Combining pegylated interferon α-2b with ruxolitinib for treating resistant polycythemia vera

A Randomized Controlled Study Evaluating the Efficacy and Safety of Pegylated Interferon α-2b in Combination With Ruxolitinib vs. Pegylated Interferon α-2b Monotherapy for Treating Hydroxyurea-resistant/Intolerant Polycythemia Vera

This study is testing if combining two medications, pegylated interferon α-2b and ruxolitinib, can help people with polycythemia vera who haven't responded well to other treatments feel better.

Quick facts

• Phase: PHASE2
• Study type: Interventional
• Enrollment: 94 (estimated)
• Ages: 18 Years and up
• Sex: All
• Sponsor: Institute of Hematology & Blood Diseases Hospital, China (other)
• Drugs / interventions: ruxolitinib
• Locations: 1 site (Tianjin)
• Trial ID: NCT05870475 on ClinicalTrials.gov

What this trial studies

This clinical trial aims to evaluate the efficacy and safety of pegylated interferon α-2b in combination with ruxolitinib compared to pegylated interferon α-2b alone in patients with polycythemia vera who are resistant or intolerant to hydroxyurea. Participants will be randomly assigned to receive either the combination treatment or pegylated interferon α-2b alone, with the option to switch treatments if necessary. The study will monitor hematologic responses and side effects over a specified treatment period.

Who should consider this trial

Good fit: Ideal candidates are adults aged 18 and older diagnosed with polycythemia vera who are resistant or intolerant to hydroxyurea.

Not a fit: Patients with symptomatic splenomegaly or significant comorbidities that affect their ability to participate may not benefit from this study.

Why it matters

Potential benefit: If successful, this treatment approach could provide a new effective option for patients with hydroxyurea-resistant or intolerant polycythemia vera.

How similar studies have performed: While the combination of these treatments has not been extensively tested together, previous studies have shown promise for each treatment individually in managing polycythemia vera.

Eligibility criteria

Inclusion Criteria:

* ≥18 years old.
* Male or Female.
* Meets the diagnostic criteria for Polycythemia Vera according to WHO-2022.
* Resistant or intolerant to hydroxyurea (based on the 2013 European LeukemiaNet criteria).
* Have not previously received interferon preparations or ruxolitinib treatment, or the washout period between the last use of interferon preparations or ruxolitinib and the first use of the study drug should not be less than 4 weeks.
* Patients with indications for cytoreductive therapy.
* During screening, female hemoglobin (HGB) ≥10g/dL, male hemoglobin (HGB) ≥11g/dL; neutrophil count ≥1.5×109/L; platelet count ≥100×109/L.
* Voluntary written informed consent.

Exclusion Criteria:

* Symptomatic splenomegaly;
* Contraindications to interferon or ruxolitinib therapy;
* Severe or significant comorbidities that may affect the participant's ability to participate in the study, as determined by the investigator;
* History of major organ transplantation;
* Pregnant or breastfeeding women;
* History or current diagnosis of autoimmune thyroid disease (patients with controlled hypothyroidism on oral thyroid hormone replacement therapy may be included);
* Documented evidence of any other autoimmune disease (such as active hepatitis, systemic lupus erythematosus, antiphospholipid antibody syndrome, or autoimmune arthritis);
* Clinically significant bacterial, fungal, mycobacterial, parasitic, or viral infection such as active hepatitis or HIV infection (patients with acute bacterial infections requiring antibiotic treatment should be deferred from screening/enrollment until completion of antibiotic treatment);
* Evidence of severe retinopathy or clinically significant ophthalmologic disease (due to diabetes or hypertension);
* Current clinically significant depression or history of depression, or any suicidal attempt or tendency during screening;
* Active bleeding or thrombotic complications;
* History of any malignant tumor within the past 5 years (except for stage 0 chronic lymphocytic leukemia \[CLL\], cured basal cell carcinoma, squamous cell carcinoma, and superficial melanoma);
* History of alcohol or substance abuse within the past year;
* Presence of blasts in the peripheral blood within the past 3 months;
* Use of any investigational drug or participation in any other clinical trial within 4 weeks prior to the first dose of the study drug, or failure to recover from any effects of previously administered study drugs;
* The investigator deems the presence of any concurrent condition that may jeopardize the safety of the participant or the compliance to the protocol.

Where this trial is running

Tianjin

• Institute of Hematology & Blood Diseases Hospital — Tianjin, China (RECRUITING)

Study contacts

• Principal investigator: Lei Zhang, MD — Institute of Hematology & Blood Diseases Hospital, China
• Study coordinator: Lei Zhang, MD

Conditions: Polycythemia Vera