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

Polycythemia Vera (PV) Clinical Trial

Official 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)

Clinical Trial Details — Status: Recruiting

Administrative data

• NCT number: NCT04655092
• Other study ID #: A20-201
• Status: Recruiting
• Phase: Phase 3
• Start date: January 19, 2021
• Est. completion date: October 31, 2024

Study information

• Verified date: March 2023
• Source: PharmaEssentia Japan K.K.
• Contact: Hiroaki Kawase
• Phone: +81-3-6910-5103
• Email: hiroaki_kawase[@]pharmaessentia.com
• Is FDA regulated: No
• Study type: Interventional

Clinical Trial Summary

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.

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.

Recruitment information / eligibility

• Status: Recruiting
• Enrollment: 67
• Est. completion date: October 31, 2024
• Est. primary completion date: October 30, 2024
• Accepts healthy volunteers: No
• Gender: All
• Age group: 20 Years and older

Eligibility

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

Related Conditions & MeSH terms

• Polycythemia
• Polycythemia Vera
• Polycythemia Vera (PV)

Intervention

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.

Locations

Country	Name	City	State
Japan	Juntendo University Hospital	Bunkyo-ku	Tokyo
Japan	University of Yamanashi Hospital	Chuo-shi	Yamanashi
Japan	Tokyo Medical University Hospital	Shinjuku-ku	Tokyo
Japan	Osaka University Hospital	Suita-shi	Osaka
Japan	Ehime University Hospital	Toon-shi	Ehime
Japan	Mie University Hospital	Tsu-shi	Mie

Sponsors (1)

Lead Sponsor	Collaborator
PharmaEssentia Japan K.K.

Country where clinical trial is conducted

Japan, 

Outcome

Type	Measure	Description	Time frame	Safety issue
Other	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
Primary	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	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
Secondary	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
Secondary	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
Secondary	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
Secondary	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
Secondary	Necessity of phlebotomy	Baseline is defined as Week 52 in Study A19-201	Through study completion, an average of 2 year
Secondary	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
Secondary	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