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| ID | Type | Description | Link |
|---|---|---|---|
| 2019-002089-11 | EudraCT Number |
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In December 2023, Novartis decided to terminate the sabatolimab clinical development program early after Phase II (MDS1) and Phase III (MDS2) studies failed to meet their primary objectives. The termination was not due to safety concerns.
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This was a Phase III multi-center, randomized, two-arm parallel-group, double-blind, placebo-controlled study of MBG453 or placebo added to azacitidine in adult participants with intermediate, high or very high-risk myelodysplastic syndrome (MDS) as per IPSS-R, or Chronic Myelomonocytic Leukemia-2 (CMML-2) who 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 was to assess clinical effects of MBG453 in combination with azacytidine in adult participants with IPSS-R intermediate, high, very high risk MDS and CMML-2.
This was a Phase III multi-center, randomized, two-arm parallel-group, double-blind, placebo-controlled study of MBG453 or placebo added to azacitidine in adult participants 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 was to compare overall survival (OS) in the MBG453 plus azacitidine arm versus placebo plus azacitidine arm where OS was the time from randomization until death due to any cause.
Participants were randomized in a 1:1 ratio to treatment arms as follow: MBG453 800 mg IV Q4W plus azacitidine, Placebo IV Q4W plus azacitidine.
The randomization was stratified into 4 groups: intermediate risk MDS, high risk MDS, very high risk MDS and CMML-2.
All participants who discontinued both study treatments were to have entered 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 participant was randomized.
Participants were receiving treatment until they experienced progression of disease (including transformation to acute leukemia per WHO 2016 classification), experienced unacceptable toxicity or discontinued the study treatment for other reasons.
Continuation of study treatment beyond progression (excluding transformation to acute leukemia: continuation in this case was not possible) could have been possible in selected participants.
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| Label | Type | Description | Intervention Names |
|---|---|---|---|
| Sabatolimab (MBG453) + Azacitidine | Experimental | Participants received sabatolimab plus Azacitidine |
|
| Placebo + Azacitidine | Placebo Comparator | Participants received placebo plus Azacitidine. |
|
| Name | Type | Description | Arm Group Labels | Other Names |
|---|---|---|---|---|
| Sabatolimab | Drug | A dose of MBG453 800 mg was administered intravenously (IV) every 4 weeks (Q4W). |
|
| Measure | Description | Time Frame |
|---|---|---|
| Overall Survival (OS) (Primary Efficacy Results) | OS is the time from randomization until death due to any cause. | up to approx. 39 months |
| Overall Survival (OS) (Final Efficacy Results) | OS is the time from randomization until death due to any cause. | up to approx. 52 months |
| Measure | Description | Time Frame |
|---|---|---|
| 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 participants. 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 to definitive deterioration of fatigue is defined as time from randomization to at least 3 points worsening from baseline in FACIT-fatigue scores with no subsequently observed improvement above this threshold, or death due to any cause, whichever occurred first. |
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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):
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:
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| Name | Affiliation | Role |
|---|---|---|
| Novartis Pharmaceuticals | Novartis Pharmaceuticals | Study Director |
| Facility | Status | City | State | ZIP | Country | Contacts |
|---|---|---|---|---|---|---|
| Yuma Regional Cancer Center | Yuma | Arizona | 85364 | United States | ||
| University of California LA |
| PubMed Identifier | Type | Citation | Retractions |
|---|---|---|---|
| 37083373 | Derived | Zeidan AM, Giagounidis A, Sekeres MA, Xiao Z, Sanz GF, Hoef MV, Ma F, Hertle S, Santini V. STIMULUS-MDS2 design and rationale: a phase III trial with the anti-TIM-3 sabatolimab (MBG453) + azacitidine in higher risk MDS and CMML-2. Future Oncol. 2023 Mar;19(9):631-642. doi: 10.2217/fon-2022-1237. Epub 2023 Apr 21. |
| Label | URL |
|---|---|
| A Plain Language Trial Summary is available on www.novctrd.com | View source |
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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 participants who have participated in the trial in line with applicable laws and regulations.
This trial data is currently available according to the process described on www.clinicalstudydatarequest.com
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This study randomized participants in 149 centers in 36 participating countries.
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| ID | Title | Description |
|---|---|---|
| FG000 | Sabatolimab (MBG453) + Azacitidine | Participants were randomized to sabatolimab plus Azacitidine |
| FG001 | Placebo + Azacitidine | Participants were randomized to placebo plus Azacitidine |
| Title | Milestones | Reasons Not Completed | |||||
|---|---|---|---|---|---|---|---|
| Overall Study |
|
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| Type | Includes Protocol | Includes SAP | Includes ICF | Document Label | Document Date | Document Uploaded Date | Document File Name |
|---|---|---|---|---|---|---|---|
| Prot | Yes | No | No | Study Protocol | Jun 23, 2022 | Sep 18, 2025 |
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Randomized, double-blind
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| Azacitidine | Drug | A dose of Azacitidine 75 mg/m2 was administered IV or subcutaneously (SC) on Day 1-7, or Day 1-5, 8 and 9. |
|
| Placebo | Drug | A dose of placebo 800 mg was administered intravenously every 4 weeks (Q4W). |
|
| up to approx. 52 months |
| Key Secondary Endpoint 2: Red Blood Cell (RBC) Annualized Transfusion Free Rate for Transfusion | Annualized transfusion free rate is defined as the average number of days in RBC transfusion-free intervals in a year (i.e., the total number of days in RBC transfusion-free intervals divided by the total days in the study multiplied by 365.25), where RBC transfusion-free intervals correspond to cumulative times of intervals with no evidence of RBC transfusion for at least 8 weeks at any point after randomization until death due to any cause. | up to approx. 52 months |
| Key Secondary Endpoint 3: Percentage of Participants With at Least 3 Point Confirmed Improvement From Baseline in FACIT-fatigue Scores | FACIT-Fatigue score is a 13-item questionnaire designed to assess fatigue in cancer participants. 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. The responder is defined as having 3 points improvement from baseline confirmed by a second improvement of 3 points at any time, regardless of preceding worsening. A participant who could not improve was considered as a non-responder. | up to approx. 52 months |
| Key Secondary Endpoint 4: Percentage of Participants With at Least 10 Point Confirmed Improvement From Baseline in Physical Functioning 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 participants. Participants' 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. The responder is defined as having 10 points improvement from baseline confirmed by a second improvement of 10 points at any time, regardless of preceding worsening. A participant who could not improve was considered as a non-responder. | up to approx. 52 months |
| Key Secondary Endpoint 5: Percentage of Participants 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 participants. Participants' 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. The responder is defined as having 10 points improvement from baseline confirmed by a second improvement of 10 points at any time, regardless of preceding worsening. A participant who could not improve was considered as a non-responder. | up to approx. 52 months |
| Percentage of Participants 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 participants with complete remission (CR), or marrow remission (mCR), or partial remission (PR), or hematologic improvement (HI). CR: where the Bone marrow: ≤ 5% blasts with normal maturation of all cell lineages and Peripheral blood: where Hgb ≥ 10 g/dL AND Platelets ≥ 100*109/L AND Neutrophils ≥ 1.0*109/L AND Peripheral blasts 0%. mCR: Bone marrow: ≤ 5% blasts and blast count decrease by ≥ 50% compared to baseline; Peripheralblood/transfusion: Marrow CR may be achieved with or without improved blood counts or with or without transfusions PR: All CR criteria except bone marrow: ≥50% decrease from baseline in blasts in bone marrow AND blast count in bone marrow >5%. HI: restoration or enhancement of the function of the body's blood cell-producing system that must last as least 8 weeks. | up to approx. 52 months |
| Percentage of Participants With Stable Disease (SD) According to International Working Group for MDS (IWG-MDS) as Per Investigator Assessment | Response rate of participants with stable disease. SD is the failure to achieve at least partial response (PR), but no evidence of progression for >8 weeks. | up to approx. 52 months |
| Progression Free Survival (PFS) | PFS is defined as the time from randomization to disease progression (including transformation to acute leukemia per WHO 2016), relapse from CR (IWG-MDS), or death. Disease progression: bone marrow blasts increase ≥ 50% over baseline, to > 5% if initially < 5%, > 10% if 5%-<10%, or > 20% if 10%-<20%. Includes a peripheral blood count decrease ≥ 50% from maximum remission/response levels: neutrophils < 1.0x109/L, platelets < 100x109/L, or hemoglobin drop ≥ 2 g/dL to < 10 g/dL, becoming transfusion-dependent. Relapse from CR: baseline bone marrow blast % return, neutrophils decrease ≥ 50% to < 1.0x109/L, platelets decrease ≥ 50% to < 100x109/L, or hemoglobin drop ≥ 1.5 g/dL to < 10 g/dL, becoming transfusion-dependent. Leukemia transformation: > 20% blasts per WHO 2016 (Arber et al 2016). | up to approx. 52 months |
| Leukemia-free Survival (LFS) | LFS is defined as the 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 | up to approx. 52 months |
| Number of Participants Who Become Red Blood Cells (RBC) Transfusion Independent After Randomization | Improvement in RBC transfusion independence as per International Working Group for MDS (IWG-MDS) criteria. RBC transfusion independence was defined as having received 0 units of RBC transfusions during at least 8 consecutive weeks after randomization. The number of participants was shown in only those with transfusion dependence at baseline (BL). | up to approx. 52 months |
| Number of Participants Who Become Platelets Transfusion Independent After Randomization | Improvement in Platelets transfusion independence as per International Working Group for MDS (IWG-MDS) criteria. Platelets transfusion independence was defined as having received 0 units of Platelets transfusions during at least 8 consecutive weeks after randomization. The number of participants was shown in only those with transfusion dependence at baseline (BL). | up to approx. 52 months |
| Pharmacokinetics of MBG453 (Parameter Cmax) | Cmax is the maximum (peak) observed drug concentration after single dose administration (mass x volume-1). | 0 hr (pre-dose) & 2 hrs (post-dose) of Cycle (C) 1 Day (D) 8 and 0 hr (pre-dose) & 2 hrs (post-dose) of C3D8 |
| Pharmacokinetics of MBG453 (Parameter AUC) | AUCinf is the AUC from time zero to infinity (mass x time x volume-1). | 0 hr (pre-dose) & 2 hrs (post-dose) of Cycle (C) 1 Day (D) 8 and 0 hr (pre-dose) & 2 hrs (post-dose) of C3D8 |
| ADA Prevalence at Baseline and ADA-positive Participants On-treatment | Anti-drug Antibody (ADA) prevalence is the number of participants with at least one sample meeting the criteria at baseline. ADA-positive participants were calculated as the number of participants with at least one on-treatment ADA-positive sample divided by the number of participants with a determinant baseline IG sample and at least one determinant post-baseline IG sample. | Baseline, up to approx. 39 months |
| 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 is being presented for EQ Index score. Index score is defined as a weighted combination of the levels of the 5-dimention scales, ranging from 0 to 1 (perfect health), with higher scores indicating better health-related quality of life. The United States value set from Pickard et al 2019 was used. | Baseline, every 3 cycles up to C48D1 (1 cycle = 28 days) |
| Change From Baseline in the European Quality of Life (EuroQoL) - 5 Dimensions, 5 Level Questionnaire (EQ-5D-5L) Visual Analogue Scale (VAS) Over Time | The EQ-5D-5L VAS records the participant'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 was presented. | Baseline, every 3 cycles up to C48D1 (1 cycle = 28 days) |
| Change From Baseline 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 participants. Participant'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 as presented. | Up to Cycle 12 Day 1 (C12D1) (1 cycle = 28 days) |
| Los Angeles |
| California |
| 90095 |
| United States |
| Yale University School Of Medicine | New Haven | Connecticut | 06520 | United States |
| Mayo Clinic Jacksonville | Jacksonville | Florida | 32224 | United States |
| University Of Miami | Miami | Florida | 33136 | United States |
| Northwestern University | Chicago | Illinois | 60611 | United States |
| Massachusetts General Hospital | Boston | Massachusetts | 02114 | United States |
| Hackensack University Medical Ctr | Hackensack | New Jersey | 07601 | United States |
| Weill Cornell Medicine NY-Presb | New York | New York | 10021 | United States |
| University of Rochester Medical Ctr | Rochester | New York | 14642 | United States |
| University of Virginia | Charlottesville | Virginia | 22903 | United States |
| Novartis Investigative Site | Pilar | Buenos Aires | B1629AHJ | Argentina |
| Novartis Investigative Site | Wooloongabba | Queensland | 4102 | Australia |
| Novartis Investigative Site | Clayton | Victoria | 3168 | Australia |
| Novartis Investigative Site | Perth | Western Australia | 6000 | Australia |
| Novartis Investigative Site | Innsbruck | Tyrol | 6020 | Austria |
| Novartis Investigative Site | Graz | 8036 | Austria |
| Novartis Investigative Site | Linz | 4020 | Austria |
| Novartis Investigative Site | Roeselare | West-Vlaanderen | 8800 | Belgium |
| Novartis Investigative Site | Brasschaat | 2930 | Belgium |
| Novartis Investigative Site | Florianópolis | Santa Catarina | 88020-210 | Brazil |
| Novartis Investigative Site | São Paulo | São Paulo | 04014-002 | Brazil |
| Novartis Investigative Site | São Paulo | São Paulo | 05319-000 | Brazil |
| Novartis Investigative Site | Calgary | Alberta | T2N 4N2 | Canada |
| Novartis Investigative Site | Toronto | Ontario | M4N 3M5 | Canada |
| Novartis Investigative Site | Viña del Mar | Valparaiso | 2540364 | Chile |
| Novartis Investigative Site | Guangzhou | Guangdong | 510080 | China |
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| Novartis Investigative Site | Alexandroupoli | 681 00 | Greece |
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| Novartis Investigative Site | Ahmedabad | Gujarat | 380009 | India |
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| Novartis Investigative Site | Barcelona | Catalonia | 08035 | Spain |
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| Novartis Investigative Site | Madrid | 28009 | Spain |
| Novartis Investigative Site | Seville | 41013 | Spain |
| Novartis Investigative Site | Valencia | 46010 | Spain |
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| Novartis Investigative Site | Bern | 3010 | Switzerland |
| Novartis Investigative Site | Zurich | 8091 | Switzerland |
| Novartis Investigative Site | Hualien City | 970 | Taiwan |
| Novartis Investigative Site | Kaohsiung City | 83301 | Taiwan |
| Novartis Investigative Site | Liouying Township | 736005 | Taiwan |
| Novartis Investigative Site | Taichung | 40447 | Taiwan |
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| Novartis Investigative Site | Songkhla | Hat Yai | 90110 | Thailand |
| Novartis Investigative Site | Khon Kaen | THA | 40002 | Thailand |
| Novartis Investigative Site | Bangkok | 10330 | Thailand |
| Novartis Investigative Site | Bangkok | 10400 | Thailand |
| Novartis Investigative Site | Bangkok | 10700 | Thailand |
| Novartis Investigative Site | Chiang Mai | 50200 | Thailand |
| Novartis Investigative Site | Samsun | Atakum | 55200 | Turkey (Türkiye) |
| Novartis Investigative Site | Edirne | Merkez | 22030 | Turkey (Türkiye) |
| Novartis Investigative Site | Istanbul | Pendik | 34899 | Turkey (Türkiye) |
| Novartis Investigative Site | Ankara | 06230 | Turkey (Türkiye) |
| Novartis Investigative Site | Izmir | 35100 | Turkey (Türkiye) |
| Novartis Investigative Site | Portsmouth | Hants | PO6 3LY | United Kingdom |
| Novartis Investigative Site | Edinburgh | Scotland | EH4 2XU | United Kingdom |
| Novartis Investigative Site | Manchester | M20 2BX | United Kingdom |
| Novartis Investigative Site | Nottingham | NG5 1PB | United Kingdom |
| Participants Treated |
|
| Participants Not treated |
|
| Discontinued from treatment |
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| Entered post-treatment |
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| Did not enter post-treatment |
|
| Discontinued from study |
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| Full Analysis See (FAS) |
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| Safety Set | Two participants (one per arm) did not receive treatment and were excluded from the Safety Set. One sabatolimab-assigned participant withdrew consent pre-treatment. Another, randomized to placebo, progressed to AML before treatment and died. One sabatolimab-assigned participant progressed to AML, received only azacitidine, and was analyzed in the placebo Safety Set. |
|
| COMPLETED | Completed = Completed treatment |
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| NOT COMPLETED |
|
|
The Full Analysis Set (FAS) comprised of all participants to whom study treatment had been assigned by randomization.
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| ID | Title | Description |
|---|---|---|
| BG000 | Sabatolimab (MBG453) + Azacitidine | Participants were randomized to sabatolimab plus Azacitidine |
| BG001 | Placebo + Azacitidine | Participants were randomized to placebo plus Azacitidine |
| BG002 | Total | Total of all reporting groups |
| Units | Counts |
|---|---|
| Participants |
|
| Title | Description | Population Description | Parameter Type | Dispersion Type | Unit of Measure | Calculate Percentage | Denominator Units Selected | Denominators | Classes | ||||||||||
|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|
| Age, Continuous | Mean | Standard Deviation | Years |
| |||||||||||||||
| Sex: Female, Male | Count of Participants | Participants |
| ||||||||||||||||
| Race/Ethnicity, Customized | Count of Participants | Participants |
| ||||||||||||||||
| ECOG performance status | Eastern Cooperative Oncology Group (ECOG) performance status scale is a widely used standard of care criteria used to assess the functional status of a patient with cancer to measure how the disease impacts the patient's daily living abilities. This scale has a range from 0 - 5. The higher the grade, the worse the patient's abilities: 0 implies fully active, able to carry on all pre-disease performance without restriction and 5 implies death. | Count of Participants | Participants |
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| Type | Title | Description | Population Description | Reporting Status | Anticipated Posting Date | Parameter Type | Dispersion Type | Unit of Measure | Calculate Percentage | Time Frame | Units Analyzed | Denominator Units Selected | Arm/Group Information | Denominators | Classes | Analyses | |||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||
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| Primary | Overall Survival (OS) (Primary Efficacy Results) | OS is the time from randomization until death due to any cause. | FAS comprised of all participants to whom study treatment had been assigned by randomization. | Posted | Median | 95% Confidence Interval | Months | up to approx. 39 months |
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| Primary | Overall Survival (OS) (Final Efficacy Results) | OS is the time from randomization until death due to any cause. | FAS comprised of all participants to whom study treatment had been assigned by randomization. | Posted | Median | 95% Confidence Interval | Months | up to approx. 52 months |
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| Secondary | 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 participants. 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 to definitive deterioration of fatigue is defined as time from randomization to at least 3 points worsening from baseline in FACIT-fatigue scores with no subsequently observed improvement above this threshold, or death due to any cause, whichever occurred first. | FAS comprised of all participants to whom study treatment had been assigned by randomization. | Posted | Median | 95% Confidence Interval | Months | up to approx. 52 months |
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| Secondary | Key Secondary Endpoint 2: Red Blood Cell (RBC) Annualized Transfusion Free Rate for Transfusion | Annualized transfusion free rate is defined as the average number of days in RBC transfusion-free intervals in a year (i.e., the total number of days in RBC transfusion-free intervals divided by the total days in the study multiplied by 365.25), where RBC transfusion-free intervals correspond to cumulative times of intervals with no evidence of RBC transfusion for at least 8 weeks at any point after randomization until death due to any cause. | FAS comprised of all participants to whom study treatment had been assigned by randomization. | Posted | Median | Full Range | days per year | up to approx. 52 months |
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| Secondary | Key Secondary Endpoint 3: Percentage of Participants With at Least 3 Point Confirmed Improvement From Baseline in FACIT-fatigue Scores | FACIT-Fatigue score is a 13-item questionnaire designed to assess fatigue in cancer participants. 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. The responder is defined as having 3 points improvement from baseline confirmed by a second improvement of 3 points at any time, regardless of preceding worsening. A participant who could not improve was considered as a non-responder. | FAS comprised of all participants to whom study treatment had been assigned by randomization. | Posted | Number | 95% Confidence Interval | Percentage of participants with response | up to approx. 52 months |
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| Secondary | Key Secondary Endpoint 4: Percentage of Participants With at Least 10 Point Confirmed Improvement From Baseline in Physical Functioning 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 participants. Participants' 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. The responder is defined as having 10 points improvement from baseline confirmed by a second improvement of 10 points at any time, regardless of preceding worsening. A participant who could not improve was considered as a non-responder. | FAS comprised of all participants to whom study treatment had been assigned by randomization. | Posted | Number | 95% Confidence Interval | Percentage of participants with response | up to approx. 52 months |
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| Secondary | Key Secondary Endpoint 5: Percentage of Participants 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 participants. Participants' 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. The responder is defined as having 10 points improvement from baseline confirmed by a second improvement of 10 points at any time, regardless of preceding worsening. A participant who could not improve was considered as a non-responder. | FAS comprised of all participants to whom study treatment had been assigned by randomization. | Posted | Number | 95% Confidence Interval | Percentage of participants | up to approx. 52 months |
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| Secondary | Percentage of Participants 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 participants with complete remission (CR), or marrow remission (mCR), or partial remission (PR), or hematologic improvement (HI). CR: where the Bone marrow: ≤ 5% blasts with normal maturation of all cell lineages and Peripheral blood: where Hgb ≥ 10 g/dL AND Platelets ≥ 100*109/L AND Neutrophils ≥ 1.0*109/L AND Peripheral blasts 0%. mCR: Bone marrow: ≤ 5% blasts and blast count decrease by ≥ 50% compared to baseline; Peripheralblood/transfusion: Marrow CR may be achieved with or without improved blood counts or with or without transfusions PR: All CR criteria except bone marrow: ≥50% decrease from baseline in blasts in bone marrow AND blast count in bone marrow >5%. HI: restoration or enhancement of the function of the body's blood cell-producing system that must last as least 8 weeks. | FAS comprised of all participants to whom study treatment had been assigned by randomization. | Posted | Number | 95% Confidence Interval | Percentage of participants | up to approx. 52 months |
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| Secondary | Percentage of Participants With Stable Disease (SD) According to International Working Group for MDS (IWG-MDS) as Per Investigator Assessment | Response rate of participants with stable disease. SD is the failure to achieve at least partial response (PR), but no evidence of progression for >8 weeks. | FAS comprised of all participants to whom study treatment had been assigned by randomization. | Posted | Number | Percentage of participants | up to approx. 52 months |
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| Secondary | Progression Free Survival (PFS) | PFS is defined as the time from randomization to disease progression (including transformation to acute leukemia per WHO 2016), relapse from CR (IWG-MDS), or death. Disease progression: bone marrow blasts increase ≥ 50% over baseline, to > 5% if initially < 5%, > 10% if 5%-<10%, or > 20% if 10%-<20%. Includes a peripheral blood count decrease ≥ 50% from maximum remission/response levels: neutrophils < 1.0x109/L, platelets < 100x109/L, or hemoglobin drop ≥ 2 g/dL to < 10 g/dL, becoming transfusion-dependent. Relapse from CR: baseline bone marrow blast % return, neutrophils decrease ≥ 50% to < 1.0x109/L, platelets decrease ≥ 50% to < 100x109/L, or hemoglobin drop ≥ 1.5 g/dL to < 10 g/dL, becoming transfusion-dependent. Leukemia transformation: > 20% blasts per WHO 2016 (Arber et al 2016). | FAS comprised of all participants to whom study treatment had been assigned by randomization. | Posted | Median | 95% Confidence Interval | Months | up to approx. 52 months |
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| Secondary | Leukemia-free Survival (LFS) | LFS is defined as the 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 | FAS comprised of all participants to whom study treatment had been assigned by randomization. | Posted | Median | 95% Confidence Interval | Months | up to approx. 52 months |
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| Secondary | Number of Participants Who Become Red Blood Cells (RBC) Transfusion Independent After Randomization | Improvement in RBC transfusion independence as per International Working Group for MDS (IWG-MDS) criteria. RBC transfusion independence was defined as having received 0 units of RBC transfusions during at least 8 consecutive weeks after randomization. The number of participants was shown in only those with transfusion dependence at baseline (BL). | Participants of the full analysis set (FAS) who were RBC transfusion dependent at baseline. | Posted | Count of Participants | Participants | up to approx. 52 months |
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| Secondary | Number of Participants Who Become Platelets Transfusion Independent After Randomization | Improvement in Platelets transfusion independence as per International Working Group for MDS (IWG-MDS) criteria. Platelets transfusion independence was defined as having received 0 units of Platelets transfusions during at least 8 consecutive weeks after randomization. The number of participants was shown in only those with transfusion dependence at baseline (BL). | Participants of the full analysis set (FAS) who were Platelets transfusion dependent at baseline. | Posted | Count of Participants | Participants | up to approx. 52 months |
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| Secondary | Pharmacokinetics of MBG453 (Parameter Cmax) | Cmax is the maximum (peak) observed drug concentration after single dose administration (mass x volume-1). | Pharmacokinetic Analysis Set (PAS) included all participants in the Safety Set, who had at least one evaluable PK concentration. Safety Set included all participants who received at least one dose of any component of the study treatment, and they were analyzed according to the study treatment they received. | Posted | Geometric Mean | Geometric Coefficient of Variation | ug/ml | 0 hr (pre-dose) & 2 hrs (post-dose) of Cycle (C) 1 Day (D) 8 and 0 hr (pre-dose) & 2 hrs (post-dose) of C3D8 |
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| Secondary | Pharmacokinetics of MBG453 (Parameter AUC) | AUCinf is the AUC from time zero to infinity (mass x time x volume-1). | Pharmacokinetic Analysis Set (PAS) included all participants in the Safety Set, who had at least one evaluable PK concentration. Safety Set included all participants who received at least one dose of any component of the study treatment, and they were analyzed according to the study treatment they received. | Posted | Geometric Mean | Geometric Coefficient of Variation | day*ug/ml | 0 hr (pre-dose) & 2 hrs (post-dose) of Cycle (C) 1 Day (D) 8 and 0 hr (pre-dose) & 2 hrs (post-dose) of C3D8 |
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| Secondary | ADA Prevalence at Baseline and ADA-positive Participants On-treatment | Anti-drug Antibody (ADA) prevalence is the number of participants with at least one sample meeting the criteria at baseline. ADA-positive participants were calculated as the number of participants with at least one on-treatment ADA-positive sample divided by the number of participants with a determinant baseline IG sample and at least one determinant post-baseline IG sample. | Immunogenicity Incidence Set includes all participants in the Immunogenicity prevalence set with a non-missing baseline ADA sample and at least one non-missing post-baseline ADA sample. The Immunogenicity prevalence set includes all participants in the Safety Set with a non-missing baseline ADA sample or at least one non-missing post-baseline ADA sample. | Posted | Count of Participants | Participants | No | Baseline, up to approx. 39 months |
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| Secondary | 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 is being presented for EQ Index score. Index score is defined as a weighted combination of the levels of the 5-dimention scales, ranging from 0 to 1 (perfect health), with higher scores indicating better health-related quality of life. The United States value set from Pickard et al 2019 was used. | FAS population with non-missing baseline EQ-5D-5L assessments. For each post-baseline timepoint, FAS population with non-missing baseline and post-baseline EQ-5D-5L assessment. FAS comprised of all patients to whom study treatment had been assigned by randomization. | Posted | Mean | Standard Deviation | Scores on a scale | Baseline, every 3 cycles up to C48D1 (1 cycle = 28 days) |
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| Secondary | Change From Baseline in the European Quality of Life (EuroQoL) - 5 Dimensions, 5 Level Questionnaire (EQ-5D-5L) Visual Analogue Scale (VAS) Over Time | The EQ-5D-5L VAS records the participant'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 was presented. | FAS population with non-missing baseline EQ-5D-5L assessments. For each post-baseline timepoint, FAS population with non-missing baseline and post-baseline EQ-5D-5L assessment. FAS comprised of all patients to whom study treatment had been assigned by randomization. | Posted | Mean | Standard Deviation | Scores on a scale | Baseline, every 3 cycles up to C48D1 (1 cycle = 28 days) |
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| Secondary | Change From Baseline 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 participants. Participant'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 as presented. | FAS population with non-missing baseline (Cycle 1 Day 1) and Cycle 12 Day 1 EORTC-QLQ-C30 assessments. FAS comprised of all patients to whom study treatment had been assigned by randomization. | Posted | Mean | Standard Deviation | Scores on a scale | Up to Cycle 12 Day 1 (C12D1) (1 cycle = 28 days) |
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| Post-Hoc | All Collected Deaths | Deaths were collected from randomization until the end of the trial, approx. 52 months, including post-treatment survival follow up period. | Clinical database population: All randomized participants: participants who died before treatment, during treatment and post-treatment. | Posted | Number | Participants | from randomization until end of trial, up to approx. 52 months |
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Adverse Events (AEs) were collected from the date of first administration of study treatment to 30 days after the date of the last administration of study treatment, up to maximum duration of 43.5 months (sabatolimab plus AZA) and 42.6 months (placebo + AZA). Deaths were collected from randomization until end of trial, approx. 52 months.
Adverse Event: Any sign or symptom that occurs during the study treatment + 30 days post treatment. All-Cause Mortality is based on all randomized participants (by randomized study treatment), Serious and Other Adverse Events are summarized for the participants who received study treatment (by study treatment received).
Not provided
| ID | Title | Description | Deaths (Affected) | Deaths (At Risk) | Serious Events (Affected) | Serious Events (At Risk) | Other Events (Affected) | Other Events (At Risk) |
|---|---|---|---|---|---|---|---|---|
| EG000 | Sabatolimab (MBG453) + Azacitidine | Participants received sabatolimab plus Azacitidine | 161 | 265 | 174 | 263 | 256 | 263 |
| EG001 | Placebo + Azacitidine | Participants received placebo plus Azacitidine | 160 | 265 | 164 | 265 | 255 | 265 |
| Term | Organ System | Source Vocabulary | Assessment Type | Notes | Statistical Information |
|---|---|---|---|---|---|
| Agranulocytosis | Blood and lymphatic system disorders | MedDRA (27.1) | Systematic Assessment |
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| Anaemia | Blood and lymphatic system disorders | MedDRA (27.1) | Systematic Assessment |
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| Anaemia of malignant disease | Blood and lymphatic system disorders | MedDRA (27.1) | Systematic Assessment |
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| Aplastic anaemia | Blood and lymphatic system disorders | MedDRA (27.1) | Systematic Assessment |
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| Autoimmune haemolytic anaemia | Blood and lymphatic system disorders | MedDRA (27.1) | Systematic Assessment |
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| Disseminated intravascular coagulation | Blood and lymphatic system disorders | MedDRA (27.1) | Systematic Assessment |
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| Febrile bone marrow aplasia | Blood and lymphatic system disorders | MedDRA (27.1) | Systematic Assessment |
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| Febrile neutropenia | Blood and lymphatic system disorders | MedDRA (27.1) | Systematic Assessment |
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| Iron deficiency anaemia | Blood and lymphatic system disorders | MedDRA (27.1) | Systematic Assessment |
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| Leukocytosis | Blood and lymphatic system disorders | MedDRA (27.1) | Systematic Assessment |
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| Lymphadenitis | Blood and lymphatic system disorders | MedDRA (27.1) | Systematic Assessment |
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| Myelosuppression | Blood and lymphatic system disorders | MedDRA (27.1) | Systematic Assessment |
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| Neutropenia | Blood and lymphatic system disorders | MedDRA (27.1) | Systematic Assessment |
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| Pancytopenia | Blood and lymphatic system disorders | MedDRA (27.1) | Systematic Assessment |
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| Thrombocytopenia | Blood and lymphatic system disorders | MedDRA (27.1) | Systematic Assessment |
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| Acute myocardial infarction | Cardiac disorders | MedDRA (27.1) | Systematic Assessment |
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| Angina pectoris | Cardiac disorders | MedDRA (27.1) | Systematic Assessment |
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| Angina unstable | Cardiac disorders | MedDRA (27.1) | Systematic Assessment |
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| Atrial fibrillation | Cardiac disorders | MedDRA (27.1) | Systematic Assessment |
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| Atrial flutter | Cardiac disorders | MedDRA (27.1) | Systematic Assessment |
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| Cardiac arrest | Cardiac disorders | MedDRA (27.1) | Systematic Assessment |
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| Cardiac failure | Cardiac disorders | MedDRA (27.1) | Systematic Assessment |
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| Left ventricular dysfunction | Cardiac disorders | MedDRA (27.1) | Systematic Assessment |
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| Mitral valve incompetence | Cardiac disorders | MedDRA (27.1) | Systematic Assessment |
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| Myocardial infarction | Cardiac disorders | MedDRA (27.1) | Systematic Assessment |
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| Pericardial effusion | Cardiac disorders | MedDRA (27.1) | Systematic Assessment |
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| Haematotympanum | Ear and labyrinth disorders | MedDRA (27.1) | Systematic Assessment |
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| Hypoacusis | Ear and labyrinth disorders | MedDRA (27.1) | Systematic Assessment |
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| Vertigo | Ear and labyrinth disorders | MedDRA (27.1) | Systematic Assessment |
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| Vision blurred | Eye disorders | MedDRA (27.1) | Systematic Assessment |
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| Abdominal pain | Gastrointestinal disorders | MedDRA (27.1) | Systematic Assessment |
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| Abdominal pain upper | Gastrointestinal disorders | MedDRA (27.1) | Systematic Assessment |
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| Ascites | Gastrointestinal disorders | MedDRA (27.1) | Systematic Assessment |
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| Colitis | Gastrointestinal disorders | MedDRA (27.1) | Systematic Assessment |
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| Constipation | Gastrointestinal disorders | MedDRA (27.1) | Systematic Assessment |
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| Diarrhoea | Gastrointestinal disorders | MedDRA (27.1) | Systematic Assessment |
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| Enteritis | Gastrointestinal disorders | MedDRA (27.1) | Systematic Assessment |
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| Enterocolitis | Gastrointestinal disorders | MedDRA (27.1) | Systematic Assessment |
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| Food poisoning | Gastrointestinal disorders | MedDRA (27.1) | Systematic Assessment |
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| Gastric dysplasia | Gastrointestinal disorders | MedDRA (27.1) | Systematic Assessment |
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| Gastric haemorrhage | Gastrointestinal disorders | MedDRA (27.1) | Systematic Assessment |
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| Gastric ulcer haemorrhage | Gastrointestinal disorders | MedDRA (27.1) | Systematic Assessment |
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| Gastritis | Gastrointestinal disorders | MedDRA (27.1) | Systematic Assessment |
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| Gastrointestinal haemorrhage | Gastrointestinal disorders | MedDRA (27.1) | Systematic Assessment |
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| Gastrointestinal motility disorder | Gastrointestinal disorders | MedDRA (27.1) | Systematic Assessment |
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| Haemorrhoids thrombosed | Gastrointestinal disorders | MedDRA (27.1) | Systematic Assessment |
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| Inguinal hernia | Gastrointestinal disorders | MedDRA (27.1) | Systematic Assessment |
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| Intestinal obstruction | Gastrointestinal disorders | MedDRA (27.1) | Systematic Assessment |
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| Melaena | Gastrointestinal disorders | MedDRA (27.1) | Systematic Assessment |
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| Obstructive pancreatitis | Gastrointestinal disorders | MedDRA (27.1) | Systematic Assessment |
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| Overflow diarrhoea | Gastrointestinal disorders | MedDRA (27.1) | Systematic Assessment |
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| Pancreatitis | Gastrointestinal disorders | MedDRA (27.1) | Systematic Assessment |
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| Proctalgia | Gastrointestinal disorders | MedDRA (27.1) | Systematic Assessment |
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| Proctitis | Gastrointestinal disorders | MedDRA (27.1) | Systematic Assessment |
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| Rectal haemorrhage | Gastrointestinal disorders | MedDRA (27.1) | Systematic Assessment |
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| Rectal ulcer | Gastrointestinal disorders | MedDRA (27.1) | Systematic Assessment |
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| Small intestinal haemorrhage | Gastrointestinal disorders | MedDRA (27.1) | Systematic Assessment |
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| Small intestinal obstruction | Gastrointestinal disorders | MedDRA (27.1) | Systematic Assessment |
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| Stomatitis | Gastrointestinal disorders | MedDRA (27.1) | Systematic Assessment |
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| Upper gastrointestinal haemorrhage | Gastrointestinal disorders | MedDRA (27.1) | Systematic Assessment |
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| Vomiting | Gastrointestinal disorders | MedDRA (27.1) | Systematic Assessment |
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| Asthenia | General disorders and administration site conditions | MedDRA (27.1) | Systematic Assessment |
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| Chest discomfort | General disorders and administration site conditions | MedDRA (27.1) | Systematic Assessment |
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| Chest pain | General disorders and administration site conditions | MedDRA (27.1) | Systematic Assessment |
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| Fatigue | General disorders and administration site conditions | MedDRA (27.1) | Systematic Assessment |
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| General physical health deterioration | General disorders and administration site conditions | MedDRA (27.1) | Systematic Assessment |
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| Multiple organ dysfunction syndrome | General disorders and administration site conditions | MedDRA (27.1) | Systematic Assessment |
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| Oedema | General disorders and administration site conditions | MedDRA (27.1) | Systematic Assessment |
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| Pain | General disorders and administration site conditions | MedDRA (27.1) | Systematic Assessment |
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| Peripheral swelling | General disorders and administration site conditions | MedDRA (27.1) | Systematic Assessment |
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| Pyrexia | General disorders and administration site conditions | MedDRA (27.1) | Systematic Assessment |
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| Sudden death | General disorders and administration site conditions | MedDRA (27.1) | Systematic Assessment |
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| Biliary colic | Hepatobiliary disorders | MedDRA (27.1) | Systematic Assessment |
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| Cholecystitis | Hepatobiliary disorders | MedDRA (27.1) | Systematic Assessment |
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| Drug-induced liver injury | Hepatobiliary disorders | MedDRA (27.1) | Systematic Assessment |
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| Hepatic failure | Hepatobiliary disorders | MedDRA (27.1) | Systematic Assessment |
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| Hepatic function abnormal | Hepatobiliary disorders | MedDRA (27.1) | Systematic Assessment |
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| Hepatitis acute | Hepatobiliary disorders | MedDRA (27.1) | Systematic Assessment |
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| Hepatotoxicity | Hepatobiliary disorders | MedDRA (27.1) | Systematic Assessment |
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| Hypertransaminasaemia | Hepatobiliary disorders | MedDRA (27.1) | Systematic Assessment |
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| Portal hypertension | Hepatobiliary disorders | MedDRA (27.1) | Systematic Assessment |
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| Portal vein thrombosis | Hepatobiliary disorders | MedDRA (27.1) | Systematic Assessment |
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| Haemophagocytic lymphohistiocytosis | Immune system disorders | MedDRA (27.1) | Systematic Assessment |
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| Abdominal infection | Infections and infestations | MedDRA (27.1) | Systematic Assessment |
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| Anal abscess | Infections and infestations | MedDRA (27.1) | Systematic Assessment |
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| Appendicitis | Infections and infestations | MedDRA (27.1) | Systematic Assessment |
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| Arthritis infective | Infections and infestations | MedDRA (27.1) | Systematic Assessment |
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| Aspergillus infection | Infections and infestations | MedDRA (27.1) | Systematic Assessment |
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| Bacteraemia | Infections and infestations | MedDRA (27.1) | Systematic Assessment |
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| Bacterial infection | Infections and infestations | MedDRA (27.1) | Systematic Assessment |
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| Bronchitis | Infections and infestations | MedDRA (27.1) | Systematic Assessment |
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| Bronchopulmonary aspergillosis | Infections and infestations | MedDRA (27.1) | Systematic Assessment |
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| COVID-19 | Infections and infestations | MedDRA (27.1) | Systematic Assessment |
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| COVID-19 pneumonia | Infections and infestations | MedDRA (27.1) | Systematic Assessment |
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| Candida infection | Infections and infestations | MedDRA (27.1) | Systematic Assessment |
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| Cellulitis | Infections and infestations | MedDRA (27.1) | Systematic Assessment |
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| Device related infection | Infections and infestations | MedDRA (27.1) | Systematic Assessment |
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| Diverticulitis | Infections and infestations | MedDRA (27.1) | Systematic Assessment |
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| Encephalitis viral | Infections and infestations | MedDRA (27.1) | Systematic Assessment |
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| Enterococcal sepsis | Infections and infestations | MedDRA (27.1) | Systematic Assessment |
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| Enterocolitis infectious | Infections and infestations | MedDRA (27.1) | Systematic Assessment |
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| Epididymitis | Infections and infestations | MedDRA (27.1) | Systematic Assessment |
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| Escherichia sepsis | Infections and infestations | MedDRA (27.1) | Systematic Assessment |
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| Eyelid infection | Infections and infestations | MedDRA (27.1) | Systematic Assessment |
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| Gastroenteritis | Infections and infestations | MedDRA (27.1) | Systematic Assessment |
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| Herpes dermatitis | Infections and infestations | MedDRA (27.1) | Systematic Assessment |
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| Herpes zoster | Infections and infestations | MedDRA (27.1) | Systematic Assessment |
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| Infection | Infections and infestations | MedDRA (27.1) | Systematic Assessment |
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| Legionella infection | Infections and infestations | MedDRA (27.1) | Systematic Assessment |
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| Lower respiratory tract infection | Infections and infestations | MedDRA (27.1) | Systematic Assessment |
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| Nasopharyngitis | Infections and infestations | MedDRA (27.1) | Systematic Assessment |
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| Necrotising fasciitis | Infections and infestations | MedDRA (27.1) | Systematic Assessment |
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| Neutropenic sepsis | Infections and infestations | MedDRA (27.1) | Systematic Assessment |
| |
| Opportunistic infection | Infections and infestations | MedDRA (27.1) | Systematic Assessment |
| |
| Otitis externa | Infections and infestations | MedDRA (27.1) | Systematic Assessment |
| |
| Perineal abscess | Infections and infestations | MedDRA (27.1) | Systematic Assessment |
| |
| Periodontitis | Infections and infestations | MedDRA (27.1) | Systematic Assessment |
| |
| Periorbital cellulitis | Infections and infestations | MedDRA (27.1) | Systematic Assessment |
| |
| Pneumocystis jirovecii pneumonia | Infections and infestations | MedDRA (27.1) | Systematic Assessment |
| |
| Pneumonia | Infections and infestations | MedDRA (27.1) | Systematic Assessment |
| |
| Pneumonia aspiration | Infections and infestations | MedDRA (27.1) | Systematic Assessment |
| |
| Pneumonia bacterial | Infections and infestations | MedDRA (27.1) | Systematic Assessment |
| |
| Pneumonia escherichia | Infections and infestations | MedDRA (27.1) | Systematic Assessment |
| |
| Pneumonia fungal | Infections and infestations | MedDRA (27.1) | Systematic Assessment |
| |
| Pneumonia klebsiella | Infections and infestations | MedDRA (27.1) | Systematic Assessment |
| |
| Pseudomonas infection | Infections and infestations | MedDRA (27.1) | Systematic Assessment |
| |
| Pulmonary mucormycosis | Infections and infestations | MedDRA (27.1) | Systematic Assessment |
| |
| Pyelonephritis | Infections and infestations | MedDRA (27.1) | Systematic Assessment |
| |
| Rectal abscess | Infections and infestations | MedDRA (27.1) | Systematic Assessment |
| |
| Renal abscess | Infections and infestations | MedDRA (27.1) | Systematic Assessment |
| |
| Respiratory tract infection | Infections and infestations | MedDRA (27.1) | Systematic Assessment |
| |
| Rhinovirus infection | Infections and infestations | MedDRA (27.1) | Systematic Assessment |
| |
| Rotavirus infection | Infections and infestations | MedDRA (27.1) | Systematic Assessment |
| |
| Sepsis | Infections and infestations | MedDRA (27.1) | Systematic Assessment |
| |
| Septic shock | Infections and infestations | MedDRA (27.1) | Systematic Assessment |
| |
| Sialoadenitis | Infections and infestations | MedDRA (27.1) | Systematic Assessment |
| |
| Skin infection | Infections and infestations | MedDRA (27.1) | Systematic Assessment |
| |
| Soft tissue infection | Infections and infestations | MedDRA (27.1) | Systematic Assessment |
| |
| Staphylococcal infection | Infections and infestations | MedDRA (27.1) | Systematic Assessment |
| |
| Staphylococcal sepsis | Infections and infestations | MedDRA (27.1) | Systematic Assessment |
| |
| Systemic infection | Infections and infestations | MedDRA (27.1) | Systematic Assessment |
| |
| Upper respiratory tract infection | Infections and infestations | MedDRA (27.1) | Systematic Assessment |
| |
| Urinary tract infection | Infections and infestations | MedDRA (27.1) | Systematic Assessment |
| |
| Urinary tract infection enterococcal | Infections and infestations | MedDRA (27.1) | Systematic Assessment |
| |
| Urosepsis | Infections and infestations | MedDRA (27.1) | Systematic Assessment |
| |
| Allergic transfusion reaction | Injury, poisoning and procedural complications | MedDRA (27.1) | Systematic Assessment |
| |
| Extradural haematoma | Injury, poisoning and procedural complications | MedDRA (27.1) | Systematic Assessment |
| |
| Fall | Injury, poisoning and procedural complications | MedDRA (27.1) | Systematic Assessment |
| |
| Hip fracture | Injury, poisoning and procedural complications | MedDRA (27.1) | Systematic Assessment |
| |
| Infusion related reaction | Injury, poisoning and procedural complications | MedDRA (27.1) | Systematic Assessment |
| |
| Lumbar vertebral fracture | Injury, poisoning and procedural complications | MedDRA (27.1) | Systematic Assessment |
| |
| Multiple injuries | Injury, poisoning and procedural complications | MedDRA (27.1) | Systematic Assessment |
| |
| Post procedural haemorrhage | Injury, poisoning and procedural complications | MedDRA (27.1) | Systematic Assessment |
| |
| Procedural haemorrhage | Injury, poisoning and procedural complications | MedDRA (27.1) | Systematic Assessment |
| |
| Procedural pain | Injury, poisoning and procedural complications | MedDRA (27.1) | Systematic Assessment |
| |
| Radius fracture | Injury, poisoning and procedural complications | MedDRA (27.1) | Systematic Assessment |
| |
| Refractoriness to platelet transfusion | Injury, poisoning and procedural complications | MedDRA (27.1) | Systematic Assessment |
| |
| Spinal compression fracture | Injury, poisoning and procedural complications | MedDRA (27.1) | Systematic Assessment |
| |
| Stoma site inflammation | Injury, poisoning and procedural complications | MedDRA (27.1) | Systematic Assessment |
| |
| Subdural haematoma | Injury, poisoning and procedural complications | MedDRA (27.1) | Systematic Assessment |
| |
| Transfusion reaction | Injury, poisoning and procedural complications | MedDRA (27.1) | Systematic Assessment |
| |
| Alanine aminotransferase increased | Investigations | MedDRA (27.1) | Systematic Assessment |
| |
| Anti-platelet antibody positive | Investigations | MedDRA (27.1) | Systematic Assessment |
| |
| Aspartate aminotransferase increased | Investigations | MedDRA (27.1) | Systematic Assessment |
| |
| Blood alkaline phosphatase increased | Investigations | MedDRA (27.1) | Systematic Assessment |
| |
| Blood bilirubin increased | Investigations | MedDRA (27.1) | Systematic Assessment |
| |
| Blood creatinine increased | Investigations | MedDRA (27.1) | Systematic Assessment |
| |
| C-reactive protein increased | Investigations | MedDRA (27.1) | Systematic Assessment |
| |
| Ejection fraction decreased | Investigations | MedDRA (27.1) | Systematic Assessment |
| |
| Liver function test increased | Investigations | MedDRA (27.1) | Systematic Assessment |
| |
| Neutrophil count decreased | Investigations | MedDRA (27.1) | Systematic Assessment |
| |
| Platelet count decreased | Investigations | MedDRA (27.1) | Systematic Assessment |
| |
| Transaminases increased | Investigations | MedDRA (27.1) | Systematic Assessment |
| |
| Troponin T increased | Investigations | MedDRA (27.1) | Systematic Assessment |
| |
| Weight decreased | Investigations | MedDRA (27.1) | Systematic Assessment |
| |
| White blood cell count decreased | Investigations | MedDRA (27.1) | Systematic Assessment |
| |
| Decreased appetite | Metabolism and nutrition disorders | MedDRA (27.1) | Systematic Assessment |
| |
| Dehydration | Metabolism and nutrition disorders | MedDRA (27.1) | Systematic Assessment |
| |
| Hyperkalaemia | Metabolism and nutrition disorders | MedDRA (27.1) | Systematic Assessment |
| |
| Hypokalaemia | Metabolism and nutrition disorders | MedDRA (27.1) | Systematic Assessment |
| |
| Lactic acidosis | Metabolism and nutrition disorders | MedDRA (27.1) | Systematic Assessment |
| |
| Arthropathy | Musculoskeletal and connective tissue disorders | MedDRA (27.1) | Systematic Assessment |
| |
| Chondrocalcinosis | Musculoskeletal and connective tissue disorders | MedDRA (27.1) | Systematic Assessment |
| |
| Foot deformity | Musculoskeletal and connective tissue disorders | MedDRA (27.1) | Systematic Assessment |
| |
| Joint effusion | Musculoskeletal and connective tissue disorders | MedDRA (27.1) | Systematic Assessment |
| |
| Osteoarthritis | Musculoskeletal and connective tissue disorders | MedDRA (27.1) | Systematic Assessment |
| |
| Pain in extremity | Musculoskeletal and connective tissue disorders | MedDRA (27.1) | Systematic Assessment |
| |
| Tenosynovitis | Musculoskeletal and connective tissue disorders | MedDRA (27.1) | Systematic Assessment |
| |
| Hepatocellular carcinoma | Neoplasms benign, malignant and unspecified (incl cysts and polyps) | MedDRA (27.1) | Systematic Assessment |
| |
| Intestinal adenocarcinoma | Neoplasms benign, malignant and unspecified (incl cysts and polyps) | MedDRA (27.1) | Systematic Assessment |
| |
| Liposarcoma | Neoplasms benign, malignant and unspecified (incl cysts and polyps) | MedDRA (27.1) | Systematic Assessment |
| |
| Lung neoplasm malignant | Neoplasms benign, malignant and unspecified (incl cysts and polyps) | MedDRA (27.1) | Systematic Assessment |
| |
| Nodular melanoma | Neoplasms benign, malignant and unspecified (incl cysts and polyps) | MedDRA (27.1) | Systematic Assessment |
| |
| Rectal adenocarcinoma | Neoplasms benign, malignant and unspecified (incl cysts and polyps) | MedDRA (27.1) | Systematic Assessment |
| |
| Small intestine adenocarcinoma | Neoplasms benign, malignant and unspecified (incl cysts and polyps) | MedDRA (27.1) | Systematic Assessment |
| |
| Altered state of consciousness | Nervous system disorders | MedDRA (27.1) | Systematic Assessment |
| |
| Central nervous system haemorrhage | Nervous system disorders | MedDRA (27.1) | Systematic Assessment |
| |
| Cerebral haemorrhage | Nervous system disorders | MedDRA (27.1) | Systematic Assessment |
| |
| Cerebrovascular accident | Nervous system disorders | MedDRA (27.1) | Systematic Assessment |
| |
| Encephalopathy | Nervous system disorders | MedDRA (27.1) | Systematic Assessment |
| |
| Haemorrhage intracranial | Nervous system disorders | MedDRA (27.1) | Systematic Assessment |
| |
| Headache | Nervous system disorders | MedDRA (27.1) | Systematic Assessment |
| |
| Orthostatic intolerance | Nervous system disorders | MedDRA (27.1) | Systematic Assessment |
| |
| Sciatica | Nervous system disorders | MedDRA (27.1) | Systematic Assessment |
| |
| Subarachnoid haemorrhage | Nervous system disorders | MedDRA (27.1) | Systematic Assessment |
| |
| Syncope | Nervous system disorders | MedDRA (27.1) | Systematic Assessment |
| |
| Confusional state | Psychiatric disorders | MedDRA (27.1) | Systematic Assessment |
| |
| Delirium | Psychiatric disorders | MedDRA (27.1) | Systematic Assessment |
| |
| Hallucination | Psychiatric disorders | MedDRA (27.1) | Systematic Assessment |
| |
| Acute kidney injury | Renal and urinary disorders | MedDRA (27.1) | Systematic Assessment |
| |
| Calculus bladder | Renal and urinary disorders | MedDRA (27.1) | Systematic Assessment |
| |
| Haematuria | Renal and urinary disorders | MedDRA (27.1) | Systematic Assessment |
| |
| Renal cyst haemorrhage | Renal and urinary disorders | MedDRA (27.1) | Systematic Assessment |
| |
| Prostatitis | Reproductive system and breast disorders | MedDRA (27.1) | Systematic Assessment |
| |
| Aspiration | Respiratory, thoracic and mediastinal disorders | MedDRA (27.1) | Systematic Assessment |
| |
| Chronic obstructive pulmonary disease | Respiratory, thoracic and mediastinal disorders | MedDRA (27.1) | Systematic Assessment |
| |
| Dyspnoea | Respiratory, thoracic and mediastinal disorders | MedDRA (27.1) | Systematic Assessment |
| |
| Emphysema | Respiratory, thoracic and mediastinal disorders | MedDRA (27.1) | Systematic Assessment |
| |
| Epistaxis | Respiratory, thoracic and mediastinal disorders | MedDRA (27.1) | Systematic Assessment |
| |
| Hypersensitivity pneumonitis | Respiratory, thoracic and mediastinal disorders | MedDRA (27.1) | Systematic Assessment |
| |
| Hypoxia | Respiratory, thoracic and mediastinal disorders | MedDRA (27.1) | Systematic Assessment |
| |
| Interstitial lung disease | Respiratory, thoracic and mediastinal disorders | MedDRA (27.1) | Systematic Assessment |
| |
| Lung infiltration | Respiratory, thoracic and mediastinal disorders | MedDRA (27.1) | Systematic Assessment |
| |
| Pleural effusion | Respiratory, thoracic and mediastinal disorders | MedDRA (27.1) | Systematic Assessment |
| |
| Pneumonitis | Respiratory, thoracic and mediastinal disorders | MedDRA (27.1) | Systematic Assessment |
| |
| Pulmonary oedema | Respiratory, thoracic and mediastinal disorders | MedDRA (27.1) | Systematic Assessment |
| |
| Pulmonary toxicity | Respiratory, thoracic and mediastinal disorders | MedDRA (27.1) | Systematic Assessment |
| |
| Respiratory failure | Respiratory, thoracic and mediastinal disorders | MedDRA (27.1) | Systematic Assessment |
| |
| Cutaneous vasculitis | Skin and subcutaneous tissue disorders | MedDRA (27.1) | Systematic Assessment |
| |
| Dermatitis | Skin and subcutaneous tissue disorders | MedDRA (27.1) | Systematic Assessment |
| |
| Erythema nodosum | Skin and subcutaneous tissue disorders | MedDRA (27.1) | Systematic Assessment |
| |
| Pyoderma gangrenosum | Skin and subcutaneous tissue disorders | MedDRA (27.1) | Systematic Assessment |
| |
| Rash | Skin and subcutaneous tissue disorders | MedDRA (27.1) | Systematic Assessment |
| |
| Rash maculo-papular | Skin and subcutaneous tissue disorders | MedDRA (27.1) | Systematic Assessment |
| |
| Skin ulcer | Skin and subcutaneous tissue disorders | MedDRA (27.1) | Systematic Assessment |
| |
| Arterial thrombosis | Vascular disorders | MedDRA (27.1) | Systematic Assessment |
| |
| Deep vein thrombosis | Vascular disorders | MedDRA (27.1) | Systematic Assessment |
| |
| Hypotension | Vascular disorders | MedDRA (27.1) | Systematic Assessment |
| |
| Peripheral artery thrombosis | Vascular disorders | MedDRA (27.1) | Systematic Assessment |
| |
| Thrombophlebitis | Vascular disorders | MedDRA (27.1) | Systematic Assessment |
| |
| White coat hypertension | Vascular disorders | MedDRA (27.1) | Systematic Assessment |
|
| Term | Organ System | Source Vocabulary | Assessment Type | Notes | Statistical Information |
|---|---|---|---|---|---|
| Anaemia | Blood and lymphatic system disorders | MedDRA (27.1) | Systematic Assessment |
| |
| Febrile neutropenia | Blood and lymphatic system disorders | MedDRA (27.1) | Systematic Assessment |
| |
| Leukopenia | Blood and lymphatic system disorders | MedDRA (27.1) | Systematic Assessment |
| |
| Neutropenia | Blood and lymphatic system disorders | MedDRA (27.1) | Systematic Assessment |
| |
| Thrombocytopenia | Blood and lymphatic system disorders | MedDRA (27.1) | Systematic Assessment |
| |
| Abdominal pain | Gastrointestinal disorders | MedDRA (27.1) | Systematic Assessment |
| |
| Constipation | Gastrointestinal disorders | MedDRA (27.1) | Systematic Assessment |
| |
| Diarrhoea | Gastrointestinal disorders | MedDRA (27.1) | Systematic Assessment |
| |
| Nausea | Gastrointestinal disorders | MedDRA (27.1) | Systematic Assessment |
| |
| Stomatitis | Gastrointestinal disorders | MedDRA (27.1) | Systematic Assessment |
| |
| Vomiting | Gastrointestinal disorders | MedDRA (27.1) | Systematic Assessment |
| |
| Asthenia | General disorders and administration site conditions | MedDRA (27.1) | Systematic Assessment |
| |
| Fatigue | General disorders and administration site conditions | MedDRA (27.1) | Systematic Assessment |
| |
| Injection site reaction | General disorders and administration site conditions | MedDRA (27.1) | Systematic Assessment |
| |
| Oedema peripheral | General disorders and administration site conditions | MedDRA (27.1) | Systematic Assessment |
| |
| Pyrexia | General disorders and administration site conditions | MedDRA (27.1) | Systematic Assessment |
| |
| COVID-19 | Infections and infestations | MedDRA (27.1) | Systematic Assessment |
| |
| Cellulitis | Infections and infestations | MedDRA (27.1) | Systematic Assessment |
| |
| Pneumonia | Infections and infestations | MedDRA (27.1) | Systematic Assessment |
| |
| Upper respiratory tract infection | Infections and infestations | MedDRA (27.1) | Systematic Assessment |
| |
| Urinary tract infection | Infections and infestations | MedDRA (27.1) | Systematic Assessment |
| |
| Fall | Injury, poisoning and procedural complications | MedDRA (27.1) | Systematic Assessment |
| |
| Alanine aminotransferase increased | Investigations | MedDRA (27.1) | Systematic Assessment |
| |
| Aspartate aminotransferase increased | Investigations | MedDRA (27.1) | Systematic Assessment |
| |
| Blood creatinine increased | Investigations | MedDRA (27.1) | Systematic Assessment |
| |
| C-reactive protein increased | Investigations | MedDRA (27.1) | Systematic Assessment |
| |
| Lymphocyte count decreased | Investigations | MedDRA (27.1) | Systematic Assessment |
| |
| Neutrophil count decreased | Investigations | MedDRA (27.1) | Systematic Assessment |
| |
| Platelet count decreased | Investigations | MedDRA (27.1) | Systematic Assessment |
| |
| Weight decreased | Investigations | MedDRA (27.1) | Systematic Assessment |
| |
| White blood cell count decreased | Investigations | MedDRA (27.1) | Systematic Assessment |
| |
| Decreased appetite | Metabolism and nutrition disorders | MedDRA (27.1) | Systematic Assessment |
| |
| Hyperglycaemia | Metabolism and nutrition disorders | MedDRA (27.1) | Systematic Assessment |
| |
| Hyperuricaemia | Metabolism and nutrition disorders | MedDRA (27.1) | Systematic Assessment |
| |
| Hypoalbuminaemia | Metabolism and nutrition disorders | MedDRA (27.1) | Systematic Assessment |
| |
| Hypocalcaemia | Metabolism and nutrition disorders | MedDRA (27.1) | Systematic Assessment |
| |
| Hypokalaemia | Metabolism and nutrition disorders | MedDRA (27.1) | Systematic Assessment |
| |
| Hyponatraemia | Metabolism and nutrition disorders | MedDRA (27.1) | Systematic Assessment |
| |
| Arthralgia | Musculoskeletal and connective tissue disorders | MedDRA (27.1) | Systematic Assessment |
| |
| Back pain | Musculoskeletal and connective tissue disorders | MedDRA (27.1) | Systematic Assessment |
| |
| Pain in extremity | Musculoskeletal and connective tissue disorders | MedDRA (27.1) | Systematic Assessment |
| |
| Dizziness | Nervous system disorders | MedDRA (27.1) | Systematic Assessment |
| |
| Headache | Nervous system disorders | MedDRA (27.1) | Systematic Assessment |
| |
| Insomnia | Psychiatric disorders | MedDRA (27.1) | Systematic Assessment |
| |
| Cough | Respiratory, thoracic and mediastinal disorders | MedDRA (27.1) | Systematic Assessment |
| |
| Dyspnoea | Respiratory, thoracic and mediastinal disorders | MedDRA (27.1) | Systematic Assessment |
| |
| Epistaxis | Respiratory, thoracic and mediastinal disorders | MedDRA (27.1) | Systematic Assessment |
| |
| Pruritus | Skin and subcutaneous tissue disorders | MedDRA (27.1) | Systematic Assessment |
| |
| Rash | Skin and subcutaneous tissue disorders | MedDRA (27.1) | Systematic Assessment |
| |
| Hypertension | Vascular disorders | MedDRA (27.1) | Systematic Assessment |
| |
| Hypotension | Vascular disorders | MedDRA (27.1) | Systematic Assessment |
|
A total of 530 participants were randomized (265 to sabatolimab+AZA, 265 to placebo+AZA), but 1 participant in each arm did not receive any study treatment and were excluded from the Safety Set. Another sabatolimab-assigned participant received only AZA and thus was summarized in the placebo+AZA arm in all safety analyses. Therefore, the Serious and Other Adverse Events were shown for 263 sabatolimab+AZA and 265 placebo+AZA participants.
The terms and conditions of Novartis' agreements with its investigators may vary. However, Novartis does not prohibit any investigator from publishing. Any publications from a single site are postponed until the publication of pooled data (i.e. data from all sites) in clinical trial or disclosure of trial results in their entirety.
| Title | Organization | Phone | Extension | |
|---|---|---|---|---|
| Study Director | Novartis Pharmaceuticals | 862-778-3000 | novartis.email@novartis.com |
| Prot_000.pdf |
| SAP | No | Yes | No | Statistical Analysis Plan | Sep 18, 2023 | Sep 18, 2025 | SAP_001.pdf |
Not provided
| ID | Term |
|---|---|
| D009190 | Myelodysplastic Syndromes |
| D015477 | Leukemia, Myelomonocytic, Chronic |
| ID | Term |
|---|---|
| D001855 | Bone Marrow Diseases |
| D006402 | Hematologic Diseases |
| D006425 | Hemic and Lymphatic Diseases |
| D007951 | Leukemia, Myeloid |
| D007938 | Leukemia |
| D009370 | Neoplasms by Histologic Type |
| D009369 | Neoplasms |
| D054437 | Myelodysplastic-Myeloproliferative Diseases |
| D002908 | Chronic Disease |
| D020969 | Disease Attributes |
| D010335 | Pathologic Processes |
| D013568 | Pathological Conditions, Signs and Symptoms |
Not provided
Not provided
| ID | Term |
|---|---|
| C000723550 | sabatolimab |
| D001374 | Azacitidine |
| ID | Term |
|---|---|
| D001372 | Aza Compounds |
| D009930 | Organic Chemicals |
| D003562 | Cytidine |
| D011741 | Pyrimidine Nucleosides |
| D011743 | Pyrimidines |
| D006573 | Heterocyclic Compounds, 1-Ring |
| D006571 | Heterocyclic Compounds |
| D009705 | Nucleosides |
| D009706 | Nucleic Acids, Nucleotides, and Nucleosides |
| D012263 | Ribonucleosides |
Not provided
Not provided
| Male |
|
| Asian |
|
| Black or African American |
|
| American Indian or Alaska Native |
|
| Unknown |
|
| ECOG performance status: 1 |
|
| ECOG performance status: 2 |
|
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