NCT06788691

Luspatercept for Clonal Cytopenias of Uncertain Significance

Efficacy of Luspatercept In Clonal Cytopenias of Uncertain Significance

• Status: Recruiting
• Phase: Phase 2
• Study type: Interventional
• Enrollment: 50 (estimated)
• Sponsor: Weill Medical College of Cornell University · Academic / Other
• Sex: All
• Age: 18 Years
• Healthy volunteers: Not accepted

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.

Conditions

• CCUS Clonal Cytopenia of Undetermined Significance
• Anemia
• Leukopenia
• Thrombocytopenia
• Neutropenia

Interventions

Type	Name	Description
DRUG	Luspatercept	Administered at 1 mg/kg once every 3 weeks

Timeline

• Start date: 2025-03-25
• Primary completion: 2027-02-01
• Completion: 2028-02-01
• First posted: 2025-01-23
• Last updated: 2026-03-24

Locations

1 site across 1 country: United States

Regulatory

• FDA-regulated drug study