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Sponsor's decision as at the completion of the primary analysis, the data were considered mature
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This is a Phase 2, two-stage study of the safety and efficacy of pracinostat in combination with azacitidine in patients with IPSS-R high and very high risk myelodysplastic syndrome (MDS) who are previously untreated with hypomethylating agents (HMAs). Enrollment in this study will be limited to high/very high risk MDS because this group represents the highest unmet need, with median survival of less than 18 months.
Stage 1a will be conducted as an open-label single arm in up to 40 subjects to assess if this regimen with a lower pracinostat dose results in a discontinuation rate that meets a predefined threshold and in efficacy that justifies expansion of enrollment into Stage 1b.
A discontinuation rate of approximately ≤10% in Stage 1a, a rate comparable to that observed with azacitidine alone in study MEI-003 (NCT01873703), supports expansion into Stage 1b.
Stage 1b will be conducted as expansion of stage 1a. Approximately 20 additional subjects will be enrolled. Study drugs should be continued until disease progression or intolerable toxicity. It is important to note that the median time to achieving a response with azacitidine alone is 4 to 5 months. Furthermore, the median time to achieving a Complete Response (CR) in study MEI-003 (NCT01873703) was 4 cycles. Therefore, early (<6 months) discontinuation of trial therapy for 'no response' should be avoided. The Medical Monitor should be contacted prior to discontinuing a subject from the study to discuss the rationale for discontinuation.
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| Label | Type | Description | Intervention Names |
|---|---|---|---|
| Stage 1a and 1b open-label pracinostat plus azacitidine | Experimental | open-label single arm pracinostat plus azacitidine. Pracinostat: 45 mg administered orally 3 days each week for 3 consecutive weeks, followed by 1 week of rest, in 28-day cycles. In later cycles (i.e., after Cycle 4), pracinostat dose reduction to 45 mg orally 3 days each week × 2 weeks (instead of 3 weeks) or dose interruption is allowed to manage toxicity such as fatigue, gastrointestinal toxicity, or myelosuppression. Azacitidine: 75 mg/m2 for 7 days of each 28-day cycle. Administration will occur by subcutaneous (SC) injection, or IV infusion if SC injections are not tolerated, on one of two schedules:
|
|
| Name | Type | Description | Arm Group Labels | Other Names |
|---|---|---|---|---|
| Pracinostat | Drug | 45 mg capsule |
|
| Measure | Description | Time Frame |
|---|---|---|
| Overall Response Rate (ORR) | Percentage of subjects with confirmed complete remission (CR), partial remission (PR) and marrow CR, as per modified International Working Group (IWG) criteria: CR: Bone marrow: ≤5% myeloblasts with normal maturation of all cell lines; Peripheral blood Hemoglobin (Hb) ≥11 g/dL; Platelets ≥100 × 10^9/L; Neutrophils ≥1.0 × 10^9/L; Blasts 0%. PR: All CR criteria if abnormal before treatment except: Bone marrow blasts decreased by ≥ 50% over pre-treatment but still >5%; Cellularity and morphology not relevant Marrow CR: Bone marrow: ≤5% myeloblasts and decrease by ≥50% over pre-treatment | 36 months |
| Measure | Description | Time Frame |
|---|---|---|
| Complete Response (CR) Rate | Percentage of subjects with confirmed CR (i.e., 2 CRs at least 28 days apart) as per modified IWG criteria: CR: Bone marrow: ≤5% myeloblasts with normal maturation of all cell lines; Peripheral blood Hb ≥11 g/dL; Platelets ≥100 × 109/L; Neutrophils ≥1.0 × 109/L; Blasts 0%. | 36 months |
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Inclusion Criteria:
Female or male subjects ≥18 years-of-age.
Histologically or cytologically documented diagnosis of MDS according to the World Health Organization (WHO) classification (Vardiman 2009, Arber 2016) with >5% and <20% bone marrow blasts by morphology and a peripheral white blood cell (WBC) count of <20,000/μL
Classified as high or very high risk according to the Revised International Prognostic Scoring System (IPSS-R) risk category. CMML-1 and CMML-2 subtypes will be considered high-risk MDS and will not require IPSS-r scoring
Bone marrow biopsy (BMBx) and/or aspirate within 28 days prior to first study treatment.
Clinical indication for treatment with azacitidine.
Previously untreated with HMAs (prior therapy with transfusions, hematopoietic growth factors, or immunosuppressive therapy is allowed).
a. subjects who require the start of an HMA (e.g., azacitidine) due to progressing MDS may receive up to 1 cycle of azacitidine within 30 days prior to planned first dose (Cycle 1 Day 1)
Eastern Cooperative Oncology Group (ECOG) performance status of 0, 1, or 2.
Adequate organ function as evidenced by:
Female subjects of childbearing potential and male subjects with female partners of childbearing potential are required to use two forms of acceptable contraception, including one barrier method, during their participation in the study and for 30 days following last dose. Female subjects of childbearing potential must not be breastfeeding, or planning to breastfeed, and must have a negative pregnancy test ≤7 days before first study drug administration.
Male subjects must also refrain from donating sperm during their participation in the study.
Voluntary written informed consent before performance of any study-related procedure not part of normal medical care.
Have the willingness and ability to understand the nature of this study and to comply with the study and follow-up procedures.
Exclusion Criteria:
Bone marrow blasts ≥20%, indicating a diagnosis of acute myeloid leukemia (AML).
Received any of the following within the specified time frame prior to administration of study medication:
Subjects who have not recovered from side effects of previous therapy.
Cardiopulmonary function criteria:
Prior treatment for MDS with histone deacetylase (HDAC) inhibitors Zolinza (vorinostat), Belenodaq (belinostat), Farydak (panobinostat), Istodax (romidepsin/depsipetide), or investigational agent with significant action as an HDAC inhibitor.
Clinical evidence of central nervous system involvement.
Subjects with gastrointestinal (GI) tract disease causing the inability to take oral medication, malabsorption syndrome, a requirement for intravenous (IV) alimentation, prior surgical procedures affecting absorption, uncontrolled inflammatory GI disease (e.g., Crohn's disease, ulcerative colitis).
Uncontrolled infection with human immunodeficiency virus (HIV) or chronic hepatitis B or C.
Life-threatening illness unrelated to cancer or any serious medical or psychiatric illness that could, in the Investigator's opinion, potentially interfere with participation in this study.
Presence of a malignant disease within the last 12 months, with the exception of adequately treated in-situ carcinomas, basal or squamous cell carcinoma, non-melanomatous skin cancer, or malignancies treated with curative intent and no evidence of active disease in prior 12 months and felt to be low risk for recurrence. Other malignancies may be considered after consultation with the Medical Monitor
An unwillingness or inability (including breastfeeding women, prohibited concomitant medications, uncontrolled infections, psychological, familial, sociological, or geographical conditions) to comply with study and/or follow-up procedures as outlined in the protocol
Known hypersensitivity to any components of pracinostat, azacitidine or mannitol
Current smoking or vaporizing of tobacco or cannabis-related products (use of patches, chewing tobacco, or nicotine gum is permitted). Subjects who stopped smoking at least 8 days prior to first pracinostat dosing can be, provided they refrain from smoking during the whole study.
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| Name | Affiliation | Role |
|---|---|---|
| Richard Ghalie, MD | MEI Pharma | Study Director |
| Ehab Atallah, MD | Medical College of Wisconsin adn Froedtert Hospital | Study Chair |
| Facility | Status | City | State | ZIP | Country | Contacts |
|---|---|---|---|---|---|---|
| City of Hope | Duarte | California | 91010 | United States | ||
| Scripps Cancer Center-Mercy |
Stage 1 was to be conducted in 20-40 evaluable subjects to assess if the pracinostat dose regimen resulted in a discontinuation rate that met a predefined threshold, and the observed efficacy justified expansion of enrollment in Stage 1b.
Stage 1b was conducted to achieve a planned total enrollment of 60 subjects evaluable for efficacy, inclusive of Stage 1 and Stage 1b enrollment.
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| ID | Title | Description |
|---|---|---|
| FG000 | Stage 1a and 1b Open-label Pracinostat Plus Azacitidine | open-label single arm pracinostat plus azacitidine. Pracinostat: 45 mg administered orally 3 days each week for 3 consecutive weeks, followed by 1 week of rest, in 28-day cycles. In later cycles (i.e., after Cycle 4), pracinostat dose reduction to 45 mg orally 3 days each week × 2 weeks (instead of 3 weeks) or dose interruption is allowed to manage toxicity such as fatigue, gastrointestinal toxicity, or myelosuppression. Azacitidine: 75 mg/m2 for 7 days of each 28-day cycle. Administration will occur by subcutaneous (SC) injection, or IV infusion if SC injections are not tolerated, on one of two schedules:
Pracinostat: 45 mg capsule Azacitidine: SC or IV injection |
| 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 25, 2018 | Dec 15, 2021 |
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Two-Stage, Open-Label
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| Azacitidine | Drug | SC or IV injection |
|
| Overall Hematologic Improvement (HI) Response Rate |
Percentage of subjects demonstrating major hematologic improvement according to modified IWG: Erythroid response (pre-treatment, <11 g/dL): Hb increase by ≥1.5 g/dL; Relevant reduction of units of RBC transfusions by an absolute number of at least 4 RBC transfusions/8 weeks compared with the pre-treatment transfusion number in the previous 8 weeks. Only RBC transfusions given for a Hb of ≤9.0 g/dL pre-treatment will count in the RBC transfusion response evaluation. Platelet response (pre-treatment, <100 × 10^9/L): Absolute increase of ≥30 × 10^9/L for patients starting with >20 × 10^9/L platelets; Increase from <20 × 10^9/L to >20 × 10^9/L and by at least 100%. Neutrophil response (pre-treatment, <1.0 × 10^9/L): At least 100% increase and an absolute increase >0.5 × 10^9/L. Progression or relapse after HI: at least 1 of the following:
|
| 36 months |
| Clinical Benefit Rate | Percentage of subjects with confirmed CR, PR, Marrow CR, and HI with clinical benefit rate, defines as rate of CR + PR + HI + Marrow CR. All subjects who achieve hematologic CR, PR, marrow CR, or hematologic improvement on the erythrocytic lineage per modified IWG response criteria will be considered responders | 36 months |
| Rate of Cytogenetic CR | Percentage of subjects with confirmed CR by cytogenetic assessment. Complete cytogenetic response is defined per modified IWG response criteria: Complete: Disappearance of the chromosomal abnormality without appearance of new ones Partial: At least 50% reduction of the chromosomal abnormality | 36 months |
| Duration of Response (DoR) | For subjects who have achieved CR, PR, Marrow CR, or HI, DoR is defined as the time from the initial determination of response to the time of disease progression or death on study, whichever occurs first. | 36 months |
| Rate of Leukemic Transformation | Percentage of subjects with leukemic transformation at landmark time point of 6 months | 6 months |
| Event-free Survival (EFS) | time from the first day of study drug administration (Day 1) to failure or death from any cause | 36 months |
| Progression-free Survival (PFS) | time from the first day of study drug administration (Day 1) to disease recurrence or progression as defined by the IWG criteria, or death on study: Disease progression for subjects with: Less than 5% blasts: ≥50% increase in blasts to >5% blasts 5%-10% blasts: ≥50% increase to >10% blasts 10%-20% blasts: ≥50% increase to >20% blasts 20%-30% blasts: ≥50% increase to >30% blasts Any of the following: At least 50% decrement from maximum remission/response in granulocytes or platelets Reduction in Hb by ≥2 g/dL Transfusion dependence | 36 months |
| Overall Survival (OS) | time from the first day of study drug administration (Day 1) to death on study | form day 1 to death on study, assessed up to 36 months |
| Rate of Leukemic Transformation | Percentage of subjects with leukemic transformation at landmark time point of 12 months | 12 months |
| Rate of Leukemic Transformation | Percentage of subjects with leukemic transformation at landmark time point of 18 months | 18 months |
| Rate of Leukemic Transformation | Percentage of subjects with leukemic transformation at landmark time point of 24 months | 24 months |
| San Diego |
| California |
| 92103 |
| United States |
| Georgia Cancer Center at Augusta University | Augusta | Georgia | 30912 | United States |
| georgia cancer Center | Augusta | Georgia | 30912 | United States |
| Pontchartrain cancer Center | Covington | Louisiana | 70433 | United States |
| RCCA MD LLC (The Center for Cancer and Blood Disorders) | Bethesda | Maryland | 20817 | United States |
| Michigan Center of Medical Research | Farmington Hills | Michigan | 48334 | United States |
| Michigan State University, Breslin Cancer Center | Lansing | Michigan | 48910 | United States |
| university of minnesota medical Center, Fairview | Minneapolis | Minnesota | 55455 | United States |
| Mercy Medical Research Institute | Springfield | Missouri | 65807 | United States |
| New Mexico Cancer care Alliance | Albuquerque | New Mexico | 87131 | United States |
| University of Rochester Medical Center | Rochester | New York | 14642 | United States |
| Stony Brook University | Stony Brook | New York | 11794 | United States |
| Duke University Medical Center | Durham | North Carolina | 27710 | United States |
| Southeastern Medical Oncology Center | Goldsboro | North Carolina | 27534 | United States |
| Oncology Hematology Care | Cincinnati | Ohio | 45242 | United States |
| University Hospitals Cleveland Medical Center | Cleveland | Ohio | 44106 | United States |
| Cancer Centers of Southwest Oklahoma | Lawton | Oklahoma | 73501 | United States |
| Oklahoma Cancer Specialists and Research Institute | Tulsa | Oklahoma | 74146 | United States |
| Providence Portland Medical center | Portland | Oregon | 97213 | United States |
| UT Southwestern Medical Center | Dallas | Texas | 75390 | United States |
| UVA Health System Division of Hematology & Oncology | Charlottesville | Virginia | 22908 | United States |
| Swedish Cancer Institute | Seattle | Washington | 98104 | United States |
| Universityof Wisconsin Clinical Science Center | Madison | Wisconsin | 53792 | United States |
| Medical College of Wisconsin | Milwaukee | Wisconsin | 53226 | United States |
| COMPLETED |
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| NOT COMPLETED |
|
|
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| ID | Title | Description |
|---|---|---|
| BG000 | Stage 1a and 1b Open-label Pracinostat Plus Azacitidine | open-label single arm pracinostat plus azacitidine. Pracinostat: 45 mg administered orally 3 days each week for 3 consecutive weeks, followed by 1 week of rest, in 28-day cycles. In later cycles (i.e., after Cycle 4), pracinostat dose reduction to 45 mg orally 3 days each week × 2 weeks (instead of 3 weeks) or dose interruption is allowed to manage toxicity such as fatigue, gastrointestinal toxicity, or myelosuppression. Azacitidine: 75 mg/m2 for 7 days of each 28-day cycle. Administration will occur by subcutaneous (SC) injection, or IV infusion if SC injections are not tolerated, on one of two schedules:
Pracinostat: 45 mg capsule Azacitidine: SC or IV injection |
| 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 |
| ||||||||||||||||||
| Ethnicity (NIH/OMB) | Count of Participants | Participants |
| ||||||||||||||||||
| Race (NIH/OMB) | Count of Participants | Participants |
| ||||||||||||||||||
| Region of Enrollment | Number | participants |
| ||||||||||||||||||
| Body Mass Index (BMI) | Mean | Standard Deviation | Kg/m^2 |
|
| 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 | ||||||||||||||||||||
|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|
| Primary | Overall Response Rate (ORR) | Percentage of subjects with confirmed complete remission (CR), partial remission (PR) and marrow CR, as per modified International Working Group (IWG) criteria: CR: Bone marrow: ≤5% myeloblasts with normal maturation of all cell lines; Peripheral blood Hemoglobin (Hb) ≥11 g/dL; Platelets ≥100 × 10^9/L; Neutrophils ≥1.0 × 10^9/L; Blasts 0%. PR: All CR criteria if abnormal before treatment except: Bone marrow blasts decreased by ≥ 50% over pre-treatment but still >5%; Cellularity and morphology not relevant Marrow CR: Bone marrow: ≤5% myeloblasts and decrease by ≥50% over pre-treatment | Posted | Number | 95% Confidence Interval | percentage of subjects | 36 months |
|
|
| ||||||||||||||||||||||||||
| Secondary | Complete Response (CR) Rate | Percentage of subjects with confirmed CR (i.e., 2 CRs at least 28 days apart) as per modified IWG criteria: CR: Bone marrow: ≤5% myeloblasts with normal maturation of all cell lines; Peripheral blood Hb ≥11 g/dL; Platelets ≥100 × 109/L; Neutrophils ≥1.0 × 109/L; Blasts 0%. | Posted | Number | 95% Confidence Interval | percentage of subjects with CR | 36 months |
|
| |||||||||||||||||||||||||||
| Secondary | Overall Hematologic Improvement (HI) Response Rate | Percentage of subjects demonstrating major hematologic improvement according to modified IWG: Erythroid response (pre-treatment, <11 g/dL): Hb increase by ≥1.5 g/dL; Relevant reduction of units of RBC transfusions by an absolute number of at least 4 RBC transfusions/8 weeks compared with the pre-treatment transfusion number in the previous 8 weeks. Only RBC transfusions given for a Hb of ≤9.0 g/dL pre-treatment will count in the RBC transfusion response evaluation. Platelet response (pre-treatment, <100 × 10^9/L): Absolute increase of ≥30 × 10^9/L for patients starting with >20 × 10^9/L platelets; Increase from <20 × 10^9/L to >20 × 10^9/L and by at least 100%. Neutrophil response (pre-treatment, <1.0 × 10^9/L): At least 100% increase and an absolute increase >0.5 × 10^9/L. Progression or relapse after HI: at least 1 of the following:
| Posted | Number | 95% Confidence Interval | percentage of subjects with HI | 36 months |
| ||||||||||||||||||||||||||||
| Secondary | Clinical Benefit Rate | Percentage of subjects with confirmed CR, PR, Marrow CR, and HI with clinical benefit rate, defines as rate of CR + PR + HI + Marrow CR. All subjects who achieve hematologic CR, PR, marrow CR, or hematologic improvement on the erythrocytic lineage per modified IWG response criteria will be considered responders | Posted | Number | 95% Confidence Interval | percentage of subjects with benefit rate | 36 months |
|
| |||||||||||||||||||||||||||
| Secondary | Rate of Cytogenetic CR | Percentage of subjects with confirmed CR by cytogenetic assessment. Complete cytogenetic response is defined per modified IWG response criteria: Complete: Disappearance of the chromosomal abnormality without appearance of new ones Partial: At least 50% reduction of the chromosomal abnormality | Posted | Number | 95% Confidence Interval | percentage of subjects with cCR | 36 months |
|
| |||||||||||||||||||||||||||
| Secondary | Duration of Response (DoR) | For subjects who have achieved CR, PR, Marrow CR, or HI, DoR is defined as the time from the initial determination of response to the time of disease progression or death on study, whichever occurs first. | Posted | Median | 95% Confidence Interval | months | 36 months |
|
| |||||||||||||||||||||||||||
| Secondary | Rate of Leukemic Transformation | Percentage of subjects with leukemic transformation at landmark time point of 6 months | Posted | Number | 95% Confidence Interval | percentage of subjects | 6 months |
|
| |||||||||||||||||||||||||||
| Secondary | Event-free Survival (EFS) | time from the first day of study drug administration (Day 1) to failure or death from any cause | Posted | Median | 95% Confidence Interval | months | 36 months |
|
| |||||||||||||||||||||||||||
| Secondary | Progression-free Survival (PFS) | time from the first day of study drug administration (Day 1) to disease recurrence or progression as defined by the IWG criteria, or death on study: Disease progression for subjects with: Less than 5% blasts: ≥50% increase in blasts to >5% blasts 5%-10% blasts: ≥50% increase to >10% blasts 10%-20% blasts: ≥50% increase to >20% blasts 20%-30% blasts: ≥50% increase to >30% blasts Any of the following: At least 50% decrement from maximum remission/response in granulocytes or platelets Reduction in Hb by ≥2 g/dL Transfusion dependence | Posted | Median | 95% Confidence Interval | months | 36 months |
| ||||||||||||||||||||||||||||
| Secondary | Overall Survival (OS) | time from the first day of study drug administration (Day 1) to death on study | Posted | Median | 95% Confidence Interval | months | form day 1 to death on study, assessed up to 36 months |
|
| |||||||||||||||||||||||||||
| Secondary | Rate of Leukemic Transformation | Percentage of subjects with leukemic transformation at landmark time point of 12 months | Posted | Number | 95% Confidence Interval | percentage of subjects | 12 months |
|
| |||||||||||||||||||||||||||
| Secondary | Rate of Leukemic Transformation | Percentage of subjects with leukemic transformation at landmark time point of 18 months | Posted | Number | 95% Confidence Interval | percentage of subjects | 18 months |
|
| |||||||||||||||||||||||||||
| Secondary | Rate of Leukemic Transformation | Percentage of subjects with leukemic transformation at landmark time point of 24 months | Posted | Number | 95% Confidence Interval | percentage of subjects | 24 months |
|
|
All AEs regardless of seriousness or relationship to pracinostat or azacitidine, from the start of study drug treatment until 30 calendar days after discontinuation or completion of treatment as defined by the clinical study for that subject, have been recorded.
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| ID | Title | Description | Deaths (Affected) | Deaths (At Risk) | Serious Events (Affected) | Serious Events (At Risk) | Other Events (Affected) | Other Events (At Risk) |
|---|---|---|---|---|---|---|---|---|
| EG000 | Stage 1a and 1b Open-label Pracinostat Plus Azacitidine | open-label single arm pracinostat plus azacitidine. Pracinostat: 45 mg administered orally 3 days each week for 3 consecutive weeks, followed by 1 week of rest, in 28-day cycles. In later cycles (i.e., after Cycle 4), pracinostat dose reduction to 45 mg orally 3 days each week × 2 weeks (instead of 3 weeks) or dose interruption is allowed to manage toxicity such as fatigue, gastrointestinal toxicity, or myelosuppression. Azacitidine: 75 mg/m2 for 7 days of each 28-day cycle. Administration will occur by subcutaneous (SC) injection, or IV infusion if SC injections are not tolerated, on one of two schedules:
Pracinostat: 45 mg capsule Azacitidine: SC or IV injection | 5 | 64 | 45 | 64 | 64 | 64 |
| Term | Organ System | Source Vocabulary | Assessment Type | Notes | Statistical Information |
|---|---|---|---|---|---|
| Febrile neutropenia | Blood and lymphatic system disorders | MedDRA 20.0 | Systematic Assessment |
| |
| Pancytopenia | Blood and lymphatic system disorders | MedDRA 20.0 | Systematic Assessment |
| |
| Thrombocytopenia | Blood and lymphatic system disorders | MedDRA 20.0 | Systematic Assessment |
| |
| Acute coronary syndrome | Cardiac disorders | MedDRA 20.0 | Systematic Assessment |
| |
| Acute myocardial infarction | Cardiac disorders | MedDRA 20.0 | Systematic Assessment |
| |
| Atrial fibrillation | Cardiac disorders | MedDRA 20.0 | Systematic Assessment |
| |
| Cardiac failure | Cardiac disorders | MedDRA 20.0 | Systematic Assessment |
| |
| Cardiac failure congestive | Cardiac disorders | MedDRA 20.0 | Systematic Assessment |
| |
| Pericardial effusion | Cardiac disorders | MedDRA 20.0 | Systematic Assessment |
| |
| Pericarditis | Cardiac disorders | MedDRA 20.0 | Systematic Assessment |
| |
| Sinus tachycardia | Cardiac disorders | MedDRA 20.0 | Systematic Assessment |
| |
| Anal fissure | Gastrointestinal disorders | MedDRA 20.0 | Systematic Assessment |
| |
| Ascites | Gastrointestinal disorders | MedDRA 20.0 | Systematic Assessment |
| |
| Dysphagia | Gastrointestinal disorders | MedDRA 20.0 | Systematic Assessment |
| |
| Gastrointestinal haemorrhage | Gastrointestinal disorders | MedDRA 20.0 | Systematic Assessment |
| |
| Haemorrhoidal haemorrhage | Gastrointestinal disorders | MedDRA 20.0 | Systematic Assessment |
| |
| Large intestine perforation | Gastrointestinal disorders | MedDRA 20.0 | Systematic Assessment |
| |
| Nausea | Gastrointestinal disorders | MedDRA 20.0 | Systematic Assessment |
| |
| Proctitis | Gastrointestinal disorders | MedDRA 20.0 | Systematic Assessment |
| |
| Rectal ulcer | Gastrointestinal disorders | MedDRA 20.0 | Systematic Assessment |
| |
| Stomatitis | Gastrointestinal disorders | MedDRA 20.0 | Systematic Assessment |
| |
| Upper gastrointestinal haemorrhage | Gastrointestinal disorders | MedDRA 20.0 | Systematic Assessment |
| |
| Vomiting | Gastrointestinal disorders | MedDRA 20.0 | Systematic Assessment |
| |
| Asthenia | General disorders | MedDRA 20.0 | Systematic Assessment |
| |
| Malaise | General disorders | MedDRA 20.0 | Systematic Assessment |
| |
| Pyrexia | General disorders | MedDRA 20.0 | Systematic Assessment |
| |
| Abscess limb | Infections and infestations | MedDRA 20.0 | Systematic Assessment |
| |
| Anal abscess | Infections and infestations | MedDRA 20.0 | Systematic Assessment |
| |
| Cellulitis | Infections and infestations | MedDRA 20.0 | Systematic Assessment |
| |
| Clostridial sepsis | Infections and infestations | MedDRA 20.0 | Systematic Assessment |
| |
| Clostridium difficile colitis | Infections and infestations | MedDRA 20.0 | Systematic Assessment |
| |
| Escherichia sepsis | Infections and infestations | MedDRA 20.0 | Systematic Assessment |
| |
| Fungal oesophagitis | Infections and infestations | MedDRA 20.0 | Systematic Assessment |
| |
| Influenza | Infections and infestations | MedDRA 20.0 | Systematic Assessment |
| |
| Lung infection | Infections and infestations | MedDRA 20.0 | Systematic Assessment |
| |
| Perirectal abscess | Infections and infestations | MedDRA 20.0 | Systematic Assessment |
| |
| Pneumonia | Infections and infestations | MedDRA 20.0 | Systematic Assessment |
| |
| Pseudomonal sepsis | Infections and infestations | MedDRA 20.0 | Systematic Assessment |
| |
| Respiratory tract infection viral | Infections and infestations | MedDRA 20.0 | Systematic Assessment |
| |
| Sepsis | Infections and infestations | MedDRA 20.0 | Non-systematic Assessment |
| |
| Sialoadenitis | Infections and infestations | MedDRA 20.0 | Systematic Assessment |
| |
| Staphylococcal infection | Infections and infestations | MedDRA 20.0 | Systematic Assessment |
| |
| Staphylococcal sepsis | Infections and infestations | MedDRA 20.0 | Systematic Assessment |
| |
| Streptococcal sepsis | Infections and infestations | MedDRA 20.0 | Systematic Assessment |
| |
| Vascular access site infection | Infections and infestations | MedDRA 20.0 | Systematic Assessment |
| |
| Fall | Injury, poisoning and procedural complications | MedDRA 20.0 | Systematic Assessment |
| |
| Laceration | Injury, poisoning and procedural complications | MedDRA 20.0 | Systematic Assessment |
| |
| Spinal compression fracture | Injury, poisoning and procedural complications | MedDRA 20.0 | Systematic Assessment |
| |
| Electrocardiogram qt prolonged | Investigations | MedDRA 20.0 | Systematic Assessment |
| |
| Neutrophil count decreased | Investigations | MedDRA 20.0 | Systematic Assessment |
| |
| Dehydration | Metabolism and nutrition disorders | MedDRA 20.0 | Systematic Assessment |
| |
| Hypercalcaemia | Metabolism and nutrition disorders | MedDRA 20.0 | Systematic Assessment |
| |
| Hyperinsulinaemic hypoglycaemia | Metabolism and nutrition disorders | MedDRA 20.0 | Systematic Assessment |
| |
| Arthralgia | Musculoskeletal and connective tissue disorders | MedDRA 20.0 | Systematic Assessment |
| |
| Presyncope | Nervous system disorders | MedDRA 20.0 | Systematic Assessment |
| |
| Syncope | Nervous system disorders | MedDRA 20.0 | Systematic Assessment |
| |
| Mania | Psychiatric disorders | MedDRA 20.0 | Systematic Assessment |
| |
| Mental disorder | Psychiatric disorders | MedDRA 20.0 | Systematic Assessment |
| |
| Acute kidney injury | Renal and urinary disorders | MedDRA 20.0 | Systematic Assessment |
| |
| Haematuria | Renal and urinary disorders | MedDRA 20.0 | Systematic Assessment |
| |
| Renal failure | Renal and urinary disorders | MedDRA 20.0 | Systematic Assessment |
| |
| Dyspnoea | Respiratory, thoracic and mediastinal disorders | MedDRA 20.0 | Systematic Assessment |
| |
| Epistaxis | Respiratory, thoracic and mediastinal disorders | MedDRA 20.0 | Systematic Assessment |
| |
| Pleural effusion | Respiratory, thoracic and mediastinal disorders | MedDRA 20.0 | Systematic Assessment |
| |
| Pulmonary embolism | Respiratory, thoracic and mediastinal disorders | MedDRA 20.0 | Systematic Assessment |
| |
| Pulmonary oedema | Respiratory, thoracic and mediastinal disorders | MedDRA 20.0 | Systematic Assessment |
| |
| Respiratory distress | Respiratory, thoracic and mediastinal disorders | MedDRA 20.0 | Systematic Assessment |
| |
| Respiratory failure | Respiratory, thoracic and mediastinal disorders | MedDRA 20.0 | Systematic Assessment |
| |
| Deep vein thrombosis | Vascular disorders | MedDRA 20.0 | Non-systematic Assessment |
| |
| Orthostatic hypotension | Vascular disorders | MedDRA 20.0 | Systematic Assessment |
| |
| Anaemia | Blood and lymphatic system disorders | MedDRA 20.0 | Systematic Assessment |
|
| Term | Organ System | Source Vocabulary | Assessment Type | Notes | Statistical Information |
|---|---|---|---|---|---|
| Anaemia | Blood and lymphatic system disorders | MedDRA 20.0 | Non-systematic Assessment |
| |
| Febrile neutropenia | Blood and lymphatic system disorders | MedDRA 20.0 | Systematic Assessment |
| |
| Leukopenia | Blood and lymphatic system disorders | MedDRA 20.0 | Systematic Assessment |
| |
| Neutropenia | Blood and lymphatic system disorders | MedDRA 20.0 | Systematic Assessment |
| |
| Pancytopenia | Blood and lymphatic system disorders | MedDRA 20.0 | Systematic Assessment |
| |
| Thrombocytopenia | Blood and lymphatic system disorders | MedDRA 20.0 | Systematic Assessment |
| |
| Tachycardia | Cardiac disorders | MedDRA 20.0 | Systematic Assessment |
| |
| Ear pain | Ear and labyrinth disorders | MedDRA 20.0 | Systematic Assessment |
| |
| Dry eye | Eye disorders | MedDRA 20.0 | Systematic Assessment |
| |
| Abdominal pain | Gastrointestinal disorders | MedDRA 20.0 | Systematic Assessment |
| |
| Constipation | Gastrointestinal disorders | MedDRA 20.0 | Systematic Assessment |
| |
| Diarrhoea | Gastrointestinal disorders | MedDRA 20.0 | Systematic Assessment |
| |
| Dry mouth | Gastrointestinal disorders | MedDRA 20.0 | Systematic Assessment |
| |
| Dyspepsia | Gastrointestinal disorders | MedDRA 20.0 | Systematic Assessment |
| |
| Dysphagia | Gastrointestinal disorders | MedDRA 20.0 | Systematic Assessment |
| |
| Nausea | Gastrointestinal disorders | MedDRA 20.0 | Systematic Assessment |
| |
| Oral pain | Gastrointestinal disorders | MedDRA 20.0 | Systematic Assessment |
| |
| Proctalgia | Gastrointestinal disorders | MedDRA 20.0 | Systematic Assessment |
| |
| Tongue discolouration | Gastrointestinal disorders | MedDRA 20.0 | Systematic Assessment |
| |
| Vomiting | Gastrointestinal disorders | MedDRA 20.0 | Systematic Assessment |
| |
| Asthenia | General disorders | MedDRA 20.0 | Systematic Assessment |
| |
| Chest pain | General disorders | MedDRA 20.0 | Systematic Assessment |
| |
| Chills | General disorders | MedDRA 20.0 | Systematic Assessment |
| |
| Fatigue | General disorders | MedDRA 20.0 | Systematic Assessment |
| |
| Injection site reaction | General disorders | MedDRA 20.0 | Systematic Assessment |
| |
| Oedema peripheral | General disorders | MedDRA 20.0 | Systematic Assessment |
| |
| Pyrexia | General disorders | MedDRA 20.0 | Systematic Assessment |
| |
| Lung infection | Infections and infestations | MedDRA 20.0 | Systematic Assessment |
| |
| Pneumonia | Infections and infestations | MedDRA 20.0 | Systematic Assessment |
| |
| Sepsis | Infections and infestations | MedDRA 20.0 | Systematic Assessment |
| |
| Sinusitis | Infections and infestations | MedDRA 20.0 | Systematic Assessment |
| |
| Upper respiratory tract infection | Infections and infestations | MedDRA 20.0 | Systematic Assessment |
| |
| Urinary tract infection | Infections and infestations | MedDRA 20.0 | Systematic Assessment |
| |
| Contusion | Injury, poisoning and procedural complications | MedDRA 20.0 | Systematic Assessment |
| |
| Fall | Injury, poisoning and procedural complications | MedDRA 20.0 | Systematic Assessment |
| |
| Blood alkaline phosphatase increased | Investigations | MedDRA 20.0 | Systematic Assessment |
| |
| Blood creatinine increased | Investigations | MedDRA 20.0 | Systematic Assessment |
| |
| International normalised ratio increased | Investigations | MedDRA 20.0 | Systematic Assessment |
| |
| Lymphocyte count decreased | Investigations | MedDRA 20.0 | Systematic Assessment |
| |
| Neutrophil count decreased | Investigations | MedDRA 20.0 | Systematic Assessment |
| |
| Weight decreased | Investigations | MedDRA 20.0 | Systematic Assessment |
| |
| White blood cell count decreased | Investigations | MedDRA 20.0 | Systematic Assessment |
| |
| Decreased appetite | Metabolism and nutrition disorders | MedDRA 20.0 | Systematic Assessment |
| |
| Dehydration | Metabolism and nutrition disorders | MedDRA 20.0 | Systematic Assessment |
| |
| Hyperglycaemia | Metabolism and nutrition disorders | MedDRA 20.0 | Systematic Assessment |
| |
| Hypoalbuminaemia | Metabolism and nutrition disorders | MedDRA 20.0 | Systematic Assessment |
| |
| Hypocalcaemia | Metabolism and nutrition disorders | MedDRA 20.0 | Systematic Assessment |
| |
| Hypoglycaemia | Metabolism and nutrition disorders | MedDRA 20.0 | Systematic Assessment |
| |
| Hypokalaemia | Metabolism and nutrition disorders | MedDRA 20.0 | Systematic Assessment |
| |
| Hyponatraemia | Metabolism and nutrition disorders | MedDRA 20.0 | Systematic Assessment |
| |
| Arthralgia | Musculoskeletal and connective tissue disorders | MedDRA 20.0 | Systematic Assessment |
| |
| Arthritis | Musculoskeletal and connective tissue disorders | MedDRA 20.0 | Systematic Assessment |
| |
| Back pain | Musculoskeletal and connective tissue disorders | MedDRA 20.0 | Systematic Assessment |
| |
| Muscle spasms | Musculoskeletal and connective tissue disorders | MedDRA 20.0 | Systematic Assessment |
| |
| Muscular weakness | Musculoskeletal and connective tissue disorders | MedDRA 20.0 | Systematic Assessment |
| |
| Musculoskeletal pain | Musculoskeletal and connective tissue disorders | MedDRA 20.0 | Systematic Assessment |
| |
| Myalgia | Musculoskeletal and connective tissue disorders | MedDRA 20.0 | Systematic Assessment |
| |
| Neck pain | Musculoskeletal and connective tissue disorders | MedDRA 20.0 | Systematic Assessment |
| |
| Pain in extremity | Musculoskeletal and connective tissue disorders | MedDRA 20.0 | Systematic Assessment |
| |
| Dizziness | Nervous system disorders | MedDRA 20.0 | Systematic Assessment |
| |
| Dysgeusia | Nervous system disorders | MedDRA 20.0 | Systematic Assessment |
| |
| Headache | Nervous system disorders | MedDRA 20.0 | Systematic Assessment |
| |
| Presyncope | Nervous system disorders | MedDRA 20.0 | Systematic Assessment |
| |
| Syncope | Nervous system disorders | MedDRA 20.0 | Systematic Assessment |
| |
| Tremor | Nervous system disorders | MedDRA 20.0 | Systematic Assessment |
| |
| Anxiety | Psychiatric disorders | MedDRA 20.0 | Systematic Assessment |
| |
| Confusional state | Psychiatric disorders | MedDRA 20.0 | Systematic Assessment |
| |
| Insomnia | Psychiatric disorders | MedDRA 20.0 | Systematic Assessment |
| |
| Acute kidney injury | Renal and urinary disorders | MedDRA 20.0 | Systematic Assessment |
| |
| Pollakiuria | Renal and urinary disorders | MedDRA 20.0 | Systematic Assessment |
| |
| Urinary retention | Renal and urinary disorders | MedDRA 20.0 | Systematic Assessment |
| |
| Cough | Respiratory, thoracic and mediastinal disorders | MedDRA 20.0 | Systematic Assessment |
| |
| Dyspnoea | Respiratory, thoracic and mediastinal disorders | MedDRA 20.0 | Systematic Assessment |
| |
| Dyspnoea exertional | Respiratory, thoracic and mediastinal disorders | MedDRA 20.0 | Systematic Assessment |
| |
| Epistaxis | Respiratory, thoracic and mediastinal disorders | MedDRA 20.0 | Systematic Assessment |
| |
| Hypoxia | Respiratory, thoracic and mediastinal disorders | MedDRA 20.0 | Systematic Assessment |
| |
| Nasal congestion | Respiratory, thoracic and mediastinal disorders | MedDRA 20.0 | Systematic Assessment |
| |
| Oropharyngeal pain | Respiratory, thoracic and mediastinal disorders | MedDRA 20.0 | Systematic Assessment |
| |
| Pleural effusion | Respiratory, thoracic and mediastinal disorders | MedDRA 20.0 | Systematic Assessment |
| |
| Rhinorrhoea | Respiratory, thoracic and mediastinal disorders | MedDRA 20.0 | Systematic Assessment |
| |
| Alopecia | Skin and subcutaneous tissue disorders | MedDRA 20.0 | Systematic Assessment |
| |
| Papule | Skin and subcutaneous tissue disorders | MedDRA 20.0 | Systematic Assessment |
| |
| Pruritus | Skin and subcutaneous tissue disorders | MedDRA 20.0 | Systematic Assessment |
| |
| Rash | Skin and subcutaneous tissue disorders | MedDRA 20.0 | Systematic Assessment |
| |
| Rash maculo-papular | Skin and subcutaneous tissue disorders | MedDRA 20.0 | Systematic Assessment |
| |
| Hypertension | Vascular disorders | MedDRA 20.0 | Systematic Assessment |
| |
| Hypotension | Vascular disorders | MedDRA 20.0 | Systematic Assessment |
|
Not provided
Not provided
| Title | Organization | Phone | Extension | |
|---|---|---|---|---|
| Francesco Scarci, Clinical Operations Manager | Helsinn Healthcare | +41 (0)91 985 2121 | francesco.scarci@helsinn.com |
| Prot_000.pdf |
| SAP | No | Yes | No | Statistical Analysis Plan | Aug 28, 2020 | Dec 15, 2021 | SAP_001.pdf |
Not provided
| ID | Term |
|---|---|
| D009190 | Myelodysplastic Syndromes |
| ID | Term |
|---|---|
| D001855 | Bone Marrow Diseases |
| D006402 | Hematologic Diseases |
| D006425 | Hemic and Lymphatic Diseases |
Not provided
Not provided
| ID | Term |
|---|---|
| C557525 | SB939 compound |
| 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
| Unknown or Not Reported |
|
| Native Hawaiian or Other Pacific Islander |
|
| Black or African American |
|
| White |
|
| More than one race |
|
| Unknown or Not Reported |
|
| Participants |
|
|
|
|
| Participants |
|
|
| Participants |
|
|
|
|
|
| Units | Counts |
|---|---|
| Participants |
|
|
|
|
|
|