Extension Study of P1101 After Completion of Phase 2 Study in PV Patients or Phase 3 Study in ET Patients
Study Details
Study Description
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.
| Condition or Disease | Intervention/Treatment | Phase |
|---|---|---|
|
Phase 3 |
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 Design
Arms and Interventions
| 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.
|
Outcome Measures
Primary Outcome Measures
- Maintenance rate of phlebotomy-free complete hematologic response (CHR) every 52 weeks [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 Outcome Measures
- Changes in hematocrit every 52 weeks over time [Through study completion, an average of 2 year]
Baseline is defined as Week 52 in Study A19-201
- Changes in white blood cell every 52 weeks over time [Through study completion, an average of 2 year]
Baseline is defined as Week 52 in Study A19-201
- Changes in platelet count every 52 weeks over time [Through study completion, an average of 2 year]
Baseline is defined as Week 52 in Study A19-201
- Changes in red blood cell count every 52 weeks over time [Through study completion, an average of 2 year]
Baseline is defined as Week 52 in Study A19-201
- Changes in spleen size every 52 weeks over time [Through study completion, an average of 2 year]
Baseline is defined as Week 52 in Study A19-201
- Necessity of phlebotomy [Through study completion, an average of 2 year]
Baseline is defined as Week 52 in Study A19-201
- Proportion of subjects without thrombotic or hemorrhagic events [Through study completion, an average of 2 year]
Baseline is defined as Week 52 in Study A19-201
- Changes in JAK2 V617F mutant allelic burden value every 52 weeks over time [Through study completion, an average of 2 year]
Baseline is defined as Week 52 in Study A19-201
Other Outcome Measures
- Bone marrow histological remission (optional) [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
Eligibility Criteria
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
Contacts and Locations
Locations
| Site | City | State | Country | Postal Code | |
|---|---|---|---|---|---|
| 1 | Ehime University Hospital | Toon-shi | Ehime | Japan | 791-0295 |
| 2 | Mie University Hospital | Tsu-shi | Mie | Japan | |
| 3 | Osaka University Hospital | Suita-shi | Osaka | Japan | 565-0871 |
| 4 | Juntendo University Hospital | Bunkyo-ku | Tokyo | Japan | 113-8431 |
| 5 | Tokyo Medical University Hospital | Shinjuku-ku | Tokyo | Japan | 160-0023 |
| 6 | University of Yamanashi Hospital | Chuo-shi | Yamanashi | Japan | 409-3898 |
Sponsors and Collaborators
- PharmaEssentia Japan K.K.
Investigators
- Principal Investigator: Keita Kirito, MD, University of Yamanashi Hospital
Study Documents (Full-Text)
None provided.More Information
Publications
None provided.- A20-201