Ropeginterferon Alfa-2b for the Treatment of Myelodysplastic Syndrome/Myeloproliferative Neoplasm Overlap Syndromes and Chronic Myelomonocytic Leukemia

NCT07468916 | Not Yet Recruiting Phase 2 DMC FDA Drug

• 2 other identifiers: 25-2633NCI-2026-01103
• Study Type: interventional
• Target: 35
• Locations: 1 country
• Sites: 1

Brief Summary

This phase II trial tests the safety, best dose, and effectiveness of ropeginterferon alfa-2b for the treatment of patients with myelodysplastic syndrome/myeloproliferative neoplasm overlap syndromes and chronic myelomonocytic leukemia. Ropeginterferon alfa-2b is a form of interferon. Interferons are a type of signaling protein normally produced by the body as part of the immune response. Interferons interfere with the division of cancer cells and can slow cancer cell growth. Ropeginterferon alfa-2b is a long-acting form of a type of interferon called interferon alfa-2b. In the body, ropeginterferon alfa-2b causes the production of proteins that modulate the immune system and have anticancer effects.

Conditions

Atypical Chronic Myeloid Leukemia; Chronic Myelomonocytic Leukemia; Myelodysplastic/Myeloproliferative Neoplasm With Ring Sideroblasts and Thrombocytosis, Not Otherwise Specified; Myelodysplastic/Myeloproliferative Neoplasm, Not Otherwise Specified; Myelodysplastic/Myeloproliferative Neoplasm

Outcome Measures

Primary Outcomes (2)

• Overall response

  Will be assessed per the 2015 international consortium proposal (ICP) myelodysplastic syndrome (MDS)/myeloproliferative neoplasm (MPN) criteria. Overall response rate is defined as the best objective 2015 ICP MDS/MPN response achieved at any time on study (complete remission, complete cytogenetic remission, partial remission, marrow response or clinical benefit). Will be summarized as proportions with corresponding 95% exact binomial confidence intervals.

  Up to 24 months

• Incidence of adverse events

  The incidence of adverse events, both hematological and non-hematological will be assessed by National Cancer Institute Common Terminology Criteria for Adverse Events version 5.0. Will be summarized as proportions with corresponding 95% exact binomial confidence intervals.

  Up to 24 months

Secondary Outcomes (14)

• Cytogenetics

  At baseline

• Mutation profile

  At baseline

• Chronic myelomonocytic leukemia-specific prognostic scoring system - molecular risk

  At baseline

• Clinical benefit

  Every 3 cycles (cycle length = 28 days)

• Progression-free survival

  From initiation of treatment to disease progression or death, assessed up to 24 months

Study Arms (1)

Treatment (ropeginterferon alfa-2b)

EXPERIMENTAL

Patients receive ropeginterferon alfa-2b SC on days 1 and 15 of each cycle. Cycles repeat every 28 days for up to 24 months (26 cycles) in the absence of disease progression or unacceptable toxicity. Patients also undergo bone marrow aspiration and biopsy, CT, and collection of blood samples throughout the trial.

Procedure: Biospecimen Collection; Procedure: Bone Marrow Aspiration; Procedure: Bone Marrow Biopsy; Procedure: Computed Tomography; Other: Questionnaire Administration; Biological: Ropeginterferon Alfa-2B

Interventions

Bone Marrow Biopsy PROCEDURE

Undergo bone marrow biopsy

Also known as: Biopsy of Bone Marrow, Biopsy, Bone Marrow

Treatment (ropeginterferon alfa-2b)

Computed Tomography PROCEDURE

Undergo CT

Also known as: CAT, CAT Scan, Computed Axial Tomography, Computerized Axial Tomography, Computerized axial tomography (procedure), Computerized Tomography, Computerized Tomography (CT) scan, CT, CT Scan, Diagnostic CAT Scan, Diagnostic CAT Scan Service Type, tomography

Treatment (ropeginterferon alfa-2b)

Questionnaire Administration OTHER

Ancillary studies

Treatment (ropeginterferon alfa-2b)

Biospecimen Collection PROCEDURE

Undergo collection of blood samples

Also known as: Biological Sample Collection, Biospecimen Collected, Specimen Collection

Treatment (ropeginterferon alfa-2b)

Bone Marrow Aspiration PROCEDURE

Undergo bone marrow aspiration

Treatment (ropeginterferon alfa-2b)

Ropeginterferon Alfa-2B BIOLOGICAL

Given SC

Also known as: AOP 2014, AOP-2014, AOP2014, Besremi, P-1101, P1101, PEG-P-IFN-Alfa-2b, PEG-P-IFN-Alpha-2b, PEG-Proline-Interferon Alfa-2b

Treatment (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:

• Male or female ≥ 18 years of age at time of consent
• Documentation of a diagnosis of MDS/MPN overlap syndrome based on World Health Organization (WHO) 2022 classification, including CMML, MDS/MPN with neutrophilia, myelodysplastic/myeloproliferative neoplasm with ring sideroblasts and thrombocytosis (MDS/MPN-RS-T), or MDS/MPN, not otherwise specified, by local pathology review, and deemed to potentially benefit from study participation by the investigator
• Written informed consent obtained from participant or participant's legal representative and ability for participant to comply with the requirements of the study
• Blast =\< 10% by marrow immunohistochemistry stain
• Platelet count of \> 50,000/uL
• Absolute neutrophils count (ANC) of \> 1000/uL
• Eastern Cooperative Oncology Group (ECOG) performance status (PS) =\< 2
• Serum creatinine =\< 2.5 mg/dL
• Serum direct bilirubin \< 2.0 mg/dL
• Serum transaminase \< 2.5 times the upper limit of the normal range (ULN) or \< 5 times ULN if the transaminase elevation was deemed related to the MDS/MPN

You may not qualify if:

• Prior therapy with interferon or pegylated interferon product, or azacitidine
• Spleen overtly enlarged by physical exam (eg. greater than 5 fingerbreadth below costal margin)
• Other standard (including erythropoietin-stimulating agents \[ESA\] or luspatercept) or experimental therapy for MDS/MPN within 28 days of starting study therapy with the exception of hydroxyurea, which is allowed to continue up to 28 days after cycle 1 day 1 (C1D1) while on protocol
• Clinically significant autoimmune disease by investigator assessment, regardless if the autoimmune phenomena is related to MDS/MPN overlap syndrome
• History of or current clinically relevant depression or anxiety per investigator's judgement. Previous suicidal ideation or attempts are not allowed to participate in interferon (IFN) therapy
• Evidence of severe retinopathy or clinically relevant ophthalmological disorder
• History of organ transplant
• Pregnant or breastfeeding women
• Active uncontrolled infection with clinical symptoms, e.g., presence of bacteria, fungal, human immunodeficiency virus (HIV), hepatitis B or C
• Active uncontrolled thromboembolic complications or hemorrhage
• History of any malignancy within 5 years (except adequately treated non-melanoma skin cancer, prostate cancer status post resection with an undetectable prostate-specific antigen \[PSA\], curative treated in-situ cancer of the cervix, ductal carcinoma in situ \[DCIS\] of the breast, stage 1 grade 1 endometrial carcinoma, or other solid tumors including lymphomas curatively treated with no evidence of disease for ≥ 1 year prior to study)
• Uncontrolled active clinically significant illness that, in the investigator's opinion, may affect the patient's participation in this study
• Active abuse of alcohol and/or illicit drugs

Sponsors & Collaborators

• Jonsson Comprehensive Cancer Center: lead
• PharmaEssentia: collaborator

Study Sites (1)

UCLA / Jonsson Comprehensive Cancer Center

Los Angeles, California, 90095, United States

Location

MeSH Terms

Conditions

Leukemia, Myeloid, Chronic, Atypical, BCR-ABL Negative; Leukemia, Myelomonocytic, Chronic; Myelodysplastic-Myeloproliferative Diseases; Thrombocytosis; Myeloproliferative Disorders

Interventions

Specimen Handling; Biopsy; peginterferon alfa-2b

Condition Hierarchy (Ancestors)

Leukemia, Myeloid; Leukemia; Neoplasms by Histologic Type; Neoplasms; Bone Marrow Diseases; Hematologic Diseases; Hemic and Lymphatic Diseases; Chronic Disease; Disease Attributes; Pathologic Processes; Pathological Conditions, Signs and Symptoms; Blood Platelet Disorders

Intervention Hierarchy (Ancestors)

Clinical Laboratory Techniques; Diagnostic Techniques and Procedures; Diagnosis; Investigative Techniques; Cytodiagnosis; Cytological Techniques; Diagnostic Techniques, Surgical; Surgical Procedures, Operative

Study Officials

• Wanxing Chai-Ho, MD

  UCLA / Jonsson Comprehensive Cancer Center

  PRINCIPAL INVESTIGATOR

Central Study Contacts

Vladimir Kustanovich

CONTACT

310-206-5756; VKustanovich@mednet.ucla.edu

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: March 9, 2026
• First Posted: March 13, 2026
• Study Start (Estimated): September 29, 2026
• Primary Completion (Estimated): September 30, 2031
• Study Completion (Estimated): September 30, 2032
• Last Updated: May 6, 2026

Record last verified: 2026-03

Locations

US (1)