NCT04182100

Efficacy and Safety of P1101 in Polycythemia Vera Patients for Whom the Standard of Treatment is Difficult to Apply

Phase 2 Single Arm Study of Efficacy and Safety of P1101 for Polycythemia Vera (PV) Patients for Whom the Current Standard of Treatment is Difficult to Apply

• Status: Completed
• Phase: Phase 2
• Study type: Interventional
• Enrollment: 29 (actual)
• Sponsor: PharmaEssentia Japan K.K. · Industry
• Sex: All
• Age: 20 Years
• Healthy volunteers: Not accepted

Summary

This is a Phase 2 single arm study to investigate efficacy and safety of P1101 for adult Japanese patients with PV.

Detailed description

Eligible patients will be treated with P1101, starting at 100 μg (or 50 μg in patients under another cytoreductive therapy). The dose should be gradually increased by 50 μg every two weeks (in parallel, other cytoreductive therapy should be decreased gradually, as appropriate) until stabilization of the hematological parameters is achieved (hematocrit \<45%, platelets \<400 x 10\^9/L and leukocytes \<10 x 10\^9/L). The maximum recommended single dose is 500 μg injected every two weeks. At week 36 (month 9) and week 52 (month 12), the primary study endpoint, phlebotomy-free CHR, will be analyzed. After completion of the 52-week study duration, provision and administration of P1101, collection of the long-term follow up information (blood parameters, molecular and cytogenetic data, safety parameters and as also the optional bone marrow data) will be continued until the drug becomes commercially available for all study subjects..

Conditions

• Polycythemia Vera (PV)

Interventions

Type	Name	Description
DRUG	P1101	P1101 (ropeginterferon alfa-2b) will be administered subcutaneously every 2 weeks at the starting dose of 100 μg every two weeks (or 50 μg in patients under another cytoreductive therapy). The dose should be gradually increased by 50 μg every two weeks (in parallel, other cytoreductive therapy should be decreased gradually, as appropriate) until stabilization of the hematological parameters is achieved (hematocrit \<45%, platelets \<400 x 10\^9/L and leukocytes \<10 x 10\^9/L). The maximum recommended single dose is 500 μg injected every two weeks. The dose will be maintained at the highest level which can be tolerated and delivers best possible disease response.
DRUG	Low-dose aspirin	Low-dose aspirin (acetylsalicylic acid) (75-150 mg/day) will be given as background therapy during the 12 months of study treatment, unless contraindicated.
PROCEDURE	Phlebotomy	Phlebotomy is performed aiming at a hematocrit \< 45%. When the hematocrit value is 45% or higher, phlebotomy is performed. The volume of phlebotomy per procedure should be 200 to 400 mL while monitoring the circulatory dynamics such as blood pressure and pulse. In the elderly and patients with cardiovascular disorders, a small volume (100-200 mL) should be considered to avoid rapid changes in hemodynamics.

Timeline

• Start date: 2019-12-20
• Primary completion: 2021-03-08
• Completion: 2021-03-08
• First posted: 2019-12-02
• Last updated: 2025-09-15
• Results posted: 2025-09-15

Locations

8 sites across 1 country: Japan

Regulatory

• FDA-regulated drug study