Trial · NCT01327053
A Phase II Study of Efficacy and Safety in Patients With Locally Advanced or Metastatic Basal Cell Carcinoma
NCT01327053, Phase 2, Completed, Novartis Pharmaceuticals; results first posted 2015-11-23
3 sourcestrials-2026-11-12-0.5.0
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ConductScience Library. A Phase II Study of Efficacy and Safety in Patients With Locally Advanced or Metastatic Basal Cell Carcinoma. Trials, record NCT01327053. Release trials-2026-11-12-0.5.0, 12 Nov 2026. https://conductscience.org/library/trials/NCT01327053
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Trial
As registered on ClinicalTrials.gov, in the graph's capture of the registry.
- Title
- A Phase II Study of Efficacy and Safety in Patients With Locally Advanced or Metastatic Basal Cell Carcinoma
- NCT number
- NCT01327053
- Status
- Completed
- Phases
- Phase 2
- Enrollment
- 230 participants, actual
- Lead sponsor
- Novartis Pharmaceuticals
- Conditions
- Basal Cell Carcinoma
- Interventions
- LDE225
- First posted
- 2011-04-01
- Last updated
- 2019-08-28
- Other registries' IDs
- eudract:2010-022629-14
- Results first posted
- 2015-11-23
- Limitations and caveats
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.
Source ClinicalTrials.gov
Registered outcomes18
Each outcome the trial registered, primary first, in ClinicalTrials.gov's words.
| List | Outcome | Time frame | Description |
|---|---|---|---|
| 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) | 6 months | 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. |
| 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) | 6 months | 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. |
| Secondary | Duration of Response (DoR) Per Central Review Using mRECIST for laBCC and RECIST 1.1 for mBCC (pEAS) | 42 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. 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. |
| Secondary | Duration of Response (DoR) Per Central Review Using mRECIST for laBCC and RECIST 1.1 for mBCC (FAS) | 42 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. 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. |
| Secondary | Complete Response Rate (CRR) Per Central Review (pEAS) | 42 months | 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. |
| Secondary | Complete Response Rate (CRR) Per Central Review (FAS) | 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 |
| Secondary | Progression-free Survival (PFS) Per Central Review Using mRECIST for laBCC and RECIST 1.1 for mBCC (pEAS) | 42 months | 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. |
| Secondary | Progression-free Survival (PFS) Per Central Review Using mRECIST for laBCC and RECIST 1.1 for mBCC (FAS) | 42 months | 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. |
| Secondary | Time to Tumor Response (TTR) Per Central Review Using mRECIST for laBCC and RECIST 1.1 for mBCC (pEAS) | 42 months | 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. |
| Secondary | Time to Tumor Response (TTR) Per Central Review Using mRECIST for laBCC and RECIST 1.1 for mBCC (FAS) | 42 months | 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. |
1 to 10 of 18Source ClinicalTrials.gov
See all 18 registered outcomesOutcomes18
Each outcome the trial posted results for, in ClinicalTrials.gov's words.
| Type | Outcome | Time frame | Population | Units | Measured as |
|---|---|---|---|---|---|
| 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) | 6 months | 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. | Percentage of participants | NUMBER |
| 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) | 6 months | The full analysis set (FAS) comprised all patients who were assigned study treatment irrespective of receiving it (all randomized patients). | Percentage of participants | NUMBER |
| SECONDARY | Duration of Response (DoR) Per Central Review Using mRECIST for laBCC and RECIST 1.1 for mBCC (pEAS) | 42 months | 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. | Months | MEDIAN |
| SECONDARY | Duration of Response (DoR) Per Central Review Using mRECIST for laBCC and RECIST 1.1 for mBCC (FAS) | 42 months | 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. | Months | MEDIAN |
| SECONDARY | Complete Response Rate (CRR) Per Central Review (pEAS) | 42 months | 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. | Percentage of participants | NUMBER |
| SECONDARY | Complete Response Rate (CRR) Per Central Review (FAS) | 6 months | 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. | Percentage of participants | NUMBER |
| SECONDARY | Progression-free Survival (PFS) Per Central Review Using mRECIST for laBCC and RECIST 1.1 for mBCC (pEAS) | 42 months | 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. | Months | MEDIAN |
| SECONDARY | Progression-free Survival (PFS) Per Central Review Using mRECIST for laBCC and RECIST 1.1 for mBCC (FAS) | 42 months | The full analysis set (FAS) comprised all patients who were assigned study treatment irrespective of receiving it (all randomized patients). | Months | MEDIAN |
| SECONDARY | Time to Tumor Response (TTR) Per Central Review Using mRECIST for laBCC and RECIST 1.1 for mBCC (pEAS) | 42 months | 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. | Months | MEDIAN |
| SECONDARY | Time to Tumor Response (TTR) Per Central Review Using mRECIST for laBCC and RECIST 1.1 for mBCC (FAS) | 42 months | The full analysis set (FAS) comprised all patients who were assigned study treatment irrespective of receiving it (all randomized patients). | Months | MEDIAN |
1 to 10 of 18Source ClinicalTrials.gov
See all 18 outcomesResults by group98
Each measurement the trial posted, group by group, in the words and numbers it posted.
| Outcome | Group | Category | Value | Spread | Units |
|---|---|---|---|---|---|
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) | LDE225 200mg laBCC | 42.9 | 95% Confidence Interval 27.7 to 59.0 | Percentage of participants | |
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) | LDE225 800mg laBCC | 37.6 | 95% Confidence Interval 27.8 to 48.3 | Percentage of participants | |
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) | LDE225 200mg mBCC | 15.4 | 95% Confidence Interval 1.9 to 45.4 | Percentage of participants | |
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) | LDE225 800mg mBCC | 17.4 | 95% Confidence Interval 5.0 to 38.8 | Percentage of participants | |
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) | LDE225 200mg laBCC | 47.0 | 95% Confidence Interval 34.6 to 59.7 | Percentage of participants | |
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) | LDE225 800mg laBCC | 35.2 | 95% Confidence Interval 26.9 to 44.1 | Percentage of participants | |
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) | LDE225 200mg mBCC | 15.4 | 95% Confidence Interval 1.9 to 45.4 | Percentage of participants | |
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) | LDE225 800mg mBCC | 17.4 | 95% Confidence Interval 5.0 to 38.8 | Percentage of participants | |
| Duration of Response (DoR) Per Central Review Using mRECIST for laBCC and RECIST 1.1 for mBCC (pEAS) | LDE225 200mg laBCC | 12.9 | 95% Confidence Interval NA to NA | Months | |
| Duration of Response (DoR) Per Central Review Using mRECIST for laBCC and RECIST 1.1 for mBCC (pEAS) | LDE225 800mg laBCC | 23.7 | 95% Confidence Interval 10.8 to 29.6 | Months |
1 to 10 of 98Source ClinicalTrials.gov
See all 98 results by groupParticipant flow14
How many started, completed or left each group, period by period.
| Period | Milestone | Group | Participants | Units counted |
|---|---|---|---|---|
| Overall Study | STARTED | LDE225 (Sonidegib) 200 mg | 79 | |
| Overall Study | STARTED | LDE225 (Sonidegib) 800 mg | 151 | |
| Overall Study | Untreated | LDE225 (Sonidegib) 200 mg | 0 | |
| Overall Study | Untreated | LDE225 (Sonidegib) 800 mg | 1 | |
| Overall Study | Patients Cont. to Next Phase of Trial | LDE225 (Sonidegib) 200 mg | 52 | |
| Overall Study | Patients Cont. to Next Phase of Trial | LDE225 (Sonidegib) 800 mg | 89 | |
| Overall Study | Survival Follow-up | LDE225 (Sonidegib) 200 mg | 33 | |
| Overall Study | Survival Follow-up | LDE225 (Sonidegib) 800 mg | 49 | |
| Overall Study | Post-treatment Follow-up | LDE225 (Sonidegib) 200 mg | 19 | |
| Overall Study | Post-treatment Follow-up | LDE225 (Sonidegib) 800 mg | 40 |
1 to 10 of 14Source ClinicalTrials.gov
See all 14 participant flowAdverse events292
Each adverse event the trial posted, group by group: the participants affected out of those at risk.
| Type | Event | Organ system | Group | Affected | At risk | Events |
|---|---|---|---|---|---|---|
| other | Anaemia | Blood and lymphatic system disorders | LDE225 200 mg qd | 4 | 79 | |
| other | Anaemia | Blood and lymphatic system disorders | LDE225 800 mg qd | 13 | 150 | |
| other | Vertigo | Ear and labyrinth disorders | LDE225 200 mg qd | 0 | 79 | |
| other | Vertigo | Ear and labyrinth disorders | LDE225 800 mg qd | 11 | 150 | |
| other | Abdominal pain | Gastrointestinal disorders | LDE225 200 mg qd | 8 | 79 | |
| other | Abdominal pain | Gastrointestinal disorders | LDE225 800 mg qd | 9 | 150 | |
| other | Abdominal pain upper | Gastrointestinal disorders | LDE225 200 mg qd | 7 | 79 | |
| other | Abdominal pain upper | Gastrointestinal disorders | LDE225 800 mg qd | 12 | 150 | |
| other | Constipation | Gastrointestinal disorders | LDE225 200 mg qd | 6 | 79 | |
| other | Constipation | Gastrointestinal disorders | LDE225 800 mg qd | 24 | 150 |
1 to 10 of 292Source ClinicalTrials.gov
See all 292 adverse eventsAdverse event totals6
The totals the trial posted for each group.
| Total | Group | Affected | At risk |
|---|---|---|---|
| Total, all-cause mortality | LDE225 200 mg qd | 1 | 79 |
| Total, all-cause mortality | LDE225 800 mg qd | 7 | 150 |
| Total, other adverse events | LDE225 200 mg qd | 76 | 79 |
| Total, other adverse events | LDE225 800 mg qd | 145 | 150 |
| Total, serious adverse events | LDE225 200 mg qd | 16 | 79 |
| Total, serious adverse events | LDE225 800 mg qd | 58 | 150 |
Source ClinicalTrials.gov
Papers4
The papers the trial lists on ClinicalTrials.gov.
Grants0
The grants NIH RePORTER links to the trial, newest funded first: NIH's, and some of AHRQ's, CDC's, FDA's and VA's. Each shows its title, principal investigators, organization and funder in its newest year.
None in this release.
Trial comparisons2
Comparisons of the trial's two groups, calculated by ConductScience from the counts it posted.
| Comparison | Source |
|---|---|
| LDE225 200 mg qd vs LDE225 800 mg qd: deaths from any cause | Trial comparisons |
| LDE225 200 mg qd vs LDE225 800 mg qd: serious adverse events | Trial comparisons |
Tests0
The ConductCognition tests this trial's registered outcomes name, as ScienceGraph matched them in trial-tests-2026-11-12-0.2.1.
None in this release.
Data dates
The day each source was copied for this release.
Sources 3
- ClinicalTrials.gov's posted results, through CTTI's AACTFreely available. Acknowledgement requested
- ClinicalTrials.govNo license stated
- NIH RePORTER's clinical studies and project filesPublic domain
Each source's terms
- ClinicalTrials.gov's posted results, through CTTI's AACTClinicalTrials.gov data via Aggregate Analysis of ClinicalTrials.gov (AACT), CTTI. CTTI makes its materials freely available and requests acknowledgement. https://aact.ctti-clinicaltrials.org/documentation/299
- ClinicalTrials.govnone stated
- NIH RePORTER's clinical studies and project filesPublic domain (NIH RePORTER)
ClinicalTrials.gov's data carry an international copyright outside the United States: https://clinicaltrials.gov/about-site/terms-conditions.
For software
Read this trial from software.
The record and each of its lists are JSON, twenty rows a page. No key.
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