Ropeginterferon Alfa-2b for Essential Thrombocythemia

(SURPASS ET Trial)

Recruiting at 76 trial locations

Overseen By Toshiaki Sato, MD/PhD

Age: 18+

Sex: Any

Trial Phase: Phase 3

Sponsor: PharmaEssentia

Must not be taking: QT-prolonging drugs

Disqualifiers: Pregnancy, Autoimmune disease, Depression, others

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

Pivotal Trial (Near Approval) This treatment is in the last trial phase before FDA approval

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

Compensation: Varies

Number of Visits: Unknown

Duration: 12 months

174 Participants Needed

Trial Summary

What is the purpose of this trial?

This is a Phase 3 open-label, multicenter, randomized, active-controlled study designed to compare the efficacy and safety and tolerability of P1101 compared with ANA after 12 months of treatment as second-line therapy for subjects with ET who have had a suboptimal or failed response to HU.

Will I have to stop taking my current medications?

The trial requires a washout period (time without taking certain medications) of at least 7 days for hydroxyurea (HU) and 14 days for interferon before starting the study. Other medications that can prolong QTc and induce low potassium levels are not allowed during the study.

What data supports the effectiveness of the drug Ropeginterferon Alfa-2b for treating essential thrombocythemia?

Ropeginterferon alfa-2b has been shown to be effective 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, although direct studies are needed. ^{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 mild to moderate side effects like hair loss, fatigue, and flu-like symptoms. Studies in both Japanese and European patients with polycythemia vera found no new safety concerns, and the treatment was well tolerated. ^{1 2 4 5 6}

How is the drug Ropeginterferon alfa-2b unique for treating essential thrombocythemia?

Ropeginterferon alfa-2b is unique because it is a long-acting, mono-PEGylated interferon that requires less frequent dosing, with injections every two weeks, and potentially every four weeks after prolonged use, which is more convenient compared to other interferons. It is also known for its ability to achieve deep molecular responses and is generally well tolerated. ^{1 2 3 4 5}

Research Team

Toshiaki Sato, MD/PhD

Principal Investigator

PharmaEssentia Japan K.K.

Craig Zimmerman, PhD

Principal Investigator

PharmaEssentia USA Corp.

Eligibility Criteria

Adults with high-risk Essential Thrombocythemia who have not responded well to Hydroxyurea treatment. They must be Interferon-naïve or negative for anti-P1101 antibodies, meet specific blood count criteria, and have proper liver and kidney function. Participants need to agree to birth control measures and cannot be pregnant or breastfeeding.

Inclusion Criteria

I have taken hydroxyurea for essential thrombocythemia and stopped at least 14 days before joining.

Written informed consent obtained from the subject and ability for the subject to comply with the requirements of the study

I have had a bad reaction or no improvement with hydroxyurea for my blood condition.

Exclusion Criteria

I don't have a history of long QT syndrome and am not on medications that can prolong QTc.

History or presence of clinically relevant depression, or previous suicide attempts or at any risk of suicide at screening, in the judgement of the Investigator

I am not allergic to IFN-α, ANA, or their ingredients.

Timeline

Screening

Participants are screened for eligibility to participate in the trial

4 weeks

Treatment

Participants receive either P1101 or Anagrelide for 12 months as second-line therapy

12 months

Regular visits for efficacy evaluations, safety assessments, and PK and immunogenicity evaluations

Follow-up

Participants are monitored for safety and effectiveness after treatment

4 weeks

Open-label extension (optional)

Participants may opt into continuation of P1101 treatment long-term

Treatment Details

Interventions

• Anagrelide
• Ropeginterferon alfa-2b

Trial Overview The trial is testing Ropeginterferon Alfa-2b against Anagrelide in patients resistant or intolerant to Hydroxyurea as a second-line therapy for Essential Thrombocythemia. It's an open-label study where participants are randomly assigned one of the two drugs for comparison over 12 months.

Participant Groups

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

Pre-filled Syringe, Q2W, SC injection

Group II: Anagrelide Active Control 2 Interventions

Capsules, Daily, p.o.

Who Is Running the Clinical Trial?

PharmaEssentia

Lead Sponsor

Trials

24

Recruited

2,800+

EPS International

Collaborator

Trials

1

Recruited

170+

Medpace, Inc.

Industry Sponsor

Trials

98

Recruited

30,400+

Dr. August J. Troendle

Medpace, Inc.

Chief Executive Officer since 1992

MD from the University of Maryland, School of Medicine; MBA from Boston University

Dr. Reinilde Heyrman

Medpace, Inc.

Chief Medical Officer since 2017

MD

Brightech International

Collaborator

Trials

1

Recruited

170+

References

1. 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]

2. 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]

3. Germany pubmed.ncbi.nlm.nih.gov

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

4. 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]

5. Spain pubmed.ncbi.nlm.nih.gov

Ropeginterferon alfa-2b for the treatment of patients with polycythemia vera. [2020]

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

Ropeginterferon alfa-2b, a novel IFNα-2b, induces high response rates with low toxicity in patients with polycythemia vera. [2021]