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NCT Number: NCT04655092

Extension Study of P1101 After Completion of Phase 2 Study in PV Patients or Phase 3 Study in ET Patients

This is a Phase 3 open-label, multicenter, single arm study designed to evaluate the efficacy and safety and tolerability of P1101 patient with PV or ET in long-term.

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Key information

Conditions

Polycythemia Vera (PV) Bone Marrow Diseases Bone Marrow Neoplasms Hematologic Diseases Hematologic Neoplasms Hemic and Lymphatic Diseases Myeloproliferative Disorders Neoplasms Neoplasms by Site Polycythemia Vera

Age range

20 year and older

Sex eligibility

All sexes

Study type

Interventional

Phase

Phase 3

Primary location

Ehime University Hospital, Toon-shi, Ehime, Japan

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About this study

The study is to evaluate the long-term safety and efficacy of P1101 in PV or ET patients who participated in Study A19-201 or Study P1101 ET. The subjects who have completed the 52-week P1101 treatment duration in Study A19-201 will start treatment with P1101 at the dose at Week 50. The subjects who have completed the follow-up/end-of-study visit in Study P1101 ET will start treatment with P1101 at the dose at Week 50. The subjects who were treated with anagrelide will start treatment with P1101 at a dose of 250 μg. The dose of P1101 during this study may be increased or decreased up to 500 μg depending on the condition.

Evaluation of safety will include assessing vital signs, clinical safety laboratory tests, physical examinations, ECG evaluation, heart ECHO, lung X-ray, ECOG performance status, ocular examination, and AEs.

Efficacy evaluations, safety assessments, and immunogenicity evaluations of P1101 will be performed.

Evaluation of efficacy will include clinical laboratory assessments, allelic burden measurements of CALR, JAK-2, and MPL, spleen size measurements, bone marrow sampling.

Who can participate

Healthy volunteers accepted: No

Only the study team can determine whether someone qualifies for participation.

Inclusion criteria

  • Patients who have completed the 52-week treatment duration in Study A19-201 and are considered by the investigator or sub investigator to be eligible for participation in this study
  • Patients who have given written informed consent to participate in this study

Exclusion criteria

  • Patients who are considered by the investigator or sub investigator to be ineligible for continued treatment with P1101

Treatment and study plan

P1101 (Ropeginterferon alfa-2b)

Biological

The subjects who have completed the 52-week treatment duration in Study A19-201 will be treated with P1101, starting at the dose at Week 50. The dose during this study may be increased or decreased up to 500 μg depending on the condition. This study will be continued as a post-marketing clinical study after acquisition of the marketing approval of P1101.

Primary outcomes

  1. Maintenance rate of phlebotomy-free complete hematologic response (CHR) every 52 weeks

    Time frame: Through study completion, an average of 2 year

    CHR will be defined as follows.

    • Hematocrit <45% phlebotomy-free (absence of phlebotomy during the previous 12 weeks)
    • Platelet count ≤ 400 x 10^9/L
    • WBC count ≤ 10 x 10^9/L

Secondary outcomes

  1. Changes in hematocrit every 52 weeks over time

    Time frame: Through study completion, an average of 2 year

    Baseline is defined as Week 52 in Study A19-201

  2. Changes in white blood cell every 52 weeks over time

    Time frame: Through study completion, an average of 2 year

    Baseline is defined as Week 52 in Study A19-201

  3. Changes in platelet count every 52 weeks over time

    Time frame: Through study completion, an average of 2 year

    Baseline is defined as Week 52 in Study A19-201

  4. Changes in red blood cell count every 52 weeks over time

    Time frame: Through study completion, an average of 2 year

    Baseline is defined as Week 52 in Study A19-201

  5. Changes in spleen size every 52 weeks over time

    Time frame: Through study completion, an average of 2 year

    Baseline is defined as Week 52 in Study A19-201

  6. Necessity of phlebotomy

    Time frame: Through study completion, an average of 2 year

    Baseline is defined as Week 52 in Study A19-201

  7. Proportion of subjects without thrombotic or hemorrhagic events

    Time frame: Through study completion, an average of 2 year

    Baseline is defined as Week 52 in Study A19-201

  8. Changes in JAK2 V617F mutant allelic burden value every 52 weeks over time

    Time frame: Through study completion, an average of 2 year

    Baseline is defined as Week 52 in Study A19-201

Other outcomes

  1. Bone marrow histological remission (optional)

    Time frame: Through study completion, an average of 2 year

    Bone marrow histological remission was defined as the disappearance of hypercellularity and trilineage growth (panmyelosis), and absence of >grade 1 reticulin fibrosis in the subjects who gave informed consent in Study A19-201

Study contacts

Contact information is provided by the study sponsor or research team.

Hiroaki Kawase

CONTACT

[email protected]

+81-3-6910-5103

Sponsors and collaborators

Lead sponsor

PharmaEssentia Japan K.K.

Industry

Registry information

Official study title

Extension Study of P1101 in Japanese Patients Who Have Completed Phase 2 Single Arm Study in Polycythemia Vera (PV) Patients (Study A19-201) or Phase 3 Study in Essential Thrombocythemia (ET) Patients (Study P1101 ET)

Important dates

Study start
2021
Primary completion
2026
Study completion
2026
First posted
Dec 7, 2020
Registry last updated
Nov 8, 2024

OpenTrials presents study information sourced from ClinicalTrials.gov. The official registry record should be consulted for the latest information.

View the official ClinicalTrials.gov record (opens in a new tab)

This listing is for discovery and informational purposes only. It is not medical advice, does not guarantee that a study is recruiting, and does not determine eligibility. Contact the study team and a qualified healthcare professional when considering participation.

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