This is an open-label, multicenter study exploring the efficacy of ivosidenib in patients with clonal cytopenia of undetermined significance (CCUS) with mutations in IDH1. The purpose is to establish proof of principle that ivosidenib is well-tolerated and potentially efficacious in improving blood count abnormalities in these patients. The study will also be offered in a decentralized, remote structure to patients.
Study Type
INTERVENTIONAL
Allocation
NA
Purpose
TREATMENT
Masking
NONE
Enrollment
20
. Patients should take ivosidenib at approximately the same time every day, with or without food, but should be instructed to avoid a high-fat meal as well as grapefruit and grapefruit products.
Washington University School of Medicine
St Louis, Missouri, United States
Cleveland Clinic - Case Comprehensive Cancer Center
Cleveland, Ohio, United States
Rate of improvement in hematologic parameters
Will be evaluated according to a modified version of the IWG 2006 Criteria for Hematologic Improvement for patients with MDS on clinical trials * Erythroid response (pretreatment, \<11 g/DL) * Hemoglobin (Hgb) increase by ≥1.5 g/dL * Relevant reduction of units of RBC transfusions by an absolute number of at least 4 RBC transfusions/8 weeks, compared with the pretreatment transfusion number in the previous 8 weeks. Only RBC transfusions given for an Hgb of ≤9.0 g/dL pretreatment will count in the RBC transfusion response evaluation * Platelet response (pretreatment, \<100 x 10\^9/L) * Absolute increase of ≥30 × 10\^9/L for patients starting with \>20 × 10\^9/L platelets. * Increase from \<20 × 10\^9/L to \>20 × 10\^9/L and by at least 100%. * Neutrophil response (pretreatment, \<1.0 x 10\^9/L): * At least 100% increase and an absolute increase \>0.5 × 10\^9/L. If pegfilgrastim being used prior to initiation of study, define response as no longer requiring pegfilgrastim to maintain ANC \>500.
Time frame: Through 30 days after completion of treatment (estimated to be 61 months)
Change in mutant IDH1 variant allele fraction
-ddPCR is a highly sensitive and accurate method for quantifying VAF. This will be performed centrally at the Washington University clinical pathology laboratory using standard procedures. To account for assay variation, the investigators will perform 10 runs using the ddPCR assay and take the mean VAF as the final measurement.
Time frame: Through completion of treatment (estimated to be 60 months)
Disease free survival
-Events include development of MDS/AML or death.
Time frame: Through 30 days after completion of treatment (estimated to be 61 months)
Number of adverse events as measured by CTCAE v 5.0
Time frame: Through 30 days after completion of treatment (estimated to be 61 months)
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Duration of hematologic response
Time frame: Through completion of treatment (estimated to be 60 months)