The IL-1β inhibitor canakinumab in previously treated lower-risk myelodysplastic syndromes: a phase 2 clinical trial.
Juan Jose Rodriguez-Sevilla, Vera Adema, Kelly S Chien...
https://pubmed.ncbi.nlm.nih.gov/39537648/Actively Recruiting
Led by M.D. Anderson Cancer Center · Updated on 2026-05-20
76
Participants Needed
1
Research Sites
N/A
Total Duration
Researchers are evaluating canakinumab, a monoclonal antibody, for treating patients with low or intermediate-1 risk myelodysplastic syndrome MDS or chronic myelomonocytic leukemia CMML. The study is a phase II trial focusing on patients with these blood disorders to assess the drugs clinical activity, safety, and effects on transfusion independence and response duration. Participants receive canakinumab through a subcutaneous injection on the first day of each 28-day cycle. Treatment continues in repeated cycles unless there is disease progression or unacceptable side effects. After completing treatment, patients will be followed up at 30 days and then every six months to monitor their health. Throughout the trial, researchers will assess hematological improvement after two cycles, monitor adverse events for four weeks, and evaluate longer-term outcomes such as transfusion independence, duration of response, progression-free survival, leukemia-free survival, and overall survival for up to two years. Participants will undergo regular assessments and safety monitoring during the study period, which lasts until disease progression or unacceptable toxicity occurs.
CONDITIONS
A Phase II, Open-Label, Study of Subcutaneous Canakinumab, an Anti-IL-1β Human Monoclonal Antibody, for Patients With Low or Int-1 Risk IPSS/IPSS-R Myelodysplastic Syndromes and Chronic Myelomonocytic Leukemia
G
Guillermo Garcia-Manero
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M.D. Anderson Cancer Center
Lead Sponsor
National Cancer Institute (NCI)
Collaborating Sponsor
This table compares participation in this trial with other options available for this condition. The information is drawn from published research and the trial’s study details and is provided for educational purposes only.
| Factors | Canakinumab (subcutaneous anti‑IL‑1β antibody) | Current standard care for lower‑risk MDS |
|---|---|---|
| What it is | An investigational subcutaneously administered anti‑IL‑1β monoclonal antibody tested as a single‑group therapy in patients with lower‑risk MDS or CCUS who have cytopenia and prior treatment failure (e.g., ESAs, HMAs) | Common current treatments for similar patients include erythropoiesis‑stimulating agents, luspatercept for ring sideroblasts, lenalidomide in del(5q) patients, hypomethylating agents or supportive care |
| Role in trial | Received by all enrolled participants as the only assigned investigational therapy | Not evaluated within this study |
| Primary use | Treatment of cytopenias in lower‑risk MDS or CCUS after failure of prior therapies | First‑line or subsequent management of cytopenias in lower‑risk MDS based on risk biomarkers and response to prior treatment |
| How it is given or performed | Subcutaneous injection of canakinumab per trial dosing schedule (dose escalation to 300 mg) | Administered as prescribed for each therapy: e.g., injections (luspatercept), orals (lenalidomide), injections (ESAs), or supportive transfusion care |
| Evidence status | Phase 2 single‑arm data suggest some hematological responses and transfusion independence in a small subset of patients | Multiple agents (ESAs, luspatercept, lenalidomide) have established response rates in lower‑risk MDS per systematic reviews and trials |
| Important risks | Risks include adverse events reported up to 4 weeks, but detailed safety profile is still under investigation | Known risks vary by therapy (e.g., thromboembolic risk with ESAs, hypertension with luspatercept, cytopenias with lenalidomide) |
| Important limitations | Single‑arm design without control group; small sample size; unclear durability of response | Treatment selection depends on biomarkers, prior responses, and not all patients are candidates for each option |
Disclaimer: This comparison is created with the help of artificial intelligence(AI) and may contain errors or missing information. It is not medical advice and does not mean one option is better or safer than another. Clinical trials are still being studied, and joining a trial is your choice. Please consult your healthcare professional before making any decisions.
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Duration - 2 to 4 weeks
Participants are screened for eligibility to participate in the trial.
1 visit (in-person)
Duration - Repeated 28-day cycles until disease progression or discontinuation
Participants receive canakinumab as a subcutaneous injection on day 1 of each cycle. Treatment cycles repeat every 28 days in the absence of disease progression or unacceptable toxicity.
1 visit every 28 days for treatment
Duration - Up to 2 years
After completion of study treatment, participants are followed up to monitor their health and treatment outcomes.
1 visit at 30 days post-treatment, then visits every 6 months
Study Type
INTERVENTIONAL
Masking
NONE
Allocation
NA
Model
SINGLE_GROUP
Primary Purpose
TREATMENT
Number of Arms
1
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Juan Jose Rodriguez-Sevilla, Vera Adema, Kelly S Chien...
https://pubmed.ncbi.nlm.nih.gov/39537648/M.D. Anderson Cancer Center
Lead Sponsor
National Cancer Institute (NCI)
Collaborating Sponsor