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Essential ThrombocythemiaPhase 2NCT05482971

A Single-arm, Multicenter Study to Assess the Efficacy, Safety, and Tolerability of P1101 in Adults With ET

A Phase 2 study testing Ropeginterferon alfa-2b-njft (P1101) in people with Essential Thrombocythemia, run by PharmaEssentia at 34 sites in 2 countries.

Active, not recruiting

Under way, not looking for participants. Everyone needed has enrolled. The study is still running.

Official record
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Trial summary

A Single-arm, Multicenter Study to Assess the Efficacy, Safety, and Tolerability of Ropeginterferon alfa-2b-njft (P1101) in Adult Patients with Essential Thrombocythemia Read more about this study

Who is it for?
Ages 18 years and older
Conditions the study is for
Essential Thrombocythemia
What is being tested?
Ropeginterferon alfa-2b-njft (P1101)
Type of treatment
Injection or infusion
Could I receive a placebo?
No, everyone receives an active treatment
How many people
91 participants
Where
34 sitesin 2 countries
Study dates
29 Sep 2022to 31 Mar 2027
Phase
Phase 2
Are there results?
Not yet: the study is under way

Key requirements

Who can take part, as listed by the sponsor. Only the study team can confirm whether you are eligible, so take this list to your doctor.

  • Ages 18 years and older
  • All sexes
  • No healthy volunteers
  • Adult, Older adult

Who may be able to join

  • Male and female subjects ≥18 years old.
  • Subjects diagnosed with ET according to the World Health Organization (WHO) 2016 criteria.
  • Subjects that are cytoreductive treatment-naïve, or pre-exposed to HU and/or ANA, as specified below (according to Investigator's judgment and documented in the patient's medical record):
  • Cytoreductive-naïve patients must be in need of cytoreductive treatment, defined as having at least one of the following:
  • Progressive leukocytosis and/or thrombocytosis
  • Disease-related symptoms (i.e., pruritus, night sweats, fatigue)
Show all 17Show fewer
  • Vasomotor/microvascular disturbances, not responsive to aspirin (including headache, chest pain or erythromelalgia, etc.)
  • High-risk (history of thrombosis at any age; or age >60 years with JAK2 mutation)
  • Patients previously exposed to HU will be classified as either:
  • Documented formal HU resistance or intolerance
  • HU stopped without documented formal resistance/intolerance due to insufficient blood count control or toxicity. The last HU dose must be >7 days prior the first dose of P1101.
  • Adequate hepatic function defined as bilirubin ≤1.5 × upper limit normal (ULN), prothrombin time (PT) (international normalized ratio, [INR]) ≤1.5 x ULN, albumin >3.5 g/dL, alanine aminotransferase (ALT) ≤2.0 x ULN, aspartate aminotransferase ≤2.0 x ULN at screening.
  • Creatinine clearance ≥40 mL/min (by Cockcroft-Gault equation).
  • Males and females of childbearing potential, as well as all women <2 years after the onset of menopause, must agree to use an acceptable form of birth control until 60 days following the last dose of the study drug, and females must agree to not breastfeed during the study.
  • Written informed consent obtained from the subject and ability for the subject to comply with the requirements of the study.
  • Platelet count >450 × 109/L at screening
  • Both ANA-naïve and ANA-pretreated subjects are eligible for the study, regardless of the reason to terminate ANA use

Who cannot join

  • Any subject requiring a legally authorized representative
  • Subjects who stopped prior interferon alfa therapy due to low efficacy or poor tolerability
  • Any contraindications or hypersensitivity to IFN-α and/or its excipients
  • Co-morbidity with severe or serious condition that, in the Investigator's opinion, would jeopardize the safety of the subject or their compliance with the protocol, including significant cardiac disease (including New York Heart Association Class III-IV congestive heart failure and clinically significant arrhythmias) and pulmonary hypertension
  • History of major organ transplantation
  • Pregnant or lactating females
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  • Subjects with any significant medical conditions that, in the opinion of the Investigator, would compromise the results of the study or may impair compliance with the requirements of the protocol, including but not limited to:
  • Documented autoimmune disease at screening or in the history (e.g., thyroid dysfunction, hepatitis, idiopathic thrombocytopenic purpura, scleroderma, psoriasis, or any arthritis of autoimmune origin)
  • Clinically relevant pulmonary infiltrates, pneumonia, and pneumonitis at screening that, in the Investigator's opinion, would jeopardize the safety of the subject or their compliance with the protocol
  • Infections with systemic manifestations (e.g., bacterial, fungal, or human immunodeficiency virus [HIV], except hepatitis B [HBV] and/or hepatitis C [HCV],at screening)
  • Evidence of severe retinopathy (e.g., cytomegalovirus retinitis [CMV], macular degeneration) or clinically relevant ophthalmological disorder (due to diabetes mellitus or hypertension)
  • History or presence of clinically relevant depression
  • Previous suicide attempts or at any risk of suicide at screening, in the judgment of the Investigator
  • History or presence of clinically significant neurologic diseases
  • History of any malignancy within 5 years (except adequately treated nonmelanoma skin cancer, prostate cancer status post resection with an undetectable prostate-specific antigen [PSA], curative treated in-situ cancer of the cervix, ductal carcinoma in-situ [DCIS] of the breast, Stage 1 Grade 1 endometrial carcinoma, or other solid tumors including lymphomas [without bone marrow involvement] curatively treated with no evidence of disease for ≥2 years prior to study)
  • History of alcohol or drug abuse within the last year
  • History or evidence of any other MPN
  • Use of any investigational drug <4 weeks prior to the first dose of study drug or not recovered from effects of prior administration of any investigational agent
  • Presence of more than one driver mutation (e.g., V617F JAK2 and CALR, CALR and MPL, V617F JAK2 and MPL)
  • Prior use of JAK inhibitors

Trial locations and contacts

34 sites in 2 countries.

34 sites in 2 countries

United States30 sites

  • The Winship Cancer Institute Emory University

    Atlanta, Georgia, 30322

  • Greater Baltimore Medical Center

    Baltimore, Maryland, 21204

  • University of Alabama at Birmingham

    Birmingham, Alabama, 35294

  • Massachusetts General Hospital

    Boston, Massachusetts, 02114

  • Dana-Farber Cancer Institute

    Boston, Massachusetts, 02215

  • University of North Carolina (UNC) - Lineberger Comprehensive Cancer Center

    Chapel Hill, North Carolina, 27514

  • University of Virginia - Emily Couric Cancer Center

    Charlottesville, Virginia, 22903

  • City of Hope National Medical Center

    Duarte, California, 91010

  • Duke University Medical Center

    Durham, North Carolina, 27710

  • Astera HealthCare

    East Brunswick, New Jersey, 08816

  • Fort Wayne Medical Oncology and Hematology

    Fort Wayne, Indiana, 46804

  • Marin Cancer Care

    Greenbrae, California, 94904

  • East Carolina University

    Greenville, North Carolina, 27858

  • John Theurer Cancer Center At Hackensack UMC

    Hackensack, New Jersey, 07601

  • MD Anderson Cancer Center

    Houston, Texas, 77030

  • USC Norris Comprehensive Cancer Center

    Los Angeles, California, 90033

  • University of Tennessee Health Science Center

    Memphis, Tennessee, 38103

  • Yale University School of Medicine - Yale Cancer Center

    New Haven, Connecticut, 06510

  • Tulane University Medical Center

    New Orleans, Louisiana, 70112

  • Weill Medical College of Cornell University

    New York, New York, 10021

  • Memorial Sloan Kettering Cancer Center

    New York, New York, 10065

  • Mercy Health - Paducah Medical Oncology and Hematology

    Paducah, Kentucky, 42003

  • Cancer Care Specialists

    Reno, Nevada, 89511

  • University of Utah

    Salt Lake City, Utah, 84132

  • Fred Hutchinson Cancer Research Center

    Seattle, Washington, 98109

  • Washington University School of Medicine - Division of Oncology

    St Louis, Missouri, 63110

  • Stony Brook University Medical Center

    Stony Brook, New York, 11794

  • Montefiore Medical Center

    The Bronx, New York, 10467

  • Georgetown University Medical Center

    Washington D.C., District of Columbia, 20057

  • Regional Medical Oncology Center

    Wilson, North Carolina, 27893

Other trials in United States

Canada4 sites

  • University of Calgary Tom Baker Cancer Centre

    Calgary, Alberta, T2N 4N2

  • Juravinski Cancer Centre

    Hamilton, Ontario

  • Princess Margaret Hospital

    Toronto, Ontario

  • St. Paul's Hospital - Providence Health Care

    Vancouver, British Columbia

Other trials in Canada

About this study

Official title: A Single-arm, Multicenter Study to Assess the Efficacy, Safety, and Tolerability of Ropeginterferon Alfa-2b-njft (P1101) in Adult Patients With Essential Thrombocythemia

A Single-arm, Multicenter Study to Assess the Efficacy, Safety, and Tolerability of Ropeginterferon alfa-2b-njft (P1101) in Adult Patients with Essential Thrombocythemia

Read the detailed description

PharmaEssentia is developing a pegylated (PEG) IFN-α product, P1101, for the treatment of Essential Thrombocythemia (ET) as lack of disease modifying therapies in essential ET constitutes a serious issue in modern hematology.

Ropeginterferon alfa-2b-njft (P1101) may represent an effective, well-tolerated treatment with the ability to provide a deeper response and superior control of important blood parameters with the potential to alter the course of the disease and prevent progression to post-ET myelofibrosis (MF) and/or secondary acute myeloid leukemia (sAML). Ropeginterferon alfa-2b-njft (P1101) is currently being evaluated in comparison to ANA in the ongoing global Phase 3 clinical study, SURPASS ET.

Enrolled patients will receive P1101 over 13 months followed by an extension period.

Being tested
Ropeginterferon alfa-2b-njft (P1101)
Keywords
MPN, Myeloproliferative, Neoplasms, Myeloproliferative Neoplasms, Essential, Thrombocythemia, Besremi, Ropeginterferon Alfa-2b-njft, P1101, Essential Thrombocythemia
Medical subject headings
Thrombocythemia, Essential, Neoplasms, Myeloproliferative Disorders, Thrombocytosis

What participants receive

What participants receive. In a randomised study each person is assigned to one group by chance.

  • Ropeginterferon alfa-2b-njft (P1101)Drug

    Also called P1101

    Ropeginterferon alfa-2b-njft (P1101)

Study groups in NCT05482971
Study groupRoleWhat they receive
Ropeginterferon alfa-2b (P1101)ExperimentalPre-filled Syringe, Q2W, SC injection
Receives: Drug: Ropeginterferon alfa-2b-njft (P1101)

What is being measured

The primary outcome is the main question the study is designed to answer.

  1. To assess efficacy of ropeginterferon alfa-2b-njft (P1101) in adult USA/Canadian patients with ET

    Efficacy will be based on peripheral blood count remission as defined by hematocrit (HCT) <45%, white blood cell (WBC) count ≤10 × 109/L, and platelets (PLT) ≤ 400 × 109/L in at least 80% of bi-weekly measurements for a consecutive 32-wek period during the 52-week core study treatment period.

    Measured: 12 months

Timeline

From the start of the study to its completion; the mark is today. Estimated dates are the sponsor’s plan.

  1. First posted1 Aug 2022
  2. Study start29 Sep 2022
  3. Primary completion9 May 2025
  4. Study completion31 Mar 2027 (estimated)

Resources

More details

For doctors and researchers: how the study is designed, who runs it, and its registry IDs.

Study design
Study type
Interventional
Phase
Phase 2
Primary purpose
Treatment
Allocation
Not applicable
Intervention model
Single group
Masking
None (open label)
Enrolment
91 participants (actual)
IDs, dates and publications
ClinicalTrials.gov ID
NCT05482971
Sponsor's protocol ID
EXCEED ET / A22-301
Status last verified
Aug 2025
First submitted
28 Jul 2022
Last updated
13 Aug 2025
Results posted
No
Data monitoring committee
No
FDA-regulated drug
Yes
FDA-regulated device
No
Individual data sharing
No

Publications

  • Reeves BN, El Chaer F, Foltz L, Tashi T, Abu-Zeinah G, Qin A, Lucas J, Halpern AB, Maze D, Safah H, O'Connell CL, Goel S, Rein L, Fang B, How J, Babu S, Li Z, Cerquozzi S, Oh ST, Hunter AM, Podoltsev N, Vachhani P, Cunningham JM, Hillis C, Bose P, Yacoub A, Mascarenhas J, Zagrijtschuk O, Castro H, Mesa RA, Masarova L. Ropeginterferon alfa-2b-njft treatment in essential thrombocythemia across different driver mutations: results from a North American, single-arm, multicentre study (EXCEED-ET). Lancet Reg Health Am. 2026 Jun 10;61:101529. doi: 10.1016/j.lana.2026.101529. eCollection 2026 Sep.

    PubMed 42306077

  • Masarova L, Reeves BN, El Chaer F, Foltz L, Tashi T, Abu-Zeinah G, Lucas J, Halpern AB, Maze D, Qin A, Safah H, Lan F, O'Connell CL, Goel S, Rein L, Fang B, How J, Babu S, Li Z, Cerquozzi S, Oh ST, Hunter AM, Podoltsev N, Vachhani P, Yacoub A, Cunningham JM, Hillis C, Otoukesh S, Zagrijtschuk O, Castro H, Bose P. A multicenter study to assess efficacy, safety, and tolerability of ropeginterferon alfa-2b-njft in patients with essential thrombocythemia in the US and Canada: EXCEED-ET trial. Front Med (Lausanne). 2025 Apr 17;12:1548590. doi: 10.3389/fmed.2025.1548590. eCollection 2025.

    PubMed 40330782

Questions about this trial

What is this clinical trial studying?

A Phase 2 study testing Ropeginterferon alfa-2b-njft (P1101) in people with Essential Thrombocythemia, run by PharmaEssentia at 34 sites in 2 countries.

The full registered description is in the About this study section and the official record.

Who may be eligible to take part?

The sponsor lists 17 inclusion and 20 exclusion criteria. Eligibility is decided by the study team after screening, so read the full criteria and contact a listed site.

Is this clinical trial currently recruiting?

The record says: under way, not looking for participants, as of the sponsor's last update on 13 Aug 2025. Status can change between updates, so confirm with the study contact before making plans.

Where is the study taking place?

The record lists 34 sites in United States, Canada. Whether a site is taking part right now should be confirmed with that site. See every site.

What are the possible benefits and risks?

They depend on the study and the treatment, and no study can promise a benefit. The study team explains the known risks during informed consent, before anyone joins; the record itself does not list them.

What does this phase mean?

Phase 2 studies give the treatment to a larger group to see whether it has the intended effect and to keep checking safety.

Not every study follows the same path, and some studies (observational studies, device studies) have no phase.

Are results available for this study?

This study is still under way. Results are not available in the source record yet.

How can a doctor refer a patient or contact the research team?

The record lists no contact. The official record names the sponsor, who can be asked directly. AzTrials does not enrol participants or take referrals.

What does the primary outcome measure?

To assess efficacy of ropeginterferon alfa-2b-njft (P1101) in adult USA/Canadian patients with ET (time frame: 12 months). It is what the study was designed to measure, not a finding.

Where can I read the original study information?

On the ClinicalTrials.gov record for NCT05482971, which also lists 2 linked publications.

Does a listing mean the treatment is approved or proven to work?

No. A listing means the study is registered. It does not show that the treatment is safe, works, or is approved by a regulator.

How often is this information updated?

AzTrials copies changed records from ClinicalTrials.gov every night and reloads the whole registry every week. The sponsor last updated this record on 13 Aug 2025.

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