Topical Sirolimus in Patients With Basal Cell Nevus Syndrome and in Healthy Participants

Phase 1

Completed

• Conditions: Neoplastic Syndrome; Non-melanomatous Skin Cancer

• Registration Number: NCT00433485
• Lead Sponsor: Yale University

Brief Summary

RATIONALE: Studying samples of blood and tissue from patients with basal cell nevus syndrome and from healthy participants in the laboratory may help doctors learn more about changes that may occur in DNA and identify biomarkers related to basal cell nevus syndrome. Chemoprevention is the use of certain drugs to keep cancer from forming, growing, or coming back. The use of sirolimus may keep basal cell skin cancer from forming in patients with basal cell nevus syndrome.

PURPOSE: This phase I trial is studying topical sirolimus in patients with basal cell nevus syndrome and in healthy participants.

Detailed Description

OBJECTIVES:

Primary

* Compare messenger RNA and protein expression patterns in patients with basal cell nevus syndrome (BCNS) vs in cultured cells of healthy participants (control) before treatment to identify a set of genes that are differentially expressed in BCNS.

* Assess the effects of topical sirolimus on gene expression (genes identified in the primary objective) in vivo using keratinocytes, fibroblasts, and lymphocytes from patients with BCNS and from healthy participants (controls) by targeted expression methods.

OUTLINE: Patients and healthy participants receive topical sirolimus ointment twice daily for 12 weeks.

Blood and skin biopsies are obtained at baseline and at week 12 for gene and protein expression studies. Alterations in RNA are measured by microarray analysis. Alterations in protein expression are measured by 2-dimensional gel electrophoresis and matrix-assisted laser desorption ionization time-of-flight mass spectrometry.

After completion of study therapy, patients and healthy participants are followed at 4 weeks.

PROJECTED ACCRUAL: A total of 16 patients and healthy participants will be accrued for this study.

Study Timeline

• Study First Submitted: 2007-02-08
• Study First Submitted QC: 2007-02-08
• Study First Posted: 2007-02-12
• Primary Completion: 2008-03
• Study Completion: 2008-03
• Status Verified: 2016-09
• Last Update Submitted: 2016-09-21
• Last Update Posted: 2016-09-23

Recruitment & Eligibility

• Status: COMPLETED
• Sex: All
• Target Recruitment: 16

Inclusion Criteria

Not provided

Exclusion Criteria

Not provided

Study & Design

• Study Type: INTERVENTIONAL
• Study Design: Not specified

Primary Outcome Measures

Name	Time	Method
Alterations in RNA as measured by microarray analysis
Alterations in protein expression as measured by 2-dimensional gel electrophoresis and matrix-assisted laser desorption ionization time-of-flight mass spectroscopy