Finding studies
Finding studies
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Vladimir Kustanovich
CONTACT
Lead
Jonsson Comprehensive Cancer Center
With
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.
Age
18–any
Sex
ALL
Healthy volunteers
Not accepted
