A Phase III b study to evaluate P1101 in Japanese PV patients
- Conditions
- Polycythemia Vera
- Registration Number
- jRCT2031230267
- Lead Sponsor
- PharmaEssentia Japan KK
- Brief Summary
Not available
- Detailed Description
Not available
Recruitment & Eligibility
- Status
- Not Recruiting
- Sex
- All
- Target Recruitment
- 20
- Male or female patients >=18 years old at the time of informed consent to participate in the study
- Patients diagnosed with PV according to the WHO 2008 or 2016 criteria
- Patients with PV who have the inadequate response to an existing therapy or whom the existing therapy is inappropriate to apply
- Patients who have given written informed consent to participate in this study
- Any contraindications to interferon alfa or hypersensitivity to interferon alfa
- Subjects who stopped prior to interferon alfa therapy due to low efficacy or poor tolerability
- Subjects with severe or serious diseases that the Investigator determines may affect the subject's participation in this study
- History of major organ transplantation
- Pregnant or breastfeeding women
Study & Design
- Study Type
- Interventional
- Study Design
- single assignment
- Primary Outcome Measures
Name Time Method the rate of phlebotomy-free complete hematologic response (CHR) Week 24 The percentage of patients who have a CHR and have not required phlebotomy in the previous 12 weeks. A responder for the primary endpoint is defined as a patient who meets all of the following criteria at Week 24:
- Hct <45% and did not require phlebotomy (no phlebotomy was performed in the previous 12 weeks)
- PLT =<400 x 10^9/L
- WBC =<10 x 10^9/L
- Secondary Outcome Measures
Name Time Method Time to achieve CHR Time to achieve CHR
Time to reach response maintenance dose Time to reach response maintenance dose (three consecutive doses of the same dose)
Numbers of phlebotomy required and changes in numbers of phlebotomy required from baseline Numbers of phlebotomy required and changes in numbers of phlebotomy required from baseline
Time to first response in peripheral blood count Time to first response in peripheral blood count (Hct, WBC, and PLT)
Rate of achieving phlebotomy-free complete hematologic response (CHR) Week 12 and Week 24 Rate of achieving "phlebotomy-free complete hematologic response (CHR)" (the same criteria as for the primary efficacy endpoint) at both Week 12 and Week 24
Duration of response in peripheral blood count Duration of response in peripheral blood count (Hct, WBC, and PLT)
Duration of response maintenance on peripheral blood count Week 12 through Week 24 Duration of response maintenance on peripheral blood count (Hct, WBC, and PLT) from Week 12 through Week 24
Improvement of symptoms assessed by MPN-SAF TSS each visit Improvement of symptoms assessed by MPN-SAF TSS at each visit
Proportion of subjects without thrombotic or hemorrhagic events Weeks 12 and 24 Proportion of subjects without thrombotic or hemorrhagic events at Weeks 12 and 24
Change from baseline to Week 24 in JAK2 V617F allele burden level baseline to Week 24 Change from baseline to Week 24 in JAK2 V617F allele burden level
Pharmacokinetics (PK) assessment Pharmacokinetics (PK) assessment