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

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)

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.

Study Overview

• Status: Recruiting
• Conditions: Polycythemia Vera (PV)
• Intervention / Treatment: Biological: P1101 (Ropeginterferon alfa-2b)

Detailed Description

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.

• Study Type: Interventional
• Enrollment (Estimated): 67
• Phase: Phase 3

Contacts and Locations

• Study Contact: Name: Hiroaki Kawase; Phone Number: +81-3-6910-5103; Email: hiroaki_kawase@pharmaessentia.com

Study Locations

• Japan
  • Ehime
    • Toon-shi, Ehime, Japan, 791-0295
      • Recruiting
      • Ehime University Hospital
  • Mie
    • Tsu-shi, Mie, Japan
      • Recruiting
      • Mie University Hospital
  • Osaka
    • Suita-shi, Osaka, Japan, 565-0871
      • Recruiting
      • Osaka University Hospital
  • Tokyo
    • Bunkyo-ku, Tokyo, Japan, 113-8431
      • Recruiting
      • Juntendo University Hospital
    • Shinjuku-ku, Tokyo, Japan, 160-0023
      • Recruiting
      • Tokyo Medical University Hospital
  • Yamanashi
    • Chuo-shi, Yamanashi, Japan, 409-3898
      • Recruiting
      • University of Yamanashi Hospital

Participation Criteria

Eligibility Criteria

• Ages Eligible for Study: 20 years and older (Adult, Older Adult)
• Accepts Healthy Volunteers: No

Description

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

Study Plan

How is the study designed?

Design Details

• Primary Purpose: Treatment
• Allocation: N/A
• Interventional Model: Single Group Assignment
• Masking: None (Open Label)

Number of Arms

1

Arms and Interventions

Participant Group / Arm

Intervention / Treatment

Experimental: P1101 (Ropeginterferon alfa-2b); Conventional treatment based on phlebotomies, lowdose aspirin (acetylsalicylic acid, 75-150 mg/day) plus the subcutaneous administration of pegylated prolineinterferon alpha-2b (P1101, Ropeginterferon alfa-2b) once every 2 weeks.

Biological: P1101 (Ropeginterferon alfa-2b); 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.

What is the study measuring?

Primary Outcome Measures

Outcome Measure	Measure Description	Time Frame
Maintenance rate of phlebotomy-free complete hematologic response (CHR) every 52 weeks	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	Through study completion, an average of 2 year

Secondary Outcome Measures

Outcome Measure	Measure Description	Time Frame
Changes in hematocrit every 52 weeks over time	Baseline is defined as Week 52 in Study A19-201	Through study completion, an average of 2 year
Changes in white blood cell every 52 weeks over time	Baseline is defined as Week 52 in Study A19-201	Through study completion, an average of 2 year
Changes in platelet count every 52 weeks over time	Baseline is defined as Week 52 in Study A19-201	Through study completion, an average of 2 year
Changes in red blood cell count every 52 weeks over time	Baseline is defined as Week 52 in Study A19-201	Through study completion, an average of 2 year
Changes in spleen size every 52 weeks over time	Baseline is defined as Week 52 in Study A19-201	Through study completion, an average of 2 year
Necessity of phlebotomy	Baseline is defined as Week 52 in Study A19-201	Through study completion, an average of 2 year
Proportion of subjects without thrombotic or hemorrhagic events	Baseline is defined as Week 52 in Study A19-201	Through study completion, an average of 2 year
Changes in JAK2 V617F mutant allelic burden value every 52 weeks over time	Baseline is defined as Week 52 in Study A19-201	Through study completion, an average of 2 year

Other Outcome Measures

Outcome Measure	Measure Description	Time Frame
Bone marrow histological remission (optional)	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	Through study completion, an average of 2 year

Collaborators and Investigators

• Sponsor: PharmaEssentia Japan K.K.
• Investigators: Principal Investigator: Keita Kirito, MD, University of Yamanashi Hospital

Study record dates

Study Major Dates

• Study Start (Actual): January 19, 2021
• Primary Completion (Estimated): June 30, 2026
• Study Completion (Estimated): June 30, 2026

Study Registration Dates

• First Submitted: November 18, 2020
• First Submitted That Met QC Criteria: December 3, 2020
• First Posted (Actual): December 7, 2020

Study Record Updates

• Last Update Posted (Estimated): November 8, 2024
• Last Update Submitted That Met QC Criteria: November 6, 2024
• Last Verified: November 1, 2024

More Information

Terms related to this study

• Keywords: Myeloproliferative Neoplasms
• Additional Relevant MeSH Terms: Neoplasms by Site; Neoplasms; Hematologic Diseases; Bone Marrow Diseases; Myeloproliferative Disorders; Bone Marrow Neoplasms; Hematologic Neoplasms; Polycythemia Vera; Polycythemia
• Other Study ID Numbers: A20-201

Plan for Individual participant data (IPD)

• Plan to Share Individual Participant Data (IPD)?: NO

Drug and device information, study documents

• Studies a U.S. FDA-regulated drug product: Yes
• Studies a U.S. FDA-regulated device product: No