Split body skin Test Site study with each patient getting 0.1% topical gentamicin to a test site wound (Test Site #1) or placebo ointment to another like sized wound (Test Site #2) and each patient getting an intradermal injection of gentamicin in an intact skin site (Test Site #3) or an intradermal injection of placebo solution to another intact skin site (Test Site #4). So, there are a total of 4 Test Skin Sites in each patient.

Gentamicin Versus Placebo

Sex: Female, Male

Race (NIH/OMB)

Region of Enrollment

Expression of C7 in Skin

Presence and Definition of Anchoring Fibrils

The expression of type VII collagen at the patients' dermal-epidermal junction was assessed by immunofluorescence (IF) using an antibody specific to type VII collagen. The expression was semi-quantitated using NIH Image J software. The IF expression of type VII collagen was assessed before treatment and at one and three months after treatment for each patient. All treated and untreated sites for all patients were also analyzed to determine statistical significance of treatment versus placebo for topical and intradermal administrations. At each assessment time point, type VII collagen expression was also measured in normal human skin. The expression of type VII collagen was then expressed as a percentage of the type VII collagen expressed in normal human skin.

0.1% Gentamicin antibiotic in vehicle ointment

Topical Gentamicin Ointment

Topical Placebo Ointment

8 mg Gentamicin in Solution (200ul Intradermal Injection)

Intradermal Gentamicin Injection

Intradermal Placebo Injection

Restoration of Full-length Type VII Collagen as Assessed by Immunofluorescence.

The expression of anchoring fibril structures at the patients' dermal-epidermal junction was assessed by immuno-electron microscopy (IEM) using an antibody specific to type VII collagen. The IEM expression of anchoring fibrils was assessed before treatment and at one and three months after treatment. At each assessment time point, anchoring fibrils were compared with normal human skin. Baseline pre-treatment and one and three month post-treatment sites were compared for the presence of anchoring fibrils after gentamicin treatment (or increase if anchoring fibrils were detected at baseline in patients). Comparisons were also made between placebo-treated and gentamicin-treated sites.

Number of Participants With Anchoring Fibrils as Assessed by Immuno-electron Microscopy

Overall Study

Dr. David Woodley

Recessive Dystrophic Epidermolysis Bullosa

Gentamicin was either formulated into a 0.1% ointment or solutions for injection were purchased directly from suppliers.

Gentamicin

There are two placebos used in this study. The ointment vehicle (same as used to formulate gentamicin) and vehicle solution for injection.

Placebo

Gentamicin Therapy for Recessive Dystrophic Epidermolysis Bullosa (RDEB) Nonsense Mutation Patients

David Woodley

Professor of Dermatology

University of Southern California

Each participant will receive gentamicin ointment, and placebo ointment to treat two separate matched wounds (each ointment used exclusively on one wound) for 14 days as well as receive a intradermal injections of gentamicin and placebo at two intact skin sites (one site for each treatment).

Ointments were formulated by an independent pharmacy and masked before receipt by investigators and delivered to participants as such. Intradermal injections were blinded by clinic staff (unrelated to the study) before injection.

epidermolysis bullosa dystrophica/erythema

Gentamicin Therapy for Recessive Dystrophic Epidermolysis Bullosa (RDEB) Nonsense Mutation Patients

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