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

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

Trial statusRecruiting

Trial phasePhase 2

Trial typeInterventional

Biological sexAll

Age18+

SponsorM.D. Anderson Cancer Center

About this trial

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.

Eligibility criteria

This trial does not accept healthy volunteers

Qualifiers

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.

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

Disqualifiers

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.

Trial design

Design model

Single group

Treatments tested in this trial

Canakinumab
Biological/Vaccine
Given SC

Treatment groups

76 Participants

are divided into 1 treatment group

Group A: Treatment (canakinumab)Experimental treatment 1 intervention

Trial outcomes

Primary outcomes

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.

Time frame

After 2 cycles (each cycle is 28 days)

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.

Time frame

Up to 4 weeks

Secondary outcomes

Transfusion independence

Time frame

Up to 2 years

Duration of response

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

Time frame

Up to 2 years

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.

Time frame

Up to 2 years

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.

Time frame

Up to 2 years

Other outcomes

Pharmacodynamic (PD) parameters of canakinumab

Will include Correlative studies.

Time frame

Up to 2 years

Sponsors and contacts

Click on the lead sponsor to view all of their trials.

M.D. Anderson Cancer Center

Lead sponsor

National Cancer Institute (NCI)

Collaborator

Novartis

Collaborator

Source ClinicalTrials.gov

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