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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 : Active, not recruiting
First Posted : April 1, 2011
Results First Posted : November 23, 2015
Last Update Posted : October 4, 2017
Sponsor:
Information provided by (Responsible Party):
Novartis ( Novartis Pharmaceuticals )

March 30, 2011
April 1, 2011
August 24, 2015
November 23, 2015
October 4, 2017
June 29, 2011
June 28, 2013   (Final data collection date for primary outcome measure)
  • 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.
  • 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.
assess the efficacy of daily oral treatment of LDE225 in patients with locally advanced or metastatic basal cell carcinoma, as measured by Objective Response Rate (ORR). [ Time Frame: When all patients enrolled have completed at least 24 weeks of treatment or discontinued ]
Complete list of historical versions of study NCT01327053 on ClinicalTrials.gov Archive Site
  • 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.

  • 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.

  • 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
  • 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
  • assess the time to the first documented tumor response (TTR) as complete or partial response [ Time Frame: When all patients enrolled have completed at least 24 weeks of treatment or discontinued. ]
  • assess duration of overall response (DoR), defined as the time from the first occurrence of complete or partial response until the date of the first documented progression or death. [ Time Frame: When all patients enrolled have completed at least 24 weeks of treatment or discontinued. ]
  • assess the effect of LDE225 treatment on progression-free survival (PFS), defined as the time from the date of enrollment to the first documented progression or death due to any cause. [ Time Frame: When all patients enrolled have completed at least 24 weeks of treatment or discontinued. ]
  • assess overall survival (OS) associated with LDE225 treatment. [ Time Frame: When all patients enrolled have completed at least 24 weeks of treatment or discontinued. ]
  • characterize the safety of LDE225 treatment. characterize the safety of LDE225 treatment. [ Time Frame: : When all patients enrolled have completed at least 24 weeks of treatment or discontinued ]
Not Provided
Not Provided
 
A Phase II Study of Efficacy and Safety in Patients With Locally Advanced or Metastatic Basal Cell Carcinoma
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
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.
Not Provided
Interventional
Phase 2
Allocation: Randomized
Intervention Model: Parallel Assignment
Masking: Quadruple (Participant, Care Provider, Investigator, Outcomes Assessor)
Primary Purpose: Treatment
Basal Cell Carcinoma
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 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.
    Intervention: Drug: LDE225
  • 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.
    Intervention: Drug: LDE225

*   Includes publications given by the data provider as well as publications identified by ClinicalTrials.gov Identifier (NCT Number) in Medline.
 
Active, not recruiting
229
120
December 22, 2017
June 28, 2013   (Final data collection date for primary outcome measure)

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

Sexes Eligible for Study: All
18 Years and older   (Adult, Senior)
No
Contact information is only displayed when the study is recruiting subjects
Australia,   Belgium,   Canada,   France,   Germany,   Greece,   Hungary,   Italy,   Spain,   Switzerland,   United Kingdom,   United States
Netherlands
 
NCT01327053
CLDE225A2201
2010-022629-14 ( EudraCT Number )
Yes
Not Provided
Not Provided
Novartis ( Novartis Pharmaceuticals )
Novartis Pharmaceuticals
Not Provided
Study Director: Novartis Pharmaceuticals Novartis Pharmaceuticals
Novartis
October 2017

ICMJE     Data element required by the International Committee of Medical Journal Editors and the World Health Organization ICTRP