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A study of azacitidine with or without MBG453 for the treatment of patients with intermediate, high or very high risk myelodysplastic syndrome (MDS), or Chronic Myelomonocytic Leukemia-2 (CMML-2)

Phase 1
Conditions
adult subjects with intermediate, high or very high risk (per IPSS-R prognostic risk categories) myelodysplastic syndrome or with Chronic Myelomonocytic Leukemia - 2 (CMML-2)
MedDRA version: 20.0Level: HLTClassification code 10028536Term: Myelodysplastic syndromesSystem Organ Class: 100000004851
MedDRA version: 21.0Level: PTClassification code 10009018Term: Chronic myelomonocytic leukaemiaSystem Organ Class: 10029104 - Neoplasms benign, malignant and unspecified (incl cysts and polyps)
Therapeutic area: Diseases [C] - Cancer [C04]
Registration Number
EUCTR2019-002089-11-DE
Lead Sponsor
ovartis Pharma AG
Brief Summary

Not available

Detailed Description

Not available

Recruitment & Eligibility

Status
Authorised-recruitment may be ongoing or finished
Sex
All
Target Recruitment
500
Inclusion Criteria

Key inclusion criteria:
• Signed informed consent must be obtained prior to participation in the study
• Age = 18 years at the date of signing the informed consent form (ICF)
• Morphologically confirmed diagnosis of myelodysplastic syndrome (MDS) based on WHO 2016 classification (Arber et al 2016) by local investigator assessment with one of the following Prognostic Risk Categories, based on the revised International Prognostic Scoring System (IPSS-R):
• Very high (> 6 points)
• High (> 4.5 - = 6 points)
• Intermediate (> 3 - = 4.5 points)
Or
Morphologically confirmed diagnosis of Chronic Myelomonocytic Leukemia -2 based on WHO 2016 classification (Arber et al 2016-persistant PB monocytosis = 1 x109/L and monocytes accounting for = 10% of the WBC differential count) by local investigator assessment with WBC < 13 x 10^9/L at time of diagnosis.
• Indication for azacitidine treatment according to the investigator, based on local standard medical practice and institutional guidelines for treatment decisions
• Not eligible at time of screening for intensive chemotherapy according to the investigator, based on local standard medical practice and institutional guidelines for treatment decisions including assessment of individual clinical factors such as age, comorbidities and performance status.
• Not eligible at time of screening for hematopoietic stem cell transplantation according to the investigator, based on local standard medical practice and institutional guidelines for treatment decisions including assessment of individual clinical factors such as age, comorbidities, performance status and donor availability.
• Eastern Cooperative Oncology Group (ECOG) performance status of 0, 1 or 2
Please refer to protocol for further details and any additional inclusion criteria.
Are the trial subjects under 18? no
Number of subjects for this age range:
F.1.2 Adults (18-64 years) yes
F.1.2.1 Number of subjects for this age range 150
F.1.3 Elderly (>=65 years) yes
F.1.3.1 Number of subjects for this age range 350

Exclusion Criteria

Key exclusion criteria:
• Prior exposure to TIM-3 directed therapy at any time. Prior therapy with immune checkpoint inhibitors (e.g, anti-CTLA4, anti-PD-1, anti-PD-L1, or anti-PD-L2), cancer vaccines is allowed except if the drug was administered within 4 months prior to randomization
• Previous first-line treatment for intermediate, high, very high risk myelodysplastic syndromes (based on IPSS-R) or CMML-2 with any antineoplastic agents including for example chemotherapy, lenalidomide and hypomethylating agents (HMAs) such as decitabine or azacitidine. However, previous treatment with hydroxyurea or leukopheresis to reduce WBC count is allowed prior to randomization.
• Investigational treatment received within 4 weeks or 5 half-lives of this investigational treatment, whatever is longer, prior to randomization. In case of a checkpoint inhibitor: a minimal interval of 4 months prior to randomization is necessary to allow randomization.
• Subjects with Myelodysplastic syndrome (MDS) based on 2016 WHO classification (Arber et al 2016) with revised International Prognostic Scoring System (IPSS-R) = 3
• Diagnosis of acute myeloid leukemia (AML) including acute promyelocytic leukemia and extra-medullary acute myeloid leukemia, primary or secondary myelofibrosis based on WHO 2016 classification (Arber et al 2016)
• Diagnosis of therapy related myeloid neoplasms based on WHO 2016 classification
(Arber et al 2016)
• History of organ or allogeneic hematopoietic stem cell transplant
Please refer to protocol for further details and any additional exclusion criteria.

Study & Design

Study Type
Interventional clinical trial of medicinal product
Study Design
Not specified
Primary Outcome Measures
NameTimeMethod
Secondary Outcome Measures
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