Ropeginterferon alfa-2b added to ruxolitinib for people with myelofibrosis

Is this trial a fit for you?

A plain-English reading of the study's rules. Only the study team can confirm whether you qualify.

What this trial studies

This open-label, single-arm Phase 2 study will enroll about 20 adults with primary or post‑MPN myelofibrosis who show a suboptimal response to ruxolitinib. Participants will remain on a stable oral ruxolitinib dose and receive subcutaneous ropeginterferon alfa-2b every two weeks. The initial treatment period comprises six cycles with a qualification assessment, and patients judged to derive clinical benefit may continue on additional iterative six-cycle periods at the treating physician’s discretion. Key entry criteria include DIPSS intermediate‑1 to high risk, ECOG 0–2, platelets ≥75 x 10^9/L, ANC ≥0.5 x 10^9/L, and peripheral blasts ≤10%.

Why it matters

Who can join: the full rules

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:

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
* 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

Where it's running

• Department of Medicine, Queen Mary Hospital
  Hong Kong, Hong Kong Recruiting

Study contacts

• Principal investigator: Harinder Gill, MD — The University of Hong Kong
• Study coordinator: Hainder Gill, MD
• Email: gillhsh@hku.hk
• Phone: 852 22555859

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Other studies for the same condition or treatment. Recruiting studies are listed first.

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How to take part

1. Review the eligibility criteria above with your treating physician.
2. Check the official listing on ClinicalTrials.gov for the latest contact details and recruitment status.
3. Contact the study coordinator or principal investigator to ask for pre-screening. Pre-screening is free and never obligates you to enroll.