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Chronic Myelomonocytic LeukemiaPhase 2NCT04239157

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

A Phase 2 study testing Canakinumab in people with Chronic Myelomonocytic Leukemia and Myelodysplastic Syndrome, run by M.D. Anderson Cancer Center at 1 site in United States.

Recruiting

Looking for participants now. Contact the study team or a site below to ask about taking part.

Sites and contacts
Official record
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Trial 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. Read more about this study

Who is it for?
Ages 18 years and older
What is being tested?
Canakinumab
Could I receive a placebo?
No, everyone receives an active treatment
How many people
76 participants
Where
1 siteUnited States
Study dates
25 Aug 2020to 31 Dec 2026
Phase
Phase 2
Are there results?
Not yet: the study is under way

Key requirements

Who can take part, as listed by the sponsor. Only the study team can confirm whether you are eligible, so take this list to your doctor.

  • Ages 18 years and older
  • All sexes
  • No healthy volunteers
  • Adult, Older adult

Who may be able to join

  • 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
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  • 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.

Who cannot join

  • 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.
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  • 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.

Trial locations and contacts

1 site in 1 country. Contact the study team or a site near you to ask about taking part.

Guillermo Garcia-Manero contact

713-794-3604

ggarciam@mdanderson.org

United States1 site, 1 recruiting

  • M D Anderson Cancer Center

    Houston, Texas, 77030

    Guillermo Garcia-Manero, MD, 713-794-3604, ggarciam@mdanderson.org

    Guillermo Garcia-Manero, MD

    Recruiting

Other trials in United States

About this study

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.

Read the detailed description

PRIMARY OBJECTIVE:

  • To assess the clinical activity of canakinumab in patients with low or intermediate-1 myelodysplastic syndrome (MDS) or CCUS.

SECONDARY OBJECTIVES:

  • To assess the safety profile of canakinumab in patients with low or intermediate-1 risk by - IPSS or IPSS-R score ≤3.5 MDS, CCUS
  • To assess the rate of transfusion independence (TI)
  • To assess duration of response

EXPLORATORY OBJECTIVE:

  • To assess pharmacodynamic (PD) parameters of canakinumab

OUTLINE:

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

After completion of study treatment, patients are followed up at 30 days and then every 6 months thereafter.

Being tested
Canakinumab
Medical subject headings
Leukemia, Myelomonocytic, Chronic, Myelodysplastic Syndromes

What participants receive

What participants receive. In a randomised study each person is assigned to one group by chance.

  • CanakinumabBiological

    Also called ACZ885, Ilaris

    Given SC

Study groups in NCT04239157
Study groupRoleWhat they receive
Treatment (canakinumab)ExperimentalPatients receive canakinumab SC on day 1. Cycles repeat every 28 days in the absence of disease progression or unacceptable toxicity.
Receives: Biological: Canakinumab

What is being measured

The primary outcome is the main question the study is designed to answer.

  1. Hematological improvement (HI)

    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.

    Measured: After 2 cycles (each cycle is 28 days)

  2. Incidence of adverse events

    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.

    Measured: Up to 4 weeks

Secondary outcomes (5)

  1. Transfusion independence

    Measured: Up to 2 years

  2. Duration of response

    Will be summarized using descriptive statistics such as mean, standard deviation, median and range.

    Measured: Up to 2 years

  3. Progression-free survival (PFS)

    Will be estimated using the method of Kaplan and Meier. Comparisons of time-to-time event endpoints by important subgroups will be made using the log-rank tests.

    Measured: Up to 2 years

  4. Leukemia-free survival (LFS)

    Will be estimated using the method of Kaplan and Meier. Comparisons of time-to-time event endpoints by important subgroups will be made using the log-rank tests.

    Measured: Up to 2 years

  5. Overall survival (OS)

    Will be estimated using the method of Kaplan and Meier. Comparisons of time-to-time event endpoints by important subgroups will be made using the log-rank tests.

    Measured: Up to 2 years

Timeline

From the start of the study to its completion; the mark is today. Estimated dates are the sponsor’s plan.

  1. First posted23 Jan 2020
  2. Study start25 Aug 2020
  3. Primary completion31 Dec 2026 (estimated)
  4. Study completion31 Dec 2026 (estimated)

Resources

More details

For doctors and researchers: how the study is designed, who runs it, and its registry IDs.

Study design
Study type
Interventional
Phase
Phase 2
Primary purpose
Treatment
Allocation
Not applicable
Intervention model
Single group
Masking
None (open label)
Enrolment
76 participants (estimated)
IDs, dates and publications
ClinicalTrials.gov ID
NCT04239157
Sponsor's protocol ID
2019-0339
Other IDs
NCI-2019-08494, 2019-0339
Status last verified
May 2026
First submitted
31 Dec 2019
Last updated
20 May 2026
Results posted
No
Data monitoring committee
No
FDA-regulated drug
Yes
FDA-regulated device
No

Publications

  • Rodriguez-Sevilla JJ, Adema V, Chien KS, Loghavi S, Ma F, Yang H, Montalban-Bravo G, Huang X, Calvo X, Joseph J, Bodden K, Garcia-Manero G, Colla S. The IL-1beta inhibitor canakinumab in previously treated lower-risk myelodysplastic syndromes: a phase 2 clinical trial. Nat Commun. 2024 Nov 13;15(1):9840. doi: 10.1038/s41467-024-54290-2.

    PubMed 39537648

Questions about this trial

What is this clinical trial studying?

A Phase 2 study testing Canakinumab in people with Chronic Myelomonocytic Leukemia and Myelodysplastic Syndrome, run by M.D. Anderson Cancer Center at 1 site in United States.

The full registered description is in the About this study section and the official record.

Who may be eligible to take part?

The sponsor lists 14 inclusion and 10 exclusion criteria. Eligibility is decided by the study team after screening, so read the full criteria and contact a listed site.

Is this clinical trial currently recruiting?

The record says: looking for participants now, as of the sponsor's last update on 20 May 2026. Status can change between updates, so confirm with the study contact before making plans.

Where is the study taking place?

The record lists 1 site in United States. Whether a site is taking part right now should be confirmed with that site. See every site.

What are the possible benefits and risks?

They depend on the study and the treatment, and no study can promise a benefit. The study team explains the known risks during informed consent, before anyone joins; the record itself does not list them.

What does this phase mean?

Phase 2 studies give the treatment to a larger group to see whether it has the intended effect and to keep checking safety.

Not every study follows the same path, and some studies (observational studies, device studies) have no phase.

Are results available for this study?

This study is still under way. Results are not available in the source record yet.

How can a doctor refer a patient or contact the research team?

Contact Guillermo Garcia-Manero (713-794-3604, ggarciam@mdanderson.org), or a site near the patient. AzTrials does not enrol participants or take referrals.

What does the primary outcome measure?

Hematological improvement (HI) (time frame: After 2 cycles (each cycle is 28 days)). The study lists 2 primary outcomes in all. It is what the study was designed to measure, not a finding.

Where can I read the original study information?

On the ClinicalTrials.gov record for NCT04239157, which also lists 1 linked publication.

Does a listing mean the treatment is approved or proven to work?

No. A listing means the study is registered. It does not show that the treatment is safe, works, or is approved by a regulator.

How often is this information updated?

AzTrials copies changed records from ClinicalTrials.gov every night and reloads the whole registry every week. The sponsor last updated this record on 20 May 2026; AzTrials last refreshed its copy on 26 Oct 2026.

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