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A Phase III b, single-arm, multicenter, optimal dose finding study to assess the efficacy and safety of P1101 in Japanese patients with polycythemia vera (PV).

A Phase III b study to evaluate P1101 in Japanese PV patients

Kawase Hiroaki

PharmaEssentia Japan KK

1-3-13 Motoakasaka, Minato-ku, Tokyo

hiroaki_kawase@pharmaessentia.com

Kawase Hiroaki

PharmaEssentia Japan KK

1-3-13 Motoakasaka, Minato-ku, Tokyo

+81-3-6910-5103

hiroaki_kawase@pharmaessentia.com

Not Recruiting

July. 29, 2023

Interventional

single arm study

open(masking not used)

uncontrolled control

single assignment

treatment purpose

1. Male or female patients >=18 years old at the time of informed consent to participate in the study
2. Patients diagnosed with PV according to the WHO 2008 or 2016 criteria
3. Patients with PV who have the inadequate response to an existing therapy or whom the existing therapy is inappropriate to apply
4. Patients who have given written informed consent to participate in this study

1. Any contraindications to interferon alfa or hypersensitivity to interferon alfa
2. Subjects who stopped prior to interferon alfa therapy due to low efficacy or poor tolerability
3. Subjects with severe or serious diseases that the Investigator determines may affect the subject's participation in this study
4. History of major organ transplantation
5. Pregnant or breastfeeding women

Both

Polycythemia Vera

The subjects will be treated with P1101, starting at a dose of 250 micrograms. The dose of P1101 will be increased to 350 micrograms 2 weeks later and to 500 micrograms another 2 weeks later, and then P1101 will be administered at a fixed dose of 500 micrograms throughout the treatment period. Although the dose may be reduced to the prior dose for reasons related to the safety or tolerability, the increased dose should be preferably maintained throughout the treatment period.
The dose of P1101 will be increased or decreased appropriately depending on the pathological condition in the range up to 500 micrograms.

Myeloproliferative nemoplasms

Primary Efficacy Endpoint (at Week 24):
The primary efficacy endpoint is "the rate of phlebotomy-free complete hematologic response (CHR)" at Week 24.
"The rate of phlebotomy-free complete hematologic response" is defined as 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 efficacy endpoint:
- 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.
- Time to achieve CHR
- 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
- Time to first response in peripheral blood count (Hct, WBC, and PLT)
- Duration of response in peripheral blood count (Hct, WBC, and PLT)
- 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 at each visit
- 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
- Pharmacokinetics (PK) assessment

+81-3-3814-5672

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