Phase 2
N=230
A Phase II Study of Efficacy and Safety in Patients With Locally Advanced or Metastatic Basal Cell Carcinoma
Basal Cell Carcinoma
Bottom Line
View on ClinicalTrials.gov: NCT01327053 ↗Enrolled (actual)
230
Serious AEs
32.3%
Results posted
Nov 2015
Primary outcome: 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) — 42.9; 37.6; 15.4; 17.4 Percentage of participants
Study Design & Population
- Study type
- Interventional
- Phase
- Phase 2
- Interventions
- LDE225 (Drug)
- Age
- Adult, Older Adult · 18+ yrs
- Sex
- All
- Sponsor
- Novartis Pharmaceuticals
- Primary completion
- Jun 2013
Outcome Measures
| Outcome | Result | p-value |
|---|---|---|
| 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) |
42.9; 37.6; 15.4; 17.4 | — |
| 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) |
47.0; 35.2; 15.4; 17.4 | — |
| SECONDARY Duration of Response (DoR) Per Central Review Using mRECIST for laBCC and RECIST 1.1 for mBCC (pEAS) |
12.9; 23.7; 24.0; NA | — |
| SECONDARY Duration of Response (DoR) Per Central Review Using mRECIST for laBCC and RECIST 1.1 for mBCC (FAS) |
26.1; 23.3; 24.0; NA | — |
| SECONDARY Complete Response Rate (CRR) Per Central Review (pEAS) |
4.8; 2.2; 0.0; 0.0 | — |
| SECONDARY Complete Response Rate (CRR) Per Central Review (FAS) |
3.0; 0.0; 0.0; 0.0 | — |
| SECONDARY Progression-free Survival (PFS) Per Central Review Using mRECIST for laBCC and RECIST 1.1 for mBCC (pEAS) |
19.0; 19.4; 13.1; 11.1 | — |
| SECONDARY Progression-free Survival (PFS) Per Central Review Using mRECIST for laBCC and RECIST 1.1 for mBCC (FAS) |
22.1; 24.9; 13.1; 11.1 | — |
| SECONDARY Time to Tumor Response (TTR) Per Central Review Using mRECIST for laBCC and RECIST 1.1 for mBCC (pEAS) |
4.0; 3.7; 9.2; 1.0 | — |
| SECONDARY Time to Tumor Response (TTR) Per Central Review Using mRECIST for laBCC and RECIST 1.1 for mBCC (FAS) |
4.0; 3.7; 9.2; 1.0 | — |
| SECONDARY 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) |
71.4; 61.3; 23.1; 34.8 | — |
| SECONDARY 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) |
71.2; 58.6; 23.1; 34.8 | — |
| SECONDARY Duration of Response (DoR) Per Site Investigator Review Using mRECIST for laBCC and RECIST 1.1 for mBCC (pEAS) |
18.2; 26.0; 18.1; 10.2 | — |
| SECONDARY Duration of Response (DoR) Per Site Investigator Review Using mRECIST for laBCC and RECIST 1.1 for mBCC (FAS) |
15.7; 26.0; 18.1; 10.2 | — |
| SECONDARY Progression-free Survival (PFS) Per Site Investigator Review Using mRECIST for laBCC and RECIST 1.1 for mBCC (pEAS) |
20.1; 28.0; 13.1; 14.3 | — |
| SECONDARY Time to Tumor Response (TTR) Per Site Investigator Review Using mRECIST for laBCC and RECIST 1.1 for mBCC (pEAS) |
1.9; 1.8; 1.0; 2.7 | — |
| SECONDARY Plasma Concentration of Sonidegib (LDE225) |
NA; NA; 207.76; 586.76; 372.26; 1012.63 | — |
| SECONDARY Overall Survival (OS) |
NA; NA; 47.6; 33.8 | — |
Summary
This study assessed the efficacy and safety of oral treatment with two dose levels of LDE225 in patients with locally advanced or metastatic BCC.
Eligibility Criteria
Inclusion Criteria
- Patients with locally advanced BCC and metastatic BCC
- Patients with adequate bone marrow, liver, and renal function
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.
Data sourced from ClinicalTrials.gov (NCT01327053). Outcome figures and adverse-event rates are extracted automatically from the registry's posted results and are provided for clinician reference, not as a substitute for the primary publication.