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The study will evaluate the efficacy, safety and tolerability of two dosing schedules of LDE225 in patients with relapsed/refractory acute leukemia or elderly patients with untreated acute leukemia.
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| Label | Type | Description | Intervention Names |
|---|---|---|---|
| LDE225-400 | Experimental | Patients who were randomized to Schedule A, and received 400 mg LDE225 twice daily for the first two weeks only and then after two weeks, received 800 mg LDE225 once daily until disease progression, toxicity, withdrawal of consent, death, discretion of the investigator or early termination of the study. |
|
| LDE225-800 | Experimental | Patients who were randomized to Schedule B, received 800 mg LDE225 once daily until disease progression, toxicity, withdrawal of consent, death, discretion of the investigator or early termination of the study. |
|
| Name | Type | Description | Arm Group Labels | Other Names |
|---|---|---|---|---|
| LDE225 | Drug | LDE225 will be supplied as 200 mg capsules by Novartis. Patients will receive study treatment on an outpatient basis. LDE225 will be dispensed every two weeks for the first four weeks and at the start of every four weeks thereafter, as needed. |
| Measure | Description | Time Frame |
|---|---|---|
| Rate of Complete Remission (CR) | Complete Response (CR) was based on the International Working Group (IWG) criteria based on weekly peripheral blood count measurements and bone marrow biopsy/aspiration collection. Efficacy assessments were performed to determine CR. A treatment cycle was defined as 4 weeks. The outcome measure for the study is based on standardized response criteria as defined by the International Working Group (IWG) for AML. The IWG was established by a group of investigators interested in the design and conduct of clinical trials in acute myeloid leukemia (AML). The criteria established by this group (a set of recommendations for response assessment) are well established, endorsed by major institutions and Health Authorities, and are widely used in clinical trials. No statistical analysis was planned for this primary outcome. | at screening, every week up to Week 9, every 2 weeks thereafter until CR, every 4 weeks after CR up to 24 months |
| Complete Remission With Incomplete Blood Count Recovery (CRi) | The other primary efficacy endpoint was CRi based on the International Working Group (IWG) criteria based on weekly peripheral blood count measurements and bone marrow biopsy/aspiration collection. Efficacy assessments were performed to determine CRi. A treatment cycle was defined as 4 weeks. No statistical analysis was planned for this primary outcome. | within 3 days after clearance of blasts from peripheral blood (PB), monthly thereafter until CR or reappearance of blasts in the PB, after CR every other month until discontination up to 24 months |
| Measure | Description | Time Frame |
|---|---|---|
| Overall Response Rate (ORR) | ORR was the rate of complete remission (CR), complete remission with incomplete blood count recovery (CRi) or partial response (PR) according to IWG criteria. CR, CRi or PR will be assessed through bone marrow biopsy/aspirate and peripheral blood blasts counts. | Every 8 weeks for the first 6 months and every 12 weeks until 53 weeks after the last patient is enrolled or until relapse up to 24 months |
Not provided
Inclusion Criteria:
Exclusion Criteria:
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| Name | Affiliation | Role |
|---|---|---|
| Novartis Pharmaceuticals | Novartis Pharmaceuticals | Study Director |
| Facility | Status | City | State | ZIP | Country | Contacts |
|---|---|---|---|---|---|---|
| Duke University Medical Center SC-5 | Durham | North Carolina | 27710 | United States | ||
| Novartis Investigative Site |
35 patients were randomized but only 34 patients received at least one dose of study drug in the LDE225-800 (schedule B ) arm.
Not provided
Not provided
| ID | Title | Description |
|---|---|---|
| FG000 | LDE225-400 | Patients who were randomized to Schedule A, and received 400 mg LDE225 twice daily for the first two weeks only and after two weeks, received 800 mg LDE225 once daily until disease progression, toxicity, withdrawal of consent, death, discretion of the investigator or early termination of the study. |
| FG001 |
| Title | Milestones | Reasons Not Completed | |||||
|---|---|---|---|---|---|---|---|
| Overall Study |
|
Not provided
Not provided
Not provided
Not provided
Not provided
| Parmacokintics (PK) Parameter: Cmax | Cmax is the maximum observed plasma concentration after drug administration.The PK parameters were determined in plasma using non-compartmental methods. A PK sample was excluded from analyses if the patient vomited within the first 4 hours following the last oral dose of study drug. Other PK samples were excluded as deemed appropriate by the pharmacokineticist. Cmax was derived from the PK concentrations collected at 0, 0.5, 1, 2, 4, 6, 8 and 24 hours post dose on W1D1 and W9D1. The PK concentration at each time point is not an endpoint in the protocol, but these concentrations are used to derive the PK parameters. | Week 1 Day 1, Week 9 Day 1 |
| Parmacokintics (PK) Parameter: Tmax | Tmax is the time to reach Cmax. The PK parameters were determined in plasma using non-compartmental methods. A PK sample was excluded from analyses if the patient vomited within the first 4 hours following the last oral dose of study drug. Other PK samples were excluded as deemed appropriate by the Pharmacokineticist. Tmax was derived from the PK concentrations collected at 0, 0.5, 1, 2, 4, 6, 8 and 24 hours post dose on W1D1 and W9D1. The PK concentration at each time point is not an endpoint in the protocol, but these concentrations are used to derive the PK parameters. | Week 1 Day 1,Week 9 Day 1 |
| Parmacokintics (PK) Parameter: AUC0-8h | AUC0-8h is the area under the concentration-time curve from time zero to 8 hours. The PK parameters were determined in plasma using non-compartmental methods. A PK sample was excluded from analyses if the patient vomited within the first 4 hours following the last oral dose of study drug. Other PK samples were excluded as deemed appropriate by the Pharmacokineticist. AUC0-8h was derived from the PK concentrations collected at 0, 0.5, 1, 2, 4, 6, 8 and 24 hours post dose on W1D1 and W9D1. The PK concentration at each time point is not an endpoint in the protocol, but these concentrations are used to derive the PK parameters. | Week 1 Day 1,Week 9 Day 1 |
| Parmacokintics (PK) Parameter: AUC0-24h | AUC0-24 is the area under the concentration-time curve from time zero to 24 hours done only on 800 mg once a day schedule. The PK parameters were determined in plasma using non-compartmental methods. A PK sample was excluded from analyses if the patient vomited within the first 4 hours following the last oral dose of study drug. Other PK samples were excluded as deemed appropriate by the Pharmacokineticist. AUC0-24h was derived from the PK concentrations collected at 0, 0.5, 1, 2, 4, 6, 8 and 24 hours post dose on W1D1 and W9D1. The PK concentration at each time point is not an endpoint in the protocol, but these concentrations are used to derive the PK parameters. | Week 1 Day 1,Week 9 Day 1 |
| Adelaide |
| South Australia |
| 5000 |
| Australia |
| Novartis Investigative Site | Prahran | Victoria | 3181 | Australia |
| Novartis Investigative Site | Salzburg | 5020 | Austria |
| Novartis Investigative Site | Vienna | 1090 | Austria |
| Novartis Investigative Site | Leuven | 3000 | Belgium |
| Novartis Investigative Site | Yvoir | 5530 | Belgium |
| Novartis Investigative Site | Montreal | Quebec | H1T 2M4 | Canada |
| Novartis Investigative Site | Montreal | Quebec | H3T 1E2 | Canada |
| Novartis Investigative Site | Dresden | 01307 | Germany |
| Novartis Investigative Site | Frankfurt | 60590 | Germany |
| Novartis Investigative Site | Magdeburg | 39120 | Germany |
| Novartis Investigative Site | Ulm | 89081 | Germany |
| Novartis Investigative Site | Debrecen | 4032 | Hungary |
| Novartis Investigative Site | Amsterdam | 1081 HV | Netherlands |
| Novartis Investigative Site | Nijmegen | 6525 GA | Netherlands |
| Novartis Investigative Site | Rotterdam | 3015 CE | Netherlands |
| Novartis Investigative Site | Rotterdam | 3075 EA | Netherlands |
| Novartis Investigative Site | Bergen | NO-5021 | Norway |
| Novartis Investigative Site | Trondheim | 7006 | Norway |
| Novartis Investigative Site | Salamanca | Castille and León | 37007 | Spain |
| Novartis Investigative Site | Valencia | Valencia | 46026 | Spain |
| Novartis Investigative Site | London | WC1E 6HX | United Kingdom |
| LDE225-800 |
Patients who were randomized to Schedule B, received 800 mg LDE225 once daily until disease progression, toxicity, withdrawal of consent, death, discretion of the investigator or early termination of the study. |
| Untreated |
|
| Treated |
|
| COMPLETED |
|
| NOT COMPLETED |
|
|
Full analysis set (FAS): comprised of all patients who were randomized to a study treatment.
According to the intent-to-treat principle, patient data were analyzed according to the treatment to which they had been randomized.
Not provided
| ID | Title | Description |
|---|---|---|
| BG000 | LDE225-400 | Patients who were randomized to Schedule A, and received 400 mg LDE225 twice daily for the first two weeks only and after two weeks, received 800 mg LDE225 once daily until disease progression, toxicity, withdrawal of consent, death, discretion of the investigator or early termination of the study. |
| BG001 | LDE225-800 | Patients who were randomized to Schedule B, received 800 mg LDE225 once daily until disease progression, toxicity, withdrawal of consent, death, discretion of the investigator or early termination of the study. |
| 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 |
|
| 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 | Rate of Complete Remission (CR) | Complete Response (CR) was based on the International Working Group (IWG) criteria based on weekly peripheral blood count measurements and bone marrow biopsy/aspiration collection. Efficacy assessments were performed to determine CR. A treatment cycle was defined as 4 weeks. The outcome measure for the study is based on standardized response criteria as defined by the International Working Group (IWG) for AML. The IWG was established by a group of investigators interested in the design and conduct of clinical trials in acute myeloid leukemia (AML). The criteria established by this group (a set of recommendations for response assessment) are well established, endorsed by major institutions and Health Authorities, and are widely used in clinical trials. No statistical analysis was planned for this primary outcome. | Full analysis set (FAS): comprised of all patients who were randomized to a study treatment. According to the intent-to-treat principle, patient data were analyzed according to the treatment to which they had been randomized. No statistical analysis were reported in this study. | Posted | Number | Participants | at screening, every week up to Week 9, every 2 weeks thereafter until CR, every 4 weeks after CR up to 24 months |
|
|
| |||||||||||||||||||||||||||||
| Primary | Complete Remission With Incomplete Blood Count Recovery (CRi) | The other primary efficacy endpoint was CRi based on the International Working Group (IWG) criteria based on weekly peripheral blood count measurements and bone marrow biopsy/aspiration collection. Efficacy assessments were performed to determine CRi. A treatment cycle was defined as 4 weeks. No statistical analysis was planned for this primary outcome. | Full analysis set (FAS): comprised of all patients who were randomized to a study treatment. According to the intent-to-treat principle, patient data were analyzed according to the treatment to which they had been randomized. | Posted | Number | Participants | within 3 days after clearance of blasts from peripheral blood (PB), monthly thereafter until CR or reappearance of blasts in the PB, after CR every other month until discontination up to 24 months |
| |||||||||||||||||||||||||||||||
| Secondary | Overall Response Rate (ORR) | ORR was the rate of complete remission (CR), complete remission with incomplete blood count recovery (CRi) or partial response (PR) according to IWG criteria. CR, CRi or PR will be assessed through bone marrow biopsy/aspirate and peripheral blood blasts counts. | Full analysis set (FAS): comprised of all patients who were randomized to a study treatment. According to the intent-to-treat principle, patient data were analyzed according to the treatment to which they had been randomized. | Posted | Number | Participants | Every 8 weeks for the first 6 months and every 12 weeks until 53 weeks after the last patient is enrolled or until relapse up to 24 months |
| |||||||||||||||||||||||||||||||
| Secondary | Parmacokintics (PK) Parameter: Cmax | Cmax is the maximum observed plasma concentration after drug administration.The PK parameters were determined in plasma using non-compartmental methods. A PK sample was excluded from analyses if the patient vomited within the first 4 hours following the last oral dose of study drug. Other PK samples were excluded as deemed appropriate by the pharmacokineticist. Cmax was derived from the PK concentrations collected at 0, 0.5, 1, 2, 4, 6, 8 and 24 hours post dose on W1D1 and W9D1. The PK concentration at each time point is not an endpoint in the protocol, but these concentrations are used to derive the PK parameters. | Pharmacokinetic analysis set (PAS): consisted of all patients who received at least one dose of sonidegib and provided at least one evaluable PK blood sample. | Posted | Mean | Standard Deviation | ng/mL | Week 1 Day 1, Week 9 Day 1 |
| ||||||||||||||||||||||||||||||
| Secondary | Parmacokintics (PK) Parameter: Tmax | Tmax is the time to reach Cmax. The PK parameters were determined in plasma using non-compartmental methods. A PK sample was excluded from analyses if the patient vomited within the first 4 hours following the last oral dose of study drug. Other PK samples were excluded as deemed appropriate by the Pharmacokineticist. Tmax was derived from the PK concentrations collected at 0, 0.5, 1, 2, 4, 6, 8 and 24 hours post dose on W1D1 and W9D1. The PK concentration at each time point is not an endpoint in the protocol, but these concentrations are used to derive the PK parameters. | Pharmacokinetic analysis set (PAS): consisted of all patients who received at least one dose of sonidegib and provided at least one evaluable PK blood sample. | Posted | Median | Inter-Quartile Range | hr | Week 1 Day 1,Week 9 Day 1 |
| ||||||||||||||||||||||||||||||
| Secondary | Parmacokintics (PK) Parameter: AUC0-8h | AUC0-8h is the area under the concentration-time curve from time zero to 8 hours. The PK parameters were determined in plasma using non-compartmental methods. A PK sample was excluded from analyses if the patient vomited within the first 4 hours following the last oral dose of study drug. Other PK samples were excluded as deemed appropriate by the Pharmacokineticist. AUC0-8h was derived from the PK concentrations collected at 0, 0.5, 1, 2, 4, 6, 8 and 24 hours post dose on W1D1 and W9D1. The PK concentration at each time point is not an endpoint in the protocol, but these concentrations are used to derive the PK parameters. | Pharmacokinetic analysis set (PAS): consisted of all patients who received at least one dose of sonidegib and provided at least one evaluable PK blood sample. | Posted | Mean | Standard Deviation | ng*hr/mL | Week 1 Day 1,Week 9 Day 1 |
| ||||||||||||||||||||||||||||||
| Secondary | Parmacokintics (PK) Parameter: AUC0-24h | AUC0-24 is the area under the concentration-time curve from time zero to 24 hours done only on 800 mg once a day schedule. The PK parameters were determined in plasma using non-compartmental methods. A PK sample was excluded from analyses if the patient vomited within the first 4 hours following the last oral dose of study drug. Other PK samples were excluded as deemed appropriate by the Pharmacokineticist. AUC0-24h was derived from the PK concentrations collected at 0, 0.5, 1, 2, 4, 6, 8 and 24 hours post dose on W1D1 and W9D1. The PK concentration at each time point is not an endpoint in the protocol, but these concentrations are used to derive the PK parameters. | Pharmacokinetic analysis set (PAS): consisted of all patients who received at least one dose of sonidegib and provided at least one evaluable PK blood sample. | Posted | Mean | Standard Deviation | ng*hr/mL | Week 1 Day 1,Week 9 Day 1 |
|
Not provided
Not provided
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 | LDE225-400 | Patients who were randomized to Schedule A, and received 400 mg LDE225 twice daily for the first two weeks only and after two weeks, received 800 mg LDE225 once daily until disease progression, toxicity, withdrawal of consent, death, discretion of the investigator or early termination of the study. | 25 | 35 | 35 | 35 | ||
| EG001 | LDE225-800 | Patients who were randomized to Schedule B, received 800 mg LDE225 once daily until disease progression, toxicity, withdrawal of consent, death, discretion of the investigator or early termination of the study. | 25 | 34 | 34 | 34 |
| Term | Organ System | Source Vocabulary | Assessment Type | Notes | Statistical Information |
|---|---|---|---|---|---|
| ANAEMIA | Blood and lymphatic system disorders | MedDRA | Systematic Assessment |
| |
| FEBRILE NEUTROPENIA | Blood and lymphatic system disorders | MedDRA | Systematic Assessment |
| |
| LEUKOCYTOSIS | Blood and lymphatic system disorders | MedDRA | Systematic Assessment |
| |
| PANCYTOPENIA | Blood and lymphatic system disorders | MedDRA | Systematic Assessment |
| |
| ABDOMINAL PAIN | Gastrointestinal disorders | MedDRA | Systematic Assessment |
| |
| ABDOMINAL PAIN UPPER | Gastrointestinal disorders | MedDRA | Systematic Assessment |
| |
| COLITIS | Gastrointestinal disorders | MedDRA | Systematic Assessment |
| |
| CONSTIPATION | Gastrointestinal disorders | MedDRA | Systematic Assessment |
| |
| DIARRHOEA | Gastrointestinal disorders | MedDRA | Systematic Assessment |
| |
| GASTRIC HAEMORRHAGE | Gastrointestinal disorders | MedDRA | Systematic Assessment |
| |
| GASTROINTESTINAL HAEMORRHAGE | Gastrointestinal disorders | MedDRA | Systematic Assessment |
| |
| GINGIVAL HYPERTROPHY | Gastrointestinal disorders | MedDRA | Systematic Assessment |
| |
| NAUSEA | Gastrointestinal disorders | MedDRA | Systematic Assessment |
| |
| OESOPHAGEAL ULCER | Gastrointestinal disorders | MedDRA | Systematic Assessment |
| |
| STOMATITIS | Gastrointestinal disorders | MedDRA | Systematic Assessment |
| |
| VOMITING | Gastrointestinal disorders | MedDRA | Systematic Assessment |
| |
| ASTHENIA | General disorders | MedDRA | Systematic Assessment |
| |
| FATIGUE | General disorders | MedDRA | Systematic Assessment |
| |
| GENERAL PHYSICAL HEALTH DETERIORATION | General disorders | MedDRA | Systematic Assessment |
| |
| MULTI-ORGAN FAILURE | General disorders | MedDRA | Systematic Assessment |
| |
| PYREXIA | General disorders | MedDRA | Systematic Assessment |
| |
| ANAL ABSCESS | Infections and infestations | MedDRA | Systematic Assessment |
| |
| ARTHRITIS INFECTIVE | Infections and infestations | MedDRA | Systematic Assessment |
| |
| BACTERAEMIA | Infections and infestations | MedDRA | Systematic Assessment |
| |
| CLOSTRIDIAL INFECTION | Infections and infestations | MedDRA | Systematic Assessment |
| |
| DEVICE RELATED INFECTION | Infections and infestations | MedDRA | Systematic Assessment |
| |
| ENTEROCOCCAL BACTERAEMIA | Infections and infestations | MedDRA | Systematic Assessment |
| |
| EPIGLOTTITIS | Infections and infestations | MedDRA | Systematic Assessment |
| |
| INFECTION | Infections and infestations | MedDRA | Systematic Assessment |
| |
| INFLUENZA | Infections and infestations | MedDRA | Systematic Assessment |
| |
| LUNG INFECTION | Infections and infestations | MedDRA | Systematic Assessment |
| |
| NEUTROPENIC INFECTION | Infections and infestations | MedDRA | Systematic Assessment |
| |
| PNEUMONIA | Infections and infestations | MedDRA | Systematic Assessment |
| |
| SEPSIS | Infections and infestations | MedDRA | Systematic Assessment |
| |
| STAPHYLOCOCCAL SEPSIS | Infections and infestations | MedDRA | Systematic Assessment |
| |
| URINARY TRACT INFECTION | Infections and infestations | MedDRA | Systematic Assessment |
| |
| FALL | Injury, poisoning and procedural complications | MedDRA | Systematic Assessment |
| |
| SUBDURAL HAEMORRHAGE | Injury, poisoning and procedural complications | MedDRA | Systematic Assessment |
| |
| BLOOD CREATINE PHOSPHOKINASE INCREASED | Investigations | MedDRA | Systematic Assessment |
| |
| MYOGLOBIN BLOOD INCREASED | Investigations | MedDRA | Systematic Assessment |
| |
| DEHYDRATION | Metabolism and nutrition disorders | MedDRA | Systematic Assessment |
| |
| FLUID OVERLOAD | Metabolism and nutrition disorders | MedDRA | Systematic Assessment |
| |
| GOUT | Metabolism and nutrition disorders | MedDRA | Systematic Assessment |
| |
| BACK PAIN | Musculoskeletal and connective tissue disorders | MedDRA | Systematic Assessment |
| |
| MUSCLE SPASMS | Musculoskeletal and connective tissue disorders | MedDRA | Systematic Assessment |
| |
| MYALGIA | Musculoskeletal and connective tissue disorders | MedDRA | Systematic Assessment |
| |
| MYOPATHY | Musculoskeletal and connective tissue disorders | MedDRA | Systematic Assessment |
| |
| OSTEONECROSIS OF JAW | Musculoskeletal and connective tissue disorders | MedDRA | Systematic Assessment |
| |
| CANCER PAIN | Neoplasms benign, malignant and unspecified (incl cysts and polyps) | MedDRA | Systematic Assessment |
| |
| LEUKAEMIC INFILTRATION | Neoplasms benign, malignant and unspecified (incl cysts and polyps) | MedDRA | Systematic Assessment |
| |
| CEREBRAL HAEMORRHAGE | Nervous system disorders | MedDRA | Systematic Assessment |
| |
| DYSGEUSIA | Nervous system disorders | MedDRA | Systematic Assessment |
| |
| HAEMORRHAGE INTRACRANIAL | Nervous system disorders | MedDRA | Systematic Assessment |
| |
| QUADRIPLEGIA | Nervous system disorders | MedDRA | Systematic Assessment |
| |
| EPISTAXIS | Respiratory, thoracic and mediastinal disorders | MedDRA | Systematic Assessment |
| |
| IDIOPATHIC PNEUMONIA SYNDROME | Respiratory, thoracic and mediastinal disorders | MedDRA | Systematic Assessment |
| |
| LARYNGEAL INFLAMMATION | Respiratory, thoracic and mediastinal disorders | MedDRA | Systematic Assessment |
| |
| PLEURAL EFFUSION | Respiratory, thoracic and mediastinal disorders | MedDRA | Systematic Assessment |
| |
| RESPIRATORY FAILURE | Respiratory, thoracic and mediastinal disorders | MedDRA | Systematic Assessment |
| |
| CIRCULATORY COLLAPSE | Vascular disorders | MedDRA | Systematic Assessment |
|
| Term | Organ System | Source Vocabulary | Assessment Type | Notes | Statistical Information |
|---|---|---|---|---|---|
| ANAEMIA | Blood and lymphatic system disorders | MedDRA | Systematic Assessment |
| |
| FEBRILE NEUTROPENIA | Blood and lymphatic system disorders | MedDRA | Systematic Assessment |
| |
| LEUKOCYTOSIS | Blood and lymphatic system disorders | MedDRA | Systematic Assessment |
| |
| LEUKOPENIA | Blood and lymphatic system disorders | MedDRA | Systematic Assessment |
| |
| NEUTROPENIA | Blood and lymphatic system disorders | MedDRA | Systematic Assessment |
| |
| THROMBOCYTOPENIA | Blood and lymphatic system disorders | MedDRA | Systematic Assessment |
| |
| ATRIAL FIBRILLATION | Cardiac disorders | MedDRA | Systematic Assessment |
| |
| ABDOMINAL PAIN | Gastrointestinal disorders | MedDRA | Systematic Assessment |
| |
| ABDOMINAL PAIN UPPER | Gastrointestinal disorders | MedDRA | Systematic Assessment |
| |
| APHTHOUS STOMATITIS | Gastrointestinal disorders | MedDRA | Systematic Assessment |
| |
| CONSTIPATION | Gastrointestinal disorders | MedDRA | Systematic Assessment |
| |
| DIARRHOEA | Gastrointestinal disorders | MedDRA | Systematic Assessment |
| |
| DYSPEPSIA | Gastrointestinal disorders | MedDRA | Systematic Assessment |
| |
| DYSPHAGIA | Gastrointestinal disorders | MedDRA | Systematic Assessment |
| |
| GINGIVAL BLEEDING | Gastrointestinal disorders | MedDRA | Systematic Assessment |
| |
| HAEMORRHOIDS | Gastrointestinal disorders | MedDRA | Systematic Assessment |
| |
| MOUTH HAEMORRHAGE | Gastrointestinal disorders | MedDRA | Systematic Assessment |
| |
| MOUTH ULCERATION | Gastrointestinal disorders | MedDRA | Systematic Assessment |
| |
| NAUSEA | Gastrointestinal disorders | MedDRA | Systematic Assessment |
| |
| ORAL DISORDER | Gastrointestinal disorders | MedDRA | Systematic Assessment |
| |
| ORAL PAIN | Gastrointestinal disorders | MedDRA | Systematic Assessment |
| |
| VOMITING | Gastrointestinal disorders | MedDRA | Systematic Assessment |
| |
| ASTHENIA | General disorders | MedDRA | Systematic Assessment |
| |
| CHILLS | General disorders | MedDRA | Systematic Assessment |
| |
| FATIGUE | General disorders | MedDRA | Systematic Assessment |
| |
| FEELING COLD | General disorders | MedDRA | Systematic Assessment |
| |
| GENERAL PHYSICAL HEALTH DETERIORATION | General disorders | MedDRA | Systematic Assessment |
| |
| MALAISE | General disorders | MedDRA | Systematic Assessment |
| |
| NON-CARDIAC CHEST PAIN | General disorders | MedDRA | Systematic Assessment |
| |
| OEDEMA PERIPHERAL | General disorders | MedDRA | Systematic Assessment |
| |
| PAIN | General disorders | MedDRA | Systematic Assessment |
| |
| PYREXIA | General disorders | MedDRA | Systematic Assessment |
| |
| INFECTION | Infections and infestations | MedDRA | Systematic Assessment |
| |
| NASOPHARYNGITIS | Infections and infestations | MedDRA | Systematic Assessment |
| |
| ORAL HERPES | Infections and infestations | MedDRA | Systematic Assessment |
| |
| SKIN INFECTION | Infections and infestations | MedDRA | Systematic Assessment |
| |
| UPPER RESPIRATORY TRACT INFECTION | Infections and infestations | MedDRA | Systematic Assessment |
| |
| URINARY TRACT INFECTION | Infections and infestations | MedDRA | Systematic Assessment |
| |
| FALL | Injury, poisoning and procedural complications | MedDRA | Systematic Assessment |
| |
| ASPARTATE AMINOTRANSFERASE INCREASED | Investigations | MedDRA | Systematic Assessment |
| |
| BLOOD CREATINE PHOSPHOKINASE INCREASED | Investigations | MedDRA | Systematic Assessment |
| |
| BLOOD CREATININE INCREASED | Investigations | MedDRA | Systematic Assessment |
| |
| BLOOD MAGNESIUM DECREASED | Investigations | MedDRA | Systematic Assessment |
| |
| MYOGLOBIN BLOOD INCREASED | Investigations | MedDRA | Systematic Assessment |
| |
| NEUTROPHIL COUNT DECREASED | Investigations | MedDRA | Systematic Assessment |
| |
| PLATELET COUNT DECREASED | Investigations | MedDRA | Systematic Assessment |
| |
| WEIGHT DECREASED | Investigations | MedDRA | Systematic Assessment |
| |
| WHITE BLOOD CELL COUNT DECREASED | Investigations | MedDRA | Systematic Assessment |
| |
| DECREASED APPETITE | Metabolism and nutrition disorders | MedDRA | Systematic Assessment |
| |
| HYPERKALAEMIA | Metabolism and nutrition disorders | MedDRA | Systematic Assessment |
| |
| HYPOALBUMINAEMIA | Metabolism and nutrition disorders | MedDRA | Systematic Assessment |
| |
| HYPOCALCAEMIA | Metabolism and nutrition disorders | MedDRA | Systematic Assessment |
| |
| HYPOKALAEMIA | Metabolism and nutrition disorders | MedDRA | Systematic Assessment |
| |
| HYPOMAGNESAEMIA | Metabolism and nutrition disorders | MedDRA | Systematic Assessment |
| |
| BACK PAIN | Musculoskeletal and connective tissue disorders | MedDRA | Systematic Assessment |
| |
| BONE PAIN | Musculoskeletal and connective tissue disorders | MedDRA | Systematic Assessment |
| |
| MUSCLE SPASMS | Musculoskeletal and connective tissue disorders | MedDRA | Systematic Assessment |
| |
| MUSCULAR WEAKNESS | Musculoskeletal and connective tissue disorders | MedDRA | Systematic Assessment |
| |
| MUSCULOSKELETAL PAIN | Musculoskeletal and connective tissue disorders | MedDRA | Systematic Assessment |
| |
| MYALGIA | Musculoskeletal and connective tissue disorders | MedDRA | Systematic Assessment |
| |
| MYOPATHY | Musculoskeletal and connective tissue disorders | MedDRA | Systematic Assessment |
| |
| PAIN IN EXTREMITY | Musculoskeletal and connective tissue disorders | MedDRA | Systematic Assessment |
| |
| DIZZINESS | Nervous system disorders | MedDRA | Systematic Assessment |
| |
| DYSGEUSIA | Nervous system disorders | MedDRA | Systematic Assessment |
| |
| HEADACHE | Nervous system disorders | MedDRA | Systematic Assessment |
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| ANXIETY | Psychiatric disorders | MedDRA | Systematic Assessment |
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| CONFUSIONAL STATE | Psychiatric disorders | MedDRA | Systematic Assessment |
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| INSOMNIA | Psychiatric disorders | MedDRA | Systematic Assessment |
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| DYSURIA | Renal and urinary disorders | MedDRA | Systematic Assessment |
| |
| COUGH | Respiratory, thoracic and mediastinal disorders | MedDRA | Systematic Assessment |
| |
| DYSPNOEA | Respiratory, thoracic and mediastinal disorders | MedDRA | Systematic Assessment |
| |
| EPISTAXIS | Respiratory, thoracic and mediastinal disorders | MedDRA | Systematic Assessment |
| |
| OROPHARYNGEAL PAIN | Respiratory, thoracic and mediastinal disorders | MedDRA | Systematic Assessment |
| |
| PRODUCTIVE COUGH | Respiratory, thoracic and mediastinal disorders | MedDRA | Systematic Assessment |
| |
| ALOPECIA | Skin and subcutaneous tissue disorders | MedDRA | Systematic Assessment |
| |
| NIGHT SWEATS | Skin and subcutaneous tissue disorders | MedDRA | Systematic Assessment |
| |
| PETECHIAE | Skin and subcutaneous tissue disorders | MedDRA | Systematic Assessment |
| |
| RASH | Skin and subcutaneous tissue disorders | MedDRA | Systematic Assessment |
| |
| SKIN LESION | Skin and subcutaneous tissue disorders | MedDRA | Systematic Assessment |
| |
| HAEMATOMA | Vascular disorders | MedDRA | Systematic Assessment |
| |
| HYPERTENSION | Vascular disorders | MedDRA | Systematic Assessment |
| |
| ORTHOSTATIC HYPOTENSION | Vascular disorders | MedDRA | Systematic Assessment |
|
Only 34 of the 35 patients in the LDE225 800 mg once daily arm received at least dose of study drug were analyzed for safety.
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 | 82-778-8300 | trialandresults.registries@novartis.com |
| ID | Term |
|---|---|
| D015470 | Leukemia, Myeloid, Acute |
| D054198 | Precursor Cell Lymphoblastic Leukemia-Lymphoma |
| D007938 | Leukemia |
| D012008 | Recurrence |
| ID | Term |
|---|---|
| D007951 | Leukemia, Myeloid |
| D009370 | Neoplasms by Histologic Type |
| D009369 | Neoplasms |
| D006402 | Hematologic Diseases |
| D006425 | Hemic and Lymphatic Diseases |
| D007945 | Leukemia, Lymphoid |
| D008232 | Lymphoproliferative Disorders |
| D008206 | Lymphatic Diseases |
| D007160 | Immunoproliferative Disorders |
| D007154 | Immune System Diseases |
| D020969 | Disease Attributes |
| D010335 | Pathologic Processes |
| D013568 | Pathological Conditions, Signs and Symptoms |
Not provided
Not provided
| ID | Term |
|---|---|
| C561435 | sonidegib |
Not provided
Not provided
Not provided
| Male |
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| Units | Counts |
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| Participants |
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