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/39537648Actively Recruiting
Phase 2
Age: 18Years +
All Genders
ID04239157
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
Led by M.D. Anderson Cancer Center · Updated on 2026-05-20
76
Participants Needed
1
Research Sites
N/A
Total Duration
On this page
Sponsors
M
M.D. Anderson Cancer Center
Lead Sponsor
N
National Cancer Institute (NCI)
Collaborating Sponsor
AI-Summary
What this Trial Is About
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 drug's 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
Brief Title
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
Who Can Participate
Age: 18Years +
All Genders
Eligibility Criteria
You may qualify if you...
- Age 18 years or older
- Diagnosis of myelodysplastic syndrome (MDS) according to WHO 2016 with low or intermediate-1 risk by IPSS or IPSS-R score 3.5
- Diagnosis of clonal cytopenia of undetermined significance (CCUS) with specific somatic pathogenic variants and cytopenia for more than 30 days
- For cohort 1: no response to prior therapy with erythropoiesis stimulating agents or hypomethylating agents after at least 4 cycles
- Hemoglobin less than 10 g/dL with symptomatic anemia or transfusion dependency as defined
- Transfusion dependency criteria for cohort 2
- Signed informed consent
- Adequate liver function with bilirubin and liver enzymes within 3 times the upper limit of normal
- Serum creatinine clearance above 30 mL/min
- ECOG performance status 2 or less
You will not qualify if you...
- Active infection not responding to antibiotics
- Prior treatment with IL-1 or IL-1 receptor inhibitors
- Absolute neutrophil count less than 0.5 x 10^9 k/ul
- Pregnant or breastfeeding female participants
- Unwillingness to follow contraception requirements if of reproductive potential
- Positive pregnancy test for females of reproductive potential
- History of active malignancy within past 2 years except certain treated cancers with life expectancy over 2 years
- Receiving other investigational agents or chemotherapy, radiotherapy, or immunotherapy within 14 days prior to study
- Known HIV infection
- Use of systemic steroids, methotrexate, or other immunosuppressive drugs during study period
AI-Screening
AI-Powered Screening
Complete this quick 3-step screening to check your eligibility
1
2
3
Your Study Journey
Screening
Duration - 2 to 4 weeks
Participants are screened for eligibility to participate in the trial.
1 visit (in-person)
Treatment
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
Follow-up
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
Trial Site Locations
Total: 1 location
1
M D Anderson Cancer Center
Houston, Texas, United States, 77030
Actively Recruiting
Loading map...
Research Team
G
Guillermo Garcia-Manero
How is the study designed?
Study Type
INTERVENTIONAL
Masking
NONE
Allocation
NA
Model
SINGLE_GROUP
Primary Purpose
TREATMENT
Number of Arms
1
Similar Trials
Frequently Asked Questions
Have more questions? Get in touch with our team for quick support
Not the Right Trial for You?
Explore thousands of other clinical trials that might be a better match.
Sign up to get personalized trial recommendations delivered to your inbox.
Already have an account? Log in here