P1101 in Treating Patients With Early PMF or Overt PMF at Low or Intermediate-1 Risk

Primary Myelofibrosis Clinical Trial

Official title:

A Randomized, Double-Blind, Placebo-Controlled Multicenter Phase III Study to Assess Efficacy and Safety of Ropeginterferon Alfa-2b (P1101) in Adult Patients With Pre-fibrotic/Early Primary Myelofibrosis (PMF) or Overt PMF at Low or Intermediate-1 Risk According to DIPSS Plus

Clinical Trial Details — Status: Not yet recruiting

Administrative data

• NCT number: NCT06468033
• Other study ID #: A23-302
• Status: Not yet recruiting
• Phase: Phase 3
• Start date: October 2024
• Est. completion date: August 2027

Study information

• Verified date: June 2024
• Source: PharmaEssentia
• Contact: Ting-Fang Wang
• Phone: +886-2-26557688
• Email: tingfang_wang[@]pharmaessentia.com
• Is FDA regulated: No
• Study type: Interventional

Clinical Trial Summary

This is a phase 3 double-blind clinical trial arm to test Ropeginterferon alfa-2b (P1101) in adult patients with Primary Myelofibrosis (PMF) at early stage or low to medium risk. Participants will receive the study drug/placebo bi-weekly and have an assessment visit every 4 weeks. The ratio of study drug to placebo group is 2:1.

Recruitment information / eligibility

• Status: Not yet recruiting
• Enrollment: 150
• Est. completion date: August 2027
• Est. primary completion date: June 2026
• Accepts healthy volunteers: No
• Gender: All
• Age group: 18 Years and older

Eligibility

Inclusion Criteria:

1. Male or female patients aged =18 years at the time of signing the informed consent form;

2. Patients with pre-fibrotic/early PMF (Pre-PMF) or overt primary myelofibrosis at low to intermediate-1 risk according to DIPSS plus, diagnosed according to WHO 2016 or 2022 classification;

3. 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;

4. Hgb =10.0 g/dL at screening;

5. Neutrophil count =1.0 × 10^9/L at screening;

6. Creatinine clearance rate =30 mL/min at screening (according to the Cockcroft-Gault formula);

7. 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;

8. Written informed consent obtained from the subject and ability for the subject to comply with the requirements of the study.

Exclusion Criteria:

1. Any known contraindications to interferon a or hypersensitivity to interferon a;

2. Patients with prior interferon therapy having poor tolerability or lack of efficacy to the previous interferon therapy per investigator's judgement;

3. Patients with an ongoing cytoreduction (e.g., HU or IFN-a) at the time of screening if, in the Investigator's opinion, randomizing them into the placebo arm will lead to immediate rebound increase of peripheral blood counts and thus may jeopardize their health status;

4. With severe or serious diseases that, in the Investigator's opinion, may affect the patient's participation in this study;

5. History of major organ transplantation;

6. Pregnant or breastfeeding women;

7. Patients with any other diseases that will affect the study results or may weaken the compliance to protocol per the Investigator's judgment;

8. 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.

9. Eligible for JAK inhibitor therapy at screening.

Related Conditions & MeSH terms

• Myeloproliferative Disorders
• Myeloproliferative Neoplasm
• Primary Myelofibrosis

Intervention

Biological:
• Ropeginterferon alfa-2b
Pre-filled Syringe. Dosage: up to 500mcg

Other:
• Placebo
Placebo is a look-alike substance with the intervention (Ropeginterferon alfa-2b) that contains no active drug.

Locations

Country	Name	City	State
n/a

Sponsors (1)

Lead Sponsor	Collaborator
PharmaEssentia

Outcome

Type	Measure	Description	Time frame	Safety issue
Primary	Clinically relevant Complete Hematologic Response (CrCHR): Platelet count, and	=400 × 10^9/L	56 weeks
Primary	CrCHR: White Blood Cells (WBC) count, and	<10 × 10^9/L	56 weeks
Primary	CrCHR: Peripheral blood: Hemoglobin (Hgb), and	>10.0 g/dL	56 weeks
Primary	CrCHR: Absence of thrombotic events, and	Absence of major thrombotic events during the observation time frame	56 weeks
Primary	CrCHR: No progression to AML, or	No progression to secondary acute myeloid leukemia (AML)	56 weeks
Primary	Symptom endpoint	No progression on clinical symptoms based on the MFSAF Total Symptom Score (TSS) v4.0	56 weeks