Luspatercept for Clonal Cytopenias of Uncertain Significance (NCT06788691) | Clinical Trial Compass
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 range
18 Years
Sex
ALL
See this in plain English?
AI-rewrites the medical criteria so a patient or caregiver can understand them. Always confirm with the trial site.
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…
Questions worth asking your doctor
Bring these to your next appointment. They're a starting point for a shared conversation — not a sign you qualify or a recommendation to enrol.
1Based on my diagnosis and history, is this trial worth exploring for me — or is there a standard treatment we should try first?
2What does this trial's phase tell us about how much is already known about its safety and benefit?
3What would taking part actually involve for me — visits, tests, time, and travel?
4What are the known and possible risks or side effects I should weigh, and how would they be monitored?
5If this trial isn't the right fit, what other options or trials would you suggest I look into?
Generated to help you prepare — always confirm anything about your own eligibility and care with the study team and your doctor.
Questions for the trial coordinator
The trial coordinator is the person who runs the study day to day. These cover the practical side — logistics, costs, and what taking part would actually mean for your life. The study team confirms whether you meet the criteria; these are questions to ask, not a sign you qualify.
1What does taking part actually involve week to week — how many visits, where, and how long does each one take?
2What costs are covered by the study, and what might I have to pay for myself, including travel, parking, or time off work?
3What happens during screening, and what happens if the study team confirms I don't meet the criteria after those tests?
4Who pays for the scans, blood work, and other tests the trial requires — the study, my insurance, or me?
5How will being in the trial affect my regular care, and will my own doctor stay informed and involved?
6Can I leave the trial at any point if I change my mind, and what would happen to my care if I do?
A starting point for the conversation — always confirm anything about your own eligibility, costs, and care with the study team and your doctor.
What they're measuring
1
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