Ropeginterferon Alfa 2b Plus Ruxolitinib for Myelofibrosis

NCT06770842 | Recruiting Phase 2

• 1 other identifier: P1101RUXMF
• Study Type: interventional
• Target: 20
• Locations: 1 country
• Sites: 1

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.

Conditions

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

Keywords

Ruxolitinib; Ropeginterferon alfa 2b; Myelofibrosis; Ruxolitinib failiure

Outcome Measures

Primary Outcomes (1)

• Adverse events

  Enumeration and description of adverse events (AEs), including determination of dose limiting toxicities (DLTs), serious adverse events (SAEs), and other AEs

  24 months

Secondary Outcomes (2)

• Spleen volume change at 24 weeks

  24 weeks

• Symptom response at 24 weeks

  24 weeks

Other Outcomes (2)

• Rate of molecular responses

  24 months

• Rate of morphologic response

  24 months

Study Arms (1)

Ropeginterferon alfa 2b

EXPERIMENTAL

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.

Drug: Ropeginterferon alfa-2b (BESREMi®)

Interventions

Ropeginterferon alfa-2b (BESREMi®) 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

Ropeginterferon alfa 2b

Eligibility Criteria

• Age: 18 Years+
• Sex: all
• Healthy Volunteers: No
• Age Groups: Adult (18-64), Older Adult (65+)

You may qualify if:

• 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.

You may not qualify if:

• 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:

Sponsors & Collaborators

• The University of Hong Kong: lead

Study Sites (1)

Department of Medicine, Queen Mary Hospital

Hong Kong, Hong Kong

RECRUITING

MeSH Terms

Conditions

Primary Myelofibrosis

Condition Hierarchy (Ancestors)

Myeloproliferative Disorders; Bone Marrow Diseases; Hematologic Diseases; Hemic and Lymphatic Diseases

Study Officials

• Harinder Gill, MD

  The University of Hong Kong

  PRINCIPAL INVESTIGATOR

Central Study Contacts

Hainder Gill, MD

CONTACT

852 22555859; gillhsh@hku.hk

Study Design

• Study Type: interventional
• Phase: phase 2
• Allocation: NA
• Masking: NONE
• Purpose: TREATMENT
• Intervention Model: SINGLE GROUP
• Sponsor Type: OTHER
• Responsible Party: SPONSOR

Study Record Dates

• First Submitted: December 20, 2024
• First Posted: January 13, 2025
• Study Start: March 1, 2025
• Primary Completion (Estimated): December 1, 2026
• Study Completion (Estimated): December 1, 2027
• Last Updated: June 11, 2025

Record last verified: 2024-12

Data Sharing

• IPD Sharing: Will not share

Locations

HK (1)