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

Draft scope assessment: unclear. Attached because the study's own record describes metastatic disease. The study was not selected for this indication by its sponsor, and its eligibility has not been reconciled against this page in full. Found because this study's registry identifier is printed in an FDA approval notice, an FDA product label or an NCI summary held by this site. The citing document and the exact position of the identifier inside it are recorded. Being cited is not a statement that the study supports this treatment.

Read the full study record and all posted results →

Linked outcomes

Measures are not ranked. Open each original source section for treatment groups, denominators, units and results.

primary · POSTED

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.

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.

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primary · POSTED

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.

The full analysis set (FAS) comprised all patients who were assigned study treatment irrespective of receiving it (all randomized patients).

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secondary · POSTED

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.

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secondary · POSTED

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.

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.

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secondary · POSTED

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.

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secondary · POSTED

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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Reported safety

Serious events are not the same as severe events. Compare the original safety groups and their at-risk populations.

Open the study safety table →

Quality of life and patient-reported measures

No linked quality-of-life measure is recorded for this setting and study.

Full draft applicability and source-link record

trial

release id: clinical-indications-draft-1
disease state id: ds-basal-cell-carcinoma-libtayo-995f9df8
nct id: NCT01327053
trial label: A Phase II Study of Efficacy and Safety in Patients With Locally Advanced or Metastatic Basal Cell Carcinoma
applicability: unclear
applicability note: Attached because the study's own record describes metastatic disease. The study was not selected for this indication by its sponsor, and its eligibility has not been reconciled against this page in full.
curation status: draft
reviewed by: Source null
reviewed at: Source null
created at: 2026-09-10T00:00:00Z
provenance class: regulator-cited
discovery note: Found because this study's registry identifier is printed in an FDA approval notice, an FDA product label or an NCI summary held by this site. The citing document and the exact position of the identifier inside it are recorded. Being cited is not a statement that the study supports this treatment.
outcome Count: 6
harms Count: 2
qol Count: 0

outcomes

release id
clinical-indications-draft-1
disease state id
ds-basal-cell-carcinoma-libtayo-995f9df8
nct id
NCT01327053
outcome key
om-0000
source section id
NCT01327053-om-0000
source outcome index
0
endpoint
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)
endpoint definition
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.
time origin
6 months
analysis role
primary
reporting status
POSTED
data cutoff
Source null
population note
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.
curation status
draft
reviewed by
Source null
reviewed at
Source null
created at
2026-09-10T00:00:00Z
release id
clinical-indications-draft-1
disease state id
ds-basal-cell-carcinoma-libtayo-995f9df8
nct id
NCT01327053
outcome key
om-0001
source section id
NCT01327053-om-0001
source outcome index
1
endpoint
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)
endpoint definition
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.
time origin
6 months
analysis role
primary
reporting status
POSTED
data cutoff
Source null
population note
The full analysis set (FAS) comprised all patients who were assigned study treatment irrespective of receiving it (all randomized patients).
curation status
draft
reviewed by
Source null
reviewed at
Source null
created at
2026-09-10T00:00:00Z
release id
clinical-indications-draft-1
disease state id
ds-basal-cell-carcinoma-libtayo-995f9df8
nct id
NCT01327053
outcome key
om-0002
source section id
NCT01327053-om-0002
source outcome index
2
endpoint
Duration of Response (DoR) Per Central Review Using mRECIST for laBCC and RECIST 1.1 for mBCC (pEAS)
endpoint definition
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.
time origin
42 months
analysis role
secondary
reporting status
POSTED
data cutoff
Source null
population note
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.
curation status
draft
reviewed by
Source null
reviewed at
Source null
created at
2026-09-10T00:00:00Z
release id
clinical-indications-draft-1
disease state id
ds-basal-cell-carcinoma-libtayo-995f9df8
nct id
NCT01327053
outcome key
om-0003
source section id
NCT01327053-om-0003
source outcome index
3
endpoint
Duration of Response (DoR) Per Central Review Using mRECIST for laBCC and RECIST 1.1 for mBCC (FAS)
endpoint definition
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.
time origin
42 months
analysis role
secondary
reporting status
POSTED
data cutoff
Source null
population note
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.
curation status
draft
reviewed by
Source null
reviewed at
Source null
created at
2026-09-10T00:00:00Z
release id
clinical-indications-draft-1
disease state id
ds-basal-cell-carcinoma-libtayo-995f9df8
nct id
NCT01327053
outcome key
om-0004
source section id
NCT01327053-om-0004
source outcome index
4
endpoint
Complete Response Rate (CRR) Per Central Review (pEAS)
endpoint definition
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.
time origin
42 months
analysis role
secondary
reporting status
POSTED
data cutoff
Source null
population note
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.
curation status
draft
reviewed by
Source null
reviewed at
Source null
created at
2026-09-10T00:00:00Z
release id
clinical-indications-draft-1
disease state id
ds-basal-cell-carcinoma-libtayo-995f9df8
nct id
NCT01327053
outcome key
om-0005
source section id
NCT01327053-om-0005
source outcome index
5
endpoint
Complete Response Rate (CRR) Per Central Review (FAS)
endpoint definition
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
time origin
6 months
analysis role
secondary
reporting status
POSTED
data cutoff
Source null
population note
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.
curation status
draft
reviewed by
Source null
reviewed at
Source null
created at
2026-09-10T00:00:00Z

arms

release id
clinical-indications-draft-1
disease state id
ds-basal-cell-carcinoma-libtayo-995f9df8
nct id
NCT01327053
outcome key
om-0000
arm key
OG000
arm label
LDE225 200mg laBCC
regimen
The efficacy and safety of LDE225 200mg laBCC cohort were 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.
analyzed count
42
analyzed unit
Participants
is comparator
Source null
source pointer
NCT01327053-om-0000#groups/OG000
release id
clinical-indications-draft-1
disease state id
ds-basal-cell-carcinoma-libtayo-995f9df8
nct id
NCT01327053
outcome key
om-0000
arm key
OG001
arm label
LDE225 800mg laBCC
regimen
The efficacy and safety of LDE225 800mg laBCC cohort were 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.
analyzed count
93
analyzed unit
Participants
is comparator
Source null
source pointer
NCT01327053-om-0000#groups/OG001
release id
clinical-indications-draft-1
disease state id
ds-basal-cell-carcinoma-libtayo-995f9df8
nct id
NCT01327053
outcome key
om-0000
arm key
OG002
arm label
LDE225 200mg mBCC
regimen
The efficacy and safety of LDE225 200mg mBCC cohort were 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.
analyzed count
13
analyzed unit
Participants
is comparator
Source null
source pointer
NCT01327053-om-0000#groups/OG002
release id
clinical-indications-draft-1
disease state id
ds-basal-cell-carcinoma-libtayo-995f9df8
nct id
NCT01327053
outcome key
om-0000
arm key
OG003
arm label
LDE225 800mg mBCC
regimen
The efficacy and safety of LDE225 800mg mBCC cohort were 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.
analyzed count
23
analyzed unit
Participants
is comparator
Source null
source pointer
NCT01327053-om-0000#groups/OG003
release id
clinical-indications-draft-1
disease state id
ds-basal-cell-carcinoma-libtayo-995f9df8
nct id
NCT01327053
outcome key
om-0001
arm key
OG000
arm label
LDE225 200mg laBCC
regimen
The efficacy and safety of LDE225 200mg laBCC cohort were 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.
analyzed count
66
analyzed unit
Participants
is comparator
Source null
source pointer
NCT01327053-om-0001#groups/OG000
release id
clinical-indications-draft-1
disease state id
ds-basal-cell-carcinoma-libtayo-995f9df8
nct id
NCT01327053
outcome key
om-0001
arm key
OG001
arm label
LDE225 800mg laBCC
regimen
The efficacy and safety of LDE225 800mg laBCC cohort were 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.
analyzed count
128
analyzed unit
Participants
is comparator
Source null
source pointer
NCT01327053-om-0001#groups/OG001
release id
clinical-indications-draft-1
disease state id
ds-basal-cell-carcinoma-libtayo-995f9df8
nct id
NCT01327053
outcome key
om-0001
arm key
OG002
arm label
LDE225 200mg mBCC
regimen
The efficacy and safety of LDE225 200mg mBCC cohort were 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.
analyzed count
13
analyzed unit
Participants
is comparator
Source null
source pointer
NCT01327053-om-0001#groups/OG002
release id
clinical-indications-draft-1
disease state id
ds-basal-cell-carcinoma-libtayo-995f9df8
nct id
NCT01327053
outcome key
om-0001
arm key
OG003
arm label
LDE225 800mg mBCC
regimen
The efficacy and safety of LDE225 800mg mBCC cohort were 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.
analyzed count
23
analyzed unit
Participants
is comparator
Source null
source pointer
NCT01327053-om-0001#groups/OG003
release id
clinical-indications-draft-1
disease state id
ds-basal-cell-carcinoma-libtayo-995f9df8
nct id
NCT01327053
outcome key
om-0002
arm key
OG000
arm label
LDE225 200mg laBCC
regimen
The efficacy and safety of LDE225 200mg laBCC cohort were 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.
analyzed count
42
analyzed unit
Participants
is comparator
Source null
source pointer
NCT01327053-om-0002#groups/OG000
release id
clinical-indications-draft-1
disease state id
ds-basal-cell-carcinoma-libtayo-995f9df8
nct id
NCT01327053
outcome key
om-0002
arm key
OG001
arm label
LDE225 800mg laBCC
regimen
The efficacy and safety of LDE225 800mg laBCC cohort were 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.
analyzed count
93
analyzed unit
Participants
is comparator
Source null
source pointer
NCT01327053-om-0002#groups/OG001
release id
clinical-indications-draft-1
disease state id
ds-basal-cell-carcinoma-libtayo-995f9df8
nct id
NCT01327053
outcome key
om-0002
arm key
OG002
arm label
LDE225 200mg mBCC
regimen
The efficacy and safety of LDE225 200mg mBCC cohort were 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.
analyzed count
13
analyzed unit
Participants
is comparator
Source null
source pointer
NCT01327053-om-0002#groups/OG002
release id
clinical-indications-draft-1
disease state id
ds-basal-cell-carcinoma-libtayo-995f9df8
nct id
NCT01327053
outcome key
om-0002
arm key
OG003
arm label
LDE225 800mg mBCC
regimen
The efficacy and safety of LDE225 800mg mBCC cohort were 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.
analyzed count
23
analyzed unit
Participants
is comparator
Source null
source pointer
NCT01327053-om-0002#groups/OG003

harms

release id
clinical-indications-draft-1
disease state id
ds-basal-cell-carcinoma-libtayo-995f9df8
nct id
NCT01327053
harm key
EG000
source section id
NCT01327053-ae
event group key
EG000
event group label
LDE225 200 mg qd
serious affected
16
serious at risk
79
other affected
76
other at risk
79
frequency threshold
5
collection period
Adverse events and serious adverse events were collected for the maximum actual duration of treatment exposure and follow up for a participant per the protocol for approximately 76.2 months.
event definition
All cause mortality (deaths) was collected for as long as participants could be contacted from First Patient First Visit (FPFV) until Last Patient Last Visit (LPLV) up to a maximum of approx. 7 years.
undenominated note
Source null
curation status
draft
reviewed by
Source null
reviewed at
Source null
created at
2026-09-10T00:00:00Z
release id
clinical-indications-draft-1
disease state id
ds-basal-cell-carcinoma-libtayo-995f9df8
nct id
NCT01327053
harm key
EG001
source section id
NCT01327053-ae
event group key
EG001
event group label
LDE225 800 mg qd
serious affected
58
serious at risk
150
other affected
145
other at risk
150
frequency threshold
5
collection period
Adverse events and serious adverse events were collected for the maximum actual duration of treatment exposure and follow up for a participant per the protocol for approximately 76.2 months.
event definition
All cause mortality (deaths) was collected for as long as participants could be contacted from First Patient First Visit (FPFV) until Last Patient Last Visit (LPLV) up to a maximum of approx. 7 years.
undenominated note
Source null
curation status
draft
reviewed by
Source null
reviewed at
Source null
created at
2026-09-10T00:00:00Z

quality of life

setting source links

Preserved source evidence · Not medical advice