A Study to Assess Efficacy, Safety, and Tolerability of P1101 in Adult Patients With PV

Sponsor

PharmaEssentia (Industry)

Overall Status

Not yet recruiting

CT.gov ID

NCT05481151

Collaborator

(none)

Enrollment

Arm

Anticipated Duration (Months)

Study Details

Study Description

Brief Summary

A Phase IIIb, Single-Arm, Multicenter Study to Assess Efficacy, Safety, and Tolerability of Ropeginterferon alfa-2b-njft (P1101) in Adult Patients with Polycythemia Vera (PV)

Condition or Disease	Intervention/Treatment	Phase
Polycythemia Vera	Drug: Ropeginterferon alfa-2b-njft (P1101)	Phase 3

Detailed Description

Polycythemia vera (PV) is the most common type of chronic myeloproliferative neoplasm (MPN), with an annual reported incidence of up to 2.6/100,000. This is a long-term debilitating and life-threatening disease because it is associated with the risk of thrombosis, bleeding, and progression to myelofibrosis (MF) and secondary acute myeloid leukemia (sAML)

Ropeginterferon alfa-2b-njft (P1101), which gained US marketing authorization in November 2021, is the only interferon alfa approved for the treatment of PV.

This study aims to evaluate the efficacy, tolerability, and safety of ropeginterferon alfa-2b-njft (P1101) in US and Canadian PV patients, utilizing an optimized dosing regimen.

Study Design

Study Type:

Interventional

Anticipated Enrollment :

64 participants

Allocation:

N/A

Intervention Model:

Single Group Assignment

Masking:

None (Open Label)

Primary Purpose:

Treatment

Official Title:

A Phase IIIb, Single-Arm, Multicenter Study to Assess Efficacy, Safety, and Tolerability of Ropeginterferon Alfa-2b-njft (P1101) in Adult Patients With Polycythemia Vera (PV)

Anticipated Study Start Date :

Sep 29, 2022

Anticipated Primary Completion Date :

Jan 11, 2024

Anticipated Study Completion Date :

Dec 28, 2025

Arms and Interventions

Arm	Intervention/Treatment
Experimental: Ropeginterferon alfa-2b-njft (P1101) Pre-filled Syringe, Q2W, SC injection	Drug: Ropeginterferon alfa-2b-njft (P1101) Ropeginterferon alfa-2b-njft (P1101)

Arm

Intervention/Treatment

Experimental: Ropeginterferon alfa-2b-njft (P1101)

Pre-filled Syringe, Q2W, SC injection

Drug: Ropeginterferon alfa-2b-njft (P1101)

Ropeginterferon alfa-2b-njft (P1101)

Outcome Measures

Primary Outcome Measures

To assess the efficacy, safety, and tolerability of ropeginterferon alfa-2b-njft as the proportion of subjects that are in a complete hematologic response (CHR) at Week 24. [24 weeks]
Hematocrit (HCT) <45%, white blood cell (WBC) count ≤10 × 109/L, platelets (PLT) ≤400 × 109/L in the absence of phlebotomy

Eligibility Criteria

Criteria

Ages Eligible for Study:

18 Years and Older

Sexes Eligible for Study:

All

Accepts Healthy Volunteers:

Inclusion Criteria:

Male or female subjects aged ≥18 years at the time of signing the informed consent form
Subjects diagnosed with PV according to the 2016 World Health Organization (WHO) criteria
With good liver function at screening, which is defined as total bilirubin ≤1.5 × upper limit of normal (ULN), international normalized ratio (INR) ≤1.5 × ULN, albumin

3.5 g/dL, alanine aminotransferase (ALT) ≤2.0 × ULN, and aspartate aminotransferase (AST) ≤2.0 × ULN

Hemoglobin (HGB) ≥10 g/dL for females, and HGB ≥11 g/dL for males at screening
Neutrophil count ≥1.5 × 109/L at screening
Creatinine clearance rate ≥40 mL/min at screening (according to the Cockcroft-Gault formula)
Males and females of childbearing potential, as well as all women <2 years after the onset of menopause, must agree to use an acceptable form of birth control until 60 days following the last dose of the study drug, and females must agree to not breastfeed during the study (see Appendix 4 for details)
Written informed consent obtained from the subject or legally authorized representative, and ability for the subject to comply with the requirements of the study

Exclusion Criteria:

Any contraindications to interferon alfa or hypersensitivity to interferon alfa
Have received previous interferon alfa or ruxolitinib therapy
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
Subjects with any other diseases that the Investigator determines will affect the study results or may weaken the compliance to protocol, including but not limited to:
Use any investigational drug <4 weeks prior to the first dose of study drug, or not recovered from effects of prior administration of any investigational drug