research has shown that PTG-300, also known as rusfertide, may help treat polycythemia vera, a condition where the body produces too many red blood cells. One study found that rusfertide helped maintain red blood cell levels below 45%, reducing the need for phlebotomies, which manage the condition. In this trial, participants will receive either PTG-300 or a placebo at different stages. Another study demonstrated that rusfertide was more effective than a placebo in controlling red blood cell levels. These findings suggest that PTG-300 could effectively manage polycythemia vera and reduce the need for frequent treatments.12678
Are You a Good Fit for This Trial?
Adults diagnosed with polycythemia vera, who've had regular phlebotomies for at least 28 weeks and are either on a stable or decreasing dose of cytoreductive therapy (like hydroxyurea, interferon, or ruxolitinib) for over 24 weeks. Those not on such therapies must have stopped them for at least 24 weeks prior.Inclusion Criteria
Exclusion Criteria
Timeline for a Trial Participant
Screening
Participants are screened for eligibility to participate in the trial
Dose Finding
28 week dose finding phase to identify a dose that maintains hematocrit <45%
Randomized Withdrawal
12 week randomized withdrawal phase to confirm the response
Open-label Extension
Participants enter an up to 3 year open label extension to investigate long term safety
Follow-up
Participants are monitored for safety and effectiveness after treatment
What Are the Treatments Tested in This Trial?
Interventions
- PTG-300
Find a Clinic Near You
Who Is Running the Clinical Trial?
Protagonist Therapeutics, Inc.
Lead Sponsor