Clinical trial NCT04655092: Extension Study of P1101 After Completion of Phase 2 Study in PV Patients or Phase 3 Study in ET Patients
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)
Study type: INTERVENTIONAL
Overall status: RECRUITING
Phase: PHASE3
Conditions: Polycythemia Vera (PV)
Keywords: Myeloproliferative Neoplasms
Lead sponsor: PharmaEssentia Japan K.K.
Sponsor class: INDUSTRY
First posted: 2020-12-07
Enrollment: 67
Sex: ALL
Minimum age (years): 20
Standard ages: ADULT
OLDER_ADULT
Accepts healthy volunteers: no
Allocation: NA
Intervention model: SINGLE_GROUP
Intervention model description: Conventional treatment based on phlebotomies, low-dose aspirin (acetylsalicylic acid, 75-150 mg/day) plus the subcutaneous administration of P1101 (ropeginterferon alfa-2b) once every 2 weeks
Primary purpose: TREATMENT

Brief 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.

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.

Eligibility criteria: 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

Primary outcomes:
measure: Maintenance rate of phlebotomy-free complete hematologic response (CHR) every 52 weeks
timeFrame: Through study completion, an average of 2 year
Secondary outcomes:
measure: Changes in hematocrit every 52 weeks over time
timeFrame: Through study completion, an average of 2 year
measure: Changes in white blood cell every 52 weeks over time
timeFrame: Through study completion, an average of 2 year
measure: Changes in platelet count every 52 weeks over time
timeFrame: Through study completion, an average of 2 year
measure: Changes in red blood cell count every 52 weeks over time
timeFrame: Through study completion, an average of 2 year
measure: Changes in spleen size every 52 weeks over time
timeFrame: Through study completion, an average of 2 year
measure: Necessity of phlebotomy
timeFrame: Through study completion, an average of 2 year
measure: Proportion of subjects without thrombotic or hemorrhagic events
timeFrame: Through study completion, an average of 2 year
measure: Changes in JAK2 V617F mutant allelic burden value every 52 weeks over time
timeFrame: Through study completion, an average of 2 year
Other outcomes:
measure: Bone marrow histological remission (optional)
timeFrame: Through study completion, an average of 2 year
Locations:
city: Toon-shi
country: Japan
facility: Ehime University Hospital
state: Ehime
city: Tsu-shi
country: Japan
facility: Mie University Hospital
state: Mie
city: Suita-shi
country: Japan
facility: Osaka University Hospital
state: Osaka
city: Bunkyo-ku
country: Japan
facility: Juntendo University Hospital
state: Tokyo
city: Shinjuku-ku
country: Japan
facility: Tokyo Medical University Hospital
state: Tokyo
city: Chuo-shi
country: Japan
facility: University of Yamanashi Hospital
state: Yamanashi
Study officials:
affiliation: University of Yamanashi Hospital
name: Keita Kirito, MD
role: PRINCIPAL_INVESTIGATOR
References: