CompletedPhase 2

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

Japan29 participantsStarted 2019-12-20

Plain-language summary

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

Age range20 Years

SexALL

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AI-rewrites the medical criteria so a patient or caregiver can understand them. Always confirm with the trial site.

✓. Male or female patients ≥20 years old
✓. Patients diagnosed with PV according to the WHO 2008 or WHO 2016 criteria
✓. PV patients for whom the current standard of treatment is difficult to apply. (Patients with a documented history of refractory to HU are excluded.)
✓. Total HU treatment duration shorter than 3 years (cumulatively) at screening
✓. For cytoreduction naïve patients only: PV in need of cytoreductive treatment, defined by fulfilling as one or more of the following criteria at baseline:
✓. Adequate hepatic function defined as bilirubin ≤1.5 x upper limit normal (ULN), international normalized ratio (INR) ≤1.5 x ULN, albumin \>3.5 g/dL, alanine aminotransferase (ALT) ≤2.0 x ULN, aspartate aminotransferase (AST) ≤2.0 x ULN at screening
✓. Hemoglobin (HGB) ≥10 g/dL at screening
✓. Neutrophil count ≥1.5 x 10\^9/L at screening

✕. Patients with symptomatic splenomegaly
✕. Previous use of IFNα for any indication
✕. Any contraindications or hypersensitivity to interferon-alfa
✕. Co-morbidity with severe or serious conditions which may impact patient participation in the study in investigator's opinion
✕. History of major organ transplantation
✕. Pregnant or lactating females
✕. Patients with any other medical conditions, which in the opinion of the Investigator would compromise the results of the study or may impair compliance with the requirements of the protocol
✕. Uncontrolled diabetes mellitus (HbA1c level of \> 7% at baseline)

Proportion of Subjects Who Achieved Durable Phlebotomy-free Complete Hematological Response (CHR) at Month 12

Timeframe: 12 months

NCT IDNCT04182100

SponsorPharmaEssentia Japan K.K.

Sponsor typeINDUSTRY

Study typeINTERVENTIONAL

Primary completion2021-03-08

Results submitted2023-01-18

Who can participate

Age range20 Years

SexALL

See this in plain English?

AI-rewrites the medical criteria so a patient or caregiver can understand them. Always confirm with the trial site.

✓. Male or female patients ≥20 years old
✓. Patients diagnosed with PV according to the WHO 2008 or WHO 2016 criteria
✓. PV patients for whom the current standard of treatment is difficult to apply. (Patients with a documented history of refractory to HU are excluded.)
✓. Total HU treatment duration shorter than 3 years (cumulatively) at screening
✓. For cytoreduction naïve patients only: PV in need of cytoreductive treatment, defined by fulfilling as one or more of the following criteria at baseline:
✓. Adequate hepatic function defined as bilirubin ≤1.5 x upper limit normal (ULN), international normalized ratio (INR) ≤1.5 x ULN, albumin \>3.5 g/dL, alanine aminotransferase (ALT) ≤2.0 x ULN, aspartate aminotransferase (AST) ≤2.0 x ULN at screening
✓. Hemoglobin (HGB) ≥10 g/dL at screening
✓. Neutrophil count ≥1.5 x 10\^9/L at screening

✕. Patients with symptomatic splenomegaly
✕. Previous use of IFNα for any indication
✕. Any contraindications or hypersensitivity to interferon-alfa
✕. Co-morbidity with severe or serious conditions which may impact patient participation in the study in investigator's opinion
✕. History of major organ transplantation
✕. Pregnant or lactating females
✕. Patients with any other medical conditions, which in the opinion of the Investigator would compromise the results of the study or may impair compliance with the requirements of the protocol
✕. Uncontrolled diabetes mellitus (HbA1c level of \> 7% at baseline)