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.
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
Allocation
NA
Purpose
TREATMENT
Masking
NONE
Enrollment
67
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.
Ehime University Hospital
Toon-shi, Ehime, Japan
RECRUITINGMie University Hospital
Tsu, Mie-ken, Japan
RECRUITINGOsaka University Hospital
Suita-shi, Osaka, Japan
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
Time frame: Through study completion, an average of 2 year
Changes in hematocrit every 52 weeks over time
Baseline is defined as Week 52 in Study A19-201
Time frame: 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
Time frame: 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
Time frame: 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
Time frame: 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
Time frame: Through study completion, an average of 2 year
Necessity of phlebotomy
Baseline is defined as Week 52 in Study A19-201
Time frame: Through study completion, an average of 2 year
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Juntendo University Hospital
Bunkyo-ku, Tokyo, Japan
RECRUITINGTokyo Medical University Hospital
Shinjuku-ku, Tokyo, Japan
RECRUITINGUniversity of Yamanashi Hospital
Chuo-shi, Yamanashi, Japan
RECRUITINGProportion of subjects without thrombotic or hemorrhagic events
Baseline is defined as Week 52 in Study A19-201
Time frame: 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
Time frame: Through study completion, an average of 2 year