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Low enrollment
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The purpose of this signal seeking study is to determine whether treatment with LDE225 demonstrates sufficient efficacy in hedgehog pathway-mutated solid tumors and/or hematologic malignancies to warrant further study
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| Label | Type | Description | Intervention Names |
|---|---|---|---|
| LDE225 | Experimental | LDE225 800 mg (hard gelatin capsules) will be administered orally once daily on a continuous dosing schedule. |
|
| Name | Type | Description | Arm Group Labels | Other Names |
|---|---|---|---|---|
| LDE225 | Drug | LDE225 800 mg (hard gelatin capsules) will be administered orally once daily on a continuous dosing schedule |
|
| Measure | Description | Time Frame |
|---|---|---|
| Summary of Overall Response (ORR) and Clinical Benefit (CBR) | Clinical benefit rate (CBR) Number and percentage of subjects with CBR (responses of CR, PR or SD ≥ 16 weeks) as assessed by investigator was reported for all patients along with 95% exact confidence interval (CI). Overall Response Rate (ORR) Overall response was to be determined by investigator assessment for each tumor in the study. For subjects with solid tumors, the assessment criteria was RECIST 1.1 and included responses of CR and/or PR. The number and percentage of subjects for different categories of overall response (e.g., for solid tumors - CR, PR, SD, PD, Not Evaluable) were to be provided for solid tumors, and each hematological tumor type (if applicable). Ninety-five percent (95%) exact CI was to be provided for the response rate(s) (e.g., for solid tumors - CRn and/or PR) as well. | 16 weeks |
| Measure | Description | Time Frame |
|---|---|---|
| Summary of Timing and Estimated Rate for Progression-free Survival (PFS) - Full Analysis Set | Progression-free survival (PFS) is the time from the date of start of treatment to the date of event defined as the first documented progression or death due to any cause within 30 days of last dose. If a subject has not had an event, progression-free survival is censored at the date of last adequate tumor assessment. |
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Inclusion Criteria:
Exclusion Criteria:
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| Facility | Status | City | State | ZIP | Country | Contacts |
|---|---|---|---|---|---|---|
| University of California Davis Cancer Center UC Davis Cancer (3) | Sacramento | California | 95817 | United States | ||
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The study was closed for accrual when the sponsor realized that not enough patients will be recruited for any meaningful stat analysis even if the study were kept open beyond the original planned accrual window.
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| ID | Title | Description |
|---|---|---|
| FG000 | Sonidegib | All the patients received sonidegib on a flat scale of 800 mg (e.g., 4 x 200 mg hard gelatin capsules) once daily on a continuous dosing cycle. |
| Title | Milestones | Reasons Not Completed | |||||||||||||||||||||
|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|
| Overall Study |
|
|
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| ID | Title | Description |
|---|---|---|
| BG000 | Sonidegib | All the patients received sonidegib on a flat scale of 800 mg (e.g., 4 x 200 mg hard gelatin capsules) once daily on a continuous dosing cycle. |
| Units | Counts |
|---|---|
| Participants |
|
| Title | Description | Population Description | Parameter Type | Dispersion Type | Unit of Measure | Calculate Percentage | Denominator Units Selected | Denominators | Classes |
|---|---|---|---|---|---|---|---|---|---|
| Age, Categorical | Count of 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 | Summary of Overall Response (ORR) and Clinical Benefit (CBR) | Clinical benefit rate (CBR) Number and percentage of subjects with CBR (responses of CR, PR or SD ≥ 16 weeks) as assessed by investigator was reported for all patients along with 95% exact confidence interval (CI). Overall Response Rate (ORR) Overall response was to be determined by investigator assessment for each tumor in the study. For subjects with solid tumors, the assessment criteria was RECIST 1.1 and included responses of CR and/or PR. The number and percentage of subjects for different categories of overall response (e.g., for solid tumors - CR, PR, SD, PD, Not Evaluable) were to be provided for solid tumors, and each hematological tumor type (if applicable). Ninety-five percent (95%) exact CI was to be provided for the response rate(s) (e.g., for solid tumors - CRn and/or PR) as well. | Full analysis set | Posted | Number | percent responders | 16 weeks |
|
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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 | LDE225 | All the patients received sonidegib on a flat scale of 800 mg (e.g., 4 x 200 mg hard gelatin capsules) once daily on a continuous dosing cycle. |
| Term | Organ System | Source Vocabulary | Assessment Type | Notes | Statistical Information |
|---|---|---|---|---|---|
| Abdominal pain | Gastrointestinal disorders | MedDRA | Systematic Assessment |
| Term | Organ System | Source Vocabulary | Assessment Type | Notes | Statistical Information |
|---|---|---|---|---|---|
| Anaemia | Blood and lymphatic system disorders | MedDRA | Systematic Assessment |
The study was closed for accrual when the sponsor realized that not enough patients will be recruited for any meaningful stat analysis even if the study were kept open beyond the original planned accrual window.
| Title | Organization | Phone | Extension | |
|---|---|---|---|---|
| Clinical Disclosure Office | Novartis Pharmaceuticals | 862-778-8300 |
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| ID | Term |
|---|---|
| D001478 | Basal Cell Nevus Syndrome |
| D019337 | Hematologic Neoplasms |
| D016889 | Endometrial Neoplasms |
| D003110 | Colonic Neoplasms |
| ID | Term |
|---|---|
| D009807 | Odontogenic Cysts |
| D007570 | Jaw Cysts |
| D001845 | Bone Cysts |
| D003560 | Cysts |
| D009369 |
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| ID | Term |
|---|---|
| C561435 | sonidegib |
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| 4 months |
| Kaplan-Meier Estimates of Progression Free Survival (PFS )Timing, Months | 4 months |
| Rocky Mountain Cancer Centers RMCC - Aurora |
| Greenwood Village |
| Colorado |
| United States |
| Lurie Children's Hospital of Chicago Developmental Therapeutics | Chicago | Illinois | 60611 | United States |
| Minnesota Oncology Hematology, P.A. Southdate Medical Center | Minneapolis | Minnesota | 55404 | United States |
| Cleveland Clinic Foundation Cleveland Clinic (19) | Cleveland | Ohio | 44195 | United States |
| Sanford Research Sanford Health | Sioux Falls | South Dakota | 57104 | United States |
| Oncology Consultants Oncology Group | Houston | Texas | 77024 | United States |
| MD Anderson Cancer Center/University of Texas MD Anderson Cancer Center (3) | Houston | Texas | 77030 | United States |
| Intermountain Medical Center Intermountain Healthcare | Murray | Utah | 84157 | United States |
| Seattle Cancer Care Alliance Skagit Valley Hospital | Seattle | Washington | 98109-1023 | United States |
| Participants |
|
| Age, Continuous | Mean | Standard Deviation | years |
|
| Sex: Female, Male | Count of Participants | Participants |
|
All the patients received sonidegib on a flat scale of 800 mg (e.g., 4 x 200 mg hard gelatin capsules) once daily on a continuous dosing cycle. |
|
|
| Secondary | Summary of Timing and Estimated Rate for Progression-free Survival (PFS) - Full Analysis Set | Progression-free survival (PFS) is the time from the date of start of treatment to the date of event defined as the first documented progression or death due to any cause within 30 days of last dose. If a subject has not had an event, progression-free survival is censored at the date of last adequate tumor assessment. | Full Analysis set | Posted | Number | 95% Confidence Interval | % progression free surviors | 4 months |
|
|
|
| Secondary | Kaplan-Meier Estimates of Progression Free Survival (PFS )Timing, Months | Posted | Median | 95% Confidence Interval | months | 4 months |
|
|
|
| 7 |
| 10 |
| 10 |
| 10 |
| Dysphagia | Gastrointestinal disorders | MedDRA | Systematic Assessment |
|
| Small intestinal obstruction | Gastrointestinal disorders | MedDRA | Systematic Assessment |
|
| Pneumonia | Infections and infestations | MedDRA | Systematic Assessment |
|
| Dehydration | Metabolism and nutrition disorders | MedDRA | Systematic Assessment |
|
| Failure to thrive | Metabolism and nutrition disorders | MedDRA | Systematic Assessment |
|
| Muscular weakness | Musculoskeletal and connective tissue disorders | MedDRA | Systematic Assessment |
|
| Chronic obstructive pulmonary disease | Respiratory, thoracic and mediastinal disorders | MedDRA | Systematic Assessment |
|
| Cough | Respiratory, thoracic and mediastinal disorders | MedDRA | Systematic Assessment |
|
| Dysphonia | Respiratory, thoracic and mediastinal disorders | MedDRA | Systematic Assessment |
|
| Dyspnoea | Respiratory, thoracic and mediastinal disorders | MedDRA | Systematic Assessment |
|
| Pulmonary embolism | Respiratory, thoracic and mediastinal disorders | MedDRA | Systematic Assessment |
|
| Abdominal pain | 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 |
|
| Nausea | Gastrointestinal disorders | MedDRA | Systematic Assessment |
|
| Vomiting | Gastrointestinal disorders | MedDRA | Systematic Assessment |
|
| Asthenia | General disorders | MedDRA | Systematic Assessment |
|
| Fatigue | General disorders | MedDRA | Systematic Assessment |
|
| Oedema peripheral | General disorders | MedDRA | Systematic Assessment |
|
| Pain | General disorders | MedDRA | Systematic Assessment |
|
| Urinary tract infection | Infections and infestations | MedDRA | Systematic Assessment |
|
| Activated partial thromboplastin time prolonged | Investigations | MedDRA | Systematic Assessment |
|
| Alanine aminotransferase increased | Investigations | MedDRA | Systematic Assessment |
|
| Aspartate aminotransferase increased | Investigations | MedDRA | Systematic Assessment |
|
| Blood alkaline phosphatase increased | Investigations | MedDRA | Systematic Assessment |
|
| Blood creatine phosphokinase increased | Investigations | MedDRA | Systematic Assessment |
|
| Blood lactate dehydrogenase increased | Investigations | MedDRA | Systematic Assessment |
|
| Blood uric acid increased | Investigations | MedDRA | Systematic Assessment |
|
| Gamma-glutamyltransferase increased | Investigations | MedDRA | Systematic Assessment |
|
| Haemoglobin 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 |
|
| Dehydration | Metabolism and nutrition disorders | MedDRA | Systematic Assessment |
|
| Hypercalcaemia | Metabolism and nutrition disorders | MedDRA | Systematic Assessment |
|
| Hyperglycaemia | Metabolism and nutrition disorders | MedDRA | Systematic Assessment |
|
| Hypoglycaemia | Metabolism and nutrition disorders | MedDRA | Systematic Assessment |
|
| Hypomagnesaemia | Metabolism and nutrition disorders | MedDRA | Systematic Assessment |
|
| Hyponatraemia | Metabolism and nutrition disorders | MedDRA | Systematic Assessment |
|
| Arthralgia | Musculoskeletal and connective tissue disorders | MedDRA | Systematic Assessment |
|
| Back pain | Musculoskeletal and connective tissue disorders | MedDRA | Systematic Assessment |
|
| Muscle spasms | Musculoskeletal and connective tissue disorders | MedDRA | Systematic Assessment |
|
| Musculoskeletal chest pain | Musculoskeletal and connective tissue disorders | MedDRA | Systematic Assessment |
|
| Myalgia | Musculoskeletal and connective tissue disorders | MedDRA | Systematic Assessment |
|
| Dysgeusia | Nervous system disorders | MedDRA | Systematic Assessment |
|
| Headache | Nervous system disorders | MedDRA | Systematic Assessment |
|
| Neuropathy peripheral | Nervous system disorders | MedDRA | Systematic Assessment |
|
| Depression | Psychiatric disorders | MedDRA | Systematic Assessment |
|
| Irritability | Psychiatric disorders | MedDRA | Systematic Assessment |
|
| Proteinuria | 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 |
|
| Pleural effusion | Respiratory, thoracic and mediastinal disorders | MedDRA | Systematic Assessment |
|
| Alopecia | Skin and subcutaneous tissue disorders | MedDRA | Systematic Assessment |
|
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 the pooled data (ie, data from all sites) in the clinical trial.
| Neoplasms |
| D002280 | Carcinoma, Basal Cell |
| D002277 | Carcinoma |
| D009375 | Neoplasms, Glandular and Epithelial |
| D009370 | Neoplasms by Histologic Type |
| D018295 | Neoplasms, Basal Cell |
| D009386 | Neoplastic Syndromes, Hereditary |
| D001848 | Bone Diseases, Developmental |
| D001847 | Bone Diseases |
| D009140 | Musculoskeletal Diseases |
| D007571 | Jaw Diseases |
| D009057 | Stomatognathic Diseases |
| D000015 | Abnormalities, Multiple |
| D000013 | Congenital Abnormalities |
| D009358 | Congenital, Hereditary, and Neonatal Diseases and Abnormalities |
| D030342 | Genetic Diseases, Inborn |
| D009371 | Neoplasms by Site |
| D006402 | Hematologic Diseases |
| D006425 | Hemic and Lymphatic Diseases |
| D014594 | Uterine Neoplasms |
| D005833 | Genital Neoplasms, Female |
| D014565 | Urogenital Neoplasms |
| D014591 | Uterine Diseases |
| D005831 | Genital Diseases, Female |
| D052776 | Female Urogenital Diseases |
| D005261 | Female Urogenital Diseases and Pregnancy Complications |
| D000091642 | Urogenital Diseases |
| D000091662 | Genital Diseases |
| D015179 | Colorectal Neoplasms |
| D007414 | Intestinal Neoplasms |
| D005770 | Gastrointestinal Neoplasms |
| D004067 | Digestive System Neoplasms |
| D004066 | Digestive System Diseases |
| D005767 | Gastrointestinal Diseases |
| D003108 | Colonic Diseases |
| D007410 | Intestinal Diseases |
| Title | Measurements |
|---|---|
|
| 4 Months |
|