Ropeginterferon Alfa 2b Plus Ruxolitinib for Myelofibrosis

NCT ID: NCT06770842

Last Updated: 2025-06-11

Basic Information

• Recruitment Status: RECRUITING
• Clinical Phase: PHASE2
• Total Enrollment: 20 participants
• Study Classification: INTERVENTIONAL
• Study Start Date: 2025-03-01
• Study Completion Date: 2027-12-31

Brief Summary

In this open-label single arm phase 2 study, approximately 20 patients with MF demonstrating suboptimal response to ruxolitinib monotherapy will be enrolled. Patients will continue to receive ruxolitinib at a stable dose and ropeginterferon alfa 2b will be added to the regimen.

Detailed Description

Ropeginterferon alfa 2b is administered subcutaneously once every 2 weeks. Ruxolitinib is self-administered orally as part of standard of care. BSubjects will continue combination treatment through the Initial Treatment Period (ITP) (first 6 cycles), which includes a Qualification Assessment. Those deriving clinical benefit in the opinion of the treating physician may continue receiving combination treatment in the Additional Treatment Period (6 cycles). Qualification Assessments will be performed at the end of each Additional Treatment Period, which is iterative, and may repeat for as long as clinical benefit is sustained, at the discretion of the treating physician.

Conditions

Primary Myelofibrosis (PMF); Post Polycythemia Myelofibrosis (PPV MF); Post Essential Thrombocythaemia Myelofibrosis (PET-MF)

Study Design

• Allocation Method: NA
• Intervention Model: SINGLE_GROUP
• Primary Study Purpose: TREATMENT
• Blinding Strategy: NONE

Study Groups

Ropeginterferon alfa 2b

Ropeginterferon alfa 2b is administered subcutaneously once every 2 weeks in addition to standard of care with Ruxolitinib which will be self-administered orally as described below. Both medications will continue uninterrupted in 28-day cycles. Subjects will continue combination treatment through the Initial Treatment Period (ITP) (first 6 cycles), which includes a Qualification Assessment. Those deriving clinical benefit in the opinion of the treating physician may continue receiving combination treatment in the Additional Treatment Period (6 cycles). Qualification Assessments will be performed at the end of each Additional Treatment Period, which is iterative, and may repeat for as long as clinical benefit is sustained, at the discretion of the treating physician.

Group Type: EXPERIMENTAL

Ropeginterferon alfa-2b (BESREMi®)

Intervention Type: DRUG

Ropeginterferon alfa 2b is administered subcutaneously once every 2 weeks. The dosing will be 250mcg at Week 0, 350mcg at Week 2, 500mcg at Week 4, and 500mcg every 2 weeks thereafter

Interventions

Ropeginterferon alfa-2b (BESREMi®)

Ropeginterferon alfa 2b is administered subcutaneously once every 2 weeks. The dosing will be 250mcg at Week 0, 350mcg at Week 2, 500mcg at Week 4, and 500mcg every 2 weeks thereafter

Intervention Type: DRUG

Eligibility Criteria

Inclusion Criteria

• Willing and able to provide informed consent
• Age ≥18 years
• Diagnosis of Overt Myelofibrosis (primary, post-ET, or post-PV) per World Health Organization (WHO) 2022 diagnostic criteria
• Intermediate-1, Intermediate-2, or high-risk disease by Dynamic International Prognostic Scoring System (DIPSS)
• Eastern Cooperative Oncology Group (ECOG) performance status of 0-2
• Platelet count ≥75 x 109/L prior to dosing on Cycle 1 Day 1
• Absolute neutrophil count ≥0.5 x 109/L prior to dosing on Cycle 1 Day 1
• Peripheral blast count ≤10% prior to dosing on Cycle 1 Day 1
• Women of childbearing potential and fertile men must agree to use an approved method of contraception from screening until 30 days after the last dose of ropeginterferon and ruxolitinib.
• Patients with suboptimal response to ruxolitinib as per one of the below:

i. Relapsed: Ruxolitinib treatment for ≥3 months with spleen regrowth, defined as <10% SVR or <30% decrease in spleen size from baseline, following an initial response* ii. Refractory: Ruxolitinib treatment for ≥3 months with <10% SVR or <30% decrease in spleen size from baseline.

• Response to ruxolitinib is defined as a ≥35% reduction in spleen volume from baseline, or a ≥50% reduction in spleen size for baseline spleen sizes >10 cm below left costal margin (LCM); a non-palpable spleen for baseline spleen sizes between 5-10 cm below LCM; or not eligible for spleen response for baseline spleen <5 cm below LCM.

Exclusion Criteria

• Prior or current use of interferon alfa (IFNα) preparations for MPN
• Patients currently on other investigational therapy (ies)
• Contraindications or hypersensitivity to IFNα preparations
• History of organ and haematopoietic stem cell transplantation
• History of splenectomy
• Pregnant or lactating females, or females planning to become pregnant at any time during the study
• Documented autoimmune disease at screening
• Infection with human immunodeficiency virus (HIV)
• Active and uncontrolled infections with hepatitis B virus (HBV) and hepatitis C virus (HCV). Please note that patients on antiviral therapy with undetectable HBV DNA and HCV RNA may be recruited.
• Evidence of severe retinopathy including but not limited to macular degeneration, diabetic retinopathy and hypertensive retinopathy.
• History of clinically significant neuropsychiatric conditions including but not limited to depression and epilepsy.
• Clinically significant neuropsychiatric conditions including but not limited to depression and epilepsy.
• Concurrent second active and non-stable malignancy (patients with a concurrent second active but stable malignancy, i.e., non-melanoma skin cancers, are eligible)
• Evidence of alcohol or drug abuse within 6 months
• Evidence at the time of Screening of significant renal or hepatic insufficiency (unless due to hemolysis) as defined by any of the following local lab parameters:

• Calculated glomerular filtration rate (GFR; using the Cockcroft-Gault equation) <40 mL/min or serum creatinine >1.5 x the local upper limit of normal
• Aspartate transaminase (AST) or alanine aminotransferase (ALT) ≥2.5 x the local upper limit of normal
• Unwilling or unable to comply with the study protocol

• Minimum Eligible Age: 18 Years
• Eligible Sex: ALL
• Accepts Healthy Volunteers: No

Sponsors

The University of Hong Kong

OTHER

Sponsor Role: lead

Responsible Party

Responsibility Role: SPONSOR

Principal Investigators

Harinder Gill, MD

Role: PRINCIPAL_INVESTIGATOR

The University of Hong Kong

Locations

Department of Medicine, Queen Mary Hospital

Hong Kong, , Hong Kong

Site Status: RECRUITING

Countries

Hong Kong

Central Contacts

Hainder Gill, MD

Role: CONTACT

gillhsh@hku.hk

852 22555859

Facility Contacts

Harinder Gill, MD

Role: primary

gillhsh@hku.hk

852 22555859

Other Identifiers

P1101RUXMF

Identifier Type: -

Identifier Source: org_study_id