Primary Myelofibrosis (PMF)Phase 2NCT06770842
Ropeginterferon Alfa 2b Plus Ruxolitinib for Myelofibrosis
A Phase 2 study testing Ropeginterferon alfa-2b (BESREMi®) in people with Primary Myelofibrosis (PMF) and Post Polycythemia Myelofibrosis (PPV MF), run by The University of Hong Kong at 1 site in Hong Kong.
Trial 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. Read more about this study
- Who is it for?
- Ages 18 years and older
- Conditions the study is for
- Primary Myelofibrosis (PMF), Post Polycythemia Myelofibrosis (PPV MF), Post Essential Thrombocythaemia Myelofibrosis (PET-MF)
- What is being tested?
- Ropeginterferon alfa-2b (BESREMi®)
- Type of treatment
- Pill or oral medicine, Injection or infusion
- Could I receive a placebo?
- No, everyone receives an active treatment
- How many people
- 20 participants
- Where
- 1 siteHong Kong
- Study dates
- 1 Mar 2025to Dec 2027
- Phase
- Phase 2
Key requirements
Who can take part, as listed by the sponsor. Only the study team can confirm whether you are eligible, so take this list to your doctor.
- Ages 18 years and older
- All sexes
- No healthy volunteers
- Adult, Older adult
Who may be able to join
- 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
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- 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:
- 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.
Who cannot join
- Subjects will not be eligible for participation if they meet any of the following 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
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- 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
Trial locations and contacts
1 site in 1 country. Contact the study team or a site near you to ask about taking part.
Hong Kong1 site, 1 recruiting
- Recruiting
Department of Medicine, Queen Mary Hospital
Hong Kong
Harinder Gill, MD, 852 22555859, gillhsh@hku.hk
Harinder Gill, MD
About this study
Official title: Safety and Efficacy of Ropeginterferon Alfa-2b in Combination With Ruxolitinib in Patients With Myelofibrosis Demonstrating Suboptimal Response to Ruxolitinib Monotherapy
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.
Read the 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)
- Being tested
- Ropeginterferon alfa-2b (BESREMi®)
- Keywords
- Ruxolitinib, Ropeginterferon alfa 2b, Myelofibrosis, Ruxolitinib failiure
- Medical subject headings
- Primary Myelofibrosis
What participants receive
What participants receive. In a randomised study each person is assigned to one group by chance.
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
| Study group | Role | What they receive |
|---|---|---|
| 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. Receives: Drug: Ropeginterferon alfa-2b (BESREMi®) |
What is being measured
The primary outcome is the main question the study is designed to answer.
Adverse events
Enumeration and description of adverse events (AEs), including determination of dose limiting toxicities (DLTs), serious adverse events (SAEs), and other AEs
Measured: 24 months
Secondary outcomes (2)
Spleen volume change at 24 weeks
Proportion of patients who experience a spleen length reduction by palpation of ≥30% OR spleen volume reduction (SVR) of ≥35% by MRI or CT by 24 weeks of treatment
Measured: 24 weeks
Symptom response at 24 weeks
Proportion of patients who describe a ≥50% reduction in symptom burden by the myelofibrosis symptom assessment form (MFSAF) by 24 weeks of treatment. The MFSAF has a scale of 0-70 where a higher score indicates worse outcome.
Measured: 24 weeks
Timeline
From the start of the study to its completion; the mark is today. Estimated dates are the sponsor’s plan.
- First posted13 Jan 2025
- Study start1 Mar 2025
- Primary completionDec 2026 (estimated)
- Study completionDec 2027 (estimated)
Resources
- Official record on ClinicalTrials.govThe registry entry this page is built from, with its full change history.
- Other trials for Primary Myelofibrosis (PMF)Studies of the same condition, open and under way.
- More trials run by The University of Hong KongThe sponsor's other registered studies.
- What the trial phases meanHow a treatment moves from first tests in people to approval.
More details
For doctors and researchers: how the study is designed, who runs it, and its registry IDs.
Study design
- Study type
- Interventional
- Phase
- Phase 2
- Primary purpose
- Treatment
- Allocation
- Not applicable
- Intervention model
- Single group
- Masking
- None (open label)
- Enrolment
- 20 participants (estimated)
Sponsor and investigators
- Lead sponsor
- The University of Hong Kong
- Sponsor type
- University, hospital or other
- Responsible party
- Sponsor
- Investigators
- Harinder Gill, MD, Principal investigator, The University of Hong Kong
IDs, dates and publications
- ClinicalTrials.gov ID
- NCT06770842
- Sponsor's protocol ID
- P1101RUXMF
- Status last verified
- Dec 2024
- First submitted
- 20 Dec 2024
- Last updated
- 11 Jun 2025
- Results posted
- No
- Data monitoring committee
- No
- FDA-regulated drug
- No
- FDA-regulated device
- No
- Individual data sharing
- No