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Trial details imported from ClinicalTrials.gov

For full trial details, please see the original record at https://clinicaltrials.gov/study/NCT01327053




Registration number
NCT01327053
Ethics application status
Date submitted
30/03/2011
Date registered
1/04/2011

Titles & IDs
Public title
A Phase II Study of Efficacy and Safety in Patients With Locally Advanced or Metastatic Basal Cell Carcinoma
Scientific title
Phase II, Randomized Double-blind Study of Efficacy and Safety of Two Dose Levels of LDE225 in Patients With Locally Advanced or Metastatic Basal Cell Carcinoma
Secondary ID [1] 0 0
2010-022629-14
Secondary ID [2] 0 0
CLDE225A2201
Universal Trial Number (UTN)
Trial acronym
BOLT
Linked study record

Health condition
Health condition(s) or problem(s) studied:
Basal Cell Carcinoma 0 0
Condition category
Condition code
Cancer 0 0 0 0
Non melanoma skin cancer
Cancer 0 0 0 0
Kidney

Intervention/exposure
Study type
Interventional
Description of intervention(s) / exposure
Treatment: Drugs - LDE225

Experimental: LDE225 200 mg - 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.

Experimental: LDE225 800 mg - 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.


Treatment: Drugs: 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.

Intervention code [1] 0 0
Treatment: Drugs
Comparator / control treatment
Control group

Outcomes
Primary outcome [1] 0 0
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)
Timepoint [1] 0 0
6 months
Primary outcome [2] 0 0
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)
Timepoint [2] 0 0
6 months
Secondary outcome [1] 0 0
Duration of Response (DoR) Per Central Review Using mRECIST for laBCC and RECIST 1.1 for mBCC (pEAS)
Timepoint [1] 0 0
42 months
Secondary outcome [2] 0 0
Duration of Response (DoR) Per Central Review Using mRECIST for laBCC and RECIST 1.1 for mBCC (FAS)
Timepoint [2] 0 0
42 months
Secondary outcome [3] 0 0
Complete Response Rate (CRR) Per Central Review (pEAS)
Timepoint [3] 0 0
42 months
Secondary outcome [4] 0 0
Complete Response Rate (CRR) Per Central Review (FAS)
Timepoint [4] 0 0
6 months
Secondary outcome [5] 0 0
Progression-free Survival (PFS) Per Central Review Using mRECIST for laBCC and RECIST 1.1 for mBCC (pEAS)
Timepoint [5] 0 0
42 months
Secondary outcome [6] 0 0
Progression-free Survival (PFS) Per Central Review Using mRECIST for laBCC and RECIST 1.1 for mBCC (FAS)
Timepoint [6] 0 0
42 months
Secondary outcome [7] 0 0
Time to Tumor Response (TTR) Per Central Review Using mRECIST for laBCC and RECIST 1.1 for mBCC (pEAS)
Timepoint [7] 0 0
42 months
Secondary outcome [8] 0 0
Time to Tumor Response (TTR) Per Central Review Using mRECIST for laBCC and RECIST 1.1 for mBCC (FAS)
Timepoint [8] 0 0
42 months
Secondary outcome [9] 0 0
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)
Timepoint [9] 0 0
42 months
Secondary outcome [10] 0 0
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)
Timepoint [10] 0 0
42 months
Secondary outcome [11] 0 0
Duration of Response (DoR) Per Site Investigator Review Using mRECIST for laBCC and RECIST 1.1 for mBCC (pEAS)
Timepoint [11] 0 0
42 months
Secondary outcome [12] 0 0
Duration of Response (DoR) Per Site Investigator Review Using mRECIST for laBCC and RECIST 1.1 for mBCC (FAS)
Timepoint [12] 0 0
42 months
Secondary outcome [13] 0 0
Progression-free Survival (PFS) Per Site Investigator Review Using mRECIST for laBCC and RECIST 1.1 for mBCC (pEAS)
Timepoint [13] 0 0
42 months
Secondary outcome [14] 0 0
Time to Tumor Response (TTR) Per Site Investigator Review Using mRECIST for laBCC and RECIST 1.1 for mBCC (pEAS)
Timepoint [14] 0 0
42 months
Secondary outcome [15] 0 0
Plasma Concentration of Sonidegib (LDE225)
Timepoint [15] 0 0
Weeks 1, 3, 5, 9, 13, 17, 21, 33, 45, 57, 69
Secondary outcome [16] 0 0
Overall Survival (OS)
Timepoint [16] 0 0
42 months

Eligibility
Key inclusion criteria
* Patients with locally advanced BCC and metastatic BCC
* Patients with adequate bone marrow, liver, and renal function
Minimum age
18 Years
Maximum age
No limit
Sex
Both males and females
Can healthy volunteers participate?
No
Key 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
Purpose of the study
Treatment
Allocation to intervention
Randomised controlled trial
Procedure for enrolling a subject and allocating the treatment (allocation concealment procedures)
Methods used to generate the sequence in which subjects will be randomised (sequence generation)
Masking / blinding
Blinded (masking used)
Who is / are masked / blinded?
The people receiving the treatment/s
The people administering the treatment/s
The people assessing the outcomes
The people analysing the results/data
Intervention assignment
Parallel
Other design features
Phase
Phase 2
Type of endpoint/s
Statistical methods / analysis

Recruitment
Recruitment status
Completed
Data analysis
Reason for early stopping/withdrawal
Other reasons
Date of first participant enrolment
Anticipated
Actual
Date of last participant enrolment
Anticipated
Actual
Date of last data collection
Anticipated
Actual
Sample size
Target
Accrual to date
Final
Recruitment in Australia
Recruitment state(s)
NSW,VIC
Recruitment hospital [1] 0 0
Novartis Investigative Site - St Leonards
Recruitment hospital [2] 0 0
Novartis Investigative Site - Geelong
Recruitment postcode(s) [1] 0 0
2065 - St Leonards
Recruitment postcode(s) [2] 0 0
3220 - Geelong
Recruitment outside Australia
Country [1] 0 0
United States of America
State/province [1] 0 0
Arkansas
Country [2] 0 0
United States of America
State/province [2] 0 0
California
Country [3] 0 0
United States of America
State/province [3] 0 0
Colorado
Country [4] 0 0
United States of America
State/province [4] 0 0
District of Columbia
Country [5] 0 0
United States of America
State/province [5] 0 0
Florida
Country [6] 0 0
United States of America
State/province [6] 0 0
Illinois
Country [7] 0 0
United States of America
State/province [7] 0 0
Massachusetts
Country [8] 0 0
United States of America
State/province [8] 0 0
Michigan
Country [9] 0 0
United States of America
State/province [9] 0 0
Missouri
Country [10] 0 0
United States of America
State/province [10] 0 0
Nevada
Country [11] 0 0
United States of America
State/province [11] 0 0
New Jersey
Country [12] 0 0
United States of America
State/province [12] 0 0
New York
Country [13] 0 0
United States of America
State/province [13] 0 0
Pennsylvania
Country [14] 0 0
United States of America
State/province [14] 0 0
Texas
Country [15] 0 0
United States of America
State/province [15] 0 0
Utah
Country [16] 0 0
Belgium
State/province [16] 0 0
Bruxelles
Country [17] 0 0
Belgium
State/province [17] 0 0
Wilrijk
Country [18] 0 0
Canada
State/province [18] 0 0
Ontario
Country [19] 0 0
Canada
State/province [19] 0 0
Quebec
Country [20] 0 0
France
State/province [20] 0 0
Lyon Cedex
Country [21] 0 0
France
State/province [21] 0 0
Marseille Cedex 05
Country [22] 0 0
France
State/province [22] 0 0
Pierre Benite Cedex
Country [23] 0 0
France
State/province [23] 0 0
Toulouse Cedex 9
Country [24] 0 0
France
State/province [24] 0 0
Villejuif Cedex
Country [25] 0 0
Germany
State/province [25] 0 0
Berlin
Country [26] 0 0
Germany
State/province [26] 0 0
Essen
Country [27] 0 0
Germany
State/province [27] 0 0
Freiburg
Country [28] 0 0
Germany
State/province [28] 0 0
Gera
Country [29] 0 0
Germany
State/province [29] 0 0
Hannover
Country [30] 0 0
Germany
State/province [30] 0 0
Kiel
Country [31] 0 0
Germany
State/province [31] 0 0
Mainz
Country [32] 0 0
Germany
State/province [32] 0 0
Muenchen
Country [33] 0 0
Germany
State/province [33] 0 0
Muenster
Country [34] 0 0
Germany
State/province [34] 0 0
Stade
Country [35] 0 0
Greece
State/province [35] 0 0
Athens
Country [36] 0 0
Hungary
State/province [36] 0 0
Budapest
Country [37] 0 0
Hungary
State/province [37] 0 0
Debrecen
Country [38] 0 0
Hungary
State/province [38] 0 0
Szeged
Country [39] 0 0
Italy
State/province [39] 0 0
TO
Country [40] 0 0
Italy
State/province [40] 0 0
Napoli
Country [41] 0 0
Spain
State/province [41] 0 0
Catalunya
Country [42] 0 0
Spain
State/province [42] 0 0
Madrid
Country [43] 0 0
Switzerland
State/province [43] 0 0
Bern
Country [44] 0 0
Switzerland
State/province [44] 0 0
Geneve
Country [45] 0 0
Switzerland
State/province [45] 0 0
Zürich
Country [46] 0 0
United Kingdom
State/province [46] 0 0
Newcastle Upon Tyne
Country [47] 0 0
United Kingdom
State/province [47] 0 0
Somerset
Country [48] 0 0
United Kingdom
State/province [48] 0 0
Cardiff
Country [49] 0 0
United Kingdom
State/province [49] 0 0
Glasgow
Country [50] 0 0
United Kingdom
State/province [50] 0 0
Leicester
Country [51] 0 0
United Kingdom
State/province [51] 0 0
London
Country [52] 0 0
United Kingdom
State/province [52] 0 0
Manchester

Funding & Sponsors
Primary sponsor type
Commercial sector/industry
Name
Novartis Pharmaceuticals
Address
Country

Ethics approval
Ethics application status

Summary
Brief summary
Trial website
Trial related presentations / publications
Public notes

Contacts
Principal investigator
Name 0 0
Novartis Pharmaceuticals
Address 0 0
Novartis Pharmaceuticals
Country 0 0
Phone 0 0
Fax 0 0
Email 0 0
Contact person for public queries
Name 0 0
Address 0 0
Country 0 0
Phone 0 0
Fax 0 0
Email 0 0
Contact person for scientific queries

Data sharing statement
Will individual participant data (IPD) for this trial be available (including data dictionaries)?
Yes
What data in particular will be shared?
When will data be available (start and end dates)?
Available to whom?
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.
Available for what types of analyses?
How or where can data be obtained?
IPD available at link: http://www.clinicalstudydatarequest.com


What supporting documents are/will be available?

No Supporting Document Provided



Results publications and other study-related documents

No documents have been uploaded by study researchers.