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| ID | Type | Description | Link |
|---|---|---|---|
| 2010-022629-14 | EudraCT Number |
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This study assessed the efficacy and safety of oral treatment with two dose levels of LDE225 in patients with locally advanced or metastatic BCC.
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| Label | Type | Description | Intervention Names |
|---|---|---|---|
| LDE225 200 mg | Experimental | The study was double blinded and enrolled at least 50 evaluable patients in the 200 mg LDE225 arm. The efficacy and safety of LDE225 was analyzed separately in each group. Patients who met all the inclusion and none of the exclusion criteria were treated with 200 mg LDE225 daily until disease progression, occurrence of intolerable toxicity, start of another anticancer treatment or withdrawal of consent. |
|
| LDE225 800 mg | Experimental | The study was double blinded and enrolled at least 100 evaluable patients in the 800 mg LDE225 arm. The efficacy and safety of LDE225 was analyzed separately in each group. Patients who met all the inclusion and none of the exclusion criteria were treated with 800 mg LDE225 daily until disease progression, occurrence of intolerable toxicity, start of another anticancer treatment or withdrawal of consent. |
|
| Name | Type | Description | Arm Group Labels | Other Names |
|---|---|---|---|---|
| LDE225 | Drug | LDE225 was administered orally, on a continuous once daily dosing schedule and was supplied as 200 mg hard gelatin capsules in bottles. Every 4 weeks on the day of study visit, patients received a prescription of an adequate drug supply for self-administration at home. The 800 mg dose patients received 4 capsules of LDE225 and 200 mg dose arm patients received 1 LDE225 capsule + 3 placebo capsules. |
| Measure | Description | Time Frame |
|---|---|---|
| Objective Response Rate (ORR) Based on Central Review According to mRECIST (for Locally Advanced Basal Cell Carcinoma (laBCC)) and RECIST 1.1 (for Metastatic Basal Cell Carcinoma (mBCC)) Per Primary Efficacy Analysis Set (pEAS) | ORR is the percentage of patient's objective response (ORR) by 6 months after starting LDE225 treatment. A responder was defined as a subject with confirmed partial response (PR) or confirmed complete response (CR) 6 months after starting LDE225 treatment.Treatment with sonidegib was considered sufficiently efficacious if the observed ORR on any treatment arm at the end of the study was 30% or higher. PR: At least a 30% decrease in the sum of diameter of all target lesions, taking as reference the baseline sum of diameters. CR: Disappearance of all non-nodal target lesions. In addition, any pathological lymph nodes assigned as target lesions must have a reduction in short axis to < 10 mm. | 6 months |
| Objective Response Rate (ORR) Based on Central Review According to mRECIST (for Locally Advanced Basal Cell Carcinoma (laBCC)) and RECIST 1.1 (Metastatic Basal Cell Carcinoma (mBCC)) Per Full Analysis Set (FAS) | ORR is the percentage of patient's objective response (ORR) by 6 months after starting LDE225 treatment. A responder was defined as a subject with confirmed partial response (PR) or confirmed complete response (CR) 6 months after starting LDE225 treatment.Treatment with sonidegib was to be considered sufficiently efficacious if the observed ORR on any treatment arm at the end of the study was 30% or higher. PR: At least a 30% decrease in the sum of diameter of all target lesions, taking as reference the baseline sum of diameters. CR: Disappearance of all non-nodal target lesions. In addition, any pathological lymph nodes assigned as target lesions must have a reduction in short axis to < 10 mm. | 6 months |
| Measure | Description | Time Frame |
|---|---|---|
| Duration of Response (DoR) Per Central Review Using mRECIST for laBCC and RECIST 1.1 for mBCC (pEAS) | Duration of response is the time from the first observed confirmed response (CR or PR) to disease progression or death due to any reason. Duration of response was for participants with ORR. PR: At least a 30% decrease in the sum of diameter of all target lesions, taking as reference the baseline sum of diameters. CR: Disappearance of all non-nodal target lesions. In addition, any pathological lymph nodes assigned as target lesions must have a reduction in short axis to < 10 mm. Progressive disease (PD): At least a 20% increase in the sum of diameter of all measured target lesions, taking as reference the smallest sum of diameter of all target lesions recorded at or after baseline. In addition to the relative increase of 20%, the sum must also demonstrate an absolute increase of at least 5 mm2. |
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Inclusion Criteria:
Exclusion Criteria:
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| Name | Affiliation | Role |
|---|---|---|
| Novartis Pharmaceuticals | Novartis Pharmaceuticals | Study Director |
| Facility | Status | City | State | ZIP | Country | Contacts |
|---|---|---|---|---|---|---|
| Highlands Oncology Group | Fayetteville | Arkansas | 72703 | United States | ||
| University of California at Los Angeles UCLA 3 |
| PubMed Identifier | Type | Citation | Retractions |
|---|---|---|---|
| 34798846 | Derived | Gutzmer R, Robert C, Loquai C, Schadendorf D, Squittieri N, Arntz R, Martelli S, Dummer R. Assessment of various efficacy outcomes using ERIVANCE-like criteria in patients with locally advanced basal cell carcinoma receiving sonidegib: results from a preplanned sensitivity analysis. BMC Cancer. 2021 Nov 19;21(1):1244. doi: 10.1186/s12885-021-08968-1. | |
| 34669179 |
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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 patients 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.
All eligible, enrolled patients were randomized in 1:2 ratio to sonidegib treatment with either 200 mg or 800 mg once-daily dose. In total, 230 patients were evaluated as FAS population: 79 and 151 patients randomized to 200mg and 800 mg sonidegib respectively. However, 1 patient randomized to 800 mg sonidegib did not receive study treatment.
Randomization was stratified across the two treatment arms according to the stage of disease (laBCC or mBCC), histological subtype (non-aggressive or aggressive for laBCC patients) and the regions (Australia, Europe, and North America).
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| ID | Title | Description |
|---|---|---|
| FG000 | LDE225 (Sonidegib) 200 mg | The study is double blinded and will enroll at least 50 evaluable patients in the 200 mg LDE225 arm. The efficacy and safety of LDE225 will be analyzed separately in each group. Patients who meet all the inclusion and none of the exclusion criteria will be treated with 200 mg LDE225 daily until disease progression, occurrence of intolerable toxicity, start of another anticancer treatment or withdrawal of consent. |
| Title | Milestones | Reasons Not Completed | |||||
|---|---|---|---|---|---|---|---|
| Overall Study |
|
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| 42 months |
| Duration of Response (DoR) Per Central Review Using mRECIST for laBCC and RECIST 1.1 for mBCC (FAS) | Duration of response is the time from the first observed confirmed response (CR or PR) to disease progression or death due to any reason. Median DoR for patients with laBCC was non-estimable for both treatment arms. Duration of response was for participants with ORR. PR: At least a 30% decrease in the sum of diameter of all target lesions, taking as reference the baseline sum of diameters. CR: Disappearance of all non-nodal target lesions. In addition, any pathological lymph nodes assigned as target lesions must have a reduction in short axis to < 10 mm. | 42 months |
| Complete Response Rate (CRR) Per Central Review (pEAS) | Rate of complete response is the percentage of patients with best overall response of complete response (CR) after starting LDE225 treatment. The rate of CR was determined according to mRECIST for laBCC and RECIST 1.1 for mBCC. Patients with best overall response of 'Unknown" were treated as non responders. Complete Response (CR): Disappearance of all non-nodal target lesions. In addition, any pathological lymph nodes assigned as target lesions must have a reduction in short axis to < 10 mm. | 42 months |
| Complete Response Rate (CRR) Per Central Review (FAS) | Rate of complete response is the proportion of patients with best overall response of complete response (CR) after starting LDE225 treatment. The rate of CR will be determined according to mRECIST for laBCC and RECIST 1.1 for mBCC. Patients with best overall response of 'Unknown" will be treated as non responders | 6 months |
| Progression-free Survival (PFS) Per Central Review Using mRECIST for laBCC and RECIST 1.1 for mBCC (pEAS) | Progression-free survival (PFS) is the time from date of randomization/start of treatment to the date of event defined as the first documented progression or death due to any cause. If a patient has not had an event, progression-free survival is censored at the date of last adequate tumor assessment. | 42 months |
| Progression-free Survival (PFS) Per Central Review Using mRECIST for laBCC and RECIST 1.1 for mBCC (FAS) | Progression-free survival (PFS) is the time from date of randomization/start of treatment to the date of event defined as the first documented progression or death due to any cause. If a patient has not had an event, PFS is censored at the date of last adequate tumor assessment. | 42 months |
| Time to Tumor Response (TTR) Per Central Review Using mRECIST for laBCC and RECIST 1.1 for mBCC (pEAS) | Time to tumor response (TTR) is defined as the time from date of enrollment to the date of first documented tumor response (CR or PR). PR: At least a 30% decrease in the sum of diameter of all target lesions, taking as reference the baseline sum of diameters. CR: Disappearance of all non-nodal target lesions. In addition, any pathological lymph nodes assigned as target lesions must have a reduction in short axis to < 10 mm. | 42 months |
| Time to Tumor Response (TTR) Per Central Review Using mRECIST for laBCC and RECIST 1.1 for mBCC (FAS) | Time to tumor response (TTR) is defined as the time from date of enrollment to the date of first documented tumor response (CR or PR). PR: At least a 30% decrease in the sum of diameter of all target lesions, taking as reference the baseline sum of diameters. CR: Disappearance of all non-nodal target lesions. In addition, any pathological lymph nodes assigned as target lesions must have a reduction in short axis to < 10 mm. | 42 months |
| Objective Response Rate (ORR) Based on Site Investigator Review According to mRECIST (for Locally Advanced Basal Cell Carcinoma (laBCC)) and RECIST 1.1 (for Metastatic Basal Cell Carcinoma (mBCC)) Per Primary Efficacy Analysis Set (pEAS) | ORR is the percentage of patient's objective response (ORR) by 42 months after starting LDE225 treatment. A responder was defined as a subject with confirmed partial response (PR) or confirmed complete response (CR) 42 months after starting LDE225 treatment. Treatment with sonidegib was considered sufficiently efficacious if the observed ORR on any treatment arm at the end of the study was 30% or higher. PR: At least a 30% decrease in the sum of diameter of all target lesions, taking as reference the baseline sum of diameters. CR: Disappearance of all non-nodal target lesions. In addition, any pathological lymph nodes assigned as target lesions must have a reduction in short axis to < 10 mm. | 42 months |
| Objective Response Rate (ORR) Based on Site Investigator Review According to mRECIST (for Locally Advanced Basal Cell Carcinoma (laBCC)) and RECIST 1.1 (Metastatic Basal Cell Carcinoma (mBCC)) Per Full Analysis Set (FAS) | ORR is the percentage of patient's objective response (ORR) by 6 months after starting LDE225 treatment. A responder was defined as a subject with confirmed partial response (PR) or confirmed complete response (CR) 42 months after starting LDE225 treatment.Treatment with sonidegib was to be considered sufficiently efficacious if the observed ORR on any treatment arm at the end of the study was 30% or higher. PR: At least a 30% decrease in the sum of diameter of all target lesions, taking as reference the baseline sum of diameters. CR: Disappearance of all non-nodal target lesions. In addition, any pathological lymph nodes assigned as target lesions must have a reduction in short axis to < 10 mm. | 42 months |
| Duration of Response (DoR) Per Site Investigator Review Using mRECIST for laBCC and RECIST 1.1 for mBCC (pEAS) | Duration of response is the time from the first observed confirmed response (CR or PR) to disease progression or death due to any reason. Duration of response was for participants with ORR. PR: At least a 30% decrease in the sum of diameter of all target lesions, taking as reference the baseline sum of diameters. CR: Disappearance of all non-nodal target lesions. In addition, any pathological lymph nodes assigned as target lesions must have a reduction in short axis to < 10 mm. Progressive disease (PD): At least a 20% increase in the sum of diameter of all measured target lesions, taking as reference the smallest sum of diameter of all target lesions recorded at or after baseline. In addition to the relative increase of 20%, the sum must also demonstrate an absolute increase of at least 5 mm2. | 42 months |
| Duration of Response (DoR) Per Site Investigator Review Using mRECIST for laBCC and RECIST 1.1 for mBCC (FAS) | Duration of response is the time from the first observed confirmed response (CR or PR) to disease progression or death due to any reason. Duration of response was for participants with ORR. PR: At least a 30% decrease in the sum of diameter of all target lesions, taking as reference the baseline sum of diameters. CR: Disappearance of all non-nodal target lesions. In addition, any pathological lymph nodes assigned as target lesions must have a reduction in short axis to < 10 mm. | 42 months |
| Progression-free Survival (PFS) Per Site Investigator Review Using mRECIST for laBCC and RECIST 1.1 for mBCC (pEAS) | Progression-free survival (PFS) is the time from date of randomization/start of treatment to the date of event defined as the first documented progression or death due to any cause. If a patient has not had an event, progression-free survival is censored at the date of last adequate tumor assessment. | 42 months |
| Time to Tumor Response (TTR) Per Site Investigator Review Using mRECIST for laBCC and RECIST 1.1 for mBCC (pEAS) | Time to tumor response (TTR) is defined as the time from date of enrollment to the date of first documented tumor response (CR or PR). PR: At least a 30% decrease in the sum of diameter of all target lesions, taking as reference the baseline sum of diameters. CR: Disappearance of all non-nodal target lesions. In addition, any pathological lymph nodes assigned as target lesions must have a reduction in short axis to < 10 mm. | 42 months |
| Plasma Concentration of Sonidegib (LDE225) | Blood PK samples were collected either by direct venipuncture or an indwelling cannula inserted in a forearm vein for the determination of trough (Cmin) plasma concentrations of sonidegib and its main circulating metabolite, LGE899, from all patients who enrolled in the study. Blood was collected in Weeks 1, 3, 5, 9 (pre-dose), and subsequently pre-dose every 4 weeks up to Week 21, and every 12 weeks thereafter up to week 69. | Weeks 1, 3, 5, 9, 13, 17, 21, 33, 45, 57, 69 |
| Overall Survival (OS) | OS is defined as the time from date of randomization to date of death due to any cause or the last date that a patient was known to be alive (censored observation) as of the data cut-off. | 42 months |
| Los Angeles |
| California |
| 90095 |
| United States |
| Stanford University Medical Center Stanford Univ 2 | Stanford | California | 94304 | United States |
| University of Colorado School of Medicine UC | Aurora | Colorado | 80045 | United States |
| Washington Hospital Center Wash Hospital | Washington D.C. | District of Columbia | 20010 | United States |
| H Lee Moffitt Cancer Center and Research Institute Cutaneous Onc Dept | Tampa | Florida | 33612 | United States |
| NorthwesternUniv.Med.School/Robert H. Lurie Comp.Cancer Ctr Study coordinator | Chicago | Illinois | 60611 | United States |
| Dana Farber Cancer Institute DFCI - MA | Boston | Massachusetts | 02215 | United States |
| Henry Ford Hospital Henry Ford | Detroit | Michigan | 48202 2689 | United States |
| Washington University School Of Medicine-Siteman Cancer Ctr Siteman | St Louis | Missouri | 63110 | United States |
| Comprehensive Cancer Centers of Nevada CCC of Nevada- Southwest (2) | Las Vegas | Nevada | 89109 | United States |
| Hackensack University Medical Center Hackensack (SC) | Hackensack | New Jersey | 07601 | United States |
| New York University Medical Center SC-2 | New York | New York | 10016 | United States |
| Penn State University / Milton S. Hershey Medical Center Hershey Medical | Hershey | Pennsylvania | 17033-085 | United States |
| University of Pittsburgh Medical Center UPMC | Pittsburgh | Pennsylvania | 15213 | United States |
| Baylor Health Care System/Sammons Cancer Center Baylor Texas Oncology | Dallas | Texas | 75246 | United States |
| Texas Oncology Tex Onc 3 | Dallas | Texas | 75246 | United States |
| Texas Oncology Texas Onc - Amarillo | Dallas | Texas | 75246 | United States |
| University of Texas MD Anderson Cancer Center MD Anderson | Houston | Texas | 77030 | United States |
| Texas Oncology Cancer Care & Research Center | Waco | Texas | 76712 | United States |
| Texoma Cancer Center Texoma Cancer Center | Wichita Falls | Texas | 76310 | United States |
| University of Utah / Huntsman Cancer Institute Huntsman/Univ UT | Salt Lake City | Utah | 84103 | United States |
| Novartis Investigative Site | St Leonards | New South Wales | 2065 | Australia |
| Novartis Investigative Site | Geelong | Victoria | 3220 | Australia |
| Novartis Investigative Site | Brussels | 1200 | Belgium |
| Novartis Investigative Site | Wilrijk | 2610 | Belgium |
| Novartis Investigative Site | Waterloo | Ontario | N2J 1C4 | Canada |
| Novartis Investigative Site | Sainte-Foy | Quebec | G1V 4T3 | Canada |
| Novartis Investigative Site | Lyon | 69373 | France |
| Novartis Investigative Site | Marseille | 13885 | France |
| Novartis Investigative Site | Pierre-Bénite | 69495 | France |
| Novartis Investigative Site | Toulouse | 31059 | France |
| Novartis Investigative Site | Villejuif | 94805 | France |
| Novartis Investigative Site | Berlin | 13353 | Germany |
| Novartis Investigative Site | Essen | 45147 | Germany |
| Novartis Investigative Site | Freiburg im Breisgau | 79106 | Germany |
| Novartis Investigative Site | Gera | 07548 | Germany |
| Novartis Investigative Site | Hanover | 30625 | Germany |
| Novartis Investigative Site | Kiel | 24105 | Germany |
| Novartis Investigative Site | Mainz | 55131 | Germany |
| Novartis Investigative Site | München | 81377 | Germany |
| Novartis Investigative Site | Münster | 48157 | Germany |
| Novartis Investigative Site | Stade | 21682 | Germany |
| Novartis Investigative Site | Athens | 161 21 | Greece |
| Novartis Investigative Site | Budapest | H-1085 | Hungary |
| Novartis Investigative Site | Debrecen | 4032 | Hungary |
| Novartis Investigative Site | Szeged | H-6725 | Hungary |
| Novartis Investigative Site | Torino | TO | 10126 | Italy |
| Novartis Investigative Site | Naples | 80131 | Italy |
| Novartis Investigative Site | Barcelona | Catalonia | 08035 | Spain |
| Novartis Investigative Site | Madrid | 28034 | Spain |
| Novartis Investigative Site | Madrid | 28046 | Spain |
| Novartis Investigative Site | Bern | 3010 | Switzerland |
| Novartis Investigative Site | Geneva | 1211 | Switzerland |
| Novartis Investigative Site | Zurich | 8091 | Switzerland |
| Novartis Investigative Site | High Heaton | Newcastle Upon Tyne | NE7 7DN | United Kingdom |
| Novartis Investigative Site | Yeovil | Somerset | BA21 4AT | United Kingdom |
| Novartis Investigative Site | Cardiff | CF14 4XW | United Kingdom |
| Novartis Investigative Site | Glasgow | G3 8SJ | United Kingdom |
| Novartis Investigative Site | Leicester | LE1 5WW | United Kingdom |
| Novartis Investigative Site | London | EC1A 7BE | United Kingdom |
| Novartis Investigative Site | Manchester | M13 9WL | United Kingdom |
| Lewis K, Dummer R, Farberg AS, Guminski A, Squittieri N, Migden M. Effects of Sonidegib Following Dose Reduction and Treatment Interruption in Patients with Advanced Basal Cell Carcinoma During 42-Month BOLT Trial. Dermatol Ther (Heidelb). 2021 Dec;11(6):2225-2234. doi: 10.1007/s13555-021-00619-4. Epub 2021 Oct 20. |
| 28846163 | Derived | Lear JT, Migden MR, Lewis KD, Chang ALS, Guminski A, Gutzmer R, Dirix L, Combemale P, Stratigos A, Plummer R, Castro H, Yi T, Mone M, Zhou J, Trefzer U, Kaatz M, Loquai C, Kudchadkar R, Sellami D, Dummer R. Long-term efficacy and safety of sonidegib in patients with locally advanced and metastatic basal cell carcinoma: 30-month analysis of the randomized phase 2 BOLT study. J Eur Acad Dermatol Venereol. 2018 Mar;32(3):372-381. doi: 10.1111/jdv.14542. Epub 2017 Nov 6. |
| 25981810 | Derived | Migden MR, Guminski A, Gutzmer R, Dirix L, Lewis KD, Combemale P, Herd RM, Kudchadkar R, Trefzer U, Gogov S, Pallaud C, Yi T, Mone M, Kaatz M, Loquai C, Stratigos AJ, Schulze HJ, Plummer R, Chang AL, Cornelis F, Lear JT, Sellami D, Dummer R. Treatment with two different doses of sonidegib in patients with locally advanced or metastatic basal cell carcinoma (BOLT): a multicentre, randomised, double-blind phase 2 trial. Lancet Oncol. 2015 Jun;16(6):716-28. doi: 10.1016/S1470-2045(15)70100-2. Epub 2015 May 14. |
| FG001 | LDE225 (Sonidegib) 800 mg | The study is double blinded and will enroll at least 100 evaluable patients in the 800 mg LDE225 arm. The efficacy and safety of LDE225 will be analyzed separately in each group. Patients who meet all the inclusion and none of the exclusion criteria will be treated with 800 mg LDE225 daily until disease progression, occurrence of intolerable toxicity, start of another anticancer treatment or withdrawal of consent. |
| Untreated |
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| Patients Cont. to Next Phase of Trial |
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| Survival Follow-up |
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| Post-treatment Follow-up |
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| COMPLETED |
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| NOT COMPLETED |
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The full analysis set (FAS) comprised all patients who were assigned study treatment irrespective of receiving it (all randomized patients). Patients were classified according to the treatment they were assigned in accordance with the intention-to-treat (ITT) principle.
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| ID | Title | Description |
|---|---|---|
| BG000 | LDE225 (Sonidegib) 200 mg | The study is double blinded and will enroll at least 50 evaluable patients in the 200 mg LDE225 arm. The efficacy and safety of LDE225 will be analyzed separately in each group. Patients who meet all the inclusion and none of the exclusion criteria will be treated with 200 mg LDE225 daily until disease progression, occurrence of intolerable toxicity, start of another anticancer treatment or withdrawal of consent. |
| BG001 | LDE225 (Sonidegib) 800 mg | The study is double blinded and will enroll at least 100 evaluable patients in the 800 mg LDE225 arm. The efficacy and safety of LDE225 will be analyzed separately in each group. Patients who meet all the inclusion and none of the exclusion criteria will be treated with 800 mg LDE225 daily until disease progression, occurrence of intolerable toxicity, start of another anticancer treatment or withdrawal of consent. |
| BG002 | Total | Total of all reporting groups |
| Units | Counts |
|---|---|
| Participants |
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| Title | Description | Population Description | Parameter Type | Dispersion Type | Unit of Measure | Calculate Percentage | Denominator Units Selected | Denominators | Classes | ||||||||||
|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|
| Age, Continuous | Mean | Standard Deviation | Years |
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| Sex: Female, Male | Count of Participants | Participants |
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| Race/Ethnicity, Customized | Number | 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 | |||||||||||||||||||||||||||||
|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|
| Primary | Objective Response Rate (ORR) Based on Central Review According to mRECIST (for Locally Advanced Basal Cell Carcinoma (laBCC)) and RECIST 1.1 (for Metastatic Basal Cell Carcinoma (mBCC)) Per Primary Efficacy Analysis Set (pEAS) | ORR is the percentage of patient's objective response (ORR) by 6 months after starting LDE225 treatment. A responder was defined as a subject with confirmed partial response (PR) or confirmed complete response (CR) 6 months after starting LDE225 treatment.Treatment with sonidegib was considered sufficiently efficacious if the observed ORR on any treatment arm at the end of the study was 30% or higher. PR: At least a 30% decrease in the sum of diameter of all target lesions, taking as reference the baseline sum of diameters. CR: Disappearance of all non-nodal target lesions. In addition, any pathological lymph nodes assigned as target lesions must have a reduction in short axis to < 10 mm. | The primary efficacy analysis set (pEAS) was a subset of the full analysis set (FAS) including patients with laBCC with tumors that were adequately assessed by MRI or photography or both, & including all patients with mBCC included in the FAS which comprised all patients who were assigned study treatment irrespective of receiving it. | Posted | Number | 95% Confidence Interval | Percentage of participants | 6 months |
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| Primary | Objective Response Rate (ORR) Based on Central Review According to mRECIST (for Locally Advanced Basal Cell Carcinoma (laBCC)) and RECIST 1.1 (Metastatic Basal Cell Carcinoma (mBCC)) Per Full Analysis Set (FAS) | ORR is the percentage of patient's objective response (ORR) by 6 months after starting LDE225 treatment. A responder was defined as a subject with confirmed partial response (PR) or confirmed complete response (CR) 6 months after starting LDE225 treatment.Treatment with sonidegib was to be considered sufficiently efficacious if the observed ORR on any treatment arm at the end of the study was 30% or higher. PR: At least a 30% decrease in the sum of diameter of all target lesions, taking as reference the baseline sum of diameters. CR: Disappearance of all non-nodal target lesions. In addition, any pathological lymph nodes assigned as target lesions must have a reduction in short axis to < 10 mm. | The full analysis set (FAS) comprised all patients who were assigned study treatment irrespective of receiving it (all randomized patients). | Posted | Number | 95% Confidence Interval | Percentage of participants | 6 months |
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| Secondary | Duration of Response (DoR) Per Central Review Using mRECIST for laBCC and RECIST 1.1 for mBCC (pEAS) | Duration of response is the time from the first observed confirmed response (CR or PR) to disease progression or death due to any reason. Duration of response was for participants with ORR. PR: At least a 30% decrease in the sum of diameter of all target lesions, taking as reference the baseline sum of diameters. CR: Disappearance of all non-nodal target lesions. In addition, any pathological lymph nodes assigned as target lesions must have a reduction in short axis to < 10 mm. Progressive disease (PD): At least a 20% increase in the sum of diameter of all measured target lesions, taking as reference the smallest sum of diameter of all target lesions recorded at or after baseline. In addition to the relative increase of 20%, the sum must also demonstrate an absolute increase of at least 5 mm2. | The primary efficacy analysis set (pEAS) was a subset of the full analysis set (FAS) including patients with laBCC with tumors that were adequately assessed by MRI or photography or both, & including all patients with mBCC included in the FAS which comprised all patients who were assigned study treatment irrespective of receiving it. | Posted | Median | 95% Confidence Interval | Months | 42 months |
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| Secondary | Duration of Response (DoR) Per Central Review Using mRECIST for laBCC and RECIST 1.1 for mBCC (FAS) | Duration of response is the time from the first observed confirmed response (CR or PR) to disease progression or death due to any reason. Median DoR for patients with laBCC was non-estimable for both treatment arms. Duration of response was for participants with ORR. PR: At least a 30% decrease in the sum of diameter of all target lesions, taking as reference the baseline sum of diameters. CR: Disappearance of all non-nodal target lesions. In addition, any pathological lymph nodes assigned as target lesions must have a reduction in short axis to < 10 mm. | The full analysis set (FAS) comprised all patients who were assigned study treatment irrespective of receiving it (all randomized patients). Patients were classified according to the treatment they were assigned in accordance with the intention-to-treat (ITT) principle. | Posted | Median | 95% Confidence Interval | Months | 42 months |
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| Secondary | Complete Response Rate (CRR) Per Central Review (pEAS) | Rate of complete response is the percentage of patients with best overall response of complete response (CR) after starting LDE225 treatment. The rate of CR was determined according to mRECIST for laBCC and RECIST 1.1 for mBCC. Patients with best overall response of 'Unknown" were treated as non responders. Complete Response (CR): Disappearance of all non-nodal target lesions. In addition, any pathological lymph nodes assigned as target lesions must have a reduction in short axis to < 10 mm. | The primary efficacy analysis set (pEAS) was a subset of the full analysis set (FAS) including patients with laBCC with tumors that were adequately assessed by MRI or photography or both, & including all patients with mBCC included in the FAS which comprised all patients who were assigned study treatment irrespective of receiving it. | Posted | Number | 95% Confidence Interval | Percentage of participants | 42 months |
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| Secondary | Complete Response Rate (CRR) Per Central Review (FAS) | Rate of complete response is the proportion of patients with best overall response of complete response (CR) after starting LDE225 treatment. The rate of CR will be determined according to mRECIST for laBCC and RECIST 1.1 for mBCC. Patients with best overall response of 'Unknown" will be treated as non responders | The full analysis set (FAS) comprised all patients who were assigned study treatment irrespective of receiving it (all randomized patients). Patients were classified according to the treatment they were assigned in accordance with the intention-to-treat (ITT) principle. | Posted | Number | 95% Confidence Interval | Percentage of participants | 6 months |
| ||||||||||||||||||||||||||||||||||||
| Secondary | Progression-free Survival (PFS) Per Central Review Using mRECIST for laBCC and RECIST 1.1 for mBCC (pEAS) | Progression-free survival (PFS) is the time from date of randomization/start of treatment to the date of event defined as the first documented progression or death due to any cause. If a patient has not had an event, progression-free survival is censored at the date of last adequate tumor assessment. | The primary efficacy analysis set (pEAS) was a subset of the full analysis set (FAS) including patients with laBCC with tumors that were adequately assessed by MRI or photography or both, & including all patients with mBCC included in the FAS which comprised all patients who were assigned study treatment irrespective of receiving it. | Posted | Median | 95% Confidence Interval | Months | 42 months |
| ||||||||||||||||||||||||||||||||||||
| Secondary | Progression-free Survival (PFS) Per Central Review Using mRECIST for laBCC and RECIST 1.1 for mBCC (FAS) | Progression-free survival (PFS) is the time from date of randomization/start of treatment to the date of event defined as the first documented progression or death due to any cause. If a patient has not had an event, PFS is censored at the date of last adequate tumor assessment. | The full analysis set (FAS) comprised all patients who were assigned study treatment irrespective of receiving it (all randomized patients). | Posted | Median | 95% Confidence Interval | Months | 42 months |
| ||||||||||||||||||||||||||||||||||||
| Secondary | Time to Tumor Response (TTR) Per Central Review Using mRECIST for laBCC and RECIST 1.1 for mBCC (pEAS) | Time to tumor response (TTR) is defined as the time from date of enrollment to the date of first documented tumor response (CR or PR). PR: At least a 30% decrease in the sum of diameter of all target lesions, taking as reference the baseline sum of diameters. CR: Disappearance of all non-nodal target lesions. In addition, any pathological lymph nodes assigned as target lesions must have a reduction in short axis to < 10 mm. | The primary efficacy analysis set (pEAS) was a subset of the full analysis set (FAS) including patients with laBCC with tumors that were adequately assessed by MRI or photography or both, & including all patients with mBCC included in the FAS which comprised all patients who were assigned study treatment irrespective of receiving it. | Posted | Median | 95% Confidence Interval | Months | 42 months |
| ||||||||||||||||||||||||||||||||||||
| Secondary | Time to Tumor Response (TTR) Per Central Review Using mRECIST for laBCC and RECIST 1.1 for mBCC (FAS) | Time to tumor response (TTR) is defined as the time from date of enrollment to the date of first documented tumor response (CR or PR). PR: At least a 30% decrease in the sum of diameter of all target lesions, taking as reference the baseline sum of diameters. CR: Disappearance of all non-nodal target lesions. In addition, any pathological lymph nodes assigned as target lesions must have a reduction in short axis to < 10 mm. | The full analysis set (FAS) comprised all patients who were assigned study treatment irrespective of receiving it (all randomized patients). | Posted | Median | 95% Confidence Interval | Months | 42 months |
| ||||||||||||||||||||||||||||||||||||
| Secondary | Objective Response Rate (ORR) Based on Site Investigator Review According to mRECIST (for Locally Advanced Basal Cell Carcinoma (laBCC)) and RECIST 1.1 (for Metastatic Basal Cell Carcinoma (mBCC)) Per Primary Efficacy Analysis Set (pEAS) | ORR is the percentage of patient's objective response (ORR) by 42 months after starting LDE225 treatment. A responder was defined as a subject with confirmed partial response (PR) or confirmed complete response (CR) 42 months after starting LDE225 treatment. Treatment with sonidegib was considered sufficiently efficacious if the observed ORR on any treatment arm at the end of the study was 30% or higher. PR: At least a 30% decrease in the sum of diameter of all target lesions, taking as reference the baseline sum of diameters. CR: Disappearance of all non-nodal target lesions. In addition, any pathological lymph nodes assigned as target lesions must have a reduction in short axis to < 10 mm. | The primary efficacy analysis set (pEAS) was a subset of the full analysis set (FAS) including patients with laBCC with tumors that were adequately assessed by MRI or photography or both, & including all patients with mBCC included in the FAS which comprised all patients who were assigned study treatment irrespective of receiving it. | Posted | Number | 95% Confidence Interval | Percentage of participants | 42 months |
| ||||||||||||||||||||||||||||||||||||
| Secondary | Objective Response Rate (ORR) Based on Site Investigator Review According to mRECIST (for Locally Advanced Basal Cell Carcinoma (laBCC)) and RECIST 1.1 (Metastatic Basal Cell Carcinoma (mBCC)) Per Full Analysis Set (FAS) | ORR is the percentage of patient's objective response (ORR) by 6 months after starting LDE225 treatment. A responder was defined as a subject with confirmed partial response (PR) or confirmed complete response (CR) 42 months after starting LDE225 treatment.Treatment with sonidegib was to be considered sufficiently efficacious if the observed ORR on any treatment arm at the end of the study was 30% or higher. PR: At least a 30% decrease in the sum of diameter of all target lesions, taking as reference the baseline sum of diameters. CR: Disappearance of all non-nodal target lesions. In addition, any pathological lymph nodes assigned as target lesions must have a reduction in short axis to < 10 mm. | The full analysis set (FAS) comprised all patients who were assigned study treatment irrespective of receiving it (all randomized patients). | Posted | Number | 95% Confidence Interval | Percentage of participants | 42 months |
| ||||||||||||||||||||||||||||||||||||
| Secondary | Duration of Response (DoR) Per Site Investigator Review Using mRECIST for laBCC and RECIST 1.1 for mBCC (pEAS) | Duration of response is the time from the first observed confirmed response (CR or PR) to disease progression or death due to any reason. Duration of response was for participants with ORR. PR: At least a 30% decrease in the sum of diameter of all target lesions, taking as reference the baseline sum of diameters. CR: Disappearance of all non-nodal target lesions. In addition, any pathological lymph nodes assigned as target lesions must have a reduction in short axis to < 10 mm. Progressive disease (PD): At least a 20% increase in the sum of diameter of all measured target lesions, taking as reference the smallest sum of diameter of all target lesions recorded at or after baseline. In addition to the relative increase of 20%, the sum must also demonstrate an absolute increase of at least 5 mm2. | The primary efficacy analysis set (pEAS) was a subset of the full analysis set (FAS) including patients with laBCC with tumors that were adequately assessed by MRI or photography or both, & including all patients with mBCC included in the FAS which comprised all patients who were assigned study treatment irrespective of receiving it. | Posted | Median | 95% Confidence Interval | Months | 42 months |
| ||||||||||||||||||||||||||||||||||||
| Secondary | Duration of Response (DoR) Per Site Investigator Review Using mRECIST for laBCC and RECIST 1.1 for mBCC (FAS) | Duration of response is the time from the first observed confirmed response (CR or PR) to disease progression or death due to any reason. Duration of response was for participants with ORR. PR: At least a 30% decrease in the sum of diameter of all target lesions, taking as reference the baseline sum of diameters. CR: Disappearance of all non-nodal target lesions. In addition, any pathological lymph nodes assigned as target lesions must have a reduction in short axis to < 10 mm. | The full analysis set (FAS) comprised all patients who were assigned study treatment irrespective of receiving it (all randomized patients). Patients were classified according to the treatment they were assigned in accordance with the intention-to-treat (ITT) principle. | Posted | Median | 95% Confidence Interval | Months | 42 months |
| ||||||||||||||||||||||||||||||||||||
| Secondary | Progression-free Survival (PFS) Per Site Investigator Review Using mRECIST for laBCC and RECIST 1.1 for mBCC (pEAS) | Progression-free survival (PFS) is the time from date of randomization/start of treatment to the date of event defined as the first documented progression or death due to any cause. If a patient has not had an event, progression-free survival is censored at the date of last adequate tumor assessment. | The primary efficacy analysis set (pEAS) was a subset of the full analysis set (FAS) including patients with laBCC with tumors that were adequately assessed by MRI or photography or both, & including all patients with mBCC included in the FAS which comprised all patients who were assigned study treatment irrespective of receiving it. | Posted | Median | 95% Confidence Interval | Months | 42 months |
| ||||||||||||||||||||||||||||||||||||
| Secondary | Time to Tumor Response (TTR) Per Site Investigator Review Using mRECIST for laBCC and RECIST 1.1 for mBCC (pEAS) | Time to tumor response (TTR) is defined as the time from date of enrollment to the date of first documented tumor response (CR or PR). PR: At least a 30% decrease in the sum of diameter of all target lesions, taking as reference the baseline sum of diameters. CR: Disappearance of all non-nodal target lesions. In addition, any pathological lymph nodes assigned as target lesions must have a reduction in short axis to < 10 mm. | The primary efficacy analysis set (pEAS) was a subset of the full analysis set (FAS) including patients with laBCC with tumors that were adequately assessed by MRI or photography or both, & including all patients with mBCC included in the FAS which comprised all patients who were assigned study treatment irrespective of receiving it. | Posted | Median | 95% Confidence Interval | Months | 42 months |
| ||||||||||||||||||||||||||||||||||||
| Secondary | Plasma Concentration of Sonidegib (LDE225) | Blood PK samples were collected either by direct venipuncture or an indwelling cannula inserted in a forearm vein for the determination of trough (Cmin) plasma concentrations of sonidegib and its main circulating metabolite, LGE899, from all patients who enrolled in the study. Blood was collected in Weeks 1, 3, 5, 9 (pre-dose), and subsequently pre-dose every 4 weeks up to Week 21, and every 12 weeks thereafter up to week 69. | The PK analysis set (PAS) consisted of all patients with at least one evaluable plasma concentration. | Posted | Geometric Mean | Geometric Coefficient of Variation | ng/mL | Weeks 1, 3, 5, 9, 13, 17, 21, 33, 45, 57, 69 |
| ||||||||||||||||||||||||||||||||||||
| Secondary | Overall Survival (OS) | OS is defined as the time from date of randomization to date of death due to any cause or the last date that a patient was known to be alive (censored observation) as of the data cut-off. | The full analysis set (FAS) comprised all patients who were assigned study treatment irrespective of receiving it (all randomized patients). Patients were classified according to the treatment they were assigned in accordance with the intention-to-treat (ITT) principle. | Posted | Median | 95% Confidence Interval | Months | 42 months |
|
Adverse events and serious adverse events were collected for the maximum actual duration of treatment exposure and follow up for a participant per the protocol for approximately 76.2 months.
All cause mortality (deaths) was collected for as long as participants could be contacted from First Patient First Visit (FPFV) until Last Patient Last Visit (LPLV) up to a maximum of approx. 7 years.
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 200 mg qd | Patients who met all the inclusion and none of the exclusion criteria were treated with 200 mg LDE225 daily until disease progression, occurrence of intolerable toxicity, start of another anticancer treatment or withdrawal of consent. | 1 | 79 | 16 | 79 | 76 | 79 |
| EG001 | LDE225 800 mg qd | Patients who met all the inclusion and none of the exclusion criteria were treated with 800 mg LDE225 daily until disease progression, occurrence of intolerable toxicity, start of another anticancer treatment or withdrawal of consent. | 7 | 150 | 58 | 150 | 145 | 150 |
| Term | Organ System | Source Vocabulary | Assessment Type | Notes | Statistical Information |
|---|---|---|---|---|---|
| Anaemia | Blood and lymphatic system disorders | MedDRA (19.1) | Systematic Assessment |
| |
| Angina pectoris | Cardiac disorders | MedDRA (19.1) | Systematic Assessment |
| |
| Atrial fibrillation | Cardiac disorders | MedDRA (19.1) | Systematic Assessment |
| |
| Bradycardia | Cardiac disorders | MedDRA (19.1) | Systematic Assessment |
| |
| Bundle branch block left | Cardiac disorders | MedDRA (19.1) | Systematic Assessment |
| |
| Cardiac arrest | Cardiac disorders | MedDRA (19.1) | Systematic Assessment |
| |
| Cardiac failure congestive | Cardiac disorders | MedDRA (19.1) | Systematic Assessment |
| |
| Cardiovascular insufficiency | Cardiac disorders | MedDRA (19.1) | Systematic Assessment |
| |
| Sinus tachycardia | Cardiac disorders | MedDRA (19.1) | Systematic Assessment |
| |
| Chronic gastritis | Gastrointestinal disorders | MedDRA (19.1) | Systematic Assessment |
| |
| Constipation | Gastrointestinal disorders | MedDRA (19.1) | Systematic Assessment |
| |
| Diarrhoea | Gastrointestinal disorders | MedDRA (19.1) | Systematic Assessment |
| |
| Duodenal stenosis | Gastrointestinal disorders | MedDRA (19.1) | Systematic Assessment |
| |
| Duodenal ulcer | Gastrointestinal disorders | MedDRA (19.1) | Systematic Assessment |
| |
| Gastric ulcer | Gastrointestinal disorders | MedDRA (19.1) | Systematic Assessment |
| |
| Gastritis | Gastrointestinal disorders | MedDRA (19.1) | Systematic Assessment |
| |
| Gastritis erosive | Gastrointestinal disorders | MedDRA (19.1) | Systematic Assessment |
| |
| Gastrointestinal pain | Gastrointestinal disorders | MedDRA (19.1) | Systematic Assessment |
| |
| Nausea | Gastrointestinal disorders | MedDRA (19.1) | Systematic Assessment |
| |
| Pancreatitis acute | Gastrointestinal disorders | MedDRA (19.1) | Systematic Assessment |
| |
| Peptic ulcer perforation | Gastrointestinal disorders | MedDRA (19.1) | Systematic Assessment |
| |
| Small intestinal obstruction | Gastrointestinal disorders | MedDRA (19.1) | Systematic Assessment |
| |
| Upper gastrointestinal haemorrhage | Gastrointestinal disorders | MedDRA (19.1) | Systematic Assessment |
| |
| Vomiting | Gastrointestinal disorders | MedDRA (19.1) | Systematic Assessment |
| |
| Cardiac death | General disorders | MedDRA (19.1) | Systematic Assessment |
| |
| Facial pain | General disorders | MedDRA (19.1) | Systematic Assessment |
| |
| General physical health deterioration | General disorders | MedDRA (19.1) | Systematic Assessment |
| |
| Non-cardiac chest pain | General disorders | MedDRA (19.1) | Systematic Assessment |
| |
| Pain | General disorders | MedDRA (19.1) | Systematic Assessment |
| |
| Cholelithiasis | Hepatobiliary disorders | MedDRA (19.1) | Systematic Assessment |
| |
| Hepatotoxicity | Hepatobiliary disorders | MedDRA (19.1) | Systematic Assessment |
| |
| Abscess | Infections and infestations | MedDRA (19.1) | Systematic Assessment |
| |
| Abscess limb | Infections and infestations | MedDRA (19.1) | Systematic Assessment |
| |
| Bronchitis | Infections and infestations | MedDRA (19.1) | Systematic Assessment |
| |
| Cellulitis | Infections and infestations | MedDRA (19.1) | Systematic Assessment |
| |
| Endocarditis | Infections and infestations | MedDRA (19.1) | Systematic Assessment |
| |
| Escherichia urinary tract infection | Infections and infestations | MedDRA (19.1) | Systematic Assessment |
| |
| Lower respiratory tract infection | Infections and infestations | MedDRA (19.1) | Systematic Assessment |
| |
| Mastoiditis | Infections and infestations | MedDRA (19.1) | Systematic Assessment |
| |
| Otitis media | Infections and infestations | MedDRA (19.1) | Systematic Assessment |
| |
| Parotitis | Infections and infestations | MedDRA (19.1) | Systematic Assessment |
| |
| Pneumonia | Infections and infestations | MedDRA (19.1) | Systematic Assessment |
| |
| Pseudomonas infection | Infections and infestations | MedDRA (19.1) | Systematic Assessment |
| |
| Sepsis | Infections and infestations | MedDRA (19.1) | Systematic Assessment |
| |
| Septic shock | Infections and infestations | MedDRA (19.1) | Systematic Assessment |
| |
| Superinfection | Infections and infestations | MedDRA (19.1) | Systematic Assessment |
| |
| Urinary tract infection | Infections and infestations | MedDRA (19.1) | Systematic Assessment |
| |
| Arthropod bite | Injury, poisoning and procedural complications | MedDRA (19.1) | Systematic Assessment |
| |
| Cervical vertebral fracture | Injury, poisoning and procedural complications | MedDRA (19.1) | Systematic Assessment |
| |
| Fall | Injury, poisoning and procedural complications | MedDRA (19.1) | Systematic Assessment |
| |
| Femoral neck fracture | Injury, poisoning and procedural complications | MedDRA (19.1) | Systematic Assessment |
| |
| Joint dislocation | Injury, poisoning and procedural complications | MedDRA (19.1) | Systematic Assessment |
| |
| Lumbar vertebral fracture | Injury, poisoning and procedural complications | MedDRA (19.1) | Systematic Assessment |
| |
| Spinal fracture | Injury, poisoning and procedural complications | MedDRA (19.1) | Systematic Assessment |
| |
| Upper limb fracture | Injury, poisoning and procedural complications | MedDRA (19.1) | Systematic Assessment |
| |
| Blood creatine phosphokinase MB increased | Investigations | MedDRA (19.1) | Systematic Assessment |
| |
| Blood creatine phosphokinase increased | Investigations | MedDRA (19.1) | Systematic Assessment |
| |
| Lipase increased | Investigations | MedDRA (19.1) | Systematic Assessment |
| |
| Myoglobin blood increased | Investigations | MedDRA (19.1) | Systematic Assessment |
| |
| Weight decreased | Investigations | MedDRA (19.1) | Systematic Assessment |
| |
| Decreased appetite | Metabolism and nutrition disorders | MedDRA (19.1) | Systematic Assessment |
| |
| Dehydration | Metabolism and nutrition disorders | MedDRA (19.1) | Systematic Assessment |
| |
| Failure to thrive | Metabolism and nutrition disorders | MedDRA (19.1) | Systematic Assessment |
| |
| Hypoglycaemia | Metabolism and nutrition disorders | MedDRA (19.1) | Systematic Assessment |
| |
| Muscle contracture | Musculoskeletal and connective tissue disorders | MedDRA (19.1) | Systematic Assessment |
| |
| Muscular weakness | Musculoskeletal and connective tissue disorders | MedDRA (19.1) | Systematic Assessment |
| |
| Myositis | Musculoskeletal and connective tissue disorders | MedDRA (19.1) | Systematic Assessment |
| |
| Osteonecrosis of jaw | Musculoskeletal and connective tissue disorders | MedDRA (19.1) | Systematic Assessment |
| |
| Rhabdomyolysis | Musculoskeletal and connective tissue disorders | MedDRA (19.1) | Systematic Assessment |
| |
| B-cell lymphoma | Neoplasms benign, malignant and unspecified (incl cysts and polyps) | MedDRA (19.1) | Systematic Assessment |
| |
| Basal cell carcinoma | Neoplasms benign, malignant and unspecified (incl cysts and polyps) | MedDRA (19.1) | Systematic Assessment |
| |
| Brain neoplasm | Neoplasms benign, malignant and unspecified (incl cysts and polyps) | MedDRA (19.1) | Systematic Assessment |
| |
| Invasive papillary breast carcinoma | Neoplasms benign, malignant and unspecified (incl cysts and polyps) | MedDRA (19.1) | Systematic Assessment |
| |
| Lung neoplasm | Neoplasms benign, malignant and unspecified (incl cysts and polyps) | MedDRA (19.1) | Systematic Assessment |
| |
| Malignant melanoma | Neoplasms benign, malignant and unspecified (incl cysts and polyps) | MedDRA (19.1) | Systematic Assessment |
| |
| Prostate cancer | Neoplasms benign, malignant and unspecified (incl cysts and polyps) | MedDRA (19.1) | Systematic Assessment |
| |
| Squamous cell carcinoma | Neoplasms benign, malignant and unspecified (incl cysts and polyps) | MedDRA (19.1) | Systematic Assessment |
| |
| Transitional cell carcinoma | Neoplasms benign, malignant and unspecified (incl cysts and polyps) | MedDRA (19.1) | Systematic Assessment |
| |
| Cerebrovascular accident | Nervous system disorders | MedDRA (19.1) | Systematic Assessment |
| |
| Dysgeusia | Nervous system disorders | MedDRA (19.1) | Systematic Assessment |
| |
| Haemorrhage intracranial | Nervous system disorders | MedDRA (19.1) | Systematic Assessment |
| |
| Paraesthesia | Nervous system disorders | MedDRA (19.1) | Systematic Assessment |
| |
| Somnolence | Nervous system disorders | MedDRA (19.1) | Systematic Assessment |
| |
| Syncope | Nervous system disorders | MedDRA (19.1) | Systematic Assessment |
| |
| Bipolar disorder | Psychiatric disorders | MedDRA (19.1) | Systematic Assessment |
| |
| Mental status changes | Psychiatric disorders | MedDRA (19.1) | Systematic Assessment |
| |
| Acute kidney injury | Renal and urinary disorders | MedDRA (19.1) | Systematic Assessment |
| |
| Bladder obstruction | Renal and urinary disorders | MedDRA (19.1) | Systematic Assessment |
| |
| Haematuria | Renal and urinary disorders | MedDRA (19.1) | Systematic Assessment |
| |
| Nephropathy toxic | Renal and urinary disorders | MedDRA (19.1) | Systematic Assessment |
| |
| Uterine prolapse | Reproductive system and breast disorders | MedDRA (19.1) | Systematic Assessment |
| |
| Acute respiratory distress syndrome | Respiratory, thoracic and mediastinal disorders | MedDRA (19.1) | Systematic Assessment |
| |
| Bronchitis chronic | Respiratory, thoracic and mediastinal disorders | MedDRA (19.1) | Systematic Assessment |
| |
| Dyspnoea | Respiratory, thoracic and mediastinal disorders | MedDRA (19.1) | Systematic Assessment |
| |
| Emphysema | Respiratory, thoracic and mediastinal disorders | MedDRA (19.1) | Systematic Assessment |
| |
| Pleural effusion | Respiratory, thoracic and mediastinal disorders | MedDRA (19.1) | Systematic Assessment |
| |
| Respiratory arrest | Respiratory, thoracic and mediastinal disorders | MedDRA (19.1) | Systematic Assessment |
| |
| Rhinorrhoea | Respiratory, thoracic and mediastinal disorders | MedDRA (19.1) | Systematic Assessment |
| |
| Sputum discoloured | Respiratory, thoracic and mediastinal disorders | MedDRA (19.1) | Systematic Assessment |
| |
| Skin lesion | Skin and subcutaneous tissue disorders | MedDRA (19.1) | Systematic Assessment |
| |
| Arterial haemorrhage | Vascular disorders | MedDRA (19.1) | Systematic Assessment |
| |
| Deep vein thrombosis | Vascular disorders | MedDRA (19.1) | Systematic Assessment |
| |
| Hypotension | Vascular disorders | MedDRA (19.1) | Systematic Assessment |
| |
| Orthostatic hypotension | Vascular disorders | MedDRA (19.1) | Systematic Assessment |
|
| Term | Organ System | Source Vocabulary | Assessment Type | Notes | Statistical Information |
|---|---|---|---|---|---|
| Anaemia | Blood and lymphatic system disorders | MedDRA (19.1) | Systematic Assessment |
| |
| Vertigo | Ear and labyrinth disorders | MedDRA (19.1) | Systematic Assessment |
| |
| Abdominal pain | Gastrointestinal disorders | MedDRA (19.1) | Systematic Assessment |
| |
| Abdominal pain upper | Gastrointestinal disorders | MedDRA (19.1) | Systematic Assessment |
| |
| Constipation | Gastrointestinal disorders | MedDRA (19.1) | Systematic Assessment |
| |
| Diarrhoea | Gastrointestinal disorders | MedDRA (19.1) | Systematic Assessment |
| |
| Dry mouth | Gastrointestinal disorders | MedDRA (19.1) | Systematic Assessment |
| |
| Nausea | Gastrointestinal disorders | MedDRA (19.1) | Systematic Assessment |
| |
| Vomiting | Gastrointestinal disorders | MedDRA (19.1) | Systematic Assessment |
| |
| Asthenia | General disorders | MedDRA (19.1) | Systematic Assessment |
| |
| Fatigue | General disorders | MedDRA (19.1) | Systematic Assessment |
| |
| Pyrexia | General disorders | MedDRA (19.1) | Systematic Assessment |
| |
| Influenza | Infections and infestations | MedDRA (19.1) | Systematic Assessment |
| |
| Nasopharyngitis | Infections and infestations | MedDRA (19.1) | Systematic Assessment |
| |
| Pneumonia | Infections and infestations | MedDRA (19.1) | Systematic Assessment |
| |
| Upper respiratory tract infection | Infections and infestations | MedDRA (19.1) | Systematic Assessment |
| |
| Urinary tract infection | Infections and infestations | MedDRA (19.1) | Systematic Assessment |
| |
| Fall | Injury, poisoning and procedural complications | MedDRA (19.1) | Systematic Assessment |
| |
| Blood creatine phosphokinase increased | Investigations | MedDRA (19.1) | Systematic Assessment |
| |
| Lipase increased | Investigations | MedDRA (19.1) | Systematic Assessment |
| |
| Weight decreased | Investigations | MedDRA (19.1) | Systematic Assessment |
| |
| Decreased appetite | Metabolism and nutrition disorders | MedDRA (19.1) | Systematic Assessment |
| |
| Arthralgia | Musculoskeletal and connective tissue disorders | MedDRA (19.1) | Systematic Assessment |
| |
| Back pain | Musculoskeletal and connective tissue disorders | MedDRA (19.1) | Systematic Assessment |
| |
| Muscle spasms | Musculoskeletal and connective tissue disorders | MedDRA (19.1) | Systematic Assessment |
| |
| Muscular weakness | Musculoskeletal and connective tissue disorders | MedDRA (19.1) | Systematic Assessment |
| |
| Musculoskeletal pain | Musculoskeletal and connective tissue disorders | MedDRA (19.1) | Systematic Assessment |
| |
| Myalgia | Musculoskeletal and connective tissue disorders | MedDRA (19.1) | Systematic Assessment |
| |
| Pain in extremity | Musculoskeletal and connective tissue disorders | MedDRA (19.1) | Systematic Assessment |
| |
| Squamous cell carcinoma | Neoplasms benign, malignant and unspecified (incl cysts and polyps) | MedDRA (19.1) | Systematic Assessment |
| |
| Ageusia | Nervous system disorders | MedDRA (19.1) | Systematic Assessment |
| |
| Dizziness | Nervous system disorders | MedDRA (19.1) | Systematic Assessment |
| |
| Dysgeusia | Nervous system disorders | MedDRA (19.1) | Systematic Assessment |
| |
| Headache | Nervous system disorders | MedDRA (19.1) | Systematic Assessment |
| |
| Hypogeusia | Nervous system disorders | MedDRA (19.1) | Systematic Assessment |
| |
| Paraesthesia | Nervous system disorders | MedDRA (19.1) | Systematic Assessment |
| |
| Depression | Psychiatric disorders | MedDRA (19.1) | Systematic Assessment |
| |
| Cough | Respiratory, thoracic and mediastinal disorders | MedDRA (19.1) | Systematic Assessment |
| |
| Oropharyngeal pain | Respiratory, thoracic and mediastinal disorders | MedDRA (19.1) | Systematic Assessment |
| |
| Alopecia | Skin and subcutaneous tissue disorders | MedDRA (19.1) | Systematic Assessment |
| |
| Pruritus | Skin and subcutaneous tissue disorders | MedDRA (19.1) | Systematic Assessment |
| |
| Hypertension | Vascular disorders | MedDRA (19.1) | Systematic Assessment |
|
230 patients evaluated in the Full Analysis Set: 79 randomized to LDE225 (Sonidegib) 200mg group & 151 randomized to LDE225 800mg group. 1 patient randomized to the 800mg group did not receive study treatment & thus was not counted in the Safety Set.
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 | 862-778-8300 | Novartis.email@novartis.com |
| ID | Term |
|---|---|
| D002280 | Carcinoma, Basal Cell |
| D012878 | Skin Neoplasms |
| ID | Term |
|---|---|
| D002277 | Carcinoma |
| D009375 | Neoplasms, Glandular and Epithelial |
| D009370 | Neoplasms by Histologic Type |
| D009369 | Neoplasms |
| D018295 | Neoplasms, Basal Cell |
| D009371 | Neoplasms by Site |
| D012871 | Skin Diseases |
| D017437 | Skin and Connective Tissue Diseases |
Not provided
Not provided
| ID | Term |
|---|---|
| C561435 | sonidegib |
Not provided
Not provided
Not provided
| Male |
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| Caucasian |
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| Other |
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The efficacy and safety of LDE225 800mg laBCC cohort were analyzed separately in each group. Patients who met all the inclusion and none of the exclusion criteria were treated with 800 mg LDE225 daily until disease progression, occurrence of intolerable toxicity, start of another anticancer treatment or withdrawal of consent. |
| OG002 | LDE225 200mg mBCC | The efficacy and safety of LDE225 200mg mBCC cohort were analyzed separately in each group. Patients who met all the inclusion and none of the exclusion criteria were treated with 200 mg LDE225 daily until disease progression, occurrence of intolerable toxicity, start of another anticancer treatment or withdrawal of consent. |
| OG003 | LDE225 800mg mBCC | The efficacy and safety of LDE225 800mg mBCC cohort were analyzed separately in each group. Patients who met all the inclusion and none of the exclusion criteria were treated with 800 mg LDE225 daily until disease progression, occurrence of intolerable toxicity, start of another anticancer treatment or withdrawal of consent. |
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| OG001 | LDE225 800mg laBCC | The efficacy and safety of LDE225 800mg laBCC cohort were analyzed separately in each group. Patients who met all the inclusion and none of the exclusion criteria were treated with 800 mg LDE225 daily until disease progression, occurrence of intolerable toxicity, start of another anticancer treatment or withdrawal of consent. |
| OG002 | LDE225 200mg mBCC | The efficacy and safety of LDE225 200mg mBCC cohort were analyzed separately in each group. Patients who met all the inclusion and none of the exclusion criteria were treated with 200 mg LDE225 daily until disease progression, occurrence of intolerable toxicity, start of another anticancer treatment or withdrawal of consent. |
| OG003 | LDE225 800mg mBCC | The efficacy and safety of LDE225 800mg mBCC cohort were analyzed separately in each group. Patients who met all the inclusion and none of the exclusion criteria were treated with 800 mg LDE225 daily until disease progression, occurrence of intolerable toxicity, start of another anticancer treatment or withdrawal of consent. |
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| OG002 | LDE225 200mg mBCC | The efficacy and safety of LDE225 200mg mBCC cohort were analyzed separately in each group. Patients who met all the inclusion and none of the exclusion criteria were treated with 200 mg LDE225 daily until disease progression, occurrence of intolerable toxicity, start of another anticancer treatment or withdrawal of consent. |
| OG003 | LDE225 800mg mBCC | The efficacy and safety of LDE225 800mg mBCC cohort were analyzed separately in each group. Patients who met all the inclusion and none of the exclusion criteria were treated with 800 mg LDE225 daily until disease progression, occurrence of intolerable toxicity, start of another anticancer treatment or withdrawal of consent. |
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| OG002 | LDE225 200mg mBCC | The efficacy and safety of LDE225 200mg mBCC cohort were analyzed separately in each group. Patients who met all the inclusion and none of the exclusion criteria were treated with 200 mg LDE225 daily until disease progression, occurrence of intolerable toxicity, start of another anticancer treatment or withdrawal of consent. |
| OG003 | LDE225 800mg mBCC | The efficacy and safety of LDE225 800mg mBCC cohort were analyzed separately in each group. Patients who met all the inclusion and none of the exclusion criteria were treated with 800 mg LDE225 daily until disease progression, occurrence of intolerable toxicity, start of another anticancer treatment or withdrawal of consent. |
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|
| OG002 | LDE225 200mg mBCC | The efficacy and safety of LDE225 200mg mBCC cohort were analyzed separately in each group. Patients who met all the inclusion and none of the exclusion criteria were treated with 200 mg LDE225 daily until disease progression, occurrence of intolerable toxicity, start of another anticancer treatment or withdrawal of consent. |
| OG003 | LDE225 800mg mBCC | The efficacy and safety of LDE225 800mg mBCC cohort were analyzed separately in each group. Patients who met all the inclusion and none of the exclusion criteria were treated with 800 mg LDE225 daily until disease progression, occurrence of intolerable toxicity, start of another anticancer treatment or withdrawal of consent. |
|
|
| OG002 | LDE225 200mg mBCC | The efficacy and safety of LDE225 200mg mBCC cohort were analyzed separately in each group. Patients who met all the inclusion and none of the exclusion criteria were treated with 200 mg LDE225 daily until disease progression, occurrence of intolerable toxicity, start of another anticancer treatment or withdrawal of consent. |
| OG003 | LDE225 800mg mBCC | The efficacy and safety of LDE225 800mg mBCC cohort were analyzed separately in each group. Patients who met all the inclusion and none of the exclusion criteria were treated with 800 mg LDE225 daily until disease progression, occurrence of intolerable toxicity, start of another anticancer treatment or withdrawal of consent. |
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| LDE225 200mg mBCC |
The efficacy and safety of LDE225 200mg mBCC cohort were analyzed separately in each group. Patients who met all the inclusion and none of the exclusion criteria were treated with 200 mg LDE225 daily until disease progression, occurrence of intolerable toxicity, start of another anticancer treatment or withdrawal of consent. |
| OG003 | LDE225 800mg mBCC | The efficacy and safety of LDE225 800mg mBCC cohort were analyzed separately in each group. Patients who met all the inclusion and none of the exclusion criteria were treated with 800 mg LDE225 daily until disease progression, occurrence of intolerable toxicity, start of another anticancer treatment or withdrawal of consent. |
|
|
| OG002 | LDE225 200mg mBCC | The efficacy and safety of LDE225 200mg mBCC cohort were analyzed separately in each group. Patients who met all the inclusion and none of the exclusion criteria were treated with 200 mg LDE225 daily until disease progression, occurrence of intolerable toxicity, start of another anticancer treatment or withdrawal of consent. |
| OG003 | LDE225 800mg mBCC | The efficacy and safety of LDE225 800mg mBCC cohort were analyzed separately in each group. Patients who met all the inclusion and none of the exclusion criteria were treated with 800 mg LDE225 daily until disease progression, occurrence of intolerable toxicity, start of another anticancer treatment or withdrawal of consent. |
|
|
| OG002 | LDE225 200mg mBCC | The efficacy and safety of LDE225 200mg mBCC cohort were analyzed separately in each group. Patients who met all the inclusion and none of the exclusion criteria were treated with 200 mg LDE225 daily until disease progression, occurrence of intolerable toxicity, start of another anticancer treatment or withdrawal of consent. |
| OG003 | LDE225 800mg mBCC | The efficacy and safety of LDE225 800mg mBCC cohort were analyzed separately in each group. Patients who met all the inclusion and none of the exclusion criteria were treated with 800 mg LDE225 daily until disease progression, occurrence of intolerable toxicity, start of another anticancer treatment or withdrawal of consent. |
|
|
| OG001 | LDE225 800mg laBCC | The efficacy and safety of LDE225 800mg laBCC cohort were analyzed separately in each group. Patients who met all the inclusion and none of the exclusion criteria were treated with 800 mg LDE225 daily until disease progression, occurrence of intolerable toxicity, start of another anticancer treatment or withdrawal of consent. |
| OG002 | LDE225 200mg mBCC | The efficacy and safety of LDE225 200mg mBCC cohort were analyzed separately in each group. Patients who met all the inclusion and none of the exclusion criteria were treated with 200 mg LDE225 daily until disease progression, occurrence of intolerable toxicity, start of another anticancer treatment or withdrawal of consent. |
| OG003 | LDE225 800mg mBCC | The efficacy and safety of LDE225 800mg mBCC cohort were analyzed separately in each group. Patients who met all the inclusion and none of the exclusion criteria were treated with 800 mg LDE225 daily until disease progression, occurrence of intolerable toxicity, start of another anticancer treatment or withdrawal of consent. |
|
|
The efficacy and safety of LDE225 800mg laBCC cohort were analyzed separately in each group. Patients who met all the inclusion and none of the exclusion criteria were treated with 800 mg LDE225 daily until disease progression, occurrence of intolerable toxicity, start of another anticancer treatment or withdrawal of consent. |
| OG002 | LDE225 200mg mBCC | The efficacy and safety of LDE225 200mg mBCC cohort were analyzed separately in each group. Patients who met all the inclusion and none of the exclusion criteria were treated with 200 mg LDE225 daily until disease progression, occurrence of intolerable toxicity, start of another anticancer treatment or withdrawal of consent. |
| OG003 | LDE225 800mg mBCC | The efficacy and safety of LDE225 800mg mBCC cohort were analyzed separately in each group. Patients who met all the inclusion and none of the exclusion criteria were treated with 800 mg LDE225 daily until disease progression, occurrence of intolerable toxicity, start of another anticancer treatment or withdrawal of consent. |
|
|
| OG001 | LDE225 800mg laBCC | The efficacy and safety of LDE225 800mg laBCC cohort were analyzed separately in each group. Patients who met all the inclusion and none of the exclusion criteria were treated with 800 mg LDE225 daily until disease progression, occurrence of intolerable toxicity, start of another anticancer treatment or withdrawal of consent. |
| OG002 | LDE225 200mg mBCC | The efficacy and safety of LDE225 200mg mBCC cohort were analyzed separately in each group. Patients who met all the inclusion and none of the exclusion criteria were treated with 200 mg LDE225 daily until disease progression, occurrence of intolerable toxicity, start of another anticancer treatment or withdrawal of consent. |
| OG003 | LDE225 800mg mBCC | The efficacy and safety of LDE225 800mg mBCC cohort were analyzed separately in each group. Patients who met all the inclusion and none of the exclusion criteria were treated with 800 mg LDE225 daily until disease progression, occurrence of intolerable toxicity, start of another anticancer treatment or withdrawal of consent. |
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|
| OG002 | LDE225 200mg mBCC | The efficacy and safety of LDE225 200mg mBCC cohort were analyzed separately in each group. Patients who met all the inclusion and none of the exclusion criteria were treated with 200 mg LDE225 daily until disease progression, occurrence of intolerable toxicity, start of another anticancer treatment or withdrawal of consent. |
| OG003 | LDE225 800mg mBCC | The efficacy and safety of LDE225 800mg mBCC cohort were analyzed separately in each group. Patients who met all the inclusion and none of the exclusion criteria were treated with 800 mg LDE225 daily until disease progression, occurrence of intolerable toxicity, start of another anticancer treatment or withdrawal of consent. |
|
|
| OG002 | LDE225 200mg mBCC | The efficacy and safety of LDE225 200mg mBCC cohort were analyzed separately in each group. Patients who met all the inclusion and none of the exclusion criteria were treated with 200 mg LDE225 daily until disease progression, occurrence of intolerable toxicity, start of another anticancer treatment or withdrawal of consent. |
| OG003 | LDE225 800mg mBCC | The efficacy and safety of LDE225 800mg mBCC cohort were analyzed separately in each group. Patients who met all the inclusion and none of the exclusion criteria were treated with 800 mg LDE225 daily until disease progression, occurrence of intolerable toxicity, start of another anticancer treatment or withdrawal of consent. |
|
|
| OG002 | LDE225 200mg mBCC | The efficacy and safety of LDE225 200mg mBCC cohort were analyzed separately in each group. Patients who met all the inclusion and none of the exclusion criteria were treated with 200 mg LDE225 daily until disease progression, occurrence of intolerable toxicity, start of another anticancer treatment or withdrawal of consent. |
| OG003 | LDE225 800mg mBCC | The efficacy and safety of LDE225 800mg mBCC cohort were analyzed separately in each group. Patients who met all the inclusion and none of the exclusion criteria were treated with 800 mg LDE225 daily until disease progression, occurrence of intolerable toxicity, start of another anticancer treatment or withdrawal of consent. |
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The efficacy and safety of LDE225 200mg mBCC cohort were analyzed separately in each group. Patients who met all the inclusion and none of the exclusion criteria were treated with 200 mg LDE225 daily until disease progression, occurrence of intolerable toxicity, start of another anticancer treatment or withdrawal of consent. |
| OG003 | LDE225 800mg mBCC | The efficacy and safety of LDE225 800mg mBCC cohort were analyzed separately in each group. Patients who met all the inclusion and none of the exclusion criteria were treated with 800 mg LDE225 daily until disease progression, occurrence of intolerable toxicity, start of another anticancer treatment or withdrawal of consent. |
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