Actively Recruiting
Efficacy of Luspatercept in Clonal Cytopenias of Uncertain Significance
Led by Weill Medical College of Cornell University · Updated on 2026-03-24
50
Participants Needed
1
Research Sites
52 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
Researchers are evaluating how well the drug luspatercept improves low blood cell counts in people with clonal cytopenias of uncertain significance (CCUS). This phase 2 clinical trial aims to answer key questions about the number of patients who experience blood count improvements within 24 weeks, the duration of these improvements, and the time until disease progression to more severe conditions such as myeloid disorders. The study also monitors changes in hemoglobin levels, transfusion needs, quality of life, and potential side effects including progression to leukemia or heart-related events. Participants will receive luspatercept injections at a dose of 1 mg/kg intravenously every three weeks. Treatment and monitoring visits also occur every three weeks. The study tracks responses at multiple time points including 12, 24, and 48 weeks, with assessments of blood counts, transfusion dependence, and quality of life scores. Safety is closely monitored throughout the treatment period. During the study, participants will visit the clinic every three weeks for their injections and evaluations. Researchers will perform blood tests to measure various blood cell levels, hemoglobin, and cardiac biomarkers. Participants will complete questionnaires to assess their well-being and energy levels. The main outcome is the number of patients achieving hematologic improvement by 24 weeks, with additional measures of response duration and safety events. The study is expected to continue until February 2028.
CONDITIONS
Brief Title
Luspatercept for Clonal Cytopenias of Uncertain Significance
Who Can Participate
Eligibility Criteria
You may qualify if you...
- Male or female 18 years of age or older
- Diagnosis of clonal cytopenia of undetermined significance (CCUS) with specific blood count criteria
- Presence of somatic mutations in myeloid malignancy-associated genes at defined levels
- Clinically significant cytopenias confirmed in two lab tests including anemia, thrombocytopenia, or neutropenia with specific thresholds
- Eastern Cooperative Oncology Group (ECOG) performance status of 0, 1, or 2
- Adequate organ function defined by bilirubin, creatinine clearance or GFR, and liver enzymes
- Females of childbearing potential must have negative pregnancy tests and agree to effective contraception or abstinence
- Males must agree to abstinence or use condoms during study and for 12 weeks after stopping treatment
You will not qualify if you...
- Active malignancy requiring systemic chemotherapy (except hormonal or targeted radiation therapy)
- Diagnosis of myelodysplastic syndrome (MDS), acute myeloid leukemia (AML), myeloproliferative neoplasm (MPN), or other myeloid malignancy
- Active uncontrolled infection
- Uncontrolled hypertension not responsive to medication
- Use of erythropoiesis-stimulating agents or growth factors within 4 weeks prior to study start
- Known risk factors for blood clots, such as recent pulmonary embolism or deep vein thrombosis, unless well-controlled with anticoagulation
- Pregnant or nursing women or women not using adequate contraception during and after treatment
- Women considered post-menopausal only after 12 months of natural amenorrhea or surgical procedures as defined in criteria
AI-Screening
AI-Powered Screening
Complete this quick 3-step screening to check your eligibility
Your Study Journey
Duration - 2 to 4 weeks
Participants are screened for eligibility to participate in the trial.
1 visit (in-person)
Duration - Up to 24 weeks
Participants receive Luspatercept administered intravenously at 1 mg/kg once every 3 weeks to treat clonal cytopenias of uncertain significance.
Approximately 8 visits (in-person) every 3 weeks
Trial Site Locations
Total: 1 location
1
Weill Cornell Medical College
New York, New York, United States, 10021
Actively Recruiting
Research Team
P
Pinkal Desai, MD
A
Ameenah Sukkur, BA
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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