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

This study is ongoing, but not recruiting participants.
Sponsor:
Information provided by (Responsible Party):
Hoffmann-La Roche
ClinicalTrials.gov Identifier:
NCT01815840
First received: March 19, 2013
Last updated: September 19, 2016
Last verified: September 2016
  Purpose
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.

Condition Intervention Phase
Basal Cell Carcinoma
Drug: Vismodegib
Drug: Placebo
Phase 2

Study Type: Interventional
Study Design: Allocation: Randomized
Endpoint Classification: Safety/Efficacy Study
Intervention Model: Parallel Assignment
Masking: Double Blind (Subject, Investigator)
Primary Purpose: Treatment
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

Resource links provided by NLM:


Further study details as provided by Hoffmann-La Roche:

Primary Outcome Measures:
  • 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 ] [ Designated as safety issue: No ]
    The total number of clinically evident basal cell carcinomas = the total number of target and/or non-target lesions present in individual participants.


Secondary Outcome Measures:
  • Percentage of Participants Who Discontinued Study Treatment Due to Tolerability Issues [ Time Frame: Baseline to Week 73 ] [ Designated as safety issue: No ]
    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 ] [ Designated as safety issue: No ]
    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 ] [ Designated as safety issue: No ]
  • Percentage of Participants With New Basal Cell Carcinomas at Week 73 [ Time Frame: Baseline; Week 73 ] [ Designated as safety issue: No ]
  • 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 ] [ Designated as safety issue: No ]
  • 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 ] [ Designated as safety issue: No ]
  • 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 ] [ Designated as safety issue: No ]
  • Percentage of Participants Experiencing Any Adverse Event [ Time Frame: Up to 125 weeks ] [ Designated as safety issue: No ]
  • Percent Change From Baseline in the Skindex-16 Symptom Domain Score at Week 73 [ Time Frame: Baseline; Week 73 ] [ Designated as safety issue: No ]
    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 ] [ Designated as safety issue: No ]
    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 ] [ Designated as safety issue: No ]
    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").


Enrollment: 229
Study Start Date: April 2013
Estimated Study Completion Date: November 2016
Primary Completion Date: August 2015 (Final data collection date for primary outcome measure)
Arms Assigned Interventions
Experimental: Vismodegib Intermittent Schedule
Vismodegib intermittent schedule of 12 weeks vismodegib followed by 8 weeks placebo, repeated 3 times with a final course of vismodegib (total 72 weeks), followed by 52 weeks treatment-free follow up
Drug: Vismodegib
Vismodegib 150 mg hard gelatin capsule orally once daily
Other Name: Erivedge®
Drug: Placebo
Vismodegib placebo orally once daily
Experimental: Vismodegib Induction Followed by Intermittent Schedule
Vismodegib beginning with 24 weeks induction followed by intermittent schedule 8 weeks placebo, 8 weeks vismodegib (total 72 weeks), followed by 52 weeks treatment-free follow up
Drug: Vismodegib
Vismodegib 150 mg hard gelatin capsule orally once daily
Other Name: Erivedge®
Drug: Placebo
Vismodegib placebo orally once daily

  Eligibility

Ages Eligible for Study:   18 Years and older   (Adult, Senior)
Genders Eligible for Study:   Both
Accepts Healthy Volunteers:   No
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
  Contacts and Locations
Choosing to participate in a study is an important personal decision. Talk with your doctor and family members or friends about deciding to join a study. To learn more about this study, you or your doctor may contact the study research staff using the Contacts provided below. For general information, see Learn About Clinical Studies.

Please refer to this study by its ClinicalTrials.gov identifier: NCT01815840

  Show 57 Study Locations
Sponsors and Collaborators
Hoffmann-La Roche
Investigators
Study Director: Clinical Trials Hoffmann-La Roche
  More Information

Responsible Party: Hoffmann-La Roche
ClinicalTrials.gov Identifier: NCT01815840     History of Changes
Other Study ID Numbers: MO28295  2012-003305-10 
Study First Received: March 19, 2013
Results First Received: June 27, 2016
Last Updated: September 19, 2016
Health Authority: United States: Food and Drug Administration

Additional relevant MeSH terms:
Carcinoma
Carcinoma, Basal Cell
Basal Cell Nevus Syndrome
Hamartoma Syndrome, Multiple
Neoplasms, Glandular and Epithelial
Neoplasms by Histologic Type
Neoplasms
Neoplasms, Basal Cell
Odontogenic Cysts
Jaw Cysts
Bone Cysts
Cysts
Neoplastic Syndromes, Hereditary
Bone Diseases, Developmental
Bone Diseases
Musculoskeletal Diseases
Jaw Diseases
Stomatognathic Diseases
Abnormalities, Multiple
Congenital Abnormalities
Genetic Diseases, Inborn
Hamartoma
Neoplasms, Multiple Primary

ClinicalTrials.gov processed this record on December 02, 2016