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Clinical Trial Details — Status: Completed

Administrative data

NCT number NCT01327053
Other study ID # CLDE225A2201
Secondary ID 2010-022629-14
Status Completed
Phase Phase 2
First received
Last updated
Start date June 29, 2011
Est. completion date June 29, 2018

Study information

Verified date August 2019
Source Novartis
Contact n/a
Is FDA regulated No
Health authority
Study type Interventional

Clinical Trial Summary

This study assessed the efficacy and safety of oral treatment with two dose levels of LDE225 in patients with locally advanced or metastatic BCC.


Recruitment information / eligibility

Status Completed
Enrollment 230
Est. completion date June 29, 2018
Est. primary completion date June 28, 2013
Accepts healthy volunteers No
Gender All
Age group 18 Years and older
Eligibility Inclusion Criteria:

- Patients with locally advanced BCC and metastatic BCC

- Patients with adequate bone marrow, liver, and renal function

Exclusion Criteria:

- Patients who had had major surgery within 4 weeks of initiation of study medication

- Patients unable to take oral drugs or with lack of physical integrity of the upper gastrointestinal tract, or known malabsorption syndromes.

- Patients with concurrent medical conditions that may interfere or potentially affect the interpretation of the study.

- Patients with neuromuscular disorders or are on concurrent treatment with drugs that may cause muscle damage.

- Patients who were on concurrent therapy with other anti-neoplastic agents.

- Patients who had taken part in an experimental drug within 4 weeks of initiation of study medication.

- Pregnant or nursing (lactating) women

- Women of child bearing potential unwilling to use 2 forms of highly effective contraception throughout the study and for 3 months after the last treatment

- Fertile males not willing to use condoms throughout the study and for 3 months after the last treatment.

- Patients who were unwilling or unable to comply with the protocol.

Study Design


Related Conditions & MeSH terms


Intervention

Drug:
LDE225
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.

Locations

Country Name City State
Australia Novartis Investigative Site Geelong Victoria
Australia Novartis Investigative Site St Leonards New South Wales
Belgium Novartis Investigative Site Bruxelles
Belgium Novartis Investigative Site Wilrijk
Canada Novartis Investigative Site Sainte-Foy Quebec
Canada Novartis Investigative Site Waterloo Ontario
France Novartis Investigative Site Lyon Cedex
France Novartis Investigative Site Marseille Cedex 05
France Novartis Investigative Site Pierre Benite Cedex
France Novartis Investigative Site Toulouse Cedex 9
France Novartis Investigative Site Villejuif Cedex
Germany Novartis Investigative Site Berlin
Germany Novartis Investigative Site Essen
Germany Novartis Investigative Site Freiburg
Germany Novartis Investigative Site Gera
Germany Novartis Investigative Site Hannover
Germany Novartis Investigative Site Kiel
Germany Novartis Investigative Site Mainz
Germany Novartis Investigative Site Muenchen
Germany Novartis Investigative Site Muenster
Germany Novartis Investigative Site Stade
Greece Novartis Investigative Site Athens
Hungary Novartis Investigative Site Budapest
Hungary Novartis Investigative Site Debrecen
Hungary Novartis Investigative Site Szeged
Italy Novartis Investigative Site Napoli
Italy Novartis Investigative Site Torino TO
Spain Novartis Investigative Site Barcelona Catalunya
Spain Novartis Investigative Site Madrid
Spain Novartis Investigative Site Madrid
Switzerland Novartis Investigative Site Bern
Switzerland Novartis Investigative Site Geneve
Switzerland Novartis Investigative Site Zürich
United Kingdom Novartis Investigative Site Cardiff
United Kingdom Novartis Investigative Site Glasgow
United Kingdom Novartis Investigative Site High Heaton Newcastle Upon Tyne
United Kingdom Novartis Investigative Site Leicester
United Kingdom Novartis Investigative Site London
United Kingdom Novartis Investigative Site Manchester
United Kingdom Novartis Investigative Site Yeovil Somerset
United States University of Colorado School of Medicine UC Aurora Colorado
United States Dana Farber Cancer Institute DFCI - MA Boston Massachusetts
United States NorthwesternUniv.Med.School/Robert H. Lurie Comp.Cancer Ctr Study coordinator Chicago Illinois
United States Baylor Health Care System/Sammons Cancer Center Baylor Texas Oncology Dallas Texas
United States Texas Oncology Tex Onc 3 Dallas Texas
United States Texas Oncology Texas Onc - Amarillo Dallas Texas
United States Henry Ford Hospital Henry Ford Detroit Michigan
United States Highlands Oncology Group Fayetteville Arkansas
United States Hackensack University Medical Center Hackensack (SC) Hackensack New Jersey
United States Penn State University / Milton S. Hershey Medical Center Hershey Medical Hershey Pennsylvania
United States University of Texas MD Anderson Cancer Center MD Anderson Houston Texas
United States Comprehensive Cancer Centers of Nevada CCC of Nevada- Southwest (2) Las Vegas Nevada
United States University of California at Los Angeles UCLA 3 Los Angeles California
United States New York University Medical Center SC-2 New York New York
United States University of Pittsburgh Medical Center UPMC Pittsburgh Pennsylvania
United States Washington University School Of Medicine-Siteman Cancer Ctr Siteman Saint Louis Missouri
United States University of Utah / Huntsman Cancer Institute Huntsman/Univ UT Salt Lake City Utah
United States Stanford University Medical Center Stanford Univ 2 Stanford California
United States H Lee Moffitt Cancer Center and Research Institute Cutaneous Onc Dept Tampa Florida
United States Texas Oncology Cancer Care & Research Center Waco Texas
United States Washington Hospital Center Wash Hospital Washington District of Columbia
United States Texoma Cancer Center Texoma Cancer Center Wichita Falls Texas

Sponsors (1)

Lead Sponsor Collaborator
Novartis Pharmaceuticals

Countries where clinical trial is conducted

United States,  Australia,  Belgium,  Canada,  France,  Germany,  Greece,  Hungary,  Italy,  Spain,  Switzerland,  United Kingdom, 

Outcome

Type Measure Description Time frame Safety issue
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. 6 months
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. 6 months
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.
42 months
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.
42 months
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. 42 months
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 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. 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. 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. 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. 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. 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. 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.
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. 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. 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. 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. 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. 42 months
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