Recruiting
Phase 2

Canakinumab

Sponsor:

M.D. Anderson Cancer Center

Code:

NCT04239157

Conditions

Chronic Myelomonocytic Leukemia

Myelodysplastic Syndrome

Recurrent Chronic Myelomonocytic Leukemia

Recurrent Myelodysplastic Syndrome

Refractory Chronic Myelomonocytic Leukemia

Eligibility Criteria

Sex: All

Age: 18+

Healthy Volunteers: Not accepted

Interventions

Canakinumab

Study Details

Brief summary:

This phase II trial studies how well canakinumab works for the treatment of low- or intermediate-risk myelodysplastic syndrome or chronic myelomonocytic leukemia. Canakinumab is a monoclonal antibody that may interfere with the ability of cancer cells to grow and spread.

Conditions

Chronic Myelomonocytic Leukemia

Myelodysplastic Syndrome

Recurrent Chronic Myelomonocytic Leukemia

Recurrent Myelodysplastic Syndrome

Refractory Chronic Myelomonocytic Leukemia

Study ID

NCT04239157

Start date

Aug 25, 2020

Status verified date

May, 2026

Completion date

Dec 31, 2026

Anticipated

Primary completion date

Dec 31, 2026

Anticipated

Eligibility Criteria

Eligibility Criteria

Sex: All

Age: 18+

Healthy Volunteers: Not accepted

Inclusion Criteria:

  • 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 Criteria:

  • 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.

Participants requiring systemic steroids, methotrexate or other immunosuppressive drugs will not be included in the study.

Study Design

Enrollment

76 participants

Anticipated

Intervention Model

Single group

Primary purpose

Treatment

Interventions and Outcome Measures

Arms

experimental: Treatment (canakinumab)

Patients receive canakinumab SC on day 1. Cycles repeat every 28 days in the absence of disease progression or unacceptable toxicity.

Interventions

Canakinumab

Given SC

Primary outcome measure

  • Hematological improvement (HI) [ Time Frame: After 2 cycles (each cycle is 28 days) ]
  • Incidence of adverse events [ Time Frame: Up to 4 weeks ]

Central Contacts and Locations

Central contacts

Guillermo Garcia-Manero

713-794-3604ggarciam@mdanderson.org

Locations

M D Anderson Cancer Center

Recruiting

Houston, Texas, United States, 77030

Contacts

Guillermo Garcia-Manero, MD

713-794-3604ggarciam@mdanderson.org

Principal Investigator:

Guillermo Garcia-Manero, MD

More Information

Sponsor

M.D. Anderson Cancer Center

Last update posted

May 20, 2026

Last verified

May, 2026

Trial information was received from ClinicalTrials.gov and was last updated on 2026-09-10. This information was provided to ClinicalTrials.gov by M.D. Anderson Cancer Center on 2026-05-20.