What side effects are associated with this type of intervention?

Common treatments for Polycythemia Vera include phlebotomy, hydroxyurea, and JAK2 inhibitors such as ruxolitinib and pacritinib. Phlebotomy is generally well-tolerated but can cause iron deficiency. Hydroxyurea can lead to cytopenias, mucocutaneous ulcers, diarrhea, peripheral neuropathy, and potential teratogenicity. Ruxolitinib is associated with increased risks of herpes zoster infection and nonmelanoma skin cancer. Pacritinib can cause hemorrhage, diarrhea, prolonged QT interval, and thrombosis. These treatments aim to manage symptoms and reduce complications but come with varying side effect profiles.

What prior FDA approvals exist?

The FDA has approved several treatments for Polycythemia Vera, including hydroxyurea, which is used to manage symptoms and reduce the risk of thrombotic events. Ruxolitinib, a JAK1/JAK2 inhibitor, is another FDA-approved drug for PV, particularly for patients who are resistant or intolerant to hydroxyurea. While PTG-300 (a hepcidin mimetic) is still under investigation, it represents a novel approach targeting iron regulation, which is different from the mechanisms of currently approved treatments.

What other types of research exist?

Promising novel alternatives to PTG-300 for Polycythemia Vera patients include JAK2 inhibitors such as ruxolitinib and fedratinib, which have shown efficacy in managing PV symptoms and reducing hematocrit levels. Additionally, investigational agents like ropeginterferon alfa-2b, which has demonstrated potential in clinical trials for PV, may offer new therapeutic options. These alternatives target different pathways and mechanisms, providing a broader range of treatment strategies for PV patients.

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Hepcidin Mimetic

Hepcidin Mimetic for Polycythemia Vera

Phase 2

Waitlist Available

Research Sponsored by Protagonist Therapeutics, Inc.

Eligibility Criteria Checklist

Specific guidelines that determine who can or cannot participate in a clinical trial

Must have

Subjects who are not receiving cytoreductive therapy must have been discontinued from any prior cytoreductive therapy for at least 24 weeks before screening and have recovered from any adverse events due to cytoreductive therapy

Must not have

Active or chronic bleeding within 4 weeks of screening

Any surgical procedure requiring general anesthesia within 1 month prior to screening or planned elective surgery during the study

Timeline

Screening 3 weeks

Treatment Varies

Follow Up 12 weeks

Summary

This trial tests PTG-300, a new medication, in patients with polycythemia vera who need regular blood removal. PTG-300 aims to keep red blood cell levels in check, reducing the need for frequent blood removal.

Who is the study for?

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.

What is being tested?

The trial is testing PTG-300, a hepcidin mimetic against a placebo in patients requiring frequent blood withdrawals due to polycythemia vera. It includes an initial phase where everyone gets PTG-300 to find the best dose followed by a blind test where neither doctors nor patients know who's getting what.

What are the potential side effects?

While specific side effects of PTG-300 aren't listed here, similar treatments may cause issues like injection site reactions, fatigue, joint pain or swelling. The study aims to monitor safety closely.

Eligibility Criteria

Inclusion Criteria

You may be eligible if you check “Yes” for the criteria below

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I stopped any previous cancer-shrinking treatments 24 weeks ago and have recovered from side effects.

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I stopped any previous cancer-shrinking treatments 24 weeks ago and have recovered from side effects.

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I stopped any previous cancer-shrinking treatments 24 weeks ago and have recovered from side effects.

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I'm sorry, I cannot provide a summary for "Main" as it does not provide any context or information about what is being referred to. Can you please provide more details?

Exclusion Criteria

You may be eligible for the trial if you check “No” for criteria below:

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I have not had any active or chronic bleeding in the last 4 weeks.

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I haven't had surgery under general anesthesia in the last month and don't plan any during the study.

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I have been diagnosed with myelofibrosis following polycythemia vera.

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I have a diagnosed immune system disorder.

Timeline

Screening ~ 3 weeks3 visits

Treatment ~ Varies

Follow Up ~ 12 weeks

Screening ~ 3 weeks

Treatment ~ Varies

Follow Up ~12 weeks

This trial's timeline: 3 weeks for screening, Varies for treatment, and 12 weeks for reporting.

Treatment Details

Study Objectives

Study objectives can provide a clearer picture of what you can expect from a treatment.

Primary study objectives

Proportion of responders during the blinded randomized withdrawal period (Week 29 to Week 41).

Secondary study objectives

Proportion of subjects achieving a response at Week 29, with response defined as having achieved the absence of "phlebotomy eligibility" during the efficacy evaluation phase beginning at Week 17 and continuing to Week 29.

Proportion of subjects with reduction in the rate of phlebotomy events beginning at the Week 17 visit and continuing to Week 29 (12 weeks) compared to each subject's historical rate.

Side effects data

From 2022 Phase 2 trial • 16 Patients • NCT04202965

31%

Injection site pain

19%

Injection site pruritus

13%

Diarrhea

13%

Fatigue

13%

Injection site erythema

13%

Dizziness

13%

Headache

13%

Hypertension

Adenocarcinoma pancreas

Dry mouth

Injection site bruising

Injection site haematoma

Injection site haemorrhage

Nausea

Vomiting

Supraventricular extrasystoles

Inflammation

Injection site discomfort

Injection site induration

Injection site swelling

Malaise

Cellulitis

Gastrointestinal bacterial overgrowth

Hand fracture

Back pain

Melanocytic naevus

Rash

Paraesthesia

Insomnia

Bronchospasm

100%

80%

60%

40%

20%

Study treatment Arm

PTG-300

Trial Design

2Treatment groups

Experimental Treatment

Group I: Dose finding PTG-300 (Part 1); Placebo (Part 2); Open label extension PTG-300 (Part 3)Experimental Treatment2 Interventions

Group II: Dose finding PTG-300 (Part 1); PTG-300 (Part 2); Open label extension PTG-300 (Part 3)Experimental Treatment1 Intervention

Treatment

First Studied

Drug Approval Stage

How many patients have taken this drug

PTG-300

2019

Completed Phase 2

~150

Placebo

1995

Completed Phase 3

~2670

0 / 8Eligibility criteria met

Research Highlights

Information in this section is not a recommendation. We encourage patients to speak with their healthcare team when evaluating any treatment decision.

Mechanism Of Action

Side Effect Profile

Prior Approvals

Other Research

The most common treatments for Polycythemia Vera (PV) include hepcidin mimetics like PTG-300, which regulate iron metabolism to reduce red blood cell production, hydroxyurea, which inhibits DNA synthesis to lower blood cell counts, and JAK2 inhibitors, which target the JAK-STAT pathway to control abnormal cell proliferation. These treatments are crucial for PV patients as they help manage symptoms, reduce the risk of thrombotic events, and prevent disease progression.