RecruitingPhase 2

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 join

As published by the study on ClinicalTrials.gov. "Not stated" means the study hasn't said — ask the study team.

Age
18 Years and older
Sex
All sexes
Healthy volunteers
Not accepted
Compensation
Not stated

Other requirements are listed under Eligibility below. Only the study team can confirm whether you can join.

What to expect

No one can promise a trial will help you personally — that is what the research is trying to find out.

What participation involves

Not stated in the study description — ask the study team about visits and procedures.

Compensation
Not stated
  • Study-related exams and procedures are typically provided at no cost. This is how many trials work in general — confirm the details for this study with the study team.
  • Participants are monitored closely by the research team throughout the study.
  • Some participants may receive access to an investigational treatment, which is still being studied.
  • Your participation helps researchers learn whether this approach could help future patients.

About this study

In plain language

This study tests how well canakinumab works for people with lower-risk myelodysplastic syndromes (MDS) or chronic myelomonocytic leukemia, which affect blood cells. It also includes a group with CCUS, a condition with low blood cell counts and certain gene changes. Canakinumab is given as a shot under the skin and may help stop cancer cells from growing and spreading.

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.

Compensation is read from the study's own description and may be missing — always confirm with the study team.

Eligibility

Who can join

  • Be age 18 or older.
  • For groups 1–3: Have MDS rated low or intermediate-1 risk using the study’s scoring rules.
  • For group 4: Have CCUS, with low blood cell counts for more than 30 days and a qualifying gene change. A bone marrow test must show no MDS or blood cancer.
  • For group 1: Prior treatment to raise red blood cells or treat MDS must have failed, or the disease must have returned or worsened. Some treatments require at least 4 cycles. People with a gene change called del 5q must also have received lenalidomide.
  • For group 1: Have hemoglobin below 10 g/dL with symptoms of anemia (low red blood cells), or have needed a blood transfusion in the past 8 weeks for hemoglobin below 8 g/dL.
  • For group 2: In the past 8 weeks, have needed at least 2 units of red blood cells for low hemoglobin or anemia symptoms, or any platelet transfusion. Platelets help blood clot.
  • Have liver and kidney test results within study limits, no end-stage kidney disease, and be able to do self-care and be up and about at least half the day.
  • You or your legal representative must sign a form agreeing to join after learning about the study.

Who can't join

  • Have an active infection that is not responding well enough to antibiotics, or a known positive HIV test.
  • Have previously received drugs that block IL-1 or its receptor, which are involved in inflammation.
  • Have a very low neutrophil count, below 0.5 × 10⁹. Neutrophils fight infection. Drugs to raise this count may be given before study treatment.
  • Be pregnant or breastfeeding, or be able to become pregnant without a negative pregnancy test at screening.
  • Be able to have children but unwilling to follow the study’s birth control rules throughout the study.
  • Have had active cancer in the past 2 years. Exceptions include certain treated cervical or skin cancers, or another cancer with an expected survival of more than 2 years.
  • Be receiving another study drug, chemotherapy, radiation, or immune-based cancer treatment within 14 days before starting study treatment.
  • Need steroids that act throughout the body, methotrexate, or other drugs that weaken the immune system.

Simplified for readability. The official criteria below are what the study team uses.

Official eligibility criteria

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 sites (1)

TX