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| Sponsor: | Children's Hospital & Research Center Oakland |
|---|---|
| Collaborator: |
Genentech |
| Information provided by: | Children's Hospital & Research Center Oakland |
| ClinicalTrials.gov Identifier: | NCT00957229 |
Purpose
The purpose of this study is to reduce the number of new surgically eligible BCCs by 50% appearing during month 3-18 of medication ingestion.
| Condition | Intervention | Phase |
|---|---|---|
|
Basal Cell Nevus Syndrome Gorlin Syndrome |
Drug: GDC-0449 |
Phase 2 |
| Study Type: | Interventional |
| Study Design: | Allocation: Randomized Endpoint Classification: Safety/Efficacy Study Intervention Model: Crossover Assignment Masking: Double Blind (Subject, Investigator, Outcomes Assessor) Primary Purpose: Treatment |
| Official Title: | A Randomized, Phase II Multicenter Trial Evaluating the Efficacy and Safety of a Systemic Hedgehog Pathway Antagonist (GDC-0449) in Patients With Basal Cell Nevus Syndrome (BCNS) |
| Estimated Enrollment: | 41 |
| Study Start Date: | August 2009 |
| Estimated Study Completion Date: | March 2013 |
| Estimated Primary Completion Date: | March 2012 (Final data collection date for primary outcome measure) |
| Arms | Assigned Interventions |
|---|---|
| Placebo Comparator: Sugar pill |
Drug: GDC-0449
capsule, 150 mg, one pill daily, 18 months
Other Names:
|
| Experimental: GDC-0449 |
Drug: GDC-0449
capsule, 150 mg, one pill daily, 18 months
Other Names:
|
This is a Phase II, 18 month, double blind, randomized placebo-controlled, two arm multicenter clinical study design. During the 18-month treatment period, the safety and chemopreventive efficacy of 150 mg/day GDC-0449 versus placebo will be assessed, and include evaluations of the skin at monthly intervals for the first three months and then every 3 months for the next 15 months. Removal of new surgically eligible BCCs (SEBs) will be done by primary skin care physicians (PSCPs) or at Study Centers. A Data Safety Monitoring Board (DSMB) will review unblinded results for an interim analysis when 20 subjects have completed 12 months of drug. This review will focus on adverse events and efficacy results. Subjects will be monitored for the development of new SEBs after they discontinue study treatment. At the end of the 18 months, given that the observed adverse events are minimal, patients on placebo will be offered the opportunity to take GDC-0449 for 18 months in an open label continuation, followed by six months observation, and patients on GDC449 will be monitored for the next 24 months for assessment of the duration of benefit after stopping the drug.
Eligibility| Ages Eligible for Study: | 35 Years to 75 Years |
| Genders Eligible for Study: | Both |
| Accepts Healthy Volunteers: | No |
Inclusion Criteria:
The subject:
Exclusion Criteria:
The subject:
Contacts and Locations| United States, California | |
| Children's Hospital Oakland Research Institiute | |
| Oakland, California, United States, 94609 | |
| Principal Investigator: | Ervin Epstein, MD | Children's Hospital & Research Center Oakland |
More Information
| Responsible Party: | Ervin Epstein, MD, Children's Hospital Oakland Research Institute |
| ClinicalTrials.gov Identifier: | NCT00957229 History of Changes |
| Other Study ID Numbers: | 2009-026, SHH-4685s |
| Study First Received: | August 5, 2009 |
| Last Updated: | July 28, 2011 |
| Health Authority: | United States: Food and Drug Administration |
|
Basal Cell Nevus Syndrome Eye Abnormalities Tooth Abnormalities Nevus Nevus, Pigmented Odontogenic Cysts Jaw Cysts Bone Cysts Cysts Neoplasms Carcinoma, Basal Cell Carcinoma Neoplasms, Glandular and Epithelial Neoplasms by Histologic Type |
Neoplasms, Basal Cell Neoplastic Syndromes, Hereditary Bone Diseases, Developmental Bone Diseases Musculoskeletal Diseases Jaw Diseases Stomatognathic Diseases Abnormalities, Multiple Congenital Abnormalities Genetic Diseases, Inborn Eye Diseases Stomatognathic System Abnormalities Tooth Diseases Nevi and Melanomas |