Atypical Chronic Myeloid LeukemiaPhase 2NCT07468916
Ropeginterferon Alfa-2b for the Treatment of Myelodysplastic Syndrome/Myeloproliferative Neoplasm Overlap Syndromes and Chronic Myelomonocytic Leukemia
A Phase 2 study testing Biospecimen Collection, Bone Marrow Aspiration and Bone Marrow Biopsy in people with Atypical Chronic Myeloid Leukemia and Chronic Myelomonocytic Leukemia, run by Jonsson Comprehensive Cancer Center at 1 site in United States.
Trial 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. Read more about this study
- Who is it for?
- Ages 18 years and older
- Conditions the study is for
- Atypical Chronic Myeloid Leukemia, Chronic Myelomonocytic Leukemia, Myelodysplastic/Myeloproliferative Neoplasm
- What is being tested?
- Biospecimen Collection, Bone Marrow Aspiration and Bone Marrow Biopsy
- Type of treatment
- Surgery or procedure
- Could I receive a placebo?
- No, everyone receives an active treatment
- How many people
- 35 participants
- Where
- 1 siteUnited States
- Study dates
- 29 Sep 2026to 30 Sep 2032
- 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
- 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
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- 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
Who cannot join
- 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
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- 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
Trial locations and contacts
1 site in 1 country. Contact the study team or a site near you to ask about taking part.
United States1 site
UCLA / Jonsson Comprehensive Cancer Center
Los Angeles, California, 90095
Vladimir Kustanovich, 310-206-5756, VKustanovich@mednet.ucla.edu
Wanxing Chai-Ho, MD
About this study
Official title: Ropeginterferon Alfa-2b for MDS/MPN Overlap Syndromes, Including CMML and MDS/MPN-RS-T
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.
Read the detailed description
PRIMARY OBJECTIVE:
- To assess the safety and efficacy (overall response, OR) of ropeginterferon alfa-2b in adult patients with myelodysplastic syndrome (MDS)/myeloproliferative neoplasm (MPN) overlap syndrome.
SECONDARY OBJECTIVES:
- To evaluate baseline cytogenetics, mutation profile, chronic myelomonocytic leukemia (CMML)-specific prognostic scoring system - molecular (CPSS-Mol) risk.
II. To assess the percentage of patient with hematological response based on 2015 international consortium proposal (ICP) MDS/MPN criteria.
III. Based on 2015 ICP MDS/MPN criteria, to assess time to complete response, time to disease progression (TTP), progression free survival (PFS), and event free survival (EFS).
IV. To assess the change from baseline in mutant allele frequencies (MAF), with special interests in ASXL1, SRSF2, NRAS, KRAS, SETBP1, RUNX1, CBL, EZH2, SF3B1 mutations; as also in non-driver mutations.
- To assess the percentage of splenomegaly changes on clinical exam and on computed tomography (CT).
VI. To assess changes in MPN symptom burden using the MPN Symptom Assessment Form (MPN-Symptom Assessment Form [SAF] total symptom score [TSS]).
VII. To assess changes in packed red blood cell (PRBC) transfusion burden. VIII. To assess changes in the bone marrow morphology and fibrosis (as assessed by reticulin staining).
IX. To assess the change of cytokine profile.
OUTLINE: This is a dose-escalation study followed by a dose-expansion study.
Patients receive ropeginterferon alfa-2b subcutaneously (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.
After completion of study treatment, patients are followed up at 28 days and then every 3 months for up to 24 months.
- Conditions
- Atypical Chronic Myeloid Leukemia, Chronic Myelomonocytic Leukemia, Myelodysplastic/Myeloproliferative Neoplasm, Myelodysplastic/Myeloproliferative Neoplasm With Ring Sideroblasts and Thrombocytosis, Not Otherwise Specified, Myelodysplastic/Myeloproliferative Neoplasm, Not Otherwise Specified
- Being tested
- Biospecimen Collection, Bone Marrow Aspiration, Bone Marrow Biopsy, Computed Tomography, Questionnaire Administration
- Medical subject headings
- Leukemia, Myeloid, Chronic, Atypical, BCR-ABL Negative, Leukemia, Myelomonocytic, Chronic, Myelodysplastic-Myeloproliferative Diseases, Thrombocytosis, Myeloproliferative Disorders
What participants receive
What participants receive. In a randomised study each person is assigned to one group by chance.
Biospecimen CollectionProcedure or surgery
Also called Biological Sample Collection, Biospecimen Collected, Specimen Collection
Undergo collection of blood samples
Bone Marrow AspirationProcedure or surgery
Undergo bone marrow aspiration
Bone Marrow BiopsyProcedure or surgery
Also called Biopsy of Bone Marrow, Biopsy, Bone Marrow
Undergo bone marrow biopsy
Computed TomographyProcedure or surgery
Also called CAT, CAT Scan, Computed Axial Tomography, Computerized Axial Tomography
Undergo CT
Questionnaire AdministrationOther
Ancillary studies
Ropeginterferon Alfa-2BBiological
Also called AOP 2014, AOP-2014, AOP2014, Besremi
Given SC
| Study group | Role | What they receive |
|---|---|---|
| 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. Receives: Procedure: Biospecimen Collection; Procedure: Bone Marrow Aspiration; Procedure: Bone Marrow Biopsy; Procedure: Computed Tomography; Other: Questionnaire Administration; Biological: Ropeginterferon Alfa-2B |
What is being measured
The primary outcome is the main question the study is designed to answer.
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.
Measured: 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.
Measured: Up to 24 months
Secondary outcomes (14)
Cytogenetics
Statistical analyses will primarily be descriptive. All other safety and efficacy endpoints will be summarized using appropriate descriptive statistics, including counts and proportions for categorical variables and means, medians, standard deviations, and ranges for continuous variables.
Measured: At baseline
Mutation profile
Statistical analyses will primarily be descriptive. All other safety and efficacy endpoints will be summarized using appropriate descriptive statistics, including counts and proportions for categorical variables and means, medians, standard deviations, and ranges for continuous variables.
Measured: At baseline
Chronic myelomonocytic leukemia-specific prognostic scoring system - molecular risk
Statistical analyses will primarily be descriptive. All other safety and efficacy endpoints will be summarized using appropriate descriptive statistics, including counts and proportions for categorical variables and means, medians, standard deviations, and ranges for continuous variables.
Measured: At baseline
Clinical benefit
Will be based on 2015 ICP MDS/MPN criteria. Statistical analyses will primarily be descriptive. All other safety and efficacy endpoints will be summarized using appropriate descriptive statistics, including counts and proportions for categorical variables and means, medians, standard deviations, and ranges for continuous variables.
Measured: Every 3 cycles (cycle length = 28 days)
Progression-free survival
Progression will be evaluated per 2015 ICP MDS/MPN criteria. Will be analyzed using the Kaplan-Meier method, with median times and associated 95% confidence intervals estimated using the Brookmeyer-Crowley method.
Measured: From initiation of treatment to disease progression or death, assessed up to 24 months
Overall survival
Will be analyzed using the Kaplan-Meier method, with median times and associated 95% confidence intervals estimated using the Brookmeyer-Crowley method.
Measured: From initiation of treatment to death, assessed up to 24 months
Change in mutant allele frequencies
Will evaluate the change from baseline mutant allele frequencies, with special interests in ASXL1, SRSF2, NRAS, KRAS, SETBP1, RUNX1, CBL, EZH2, SF3B1 mutations; as also in non-driver mutations. Statistical analyses will primarily be descriptive. All other safety and efficacy endpoints will be summarized using appropriate descriptive statistics, including counts and proportions for categorical variables and means, medians, standard deviations, and ranges for continuous variables.
Measured: From baseline to every 3rd cycle (cycle length = 28 days)
Percent change in splenomegaly
Will be evaluated by clinical exam and on computed tomography. Statistical analyses will primarily be descriptive. All other safety and efficacy endpoints will be summarized using appropriate descriptive statistics, including counts and proportions for categorical variables and means, medians, standard deviations, and ranges for continuous variables.
Measured: Every cycle (for clinical exam) and on day 1 of cycles 4 and 7 and at end of treatment (for computed tomography) (cycle length = 28 days)
Changes in myeloproliferative neoplasm symptom burden
Changes in symptom burden will be evaluated using the Myelofibrosis Symptom Assessment Form total symptom score. Statistical analyses will primarily be descriptive. All other safety and efficacy endpoints will be summarized using appropriate descriptive statistics, including counts and proportions for categorical variables and means, medians, standard deviations, and ranges for continuous variables.
Measured: From baseline to day 1 of cycles 1, 2, and 4, and then every 3 cycles (cycle length = 28 days)
Time to response
Will be analyzed using the Kaplan-Meier method, with median times and associated 95% confidence intervals estimated using the Brookmeyer-Crowley method.
Measured: Up to 24 months
Duration of response
Will be analyzed using the Kaplan-Meier method, with median times and associated 95% confidence intervals estimated using the Brookmeyer-Crowley method.
Measured: From date of response to date of first evidence of disease recurrence or treatment failure, assessed up to 24 months
Changes in packed red blood cell transfusion burden
Statistical analyses will primarily be descriptive. All other safety and efficacy endpoints will be summarized using appropriate descriptive statistics, including counts and proportions for categorical variables and means, medians, standard deviations, and ranges for continuous variables.
Measured: Up to 24 months
Changes in bone marrow morphology and fibrosis
Changes in bone marrow morphology will include cellularity and blast burden and fibrosis will be assessed by reticulin staining. Statistical analyses will primarily be descriptive. All other safety and efficacy endpoints will be summarized using appropriate descriptive statistics, including counts and proportions for categorical variables and means, medians, standard deviations, and ranges for continuous variables.
Measured: From baseline to day 1 of cycles 4 and 7 and at end of treatment (cycle length = 28 days)
Change in cytokine profile
Statistical analyses will primarily be descriptive. All other safety and efficacy endpoints will be summarized using appropriate descriptive statistics, including counts and proportions for categorical variables and means, medians, standard deviations, and ranges for continuous variables.
Measured: From baseline to weeks 25 and 49
Timeline
From the start of the study to its completion; the mark is today. Estimated dates are the sponsor’s plan.
- First posted13 Mar 2026
- Study start29 Sep 2026 (estimated)
- Primary completion30 Sep 2031 (estimated)
- Study completion30 Sep 2032 (estimated)
Resources
- Official record on ClinicalTrials.govThe registry entry this page is built from, with its full change history.
- Other trials for Atypical Chronic Myeloid LeukemiaStudies of the same condition, open and under way.
- 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
- 35 participants (estimated)
Sponsor and investigators
- Lead sponsor
- Jonsson Comprehensive Cancer Center
- Sponsor type
- University, hospital or other
- Collaborators
- PharmaEssentia
- Responsible party
- Sponsor
- Investigators
- Wanxing Chai-Ho, MD, Principal investigator, UCLA / Jonsson Comprehensive Cancer Center
IDs, dates and publications
- ClinicalTrials.gov ID
- NCT07468916
- Sponsor's protocol ID
- 25-2633
- Other IDs
- NCI-2026-01103
- Status last verified
- Mar 2026
- First submitted
- 9 Mar 2026
- Last updated
- 6 May 2026
- Results posted
- No
- Data monitoring committee
- Yes
- FDA-regulated drug
- Yes
- FDA-regulated device
- No