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A Phase II Study of Efficacy and Safety in Patients With Locally Advanced or Metastatic Basal Cell Carcinoma (BOLT)

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ClinicalTrials.gov Identifier: NCT01327053
Recruitment Status : Completed
First Posted : April 1, 2011
Results First Posted : November 23, 2015
Last Update Posted : August 7, 2018
Sponsor:
Information provided by (Responsible Party):
Novartis ( Novartis Pharmaceuticals )

Brief Summary:
This study will assess the efficacy and safety of oral treatment with two dose levels of LDE225 in patients with locally advanced or metastatic BCC.

Condition or disease Intervention/treatment Phase
Basal Cell Carcinoma Drug: LDE225 Phase 2

Study Type : Interventional  (Clinical Trial)
Actual Enrollment : 229 participants
Allocation: Randomized
Intervention Model: Parallel Assignment
Masking: Quadruple (Participant, Care Provider, Investigator, Outcomes Assessor)
Primary Purpose: Treatment
Official 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
Actual Study Start Date : June 29, 2011
Actual Primary Completion Date : June 28, 2013
Actual Study Completion Date : December 22, 2017

Resource links provided by the National Library of Medicine

Drug Information available for: Sonidegib

Arm Intervention/treatment
Experimental: LDE225 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.
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.
Other Name: Sonidegib

Experimental: LDE225 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.
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.
Other Name: Sonidegib




Primary Outcome Measures :
  1. 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). [ Time Frame: 6 months ]
    ORR is the proportion of patient's objective response (ORR) by 6 months after starting LDE225 treatment. A responder will be 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.

  2. 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 Primary Efficacy Analysis Set (pEAS) [ Time Frame: 6 months ]
    ORR is the proportion of patient's objective response (ORR) by 6 months after starting LDE225 treatment. A responder will be 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.


Secondary Outcome Measures :
  1. Duration of Response (DoR) Per Central Review Using mRECIST for laBCC and RECIST 1.1 for mBCC -Per pEAS [ Time Frame: 6 months ]

    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 as limited numbers of progressive disease (PD) or deaths were observed as of the 28-Jun-2013 data cut-off date. Median DoR was non-estimable for patients with mBCC receiving treatment with sonidegib 200 mg.

    Duration of response was for participants with ORR.


  2. Duration of Response (DoR) Per Central Review Using mRECIST for laBCC and RECIST 1.1 for mBCC Per FAS [ Time Frame: 6 months ]

    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. Median DoR was non-estimable for patients with mBCC receiving treatment with sonidegib 200 mg.

    Duration of response was for participants with ORR.


  3. Complete Response Rate (CRR) Per Central Review - Per pEAS [ Time Frame: 6 months ]
    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

  4. Complete Response Rate (CRR) Per Central Review - Per FAS [ Time Frame: 6 months ]
    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



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Ages Eligible for Study:   18 Years and older   (Adult, Older Adult)
Sexes Eligible for Study:   All
Accepts Healthy Volunteers:   No
Criteria

Inclusion Criteria:

  • Patients with locally advanced BCC and metastatic BCC
  • Patients with adequate bone marrow, liver, and renal function

Exclusion Criteria:

  • Patients who have 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 are on concurrent therapy with other anti-neoplastic agents.
  • Patients who have 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 are unwilling or unable to comply with the protocol.

Other protocol-defined inclusion/exclusion criteria may apply


Information from the National Library of Medicine

To learn more about this study, you or your doctor may contact the study research staff using the contact information provided by the sponsor.

Please refer to this study by its ClinicalTrials.gov identifier (NCT number): NCT01327053


  Hide Study Locations
Locations
United States, Arkansas
Highlands Oncology Group Dept of Highlands Oncology Grp
Fayetteville, Arkansas, United States, 72703
United States, California
University of California at Los Angeles UCLA 3
Los Angeles, California, United States, 90095
Stanford University Medical Center Stanford Univ 2
Stanford, California, United States, 94304
United States, Colorado
University of Colorado UC
Aurora, Colorado, United States, 80045
United States, District of Columbia
Washington Hospital Center Wash Hospital
Washington, District of Columbia, United States, 20010
United States, Florida
H. Lee Moffitt Cancer Center & Research Institute Cutaneous Onc Dept
Tampa, Florida, United States, 33612
United States, Illinois
NorthwesternUniv.Med.School/Robert H. Lurie Comp.Cancer Ctr Study coordinator
Chicago, Illinois, United States, 60611
United States, Massachusetts
Dana Farber Cancer Institute DFCI - MA
Boston, Massachusetts, United States, 02215
United States, Michigan
Henry Ford Hospital Henry Ford
Detroit, Michigan, United States, 48202-2689
United States, Missouri
Washington University School Of Medicine-Siteman Cancer Ctr Siteman
Saint Louis, Missouri, United States, 63110
United States, Nevada
Comprehensive Cancer Centers of Nevada CCC of Nevada- Southwest (2)
Las Vegas, Nevada, United States, 89109
United States, New Jersey
Hackensack University Medical Center Hackensack (SC)
Hackensack, New Jersey, United States, 07601
United States, New York
New York University Medical Center SC-2
New York, New York, United States, 10016
United States, Pennsylvania
Penn State University / Milton S. Hershey Medical Center Hershey Medical
Hershey, Pennsylvania, United States, 17033-085
University of Pittsburgh Medical Center UPMC
Pittsburgh, Pennsylvania, United States, 15213
United States, Texas
Baylor Health Care System/Sammons Cancer Center Baylor Texas Oncology
Dallas, Texas, United States, 75246
Texas Oncology Tex Onc 3
Dallas, Texas, United States, 75246
Texas Oncology Texas Onc - Amarillo
Dallas, Texas, United States, 75246
University of Texas/MD Anderson Cancer Center MD Anderson
Houston, Texas, United States, 77030-4009
Texas Oncology Cancer Care & Research Center
Waco, Texas, United States, 76712
Texoma Cancer Center Texoma Cancer Center
Wichita Falls, Texas, United States, 76310
United States, Utah
University of Utah / Huntsman Cancer Institute Huntsman/Univ UT
Salt Lake City, Utah, United States, 84103
Australia, New South Wales
Novartis Investigative Site
St. Leonards, New South Wales, Australia, 2065
Australia, Victoria
Novartis Investigative Site
Geelong, Victoria, Australia, 3220
Belgium
Novartis Investigative Site
Bruxelles, Belgium, 1200
Novartis Investigative Site
Wilrijk, Belgium, 2610
Canada, Ontario
Novartis Investigative Site
Waterloo, Ontario, Canada, N2J 1C4
Canada, Quebec
Novartis Investigative Site
Sainte-Foy, Quebec, Canada, G1V 4T3
France
Novartis Investigative Site
Lyon Cedex, France, 69373
Novartis Investigative Site
Marseille Cedex 05, France, 13885
Novartis Investigative Site
Pierre-Benite Cedex, France, 69495
Novartis Investigative Site
Toulouse Cedex 9, France, 31059
Novartis Investigative Site
Villejuif Cedex, France, 94805
Germany
Novartis Investigative Site
Berlin, Germany, 10098
Novartis Investigative Site
Essen, Germany, 45147
Novartis Investigative Site
Freiburg, Germany, 79106
Novartis Investigative Site
Gera, Germany, 07548
Novartis Investigative Site
Hannover, Germany, 30625
Novartis Investigative Site
Kiel, Germany, 24105
Novartis Investigative Site
Mainz, Germany, 55131
Novartis Investigative Site
Muenchen, Germany, 80336
Novartis Investigative Site
Muenster, Germany, 48157
Novartis Investigative Site
Stade, Germany, 21682
Greece
Novartis Investigative Site
Athens, Greece, 161 21
Hungary
Novartis Investigative Site
Budapest, Hungary, H-1085
Novartis Investigative Site
Debrecen, Hungary, 4032
Novartis Investigative Site
Szeged, Hungary, H-6725
Italy
Novartis Investigative Site
Torino, TO, Italy, 10126
Novartis Investigative Site
Napoli, Italy, 80131
Spain
Novartis Investigative Site
Barcelona, Catalunya, Spain, 08035
Novartis Investigative Site
Madrid, Spain, 28034
Novartis Investigative Site
Madrid, Spain, 28046
Switzerland
Novartis Investigative Site
Bern, Switzerland, 3010
Novartis Investigative Site
Genève, Switzerland, 1211
Novartis Investigative Site
Zürich, Switzerland, 8091
United Kingdom
Novartis Investigative Site
High Heaton, Newcastle Upon Tyne, United Kingdom, NE7 7DN
Novartis Investigative Site
Yeovil, Somerset, United Kingdom, BA21 4AT
Novartis Investigative Site
Cardiff, United Kingdom, CF14 4XW
Novartis Investigative Site
Glasgow, United Kingdom, G3 8SJ
Novartis Investigative Site
Leicester, United Kingdom, LE1 5WW
Novartis Investigative Site
London, United Kingdom, EC1A 7BE
Novartis Investigative Site
Manchester, United Kingdom, M13 9WL
Sponsors and Collaborators
Novartis Pharmaceuticals
Investigators
Study Director: Novartis Pharmaceuticals Novartis Pharmaceuticals

Publications automatically indexed to this study by ClinicalTrials.gov Identifier (NCT Number):
Responsible Party: Novartis Pharmaceuticals
ClinicalTrials.gov Identifier: NCT01327053     History of Changes
Other Study ID Numbers: CLDE225A2201
2010-022629-14 ( EudraCT Number )
First Posted: April 1, 2011    Key Record Dates
Results First Posted: November 23, 2015
Last Update Posted: August 7, 2018
Last Verified: July 2018

Keywords provided by Novartis ( Novartis Pharmaceuticals ):
locally advanced basal cell carcinoma,
metastatic basal cell carcinoma

Additional relevant MeSH terms:
Carcinoma
Carcinoma, Basal Cell
Neoplasms, Glandular and Epithelial
Neoplasms by Histologic Type
Neoplasms
Neoplasms, Basal Cell