Luspatercept for Clonal Cytopenia

Overseen By Ameenah Sukkur, BA

Age: 18+

Sex: Any

Travel: May Be Covered

Time Reimbursement: Varies

Trial Phase: Phase 2

Waitlist Available

Sponsor: Weill Medical College of Cornell University

No Placebo Group

Prior Safety Data

Trial Summary

What is the purpose of this trial?

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.

Research Team

Pinkal Desai, MD

Principal Investigator

Weill Medical College of Cornell University

Eligibility Criteria

This trial is for people with clonal cytopenias of uncertain significance (CCUS), which means they have low counts of red cells, platelets, or white cells without a clear reason. Participants should not have progressed to more severe blood disorders and must be able to attend clinic visits every three weeks.

Inclusion Criteria

I am 18 years old or older.

I can have children, have had two negative pregnancy tests, and will use effective birth control or abstain from sex.

Documentation of a CCUS diagnosis

Exclusion Criteria

I am currently receiving treatment for another cancer.

I haven't used any blood cell growth boosters in the last 4 weeks.

I am at risk for blood clots.

Treatment Details

Interventions

• Luspatercept (Growth Factor)

Trial Overview The study tests luspatercept injections given every three weeks to see if it improves blood counts in CCUS patients. It measures the number of patients who improve within 24 weeks, duration of improvement, time until disease progression, changes in hemoglobin levels, transfusion needs, and overall well-being.

Participant Groups

Group I: Luspatercept Experimental Treatment 1 Intervention

Luspatercept administered at 1 mg/kg IV once every 3 weeks

Who Is Running the Clinical Trial?

Weill Medical College of Cornell University

Lead Sponsor

Trials

1,103

Recruited

1,157,000+

Bristol-Myers Squibb

Industry Sponsor

Trials

2,731

Recruited

4,127,000+

Headquarters

New York City, USA

Known For

Oncology & Cardiovascular

Top Products

Eliquis, Opdivo, Revlimid, Orencia

Christopher Boerner

Bristol-Myers Squibb

Chief Executive Officer since 2023

PhD in Business Administration from the Haas School of Business, University of California, Berkeley; BA in Economics and History from Washington University in St. Louis

Deepak L. Bhatt

Bristol-Myers Squibb

Chief Medical Officer since 2024

MD from Yale University; MSc in Clinical Epidemiology from the University of Pennsylvania