P1101 for Essential Thrombocythemia

Recruiting at 41 trial locations

Overseen By Jewell Jessup, PhD

Age: 18+

Sex: Any

Trial Phase: Phase 2

Sponsor: PharmaEssentia

Must not be taking: Interferon alfa

Disqualifiers: Severe cardiac disease, Autoimmune disease, Infections, Depression, others

No Placebo Group All trial participants will receive the active study treatment (no placebo)

Prior Safety Data This treatment has passed at least one previous human trial

Compensation: Varies

Number of Visits: Unknown

Duration: 52 weeks

91 Participants Needed

Trial Summary

What is the purpose of this trial?

This trial is testing a new medication called Ropeginterferon alfa-2b-njft (P1101) for adults with Essential Thrombocythemia, a blood disorder. The medication helps the body control blood cell production to prevent the disease from worsening. Interferons have been studied in similar conditions for over 30 years, but earlier versions had safety and tolerability issues.

Do I need to stop my current medications to join the trial?

The trial does not specify if you need to stop taking your current medications. However, if you have been on hydroxyurea (HU), you must stop it at least 7 days before starting the trial medication.

What data supports the effectiveness of the drug Ropeginterferon alfa-2b-njft (P1101) for treating Essential Thrombocythemia?

Ropeginterferon alfa-2b has shown effectiveness in treating polycythemia vera, a condition similar to essential thrombocythemia, by maintaining blood cell levels and reducing genetic markers associated with the disease. This suggests it may also be effective for essential thrombocythemia. ^{1 2 3 4 5}

Is Ropeginterferon alfa-2b safe for humans?

Ropeginterferon alfa-2b has been shown to be generally safe in humans, with common side effects including hair loss, fatigue, and flu-like symptoms, which are mostly mild or moderate. However, it has a warning for potentially serious effects like mental health issues, autoimmune disorders, and infections, so patients should be closely monitored. ^{1 3 6 7 8}

How is the drug Ropeginterferon alfa-2b-njft (P1101) unique for treating essential thrombocythemia?

Ropeginterferon alfa-2b-njft (P1101) is unique because it is a long-acting form of interferon that allows for less frequent dosing, which can improve patient convenience and adherence compared to other interferon treatments. It is also being explored for its potential to optimize dosing strategies to enhance patient outcomes. ^{2 3 9 10 11}

Research Team

Oleh Zagrijtschuk, MD, PhD

Principal Investigator

PharmaEssentia Corporation

Eligibility Criteria

Adults diagnosed with Essential Thrombocythemia (ET) as per WHO criteria, with a platelet count over 450 × 10^9/L and good liver and kidney function. Participants must be at least 18 years old, agree to use birth control, and not breastfeed during the study. Those previously treated with ANA or HU are eligible but not if they've had poor responses or intolerance to interferon alfa therapy.

Inclusion Criteria

My liver is functioning well according to recent tests.

I haven't had treatment to reduce my blood cell counts, or I've only had hydroxyurea and/or anagrelide.

I have or have not been treated with ANA before, regardless of why I stopped.

Exclusion Criteria

I am not allergic to IFN-α or its ingredients.

Pregnant or lactating females

I need someone legally authorized to make decisions for me.

Timeline

Screening

Participants are screened for eligibility to participate in the trial

2-4 weeks

Treatment

Participants receive Ropeginterferon alfa-2b-njft (P1101) for the treatment of Essential Thrombocythemia

52 weeks

Bi-weekly visits for injections and monitoring

Follow-up

Participants are monitored for safety and effectiveness after treatment

4 weeks

Extension

Participants may continue receiving treatment in an extension period

Treatment Details

Interventions

• Ropeginterferon alfa-2b-njft (P1101)

Trial Overview The trial is testing Ropeginterferon alfa-2b-njft (P1101) in adults with ET to evaluate its effectiveness, safety, and how well patients tolerate it. This single-arm study means all participants will receive P1101 without comparison to another treatment.

Participant Groups

Group I: Ropeginterferon alfa-2b (P1101) Experimental Treatment 1 Intervention

Pre-filled Syringe, Q2W, SC injection

Who Is Running the Clinical Trial?

PharmaEssentia

Lead Sponsor

Trials

24

Recruited

2,800+

Findings from Research

Ropeginterferon alfa-2b is an effective treatment for polycythemia vera (PV), showing long-term hematological response and reduction in JAK2V617F allele burden, making it a promising option for patients without symptomatic splenomegaly.

This treatment offers improved tolerability and safety compared to traditional therapies, with benefits such as extended dosing intervals and a potential increase in overall survival by lowering the risk of disease progression.

NCBI (Pubmed)

pubmed.ncbi.nlm.nih.gov

A pharmacokinetic evaluation of ropeginterferon alfa-2b in the treatment of polycythemia vera. Illés, Á., Pinczés, LI., Egyed, M.[2022]

References

1. Germany pubmed.ncbi.nlm.nih.gov

Ropeginterferon phase 2 randomized study in low-risk polycythemia vera: 5-year drug survival and efficacy outcomes. [2023]

2. Thailand pubmed.ncbi.nlm.nih.gov

Interferon apha 2b for treating patients with JAK2V617F positive polycythemia vera and essential thrombocytosis. [2019]

3. Switzerland pubmed.ncbi.nlm.nih.gov

An alternative dosing strategy for ropeginterferon alfa-2b may help improve outcomes in myeloproliferative neoplasms: An overview of previous and ongoing studies with perspectives on the future. [2023]

4. England pubmed.ncbi.nlm.nih.gov

A pharmacokinetic evaluation of ropeginterferon alfa-2b in the treatment of polycythemia vera. [2022]

5. England pubmed.ncbi.nlm.nih.gov

Optimal therapy for polycythemia vera and essential thrombocythemia: Preferred use of interferon therapy based on phase 2 trials. [2017]

6. Japan pubmed.ncbi.nlm.nih.gov

Efficacy and safety of ropeginterferon alfa-2b in Japanese patients with polycythemia vera: an open-label, single-arm, phase 2 study. [2022]

7. New Zealand pubmed.ncbi.nlm.nih.gov

Pharmacokinetics and Pharmacodynamics of Ropeginterferon Alfa-2b in Healthy Japanese and Caucasian Subjects After Single Subcutaneous Administration. [2022]

8. United States pubmed.ncbi.nlm.nih.gov

New Treatment for Polycythemia Vera. [2023]

9. United States pubmed.ncbi.nlm.nih.gov

Pegylated interferon alfa-2a yields high rates of hematologic and molecular response in patients with advanced essential thrombocythemia and polycythemia vera. [2023]

10. Russia (Federation) pubmed.ncbi.nlm.nih.gov

[Efficiency of interferon therapy in patients with essential thrombocythemia or polycythemia vera]. [2018]

11. Germany pubmed.ncbi.nlm.nih.gov

A long-term clinical trial of interferon alpha-therapy in essential thrombocythemia. [2019]