Phase 2
N=229
A Study of Two Vismodegib Regimens in Participants With Multiple Basal Cell Carcinomas
Basal Cell Carcinoma
Bottom Line
View on ClinicalTrials.gov: NCT01815840 ↗Enrolled (actual)
229
Serious AEs
20.7%
Results posted
Aug 2016
Primary outcome: Primary: Mean Percent Change From Baseline in the Number of Clinically Evident Basal Cell Carcinomas at Week 73 (After 72 Weeks of Treatment) — 62.9; 54.9 percent change
Study Design & Population
- Study type
- Interventional
- Phase
- Phase 2
- Interventions
- Vismodegib (Drug); Placebo (Drug)
- Age
- Adult, Older Adult · 18+ yrs
- Sex
- All
- Sponsor
- Hoffmann-La Roche
- Primary completion
- Aug 2015
Outcome Measures
| Outcome | Result | p-value |
|---|---|---|
| PRIMARY Mean Percent Change From Baseline in the Number of Clinically Evident Basal Cell Carcinomas at Week 73 (After 72 Weeks of Treatment) |
62.9; 54.9 | — |
| SECONDARY Percentage of Participants Who Discontinued Study Treatment Due to Tolerability Issues |
37.1; 41.6; 20.7; 27.4; 6.0; 2.7 | — |
| SECONDARY Mean Percent Change From Baseline in Total Size of Three Target Basal Cell Carcinoma Lesions in Individual Participants at Week 73 |
82.9; 68.0 | — |
| SECONDARY Percentage of Participants With at Least 50% Reduction in the Number of Basal Cell Carcinomas at Week 73 |
65.5; 50.4 | — |
| SECONDARY Percentage of Participants With New Basal Cell Carcinomas at Week 73 |
76.6; 74.4; 10.6; 11.6; 5.3; 5.8 | — |
| SECONDARY Percent Change in Total Number of Basal Cell Carcinomas Relative to Baseline at Week 85 (12 Weeks Following End of Treatment) (Recurrence Rate) |
35.7; 38.5 | — |
| SECONDARY Percent Change in Total Number of Basal Cell Carcinomas Relative to Baseline at Week 97 (24 Weeks Following End of Treatment) (Recurrence Rate) |
36.0; 42.1 | — |
| SECONDARY Percent Change in Total Number of Basal Cell Carcinomas Relative to Baseline at Week 125 (52 Weeks Following End of Treatment) (Recurrence Rate) |
41.2; 44.0 | — |
| SECONDARY Percentage of Participants Experiencing Any Adverse Event |
99.1; 97.3 | — |
| SECONDARY Percent Change From Baseline in the Skindex-16 Symptom Domain Score at Week 73 |
-14.9; -12.6 | — |
| SECONDARY Percent Change From Baseline in the Skindex-16 Emotion Domain Score at Week 73 |
-27.4; -28.9 | — |
| SECONDARY Percent Change From Baseline in the Skindex-16 Function Domain Score at Week 73 |
-9.5; -10.3 | — |
Summary
This randomized, double-blind, regimen-controlled, phase II, multicenter study will assess the efficacy and safety of two different vismodegib regimens in participants with multiple basal cell carcinoma. Participants will receive vismodegib 150 mg orally once daily either in an intermittent schedule of 12 weeks vismodegib followed by 8 weeks placebo (Arm A) or as 24 weeks induction followed by an intermittent schedule of 8 weeks placebo followed by 8 weeks vismodegib (Arm B). Anticipated time on study treatment is 72 weeks.
Eligibility Criteria
Inclusion Criteria
- Adult participants, >/= 18 years of age
- Participants with multiple basal cell carcinomas, including participants with Gorlin syndrome, with at least 6 clinically evident basal cell carcinomas at the time of randomization, of which 3 measure 5 mm or more in diameter and are considered target lesions. All other lesions are considered to be non-target lesions
- Histopathologic confirmation that at least one of the three target lesions is basal cell carcinoma
- Eastern Cooperative Oncology Group (ECOG) performance status of 0, 1 or 2
- Adequate renal and hepatic function and hematopoietic capacity
- Women of childbearing potential must agree to use contraception as defined by protocol during treatment and for at least 9 months after completion of study treatment
- Male participants with female partners of childbearing potential must agree to use contraception as defined by protocol during treatment and for 2 months after completion of study treatment
Exclusion Criteria
- Inability or unwillingness to swallow capsules
- Pregnant or breastfeeding women
- Any metastatic basal cell carcinoma
- Locally advanced basal cell carcinoma lesion that is considered to be inoperable or to have medical contraindications to surgery
- Recent (i.e., within the past 28 days prior to randomization) or current participation in another experimental drug study
- Known or suspected alcohol abuse
- One of the following known rare hereditary conditions: galactose intolerance, primary hypolactasia or glucose-galactose malabsorption
Data sourced from ClinicalTrials.gov (NCT01815840). 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.