NCT05641831 · Uma Borate
Canakinumab for the Prevention of Progression to Cancer in Patients With Clonal Cytopenias of Unknown Significance, IMPACT Study
What this study is about
This phase II trial tests how well canakinumab works to prevent progression to cancer in patients with clonal cytopenias of unknown significance (CCUS). CCUS is a blood condition defined by a decrease in blood cells. Blood cells are composed of either red blood cells, white blood cells, or platelets. In patients with CCUS, blood counts have been low for a long period of time.
View original scientific description
This phase II trial tests how well canakinumab works to prevent progression to cancer in patients with clonal cytopenias of unknown significance (CCUS). CCUS is a blood condition defined by a decrease in blood cells. Blood cells are composed of either red blood cells, white blood cells, or platelets. In patients with CCUS, blood counts have been low for a long period of time. Patients with CCUS also have a mutation in one of the genes that are responsible for helping blood cells develop.
Interventions
PROCEDURE
Biospecimen Collection
Undergo peripheral blood collection
PROCEDURE
Bone Marrow Aspiration and Biopsy
Undergo bone marrow biopsy and aspiration
DRUG
Canakinumab
Given SC
PROCEDURE
Chest Radiography
Undergo chest x-ray
PROCEDURE
Echocardiography
Undergo ECHO
DRUG
Placebo Administration
Given SC
OTHER
Quality-of-Life Assessment
Ancillary studies
Primary outcome measures
Time to overt myeloid malignancy
Time frame: From the date of randomization until the first date of overt myeloid malignancy diagnosis, assessed up to 6 years
Will be estimated with the non-parametric Kaplan-Meier method to compute the median time as well as the percentage of study participants with a diagnosed hematologic malignancy of myelodysplastic syndrome (MDS), myeloproliferative neoplasm (MPN), chronic myelomonocytic leukemia (CMML)/ acute myeloid leukemia (AML) at landmark time points (e.g., 1-year, 2-years) with corresponding 95% confidence intervals. Since this method will censor patients who die without having developed MDS/MPN/CMML/AML, we will also compute the cumulative incidence of overt myeloid malignancy that accounts for the competing risk of death in the absence of a hematologic malignancy. All randomized patients will be included in the primary endpoint analysis in the arm to which they were randomized (ie, intent-to-treat population).
Who can participate
This study lists these criteria on ClinicalTrials.gov. A study coordinator reviews eligibility during screening — this page does not determine whether you qualify.
Inclusion criteria
- Patients with age \>= 18 with high-risk CCUS
- Must meet ALL the following criteria:
- Unexplained, clinically meaningful cytopenias (greater than 4 months) in one or more of the following lineages: erythroid cells, neutrophils, platelets. Clinically meaningful cytopenia is institution specific and threshold may vary on age, sex, and race. Decision-making should depend upon lab values specific to the institution and supersede public works. Based upon published work, significant cytopenias are defined as the following:
- Erythroid Cells:
- Hemoglobin \< 11 g/dL
- White Blood Cells:
- Absolute Neutrophil Count \< 1800/microL and \> 500/microL
- Platelet Count \< 150,000/microL and \> 50,000/microL
- MDS criteria not fulfilled
- No other evidence of hematological malignancy
- No or only mild (\< 10%) bone marrow dysplasia
- Blast cells \< 5% detected via morphologic examination of blood and/or bone marrow sme
Where
- Miami, Florida
- New York, New York
- Columbus, Ohio
- Nashville, Tennessee
- Dallas, Texas
Related conditions & keywords
Frequently asked questions
What is a clinical trial?
A clinical trial is a research study that tests new medical treatments, drugs, devices, or procedures to determine their safety and effectiveness. Trials are carefully designed and monitored to protect participants while advancing medical knowledge.
Is it safe to participate?
Clinical trials follow strict safety guidelines and ethical standards. Trials must be reviewed and approved, and participants are closely monitored by medical professionals throughout the study. You can withdraw at any time if you choose.
Will I be compensated?
Many clinical trials offer compensation for your time, travel expenses, and inconvenience. The specific compensation varies by study and will be discussed during the screening process. All study-related medical care is typically provided at no cost to participants.
Will I receive a placebo instead of treatment?
When effective treatment exists, participants typically receive either the standard treatment plus the study intervention, or the standard treatment plus placebo. You would not be denied effective care. Placebos are primarily used when no proven treatment is available, or in addition to standard care. Your trial consent form will clearly explain what treatments you may receive.
Can I leave a trial if I change my mind?
Absolutely. Participation in clinical trials is completely voluntary. You have the right to withdraw from the study at any time, for any reason, without penalty or loss of benefits to which you are otherwise entitled.
How long does a clinical trial last?
Trial duration varies widely depending on the study design and purpose. Some trials last just a few weeks, while others may continue for months or years. The study coordinator will provide specific timeline information during your screening call.
Data: ClinicalTrials.gov · synced Jan 2, 2026 · Source of record for eligibility and locations