Clinical Trial: A Study of Two Vismodegib Regimens in Participants With Multiple Basal Cell Carcinomas

Study Status: Active, not recruiting
Recruit Status: Active, not recruiting
Study Type: Interventional

Official Title: A Randomized, Double-blinded, Regimen-controlled, Phase II, Multicenter Study to Assess the Efficacy and Safety of Two Different Vismodegib Regimens in Patients With Multiple Basal Cell Carcinomas

Brief 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.

Detailed Summary:
Sponsor: Hoffmann-La Roche

Current Primary Outcome: Mean Percent Change From Baseline in the Number of Clinically Evident Basal Cell Carcinomas at Week 73 (After 72 Weeks of Treatment) [ Time Frame: Baseline; Week 73 ]

The total number of clinically evident basal cell carcinomas = the total number of target and/or non-target lesions present in individual participants.


Original Primary Outcome: Relative reduction (%) from baseline in the number of clinically evident basal cell carcinomas at Week 73 (after 72 weeks of treatment) [ Time Frame: from baseline to Week 73 ]

Current Secondary Outcome:

  • Percentage of Participants Who Discontinued Study Treatment Due to Tolerability Issues [ Time Frame: Baseline to Week 73 ]
    The percentage of participants who discontinued study treatment (due either to adverse event, refusal of treatment, or withdrawal of consent) was summarized by treatment group.
  • Mean Percent Change From Baseline in Total Size of Three Target Basal Cell Carcinoma Lesions in Individual Participants at Week 73 [ Time Frame: Baseline; Week 73 ]
    The three target basal cell carcinoma lesions = the three largest visible lesions, at least 5 mm in the longest diameter, in individual participants.
  • Percentage of Participants With at Least 50% Reduction in the Number of Basal Cell Carcinomas at Week 73 [ Time Frame: Baseline; Week 73 ]
  • Percentage of Participants With New Basal Cell Carcinomas at Week 73 [ Time Frame: Baseline; Week 73 ]
  • Percent Change in Total Number of Basal Cell Carcinomas Relative to Baseline at Week 85 (12 Weeks Following End of Treatment) (Recurrence Rate) [ Time Frame: Baseline; Week 85 ]
  • Percent Change in Total Number of Basal Cell Carcinomas Relative to Baseline at Week 97 (24 Weeks Following End of Treatment) (Recurrence Rate) [ Time Frame: Baseline; Week 97 ]
  • Percent Change in Total Number of Basal Cell Carcinomas Relative to Baseline at Week 125 (52 Weeks Following End of Treatment) (Recurrence Rate) [ Time Frame: Baseline; Week 125 ]
  • Percentage of Participants Experiencing Any Adverse Event [ Time Frame: Up to 125 weeks ]
  • Percent Change From Baseline in the Skindex-16 Symptom Domain Score at Week 73 [ Time Frame: Baseline; Week 73 ]
    The Skindex-16 is a patient-reported outcome health questionnaire. Participants were asked about their symptoms, and their answers were combined into a composite Symptom Domain Score. Scores range from 0 ("never bothered") to 100 ("always bothered").
  • Percent Change From Baseline in the Skindex-16 Emotion Domain Score at Week 73 [ Time Frame: Baseline; Week 73 ]
    The Skindex-16 is a patient-reported outcome health questionnaire. Participants were asked about their emotional state, and their answers were combined into a composite Emotion Domain Score. Scores range from 0 ("never bothered") to 100 ("always bothered").
  • Percent Change From Baseline in the Skindex-16 Function Domain Score at Week 73 [ Time Frame: Baseline; Week 73 ]
    The Skindex-16 is a patient-reported outcome health questionnaire. Participants were asked about their ability to function, and answers were combined into a composite Function Domain Score. Scores range from 0 ("never bothered") to 100 ("always bothered").


Original Secondary Outcome:

  • Drop-out rate: Discontinuation of study treatment prior to Week 73 for either adverse events or withdrawal of consent by the patient [ Time Frame: approximately 3.5 years ]
  • Relative reduction (%) from baseline in total size of three target basal cell carcinoma lesions in individual patients at Week 73 [ Time Frame: from baseline to Week 73 ]
  • Proportion of patients with at least 50% reduction in the number of basal cell carcinomas at Week 73 in the two treatment regimens [ Time Frame: approximately 3.5 years ]
  • Number of new basal cell carcinomas at Week 73 in the two treatment groups [ Time Frame: approximately 3.5 years ]
  • Recurrence rate up to Week 125 (52 weeks after study drug discontinuation) [ Time Frame: approximately 3.5 years ]
  • Safety: Incidence of adverse events [ Time Frame: approximately 3.5 years ]
  • Patient reported outcomes: Skindex-16 tool [ Time Frame: approximately 3.5 years ]


Information By: Hoffmann-La Roche

Dates:
Date Received: March 19, 2013
Date Started: April 2013
Date Completion: November 2016
Last Updated: September 19, 2016
Last Verified: September 2016