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

ConditionsAtypical Chronic Myeloid LeukemiaChronic Myelomonocytic LeukemiaMyelodysplastic/Myeloproliferative NeoplasmMyelodysplastic/Myeloproliferative Neoplasm With Ring Sideroblasts and Thrombocytosis, Not Otherwise SpecifiedMyelodysplastic/Myeloproliferative Neoplasm, Not Otherwise Specified
Trial statusNot yet recruiting
Trial phasePhase 2
Trial typeInterventional
Biological sexAll
Age18+
SponsorJonsson Comprehensive Cancer Center

About this trial

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.

Eligibility criteria

This trial does not accept healthy volunteers

Qualifiers

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

Disqualifiers

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

Trial design

Design model

Single group

Treatments tested in this trial

  • Biospecimen Collection

    Procedure/Surgery

    Undergo collection of blood samples

  • Bone Marrow Aspiration

    Procedure/Surgery

    Undergo bone marrow aspiration

  • Bone Marrow Biopsy

    Procedure/Surgery

    Undergo bone marrow biopsy

  • Computed Tomography

    Procedure/Surgery

    Undergo CT

  • Questionnaire Administration

    Other intervention

    Ancillary studies

  • Ropeginterferon Alfa-2B

    Biological/Vaccine

    Given SC

Treatment groups

35 Participants
are divided into 1 treatment group
Group A: Treatment (ropeginterferon alfa-2b)Experimental treatment 6 interventions

Trial outcomes

Primary outcomes

1

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.

Time frame
Up to 24 months
2

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.

Time frame
Up to 24 months

Secondary outcomes

1

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.

Time frame
At baseline
2

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.

Time frame
At baseline
3

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.

Time frame
At baseline
4

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.

Time frame
Every 3 cycles (cycle length = 28 days)

Other outcomes

Sponsors and contacts

Click on the lead sponsor to view all of their trials.

Jonsson Comprehensive Cancer Center

Lead sponsor

PharmaEssentia

Collaborator

Source ClinicalTrials.gov

This trial is not recruiting at the moment. You can still explore other options: