RecruitingPhase 2

Luspatercept for Clonal Cytopenias of Uncertain Significance

United States50 participantsStarted 2025-03-25

Plain-language summary

The purpose of this clinical trial is to test how well the drug luspatercept works in improving low blood cell counts in people with clonal cytopenias of uncertain significance (CCUS). The main questions the study seeks to answer include: * How many patients experience improvements in their low blood counts (red cells, platelets, or white cells) within 24 weeks, based on specific criteria for blood conditions like myelodysplastic syndromes (MDS)? * How long these improvements last before the condition worsens or changes. * The percentage of participants showing improvements at 12, 24, and 48 weeks. * How long it takes for the condition to progress to more severe diseases like myeloid disorders. * How long red blood cell responses last and how quickly these responses are seen. * The average change in hemoglobin levels over 24 weeks. * How many patients need blood transfusions during the study and how soon transfusions are required. * Changes in participants' well-being and energy levels based on a standardized questionnaire. * Monitoring for any side effects, including progression to MDS or leukemia, heart-related issues, or sudden increases in hemoglobin. Participants will: * Receive luspatercept as an injection every three weeks. * Visit the clinic every three weeks for treatment and monitoring.

Who can participate

Age range18 Years

SexALL

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Inclusion Criteria: * Male or female ≥ 18 years of age. * Documentation of a CCUS diagnosis. * Clonal cytopenia of undetermined significance (CCUS) is defined as clonal hematopoiesis of indeterminate potential (CHIP) detected in the presence of one or more persistent cytopenias that are otherwise unexplained by hematologic or non-hematologic conditions and that do not meet diagnostic criteria for defined myeloid neoplasms. Cytopenia definitions for diagnosis of CCUS include Hb \<13 g/dL in males and \<12 g/dL in females for anemia, absolute neutrophil count \<1.8 ×109/L for leukopenia, and platelets \<150 × 109/L for thrombocytopenia. * Patients should harbor somatic mutations of myeloid malignancy-associated genes detected in the blood or bone marrow at a variant allele fraction (VAF) of ≥ 2% (≥4% for X-linked gene mutations in males * Clinically significant cytopenias demonstrated in two separate lab draws and defined as cytopenia in any one of the following: * Anemia: Transfusion dependent (LTD or HTD for Hb \< 9 g/dL based on IWG 2018 criteria). Exception for higher threshold up to 10g/dL for documented moderate or severe angina pectoris, cardiac or pulmonary insufficiency, or ischemic neurologic diseases (per IWG 2018 consensus recommendation). * Anemia NTD: symptomatic NTD CCUS with Hb \<10 g/dl, symptomatic defined as moderate or worse on ≥ 1 Patient Global Impression of Severity (PGI-S) item (fatigue, shortness of breath, weakness, or dizziness) * Thrombo…

What they're measuring

Number of patients achieving cytopenia responses as defined by HI-E, HI-P, HI-N, mHI-E responses

Timeframe: 24 Weeks

Trial details

NCT IDNCT06788691

SponsorWeill Medical College of Cornell University

Sponsor typeOTHER

Study typeINTERVENTIONAL

Primary completion2027-02

Contact for this trial

Pinkal Desai, MD

646-962-2700 pid9006@med.cornell.edu