Ropeginterferon Alfa-2b for Essential Thrombocythemia

(SURPASS ET Trial)

Not currently recruiting at 102 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

What You Need to Know Before You Apply

What is the purpose of this trial?

This trial tests Ropeginterferon alfa-2b for individuals with essential thrombocythemia (ET), a condition that causes an excess of platelets in the blood, potentially leading to clotting issues. The trial aims to evaluate the effectiveness and safety of this treatment compared to Anagrelide. It targets those who have tried hydroxyurea but experienced poor responses or intolerance. This trial may suit individuals with ET who have faced issues like blood clots or bleeding and have not found success with previous treatments. As a Phase 3 trial, this treatment represents the final step before FDA approval, offering hope for those seeking new, effective options.

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.

Is there any evidence suggesting that this trial's treatments are likely to be safe?

Research has shown that ropeginterferon alfa-2b is generally well-tolerated by people with essential thrombocythemia. Studies have found no serious side effects directly related to the medication. Most participants managed the side effects, indicating that the treatment is relatively safe for humans.

In comparison, another study found that ropeginterferon alfa-2b had better safety outcomes than anagrelide, another treatment option for this condition. These findings, based on research, can help provide some peace of mind about the treatment's safety. ^{1 2 3 4 5}

Why do researchers think this study treatment might be promising for essential thrombocythemia?

Ropeginterferon alfa-2b is unique because it offers a new approach for managing essential thrombocythemia by using a long-acting interferon. Unlike standard treatments like hydroxyurea and anagrelide, which primarily target platelet production, ropeginterferon alfa-2b works by modulating the immune system to reduce platelet levels. Additionally, it is administered via a subcutaneous injection every two weeks, potentially improving patient compliance compared to daily oral medications. Researchers are excited about its potential for fewer side effects and long-term benefits in controlling the disease.

What evidence suggests that this trial's treatments could be effective for essential thrombocythemia?

Studies have shown that ropeginterferon alfa-2b, a treatment tested in this trial, effectively treats essential thrombocythemia (ET). One study found that 42.9% of patients experienced strong, lasting improvements at both 9 and 12 months. Research also indicates that ropeginterferon alfa-2b outperforms anagrelide, the other treatment option in this trial, particularly for those who did not respond well to previous treatments. This treatment has proven both effective and safe, making it a promising option for people with ET. ^{1 2 3 6 7}

Who Is on the Research Team?

Toshiaki Sato, MD/PhD

Principal Investigator

PharmaEssentia Japan K.K.

Craig Zimmerman, PhD

Principal Investigator

PharmaEssentia USA Corp.

Are You a Good Fit for This Trial?

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 for a Trial Participant

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

What Are the Treatments Tested in This Trial?

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.

How Is the Trial Designed?

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+

Citations

1. targetedonc.com targetedonc.com/view/ropeginterferon-alfa-2b-succeeds-in-phase-3-essential-thrombocythemia-trial

Ropeginterferon Alfa-2b Succeeds in Phase 3 Essential ... In the intent-to-treat (ITT) population, 42.9% (39/91) of patients treated with ropeginterferon alfa-2b had durable responses at 9 and 12 months ...

2. ascopubs.org ascopubs.org/doi/10.1200/JCO.2025.43.16_suppl.6500

Topline results of the phase 3 SURPASS-ET trial. Conclusions: Ropeg showed superior efficacy and safety compared to anagrelide as second-line therapy for ET. It represents a potential new ...

3. onclive.com onclive.com/view/ropeginterferon-alfa-2b-generates-superior-efficacy-vs-anagrelide-in-high-risk-essential-thrombocythemia

Ropeginterferon Alfa-2b Generates Superior Efficacy vs ... Ropeginterferon alfa-2b showed superior efficacy over anagrelide in achieving durable responses in high-risk essential thrombocythemia patients.

4. pmc.ncbi.nlm.nih.gov pmc.ncbi.nlm.nih.gov/articles/PMC12054645/

A multicenter study to assess efficacy, safety, and tolerability of ... A multicenter study to assess efficacy, safety, and tolerability of ropeginterferon alfa-2b-njft in patients with essential thrombocythemia in the US and ...

5. sciencedirect.com sciencedirect.com/science/article/abs/pii/S2152265025002162

Real-World Evidence on Outcomes and Safety of ... Ropeginterferon alfa-2b-njft (ropegIFN) has demonstrated superior efficacy over hydroxyurea in polycythemia vera (PV); however, real-world ...

6. dailynews.ascopubs.org dailynews.ascopubs.org/do/ropeginterferon-alfa-2b-demonstrates-superior-efficacy-and-safety-anagrelide-second

Ropeginterferon Alfa-2b Demonstrates Superior Efficacy ... In the phase 3 SURPASS-ET trial, ropeg showed superior efficacy and safety compared with anagrelide as second-line therapy for essential thrombocythemia.

7. ashpublications.org ashpublications.org/ashclinicalnews/news/8644/Ropeg-May-Provide-New-Treatment-Option-for

Ropeg May Provide New Treatment Option for Essential ... Dr. Gill noted that 27.8% of patients in the ropeg arm achieved a partial molecular response, and 2.8% achieved a complete response, while none ...