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| ID | Type | Description | Link |
|---|---|---|---|
| NCI-2026-01103 | Registry Identifier | CTRP (Clinical Trial Reporting Program) |
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| Name | Class |
|---|---|
| PharmaEssentia | INDUSTRY |
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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.
PRIMARY OBJECTIVE:
I. 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:
I. 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.
V. 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.
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| Label | Type | Description | Intervention Names |
|---|---|---|---|
| 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. |
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| Name | Type | Description | Arm Group Labels | Other Names |
|---|---|---|---|---|
| Biospecimen Collection | Procedure | Undergo collection of blood samples |
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| Measure | Description | Time Frame |
|---|---|---|
| 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 |
| Measure | Description | Time Frame |
|---|---|---|
| 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. | At baseline |
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Inclusion Criteria:
Exclusion Criteria:
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| Name | Role | Phone | Extension | |
|---|---|---|---|---|
| Vladimir Kustanovich | Contact | 310-206-5756 | VKustanovich@mednet.ucla.edu |
| Name | Affiliation | Role |
|---|---|---|
| Wanxing Chai-Ho, MD | UCLA / Jonsson Comprehensive Cancer Center | Principal Investigator |
| Facility | Status | City | State | ZIP | Country | Contacts |
|---|---|---|---|---|---|---|
| UCLA / Jonsson Comprehensive Cancer Center | Los Angeles | California | 90095 | United States |
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| Bone Marrow Aspiration | Procedure | Undergo bone marrow aspiration |
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| Bone Marrow Biopsy | Procedure | Undergo bone marrow biopsy |
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| Computed Tomography | Procedure | Undergo CT |
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| Questionnaire Administration | Other | Ancillary studies |
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| Ropeginterferon Alfa-2B | Biological | Given SC |
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| 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. |
| 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. | 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. | 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. | 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. | 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. | 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. | 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. | 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. | 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. | 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. | 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. | 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. | From baseline to weeks 25 and 49 |
| ID | Term |
|---|---|
| D054438 | Leukemia, Myeloid, Chronic, Atypical, BCR-ABL Negative |
| D015477 | Leukemia, Myelomonocytic, Chronic |
| D054437 | Myelodysplastic-Myeloproliferative Diseases |
| D013922 | Thrombocytosis |
| D009196 | Myeloproliferative Disorders |
| ID | Term |
|---|---|
| D007951 | Leukemia, Myeloid |
| D007938 | Leukemia |
| D009370 | Neoplasms by Histologic Type |
| D009369 | Neoplasms |
| D001855 | Bone Marrow Diseases |
| D006402 | Hematologic Diseases |
| D006425 | Hemic and Lymphatic Diseases |
| D002908 | Chronic Disease |
| D020969 | Disease Attributes |
| D010335 | Pathologic Processes |
| D013568 | Pathological Conditions, Signs and Symptoms |
| D001791 | Blood Platelet Disorders |
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| ID | Term |
|---|---|
| D013048 | Specimen Handling |
| D001706 | Biopsy |
| C417083 | peginterferon alfa-2b |
| ID | Term |
|---|---|
| D019411 | Clinical Laboratory Techniques |
| D019937 | Diagnostic Techniques and Procedures |
| D003933 | Diagnosis |
| D008919 | Investigative Techniques |
| D003581 | Cytodiagnosis |
| D003584 | Cytological Techniques |
| D003949 | Diagnostic Techniques, Surgical |
| D013514 | Surgical Procedures, Operative |
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