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Study of Efficacy and Safety of MBG453 in Combination With Azacitidine in Subjects With Intermediate, High or Very High Risk Myelodysplastic Syndrome (MDS) as Per IPSS-R, or Chronic Myelomonocytic Leukemia-2 (CMML-2) (STIMULUS-MDS2)

Primary Purpose

Myelodysplastic Syndromes, Leukemia, Myelomonocytic, Chronic

Status
Active
Phase
Phase 3
Locations
International
Study Type
Interventional
Intervention
MBG453
Azacitidine
Placebo
Sponsored by
Novartis Pharmaceuticals
About
Eligibility
Locations
Arms
Outcomes
Full info

About this trial

This is an interventional treatment trial for Myelodysplastic Syndromes focused on measuring MBG453, Phase III, TIM-3, azacitidine, myelodysplastic syndrome, MDS, chronic myelomonocytic leukemia, CMML-2, Sabatolimab

Eligibility Criteria

18 Years - undefined (Adult, Older Adult)All SexesDoes not accept healthy volunteers

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

  • 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, including persistent monocytosis) by local investigator assessment with WBC < 13 x 109/L at time of initial 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 (HSCT) 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

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 with any antineoplastic agents including for example chemotherapy, lenalidomide and hypomethylating agents (HMAs) such as decitibine and 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

Other protocol-defined Inclusion/Exclusion Criteria may apply.

Sites / Locations

  • Yuma Regional Cancer Center
  • University Of California Los Angeles .
  • Yale University School Of Medicine Smilow Cancer Hospital
  • Mayo Clinic Jacksonville .
  • University Of Miami .
  • Northwestern University Div of Hematology/Oncology
  • Massachusetts General Hospital .
  • Hackensack University Medical Center SC
  • Weill Cornell Medicine NY-Presb .
  • University of Rochester Medical Center Dept.ofUniv.Rochester/JPWilmot
  • University of Virginia University of Virginia
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Arms of the Study

Arm 1

Arm 2

Arm Type

Experimental

Placebo Comparator

Arm Label

MBG453 + Azacitidine

Placebo + Azacitidine

Arm Description

Participants will receive MBG453 plus Azacitidine

Participants will receive Placebo plus Azacitidine

Outcomes

Primary Outcome Measures

Overall Survival
Time from randomization until death due to any cause

Secondary Outcome Measures

Key secondary endpoint 1: Time to definitive deterioration of fatigue using Functional Assessment of Cancer Therapy (FACIT)-Fatigue score
FACIT-Fatigue score is a 13-item questionnaire designed to assess fatigue in cancer patients. All items use a 5-point scale ranging from 0 to 4 (0=Not at All to 4=Very Much). The total score ranges from 0 to 52 with higher values representing better quality of life. Time from randomization to at least 3 points worsening from baseline will be presented.
Key secondary endpoint 2: Red Blood Cell transfusion-free intervals
Cumulative times of intervals with no evidence of Red Blood Cell (RBC) transfusion for at least 8 weeks after randomization
Key secondary endpoint: Percent of subjects with at least 3 point confirmed improvement from baseline in FACIT-fatigue scoresscore
FACIT-Fatigue score is a 13-item questionnaire designed to assess fatigue in cancer patients. All items use a 5-point scale ranging from 0 to 4 (0=Not at All to 4=Very Much). The total score ranges from 0 to 52 with higher values representing better quality of life. Percentage of subjects with at least 3 point confirmed improvement from baseline will be presented.
Key secondary endpoint 4: Percent of subjects with at least 10 point confirmed improvement from baseline in physical functioning using European Or ganization for Research and Treatment of Cancer's Core Quality of Life Questionnaire (EORTC QLQ-C30)
EORTC-QLQ-C30 is a 30-item questionnaire developed to assess the quality of life of cancer patients. Subject's responses to 5 questions about their physical functioning (Items 1-5) are scored on a 4-point scale (1=Not at All to 4=Very Much). Using linear transformation, raw scores are standardized, so that scores range from 0 to 100. A high score indicates a high / healthy level of functioning. Percentage of subjects with at least 10 point confirmed improvement from baseline in physical functioning will be presented.
Key secondary endpoint 5: Percent of subjects with at least 10 point confirmed improvement from baseline in emotional functioning using EORTC-QLQ-C30
EORTC-QLQ-C30 is a 30-item questionnaire developed to assess the quality of life of cancer patients. Subject's responses to 4 questions about their emotional functioning (Items 21-24) are scored on a 4-point scale (1=Not at All to 4=Very Much). Using linear transformation, raw scores are standardized, so that scores range from 0 to 100. A high score indicates a high / healthy level of functioning. Percentage of subjects with at least 10 point confirmed improvement from baseline in emotional functioning will be presented.
Percentage of subjects with either CR, or mCR, or PR, or HI in each treatment arm according to International Working Group for MDS (IWG-MDS) as per investigator assessment
Response rate of subjects with complete remission (CR), or marrow remission (mCR), or partial remission (PR), or hematologic improvement (HI)
Percentage of subjects with SD in each treatment arm according to International Working Group for MDS (IWG-MDS) as per investigator assessment
Response rate of subjects with stable disease (SD)
Progression Free Survival (PFS)
Time from randomization to disease progression (including transformation to acute leukemia per WHO 2016 classification), relapse from CR according to International Working Group for MDS (IWG-MDS), or death due to any cause, whichever occurs first, as per investigator assessment
Leukemia-free survival
Time from randomization to ≥ 20% blasts in bone marrow/peripheral blood (per WHO 2016 classification) or diagnosis of extramedullary acute leukemia, or death due to any cause
Number of transfusion dependent subjects at baseline who become Red Blood Cells/platelets transfusion independent after randomization
Improvement in RBC/Platelets transfusion independence as per International Working Group for MDS (IWG-MDS) criteria
Percentage of transfusion dependent subjects at baseline who become Red Blood Cells/platelets transfusion independent after randomization
Improvement in RBC/Platelets transfusion Independence as per International Working Group for MDS (IWG-MDS) criteria
Pharmacokinetics of MBG453 (parameter Cmax)
Maximum (peak) MBG453 concentration [Cmax]
Pharmacokinetics of MBG453 (parameter AUC)
Area Under the Curve [AUC]
Anti-drug Antibody (ADA) prevalence at baseline and ADA incidence on-treatment
Immunogenicity of MBG453
Change from baseline in the European Quality of Life (EuroQol) - 5 Dimensions, 5 Level Questionnaire (EQ-5D-5L) score over time
The EQ-5D-5L comprises 5 dimensions: mobility, self-care, usual activities, pain/discomfort, anxiety/depression. For each of the 5 dimensions, subject's responses are scored on a 5-point scale (1=no problem to 5=extreme problems). Change from baseline will be presented.
Change from baseline in the European Quality of Life (EuroQoL) - 5 Dimensions, 5 Level Questionnaire (EQ-5D-5L) Visual Analogue Scale over time
The EQ-5D-5L VAS records the subject's self-rated health on a visual analogue scale numbered from 0 to 100, with 0 being "the worst health you can imagine" and 100 being "the best health you can imagine". Change from baseline will be presented.
Change from baseline to C12D1 of Global Health Status/Quality of Life scores using European Organization for Research and Treatment of Cancer's Core Quality of Life Questionnaire (EORTC-QLQ-C30)
EORTC-QLQ-C30 is a 30-item questionnaire developed to assess the quality of life of cancer patients. Subject's responses to 2 questions (Items 29+30: "How would you rate your overall health during the past week?" and "How would you rate your overall quality of life during the past week?") are scored on a 7-point scale (1=Very Poor to 7=Excellent). Using linear transformation, raw scores are standardized, so that scores range from 0 to 100. A higher score indicates a better overall outcome. Change from baseline to Cycle 12 Day 1 will be presented.

Full Information

First Posted
January 21, 2020
Last Updated
October 17, 2023
Sponsor
Novartis Pharmaceuticals
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1. Study Identification

Unique Protocol Identification Number
NCT04266301
Brief Title
Study of Efficacy and Safety of MBG453 in Combination With Azacitidine in Subjects With Intermediate, High or Very High Risk Myelodysplastic Syndrome (MDS) as Per IPSS-R, or Chronic Myelomonocytic Leukemia-2 (CMML-2)
Acronym
STIMULUS-MDS2
Official Title
A Randomized, Double-blind, Placebo-controlled Phase III Multi-center Study of Azacitidine With or Without MBG453 for the Treatment of Patients With Intermediate, High or Very High Risk Myelodysplastic Syndrome (MDS) as Per IPSS-R, or Chronic Myelomonocytic Leukemia-2 (CMML-2)
Study Type
Interventional

2. Study Status

Record Verification Date
October 2023
Overall Recruitment Status
Active, not recruiting
Study Start Date
June 8, 2020 (Actual)
Primary Completion Date
January 15, 2027 (Anticipated)
Study Completion Date
January 15, 2027 (Anticipated)

3. Sponsor/Collaborators

Responsible Party, by Official Title
Sponsor
Name of the Sponsor
Novartis Pharmaceuticals

4. Oversight

Studies a U.S. FDA-regulated Drug Product
Yes
Studies a U.S. FDA-regulated Device Product
No
Data Monitoring Committee
Yes

5. Study Description

Brief Summary
This is a Phase III multi-center, randomized, two-arm parallel-group, double-blind, placebo-controlled study of MBG453 or placebo added to azacitidine in adult subjects with intermediate, high or very high risk myelodysplastic syndrome (MDS) as per IPSS-R, or Chronic Myelomonocytic Leukemia-2 (CMML-2) who have an indication for treatment with azacitidine in first-line setting and are not eligible for intensive chemotherapy or hematopoietic stem cell transplantation (HSCT) according to medical judgment by the investigator. The purpose of the current study is to assess clinical effects of MBG453 in combination with azacitidine in adult subjects with IPSS-R intermediate, high, very high risk MDS and CMML-2.
Detailed Description
This is a Phase III multi-center, randomized, two-arm parallel-group, double-blind, placebo controlled study of MBG453 or placebo added to azacitidine in adult subjects with intermediate, high or very high risk myelodysplastic syndrome (MDS) as per IPSS-R, or Chronic Myelomonocytic Leukemia-2 (CMML-2). The primary objective of this study is to compare overall survival (OS) in the MBG453 plus azacitidine arm versus placebo plus azacitidine arm where OS is the time from randomization until death due to any cause. Subjects will be randomized in a 1:1 ratio to treatment arms as follow: MBG453 800 mg IV Q4W plus azacytidine, Placebo IV Q4W plus azacitidine The randomization will be stratified into 4 groups: intermediate risk MDS, high risk MDS, very high risk MDS and CMML-2. All subjects who discontinue both study treatments will enter a long-term post-treatment follow-up including response and PRO assessments, and/or survival follow-up for up to 5 years after the last subject was randomized. Subjects will be treated until they experience progression of disease (including transformation to acute leukemia per WHO 2016 classification), experience unacceptable toxicity or discontinue the study treatment for other reasons. Continuation of study treatment beyond progression (excluding transformation to acute leukemia: continuation in this case is not possible) may be possible in selected subjects.

6. Conditions and Keywords

Primary Disease or Condition Being Studied in the Trial, or the Focus of the Study
Myelodysplastic Syndromes, Leukemia, Myelomonocytic, Chronic
Keywords
MBG453, Phase III, TIM-3, azacitidine, myelodysplastic syndrome, MDS, chronic myelomonocytic leukemia, CMML-2, Sabatolimab

7. Study Design

Primary Purpose
Treatment
Study Phase
Phase 3
Interventional Study Model
Parallel Assignment
Model Description
Randomized, double-blind
Masking
ParticipantCare ProviderInvestigator
Allocation
Randomized
Enrollment
530 (Actual)

8. Arms, Groups, and Interventions

Arm Title
MBG453 + Azacitidine
Arm Type
Experimental
Arm Description
Participants will receive MBG453 plus Azacitidine
Arm Title
Placebo + Azacitidine
Arm Type
Placebo Comparator
Arm Description
Participants will receive Placebo plus Azacitidine
Intervention Type
Drug
Intervention Name(s)
MBG453
Intervention Description
A dose of MBG453 800 mg will be administered intravenously (IV) every 4 weeks (Q4W).
Intervention Type
Drug
Intervention Name(s)
Azacitidine
Intervention Description
A dose of Azacitidine 75 mg/m2 will be administered IV or subcutaneously (SC) on Day 1-7, or Day 1-5, 8 and 9.
Intervention Type
Drug
Intervention Name(s)
Placebo
Intervention Description
A dose of Placebo 800 mg will be administered intravenously every 4 weeks (Q4W).
Primary Outcome Measure Information:
Title
Overall Survival
Description
Time from randomization until death due to any cause
Time Frame
Up to 5 years after last patient randomized
Secondary Outcome Measure Information:
Title
Key secondary endpoint 1: Time to definitive deterioration of fatigue using Functional Assessment of Cancer Therapy (FACIT)-Fatigue score
Description
FACIT-Fatigue score is a 13-item questionnaire designed to assess fatigue in cancer patients. All items use a 5-point scale ranging from 0 to 4 (0=Not at All to 4=Very Much). The total score ranges from 0 to 52 with higher values representing better quality of life. Time from randomization to at least 3 points worsening from baseline will be presented.
Time Frame
Up to 5 years after last patient randomized
Title
Key secondary endpoint 2: Red Blood Cell transfusion-free intervals
Description
Cumulative times of intervals with no evidence of Red Blood Cell (RBC) transfusion for at least 8 weeks after randomization
Time Frame
Up to 5 years after last patient randomized
Title
Key secondary endpoint: Percent of subjects with at least 3 point confirmed improvement from baseline in FACIT-fatigue scoresscore
Description
FACIT-Fatigue score is a 13-item questionnaire designed to assess fatigue in cancer patients. All items use a 5-point scale ranging from 0 to 4 (0=Not at All to 4=Very Much). The total score ranges from 0 to 52 with higher values representing better quality of life. Percentage of subjects with at least 3 point confirmed improvement from baseline will be presented.
Time Frame
Up to 5 years after last patient randomized
Title
Key secondary endpoint 4: Percent of subjects with at least 10 point confirmed improvement from baseline in physical functioning using European Or ganization for Research and Treatment of Cancer's Core Quality of Life Questionnaire (EORTC QLQ-C30)
Description
EORTC-QLQ-C30 is a 30-item questionnaire developed to assess the quality of life of cancer patients. Subject's responses to 5 questions about their physical functioning (Items 1-5) are scored on a 4-point scale (1=Not at All to 4=Very Much). Using linear transformation, raw scores are standardized, so that scores range from 0 to 100. A high score indicates a high / healthy level of functioning. Percentage of subjects with at least 10 point confirmed improvement from baseline in physical functioning will be presented.
Time Frame
Up to 5 years after last patient randomized
Title
Key secondary endpoint 5: Percent of subjects with at least 10 point confirmed improvement from baseline in emotional functioning using EORTC-QLQ-C30
Description
EORTC-QLQ-C30 is a 30-item questionnaire developed to assess the quality of life of cancer patients. Subject's responses to 4 questions about their emotional functioning (Items 21-24) are scored on a 4-point scale (1=Not at All to 4=Very Much). Using linear transformation, raw scores are standardized, so that scores range from 0 to 100. A high score indicates a high / healthy level of functioning. Percentage of subjects with at least 10 point confirmed improvement from baseline in emotional functioning will be presented.
Time Frame
Up to 5 years after last patient randomized
Title
Percentage of subjects with either CR, or mCR, or PR, or HI in each treatment arm according to International Working Group for MDS (IWG-MDS) as per investigator assessment
Description
Response rate of subjects with complete remission (CR), or marrow remission (mCR), or partial remission (PR), or hematologic improvement (HI)
Time Frame
Up to 5 years after last patient randomized
Title
Percentage of subjects with SD in each treatment arm according to International Working Group for MDS (IWG-MDS) as per investigator assessment
Description
Response rate of subjects with stable disease (SD)
Time Frame
Up to 5 years after last patient randomized
Title
Progression Free Survival (PFS)
Description
Time from randomization to disease progression (including transformation to acute leukemia per WHO 2016 classification), relapse from CR according to International Working Group for MDS (IWG-MDS), or death due to any cause, whichever occurs first, as per investigator assessment
Time Frame
Up to 5 years after last patient randomized
Title
Leukemia-free survival
Description
Time from randomization to ≥ 20% blasts in bone marrow/peripheral blood (per WHO 2016 classification) or diagnosis of extramedullary acute leukemia, or death due to any cause
Time Frame
Up to 5 years after last patient randomized
Title
Number of transfusion dependent subjects at baseline who become Red Blood Cells/platelets transfusion independent after randomization
Description
Improvement in RBC/Platelets transfusion independence as per International Working Group for MDS (IWG-MDS) criteria
Time Frame
Up to 5 years after last patient randomized as per IWG-MDS criteria
Title
Percentage of transfusion dependent subjects at baseline who become Red Blood Cells/platelets transfusion independent after randomization
Description
Improvement in RBC/Platelets transfusion Independence as per International Working Group for MDS (IWG-MDS) criteria
Time Frame
Up to 5 years after last patient randomized
Title
Pharmacokinetics of MBG453 (parameter Cmax)
Description
Maximum (peak) MBG453 concentration [Cmax]
Time Frame
At Day 8 of each cycle (1 cycle = 28 days) until cycle 9, at day 8 of cycle 12 and every 6 cycles thereafter up to 150 day after end of study drug
Title
Pharmacokinetics of MBG453 (parameter AUC)
Description
Area Under the Curve [AUC]
Time Frame
At Day 8 of each cycle (1 cycle = 28 days) until cycle 9, at day 8 of cycle 12 and every 6 cycles thereafter up to 150 day after end of study drug
Title
Anti-drug Antibody (ADA) prevalence at baseline and ADA incidence on-treatment
Description
Immunogenicity of MBG453
Time Frame
At Day 8 of each cycle (1 cycle = 28 days) until cycle 9, at day 8 of cycle 12 and every 6 cycles thereafter up to 150 day after end of study drug
Title
Change from baseline in the European Quality of Life (EuroQol) - 5 Dimensions, 5 Level Questionnaire (EQ-5D-5L) score over time
Description
The EQ-5D-5L comprises 5 dimensions: mobility, self-care, usual activities, pain/discomfort, anxiety/depression. For each of the 5 dimensions, subject's responses are scored on a 5-point scale (1=no problem to 5=extreme problems). Change from baseline will be presented.
Time Frame
Up to 5 years after last patient randomized
Title
Change from baseline in the European Quality of Life (EuroQoL) - 5 Dimensions, 5 Level Questionnaire (EQ-5D-5L) Visual Analogue Scale over time
Description
The EQ-5D-5L VAS records the subject's self-rated health on a visual analogue scale numbered from 0 to 100, with 0 being "the worst health you can imagine" and 100 being "the best health you can imagine". Change from baseline will be presented.
Time Frame
Up to 5 years after last patient randomized
Title
Change from baseline to C12D1 of Global Health Status/Quality of Life scores using European Organization for Research and Treatment of Cancer's Core Quality of Life Questionnaire (EORTC-QLQ-C30)
Description
EORTC-QLQ-C30 is a 30-item questionnaire developed to assess the quality of life of cancer patients. Subject's responses to 2 questions (Items 29+30: "How would you rate your overall health during the past week?" and "How would you rate your overall quality of life during the past week?") are scored on a 7-point scale (1=Very Poor to 7=Excellent). Using linear transformation, raw scores are standardized, so that scores range from 0 to 100. A higher score indicates a better overall outcome. Change from baseline to Cycle 12 Day 1 will be presented.
Time Frame
Up to cycle 12 day 1 (C12D1)(1 cycle = 28 days)

10. Eligibility

Sex
All
Minimum Age & Unit of Time
18 Years
Accepts Healthy Volunteers
No
Eligibility Criteria
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 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, including persistent monocytosis) by local investigator assessment with WBC < 13 x 109/L at time of initial 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 (HSCT) 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 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 with any antineoplastic agents including for example chemotherapy, lenalidomide and hypomethylating agents (HMAs) such as decitibine and 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 Other protocol-defined Inclusion/Exclusion Criteria may apply.
Overall Study Officials:
First Name & Middle Initial & Last Name & Degree
Novartis Pharmaceuticals
Organizational Affiliation
Novartis Pharmaceuticals
Official's Role
Study Director
Facility Information:
Facility Name
Yuma Regional Cancer Center
City
Yuma
State/Province
Arizona
ZIP/Postal Code
85364
Country
United States
Facility Name
University Of California Los Angeles .
City
Los Angeles
State/Province
California
ZIP/Postal Code
90095
Country
United States
Facility Name
Yale University School Of Medicine Smilow Cancer Hospital
City
New Haven
State/Province
Connecticut
ZIP/Postal Code
06520
Country
United States
Facility Name
Mayo Clinic Jacksonville .
City
Jacksonville
State/Province
Florida
ZIP/Postal Code
32224
Country
United States
Facility Name
University Of Miami .
City
Miami
State/Province
Florida
ZIP/Postal Code
33136
Country
United States
Facility Name
Northwestern University Div of Hematology/Oncology
City
Chicago
State/Province
Illinois
ZIP/Postal Code
60611
Country
United States
Facility Name
Massachusetts General Hospital .
City
Boston
State/Province
Massachusetts
ZIP/Postal Code
02114
Country
United States
Facility Name
Hackensack University Medical Center SC
City
Hackensack
State/Province
New Jersey
ZIP/Postal Code
07601
Country
United States
Facility Name
Weill Cornell Medicine NY-Presb .
City
New York
State/Province
New York
ZIP/Postal Code
10021
Country
United States
Facility Name
University of Rochester Medical Center Dept.ofUniv.Rochester/JPWilmot
City
Rochester
State/Province
New York
ZIP/Postal Code
14642
Country
United States
Facility Name
University of Virginia University of Virginia
City
Charlottesville
State/Province
Virginia
ZIP/Postal Code
22903
Country
United States
Facility Name
Novartis Investigative Site
City
Pilar
State/Province
Buenos Aires
ZIP/Postal Code
B1629AHJ
Country
Argentina
Facility Name
Novartis Investigative Site
City
Woolloongabba
State/Province
Queensland
ZIP/Postal Code
4102
Country
Australia
Facility Name
Novartis Investigative Site
City
Clayton
State/Province
Victoria
ZIP/Postal Code
3168
Country
Australia
Facility Name
Novartis Investigative Site
City
Perth
State/Province
Western Australia
ZIP/Postal Code
6000
Country
Australia
Facility Name
Novartis Investigative Site
City
Graz
ZIP/Postal Code
8036
Country
Austria
Facility Name
Novartis Investigative Site
City
Innsbruck
ZIP/Postal Code
A-6020
Country
Austria
Facility Name
Novartis Investigative Site
City
Linz
ZIP/Postal Code
A-4010
Country
Austria
Facility Name
Novartis Investigative Site
City
Brasschaat
ZIP/Postal Code
2930
Country
Belgium
Facility Name
Novartis Investigative Site
City
Roeselare
ZIP/Postal Code
8800
Country
Belgium
Facility Name
Novartis Investigative Site
City
Florianopolis
State/Province
SC
ZIP/Postal Code
88034-000
Country
Brazil
Facility Name
Novartis Investigative Site
City
Sao Paulo
State/Province
SP
ZIP/Postal Code
04014-002
Country
Brazil
Facility Name
Novartis Investigative Site
City
Sao Paulo
State/Province
SP
ZIP/Postal Code
05319-000
Country
Brazil
Facility Name
Novartis Investigative Site
City
Calgary
State/Province
Alberta
ZIP/Postal Code
T2N 4N2
Country
Canada
Facility Name
Novartis Investigative Site
City
Toronto
State/Province
Ontario
ZIP/Postal Code
M4N 3M5
Country
Canada
Facility Name
Novartis Investigative Site
City
Vina del Mar
State/Province
Valparaiso
ZIP/Postal Code
2540364
Country
Chile
Facility Name
Novartis Investigative Site
City
Guangzhou
State/Province
Guangdong
ZIP/Postal Code
51000
Country
China
Facility Name
Novartis Investigative Site
City
Guangzhou
State/Province
Guangdong
ZIP/Postal Code
510515
Country
China
Facility Name
Novartis Investigative Site
City
Shenzhen
State/Province
Guangdong
ZIP/Postal Code
518037
Country
China
Facility Name
Novartis Investigative Site
City
Wuhan
State/Province
Hubei
ZIP/Postal Code
430022
Country
China
Facility Name
Novartis Investigative Site
City
Wuhan
State/Province
Hubei
ZIP/Postal Code
430030
Country
China
Facility Name
Novartis Investigative Site
City
Suzhou
State/Province
Jiangsu
ZIP/Postal Code
215006
Country
China
Facility Name
Novartis Investigative Site
City
Chang Chun
State/Province
Jilin
ZIP/Postal Code
130021
Country
China
Facility Name
Novartis Investigative Site
City
Tianjin
State/Province
Tianjin
ZIP/Postal Code
300020
Country
China
Facility Name
Novartis Investigative Site
City
Hangzhou
State/Province
Zhejiang
ZIP/Postal Code
310003
Country
China
Facility Name
Novartis Investigative Site
City
Beijing
ZIP/Postal Code
100029
Country
China
Facility Name
Novartis Investigative Site
City
Beijing
ZIP/Postal Code
100730
Country
China
Facility Name
Novartis Investigative Site
City
Chengdu
ZIP/Postal Code
610041
Country
China
Facility Name
Novartis Investigative Site
City
Jinan
ZIP/Postal Code
250012
Country
China
Facility Name
Novartis Investigative Site
City
Shanghai
ZIP/Postal Code
200025
Country
China
Facility Name
Novartis Investigative Site
City
Shanghai
ZIP/Postal Code
200233
Country
China
Facility Name
Novartis Investigative Site
City
Tianjin
ZIP/Postal Code
300052
Country
China
Facility Name
Novartis Investigative Site
City
Rionegro
State/Province
Antioquia
ZIP/Postal Code
054047
Country
Colombia
Facility Name
Novartis Investigative Site
City
Bogota
ZIP/Postal Code
110231
Country
Colombia
Facility Name
Novartis Investigative Site
City
Praha 2
State/Province
Czech Republic
ZIP/Postal Code
128 20
Country
Czechia
Facility Name
Novartis Investigative Site
City
Hradec Kralove
State/Province
CZE
ZIP/Postal Code
500 05
Country
Czechia
Facility Name
Novartis Investigative Site
City
Brno - Bohunice
ZIP/Postal Code
625 00
Country
Czechia
Facility Name
Novartis Investigative Site
City
Praha
ZIP/Postal Code
12808
Country
Czechia
Facility Name
Novartis Investigative Site
City
Helsinki
ZIP/Postal Code
FIN 00290
Country
Finland
Facility Name
Novartis Investigative Site
City
Kuopio
ZIP/Postal Code
70211
Country
Finland
Facility Name
Novartis Investigative Site
City
Grenoble
ZIP/Postal Code
38043
Country
France
Facility Name
Novartis Investigative Site
City
Lille Cedex
ZIP/Postal Code
59037
Country
France
Facility Name
Novartis Investigative Site
City
Paris 10
ZIP/Postal Code
75475
Country
France
Facility Name
Novartis Investigative Site
City
Toulouse
ZIP/Postal Code
31059
Country
France
Facility Name
Novartis Investigative Site
City
Tours
ZIP/Postal Code
37044
Country
France
Facility Name
Novartis Investigative Site
City
Vandoeuvre Les Nancy
ZIP/Postal Code
54511
Country
France
Facility Name
Novartis Investigative Site
City
Velbert
State/Province
North Rhine-Westphalia
ZIP/Postal Code
42551
Country
Germany
Facility Name
Novartis Investigative Site
City
Dresden
State/Province
Sachsen
ZIP/Postal Code
01307
Country
Germany
Facility Name
Novartis Investigative Site
City
Augsburg
ZIP/Postal Code
86179
Country
Germany
Facility Name
Novartis Investigative Site
City
Duesseldorf
ZIP/Postal Code
40479
Country
Germany
Facility Name
Novartis Investigative Site
City
Frankfurt
ZIP/Postal Code
60590
Country
Germany
Facility Name
Novartis Investigative Site
City
Greifswald
ZIP/Postal Code
17475
Country
Germany
Facility Name
Novartis Investigative Site
City
Jena
ZIP/Postal Code
07740
Country
Germany
Facility Name
Novartis Investigative Site
City
Kiel
ZIP/Postal Code
24116
Country
Germany
Facility Name
Novartis Investigative Site
City
Ulm
ZIP/Postal Code
89081
Country
Germany
Facility Name
Novartis Investigative Site
City
Alexandroupolis
State/Province
Evros
ZIP/Postal Code
681 00
Country
Greece
Facility Name
Novartis Investigative Site
City
Patras
ZIP/Postal Code
265 00
Country
Greece
Facility Name
Novartis Investigative Site
City
Ahmedabad
State/Province
Gujrat
ZIP/Postal Code
380009
Country
India
Facility Name
Novartis Investigative Site
City
Faridabad
State/Province
Haryana
ZIP/Postal Code
121 001
Country
India
Facility Name
Novartis Investigative Site
City
Madurai
State/Province
Tamil NADU
ZIP/Postal Code
625107
Country
India
Facility Name
Novartis Investigative Site
City
Kolkata
State/Province
West Bengal
ZIP/Postal Code
700014
Country
India
Facility Name
Novartis Investigative Site
City
Kolkata
State/Province
West Bengal
ZIP/Postal Code
700160
Country
India
Facility Name
Novartis Investigative Site
City
Delhi
ZIP/Postal Code
110 085
Country
India
Facility Name
Novartis Investigative Site
City
Afula
ZIP/Postal Code
1834111
Country
Israel
Facility Name
Novartis Investigative Site
City
Tel Aviv
ZIP/Postal Code
6423906
Country
Israel
Facility Name
Novartis Investigative Site
City
Bologna
State/Province
BO
ZIP/Postal Code
40138
Country
Italy
Facility Name
Novartis Investigative Site
City
Catania
State/Province
CT
ZIP/Postal Code
95123
Country
Italy
Facility Name
Novartis Investigative Site
City
Firenze
State/Province
FI
ZIP/Postal Code
50134
Country
Italy
Facility Name
Novartis Investigative Site
City
Genova
State/Province
GE
ZIP/Postal Code
16132
Country
Italy
Facility Name
Novartis Investigative Site
City
Milano
State/Province
MI
ZIP/Postal Code
20162
Country
Italy
Facility Name
Novartis Investigative Site
City
Rozzano
State/Province
MI
ZIP/Postal Code
20089
Country
Italy
Facility Name
Novartis Investigative Site
City
Reggio Calabria
State/Province
RC
ZIP/Postal Code
89124
Country
Italy
Facility Name
Novartis Investigative Site
City
Roma
State/Province
RM
ZIP/Postal Code
00133
Country
Italy
Facility Name
Novartis Investigative Site
City
Nagoya
State/Province
Aichi
ZIP/Postal Code
464 8681
Country
Japan
Facility Name
Novartis Investigative Site
City
Kashiwa
State/Province
Chiba
ZIP/Postal Code
277 8577
Country
Japan
Facility Name
Novartis Investigative Site
City
Fukuoka city
State/Province
Fukuoka
ZIP/Postal Code
812-8582
Country
Japan
Facility Name
Novartis Investigative Site
City
Sapporo
State/Province
Hokkaido
ZIP/Postal Code
064 0804
Country
Japan
Facility Name
Novartis Investigative Site
City
Isehara
State/Province
Kanagawa
ZIP/Postal Code
259-1193
Country
Japan
Facility Name
Novartis Investigative Site
City
Sendai city
State/Province
Miyagi
ZIP/Postal Code
980 8574
Country
Japan
Facility Name
Novartis Investigative Site
City
Nagasaki-city
State/Province
Nagasaki
ZIP/Postal Code
852-8501
Country
Japan
Facility Name
Novartis Investigative Site
City
Osaka Sayama
State/Province
Osaka
ZIP/Postal Code
589 8511
Country
Japan
Facility Name
Novartis Investigative Site
City
Bunkyo-ku
State/Province
Tokyo
ZIP/Postal Code
113-8603
Country
Japan
Facility Name
Novartis Investigative Site
City
Osaka
ZIP/Postal Code
545-8586
Country
Japan
Facility Name
Novartis Investigative Site
City
Yamagata
ZIP/Postal Code
990 9585
Country
Japan
Facility Name
Novartis Investigative Site
City
Seoul
State/Province
Seocho Gu
ZIP/Postal Code
06591
Country
Korea, Republic of
Facility Name
Novartis Investigative Site
City
Seoul
ZIP/Postal Code
03080
Country
Korea, Republic of
Facility Name
Novartis Investigative Site
City
Seoul
ZIP/Postal Code
03722
Country
Korea, Republic of
Facility Name
Novartis Investigative Site
City
Seoul
ZIP/Postal Code
05505
Country
Korea, Republic of
Facility Name
Novartis Investigative Site
City
Seoul
ZIP/Postal Code
06351
Country
Korea, Republic of
Facility Name
Novartis Investigative Site
City
Beirut
ZIP/Postal Code
1107 2020
Country
Lebanon
Facility Name
Novartis Investigative Site
City
Vilnius
ZIP/Postal Code
LT-08661
Country
Lithuania
Facility Name
Novartis Investigative Site
City
Kuching
State/Province
Sarawak
ZIP/Postal Code
93586
Country
Malaysia
Facility Name
Novartis Investigative Site
City
Kuala Lumpur
ZIP/Postal Code
59100
Country
Malaysia
Facility Name
Novartis Investigative Site
City
Pulau Pinang
ZIP/Postal Code
10990
Country
Malaysia
Facility Name
Novartis Investigative Site
City
Selangor
ZIP/Postal Code
68000
Country
Malaysia
Facility Name
Novartis Investigative Site
City
Mexico
State/Province
Distrito Federal
ZIP/Postal Code
06726
Country
Mexico
Facility Name
Novartis Investigative Site
City
Satelite
State/Province
Edo Mexico
ZIP/Postal Code
53100
Country
Mexico
Facility Name
Novartis Investigative Site
City
Morelia
State/Province
Michoacan
ZIP/Postal Code
58260
Country
Mexico
Facility Name
Novartis Investigative Site
City
Estado de Mexico
ZIP/Postal Code
52787
Country
Mexico
Facility Name
Novartis Investigative Site
City
Groningen
ZIP/Postal Code
9713 GZ
Country
Netherlands
Facility Name
Novartis Investigative Site
City
Muscat
ZIP/Postal Code
123
Country
Oman
Facility Name
Novartis Investigative Site
City
Lisboa
ZIP/Postal Code
1099 023
Country
Portugal
Facility Name
Novartis Investigative Site
City
Porto
ZIP/Postal Code
4200-072
Country
Portugal
Facility Name
Novartis Investigative Site
City
Saint Petersburg
ZIP/Postal Code
197022
Country
Russian Federation
Facility Name
Novartis Investigative Site
City
St Petersburg
ZIP/Postal Code
191024
Country
Russian Federation
Facility Name
Novartis Investigative Site
City
Riyadh
ZIP/Postal Code
11211
Country
Saudi Arabia
Facility Name
Novartis Investigative Site
City
Singapore
ZIP/Postal Code
119228
Country
Singapore
Facility Name
Novartis Investigative Site
City
Singapore
ZIP/Postal Code
169608
Country
Singapore
Facility Name
Novartis Investigative Site
City
Sevilla
State/Province
Andalucia
ZIP/Postal Code
41013
Country
Spain
Facility Name
Novartis Investigative Site
City
Oviedo
State/Province
Asturias
ZIP/Postal Code
33011
Country
Spain
Facility Name
Novartis Investigative Site
City
Salamanca
State/Province
Castilla Y Leon
ZIP/Postal Code
37007
Country
Spain
Facility Name
Novartis Investigative Site
City
Badalona
State/Province
Catalunya
ZIP/Postal Code
08916
Country
Spain
Facility Name
Novartis Investigative Site
City
Barcelona
State/Province
Catalunya
ZIP/Postal Code
08035
Country
Spain
Facility Name
Novartis Investigative Site
City
Valencia
State/Province
Comunidad Valenciana
ZIP/Postal Code
46010
Country
Spain
Facility Name
Novartis Investigative Site
City
Valencia
State/Province
Comunidad Valenciana
ZIP/Postal Code
46026
Country
Spain
Facility Name
Novartis Investigative Site
City
Madrid
ZIP/Postal Code
28009
Country
Spain
Facility Name
Novartis Investigative Site
City
Bern
ZIP/Postal Code
3010
Country
Switzerland
Facility Name
Novartis Investigative Site
City
Zürich
ZIP/Postal Code
8091
Country
Switzerland
Facility Name
Novartis Investigative Site
City
Hualien City
State/Province
Hualien
ZIP/Postal Code
970
Country
Taiwan
Facility Name
Novartis Investigative Site
City
Kaohsiung
ZIP/Postal Code
83301
Country
Taiwan
Facility Name
Novartis Investigative Site
City
Liouying Township
ZIP/Postal Code
736005
Country
Taiwan
Facility Name
Novartis Investigative Site
City
Taichung
ZIP/Postal Code
40447
Country
Taiwan
Facility Name
Novartis Investigative Site
City
Taipei
ZIP/Postal Code
10002
Country
Taiwan
Facility Name
Novartis Investigative Site
City
Taoyuan
ZIP/Postal Code
33305
Country
Taiwan
Facility Name
Novartis Investigative Site
City
Songkhla
State/Province
Hat Yai
ZIP/Postal Code
90110
Country
Thailand
Facility Name
Novartis Investigative Site
City
Khon Kaen
State/Province
THA
ZIP/Postal Code
40002
Country
Thailand
Facility Name
Novartis Investigative Site
City
Bangkok
ZIP/Postal Code
10330
Country
Thailand
Facility Name
Novartis Investigative Site
City
Bangkok
ZIP/Postal Code
10400
Country
Thailand
Facility Name
Novartis Investigative Site
City
Bangkok
ZIP/Postal Code
10700
Country
Thailand
Facility Name
Novartis Investigative Site
City
Chiang Mai
ZIP/Postal Code
50200
Country
Thailand
Facility Name
Novartis Investigative Site
City
Ankara
ZIP/Postal Code
06100
Country
Turkey
Facility Name
Novartis Investigative Site
City
Edirne
ZIP/Postal Code
22030
Country
Turkey
Facility Name
Novartis Investigative Site
City
Istanbul
ZIP/Postal Code
34890
Country
Turkey
Facility Name
Novartis Investigative Site
City
Izmir
ZIP/Postal Code
35040
Country
Turkey
Facility Name
Novartis Investigative Site
City
Samsun
ZIP/Postal Code
55139
Country
Turkey
Facility Name
Novartis Investigative Site
City
Portsmouth
State/Province
Hants
ZIP/Postal Code
PO6 3LY
Country
United Kingdom
Facility Name
Novartis Investigative Site
City
Edinburgh
ZIP/Postal Code
EH4 2XU
Country
United Kingdom
Facility Name
Novartis Investigative Site
City
Manchester
ZIP/Postal Code
M20 4BX
Country
United Kingdom
Facility Name
Novartis Investigative Site
City
Nottingham
ZIP/Postal Code
NG5 1PB
Country
United Kingdom

12. IPD Sharing Statement

Plan to Share IPD
Yes
IPD Sharing Plan Description
Novartis is committed to sharing with qualified external researchers, access to patient-level data and supporting clinical documents from eligible studies. These requests are reviewed and approved by an independent expert panel on the basis of scientific merit. All data provided is anonymized to respect the privacy of patients who have participated in the trial in line with applicable laws and regulations.

Learn more about this trial

Study of Efficacy and Safety of MBG453 in Combination With Azacitidine in Subjects With Intermediate, High or Very High Risk Myelodysplastic Syndrome (MDS) as Per IPSS-R, or Chronic Myelomonocytic Leukemia-2 (CMML-2)

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