Actively Recruiting

Phase 2
Age: 18Years +
All Genders
NCT06788691

Luspatercept for Clonal Cytopenias of Uncertain Significance

Led by Weill Medical College of Cornell University · Updated on 2026-03-24

50

Participants Needed

1

Research Sites

149 weeks

Total Duration

On this page

Sponsors

W

Weill Medical College of Cornell University

Lead Sponsor

B

Bristol-Myers Squibb

Collaborating Sponsor

AI-Summary

What this Trial Is About

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

Official Title

Luspatercept for Clonal Cytopenias of Uncertain Significance

Who Can Participate

Age: 18Years +
All Genders

Eligibility Criteria

Eligible

You may qualify if you...

  • Male or female 18 years of age or older
  • Diagnosis of clonal cytopenia of undetermined significance (CCUS) confirmed by specific blood test criteria
  • Presence of clinically significant low blood counts confirmed in two separate lab tests including anemia, thrombocytopenia, or neutropenia under defined thresholds
  • ECOG performance status of 0, 1, or 2
  • Adequate organ function including bilirubin, kidney, and liver function within specified limits
  • Females of childbearing potential must have negative pregnancy tests before and during the study and agree to use effective contraception
  • Male participants must agree to use condoms or practice abstinence during the study and for 12 weeks after treatment
Not Eligible

You will not qualify if you...

  • Active cancers requiring chemotherapy other than hormonal therapy or targeted radiation
  • Previous diagnosis of myelodysplastic syndromes (MDS), acute myeloid leukemia (AML), myeloproliferative neoplasms (MPN), or other myeloid malignancies
  • Uncontrolled infections or uncontrolled high blood pressure deemed unsafe by the investigator
  • Use of erythropoiesis-stimulating agents or growth factors within four weeks prior to study start
  • Known risk factors for blood clots such as recent pulmonary embolism or deep vein thrombosis unless well controlled with medication
  • Pregnant or nursing women or women not using required contraception methods
  • Women who are not postmenopausal or have not had surgical sterilization as defined in the criteria

AI-Screening

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Trial Site Locations

Total: 1 location

1

Weill Cornell Medical College

New York, New York, United States, 10021

Actively Recruiting

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Research Team

P

Pinkal Desai, MD

CONTACT

A

Ameenah Sukkur, BA

CONTACT

How is the study designed?

Study Type

INTERVENTIONAL

Masking

NONE

Allocation

NA

Model

SINGLE_GROUP

Primary Purpose

TREATMENT

Number of Arms

1

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