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New treatment option for CCUS Clonal Cytopenia of Undetermined Significance
Official title Luspatercept for Clonal Cytopenias of Uncertain Significance
ClinicalTrials.gov ID: NCT06788691
What this study is testing
What is Luspatercept?
Luspatercept is an investigational medicine, given as an injectable medicine, being studied as a potential treatment for ccus clonal cytopenia of undetermined significance.
Plain-language explanation of the investigational treatment - it is being studied and is not an approved or proven therapy. The study team can confirm the details.
- What it's testing
- 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)?
- Phase 2: a mid-size study of how well it works
- Time commitment: about 24 weeks
A plain-language read of the study's public ClinicalTrials.gov listing. The study team confirms the details.
Who can take part
Ages 18 and older
You may be able to join if
- 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...
- Patients should harbor somatic mutations of myeloid malignancy-associated genes detected in the blood or bone marrow at a variant allele fraction...
- Clinically significant cytopenias demonstrated in two separate lab draws and defined as cytopenia in any one of the following:
You likely can't join if
- Concurrent malignancy requiring active concurrent systemic chemotherapy. Hormonal therapy for malignancy and targeted radiation is allowed. If...
- Diagnosis of MDS, AML, MPN or any other myeloid malignancy in the patient's lifetime
- Active uncontrolled infection that in the investigators opinion will affect study procedures and/or results
- Active uncontrolled hypertension not responding to blood pressure lowering medications which in the investigator's opinion will be harmful for the...
- Use of ESA or growth factors within four weeks prior to the start of the study
- Known risk factors for thromboembolism (splenectomy, concomitant use of hormone replacement therapy or recent uncontrolled pulmonary embolism or DVT...
See the full eligibility criteria
Who can join
- 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...
- 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...
- 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)
- Thrombocytopenia: platelet count less than 30,000 /microL or \< 50,000/microL with documented bleeding events or high risk for bleeding, for example on blood thinners or drugs that inhibit platelet function for other...
- Neutropenia: Neutropenia below 750/microl are included in the study. For people with neutropenia between 750-1000/microl, people should have neutropenia AND a history of serious infection(s).
- Eastern Cooperative Oncology Group (ECOG) performance status 0, 1, or 2
- Adequate organ function as defined by:
- Direct bilirubin \< 3 x ULN. Indirect hyperbilirubinemia from hemolysis or Gilberts disease are not considered as impaired.
- Estimated Creatinine clearance or GFR \>30 ml/min by institutional standards (for example MDRD or Cockcroft Gault or measured by 24 hour urine clearance).
- ALT and AST \< 3 x ULN
- Females of childbearing potential (FCBP), defined as a sexually mature woman who: 1) has achieved menarche at some point, 2) not undergone a hysterectomy or bilateral oophorectomy or 3) has not been naturally...
- Have two negative pregnancy tests (serum or urine) as verified by the investigator prior to starting study therapy (unless the screening pregnancy test was done within 72 hours of W1D1). She must agree to ongoing...
- Either commit to true abstinence1 from heterosexual contact (which must be reviewed on a monthly basis and source documented) or agree to use, and be able to comply with, highly effective contraception2 without...
- True abstinence is acceptable when this is in line with the preferred and usual lifestyle of the subject. [Periodic abstinence (e.g., calendar, ovulation, symptothermal, post-ovulation methods) and withdrawal are not...
- Highly effective contraception is defined in this protocol as the following (information will also appear in the ICF): Hormonal contraception (for example, birth control pills, injection, implant, transdermal patch...
What rules you out
- Concurrent malignancy requiring active concurrent systemic chemotherapy. Hormonal therapy for malignancy and targeted radiation is allowed. If patients after enrollment, have a clinical need for chemotherapy after...
- Diagnosis of MDS, AML, MPN or any other myeloid malignancy in the patient's lifetime
- Active uncontrolled infection that in the investigators opinion will affect study procedures and/or results
- Active uncontrolled hypertension not responding to blood pressure lowering medications which in the investigator's opinion will be harmful for the patient.
- Use of ESA or growth factors within four weeks prior to the start of the study
- Known risk factors for thromboembolism (splenectomy, concomitant use of hormone replacement therapy or recent uncontrolled pulmonary embolism or DVT in the last 6 months). people adequately controlled on anticoagulation...
- Pregnant or nursing women, where pregnancy is defined as the state of a female after conception and until the termination of gestation, confirmed by a positive hCG laboratory test. Women of child-bearing potential...
- Women are considered post-menopausal and not of childbearing potential if they have had 12 months of natural (spontaneous) amenorrhea with an appropriate clinical profile (i.e. age appropriate, history of vasomotor...
The study team makes the final eligibility decision.
Where it's taking place
- New York, New York, United States
Questions & answers
Do participants get paid in this trial?
This listing doesn't specify compensation. Many trials still reimburse travel or offer a stipend, so it's worth asking the study team when you connect.
Is it free to join, and do I need insurance?
Searching and applying through BridgeMD is free. In clinical trials the study-related treatment and visits are generally provided at no cost to you. You usually don't need insurance to take part - confirm specifics with the study team.
How long does this study last?
The study runs about 24 weeks per participant, based on its public description. The team confirms the exact schedule and number of visits before you enroll.
Who can join this trial?
This study is enrolling all sexes, 18 years and older. The study team makes the final eligibility decision.
Where is this trial taking place?
Study sites include New York, New York, United States. Enter your location above to see the nearest site and check your eligibility.
Explore other conditions
BridgeMD is an information and trial-matching tool - not medical advice, and not the study sponsor. Details come from ClinicalTrials.gov; the study team decides eligibility.