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Phase II Study of Canakinumab for Myelodysplastic Syndromes and Chronic Myelomonocytic Leukemia

This Phase II study is testing a drug called canakinumab for people with low- or intermediate-risk myelodysplastic syndrome (MDS) or chronic myelomonocytic leukemia (CMML). Canakinumab is a type of antibody that might help stop cancer cells from growing. The study aims to see how well canakinumab works by measuring improvements in blood counts and how safe it is. You might be able to join if you are 18 or older and have been diagnosed with MDS or CMML that fits specific risk criteria. The study plans to enroll about 76 participants.

Study design
This is an open-label, Phase II study, meaning both you and your doctors will know you are receiving canakinumab. It plans to enroll 76 participants.
What's involved
You will receive canakinumab as an injection under the skin on day 1 of each 28-day cycle. You will continue treatment as long as your condition doesn't worsen or side effects are manageable.
Compensation
Not stated in the trial record.
Follow-up
After your study treatment ends, you will have a follow-up visit 30 days later, and then every 6 months after that.

AI-generated from the public study record. Only the study team can confirm whether you're eligible — confirm details with them before making decisions.

NCT04239157

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

Recruiting
PHASE2Ages 18+InterventionalTreatment
M.D. Anderson Cancer Center
~76 participants
Updated 2026-05-20 on ClinicalTrials.gov
What's tested:Canakinumab

At a glance

Recruiting sites
1 of 1 listed site is recruiting right now
RecruitingSuspended, closed, or not yet open
What they're measuring
Hematological improvement (HI)
Measured over After 2 cycles (each cycle is 28 days)
+1 more outcome measured

Conditions

Chronic Myelomonocytic Leukemia
Myelodysplastic Syndrome
Recurrent Chronic Myelomonocytic Leukemia
Recurrent Myelodysplastic Syndrome
Refractory Chronic Myelomonocytic Leukemia
Refractory Myelodysplastic Syndrome

Explore related trials

Chronic Myelomonocytic LeukemiaClonal Cytopenia of Undetermined SignificanceMyelodysplastic SyndromeSomatic pathogenic variant

Where it's being run

1 sites across 1 states
Texas1

Study leadership

  • Guillermo Garcia-Manero · PRINCIPAL_INVESTIGATOR · M.D. Anderson Cancer Center

Who to contact

Guillermo Garcia-Manero
[email protected]
713-794-3604
Email the study team

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Do you actually qualify for this trial?

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Eligibility criteria

Inclusion

Age ≥ 18 years as MDS and CCUS are very rare conditions in the pediatric setting.
Cohorts 1-3: Diagnosis of MDS according to WHO 2016 classification and low or intermediate-1 risk by IPSS or IPSS-R with a score of ≤ 3.5.
Cohort 4: Diagnosis of CCUS defined as:
Presence of a somatic pathogenic variant associated with hematological malignancy without morphological evidence of myelodysplasia
Variant allele fraction of greater than or equal to 2% in at least one identified somatic pathogenic variant
Bone marrow aspirate excluding hematological malignancy and MDS
Presence of a cytopenia for \>30 days. Cytopenia will be defined using accepted CHRS (Clonal Hematopoiesis Risk Score) criteria (Weeks et al, NEJM Evidence in press): ANC \<1.8 or hgb \<12 in females and \<13 in males or a platelet count of \<150.
Cohort 1: Participants need to have not responded to prior therapy with ESAs or hypomethylating agents (HMAs). These could include azacitidine, decitabine, SGI-110, ASTX727, or CC-486. Patients will need to have received at least 4 cycles of HMA. Participants with relapse or progression after any number of cycles of HMA by IWG 2006 criteria will also be candidates. Participants with evidence of del 5q alteration also are required to have been treated with Lenalidomide.
Cohort 1: Hemoglobin \<10g/dL with symptomatic anemia or transfusion dependency defined as the need for prior transfusion in the past 8 weeks for a hemoglobin level less than 8g/dl.
Cohort 2: Transfusion dependency defined as the need for prior transfusion in the past 8 weeks of (1) at least 2 units of PRBC for a hemoglobin level less than 8g/dl or symptomatic anemia (hemoglobin \<10g/dL), or (2) any platelet transfusion.
Participants (or patient's legally authorized representative) must have signed an informed consent document indicating that the patient understands the purpose of and procedures required for the study and is willing to participate in the study.
Adequate hepatic function with total bilirubin \</=3 x ULN, AST or ALT \</= 3xULN.
Serum creatinine clearance \>30mL/min and no end/stage renal disease (using Cockcroft-Gault).
ECOG performance status \</=2.

Exclusion

Active infection not adequately responding to appropriate antibiotics.
Prior treatment with IL-1/IL-1r inhibitors
Absolute neutrophil count (ANC) \<0.5x109 k/ul; colony-stimulating factors can be administered prior to study drug initiation.
Female participants who are pregnant or lactating.
Participants with reproductive potential who are unwilling to following contraception requirements (including condom use for males with sexual partners, and for females: prescription oral contraceptives \[birth control pills\], contraceptive injections, intrauterine devices \[IUD\], double-barrier method \[spermicidal jelly or foam with condoms or diaphragm\], contraceptive patch, or surgical sterilization) throughout the study. Reproductive potential is defined as no previous surgical sterilization or females that are not post-menopausal for 12 months.
Female participants with reproductive potential who do not have a negative urine or blood beta-human chorionic gonadotropin (beta HCG) pregnancy test at screening.
History of an active malignancy within the past 2 years prior to study entry, with the exception of: a. Adequately treated in situ carcinoma of the cervix uteri b. Adequately treated basal cell carcinoma or localized squamous cell carcinoma of the skin or any other malignancy with a life expectancy of more than 2 years.
Participants receiving any other concurrent investigational agent or chemotherapy, radiotherapy, or immunotherapy (within 14 days of initiating study treatment).
Known history of testing positive for Human Immunodeficiency Virus (HIV) infections.

What this trial measures

  • Hematological improvement (HI)After 2 cycles (each cycle is 28 days)

    Will be monitored simultaneously using the Bayesian approach of Thall, Simon, Estey. Will estimate the HI rate for canakinumab, along with the 95% credible intervals. The association between HI rate and patient's clinical characteristics will be examined by Wilcoxon's rank sum test or Fisher's exact test.

  • Incidence of adverse eventsUp to 4 weeks

    Will be monitored simultaneously using the Bayesian approach of Thall, Simon, Estey. Safety data of the patients will be summarized using descriptive statistics such as mean, standard deviation, median and range. Toxicity type, severity and attribution will be summarized for each patient using frequency tables.