Gentamicin Therapy for Recessive Dystrophic Epidermolysis Bullosa (RDEB) Nonsense Mutation Patients
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|ClinicalTrials.gov Identifier: NCT02698735|
Recruitment Status : Completed
First Posted : March 4, 2016
Results First Posted : August 14, 2019
Last Update Posted : October 29, 2019
|Condition or disease||Intervention/treatment||Phase|
|Recessive Dystrophic Epidermolysis Bullosa||Drug: Gentamicin Drug: Placebo||Phase 1 Phase 2|
|Study Type :||Interventional (Clinical Trial)|
|Actual Enrollment :||5 participants|
|Intervention Model:||Parallel Assignment|
|Intervention Model Description:||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).|
|Masking:||Triple (Participant, Investigator, Outcomes Assessor)|
|Masking Description:||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.|
|Actual Study Start Date :||February 25, 2016|
|Actual Primary Completion Date :||March 31, 2017|
|Actual Study Completion Date :||June 30, 2017|
Gentamicin was either formulated into a 0.1% ointment or solutions for injection were purchased directly from suppliers.
Other Name: Gentamicin Sulfate, Garamycin
Placebo Comparator: Placebo
There are two placebos used in this study. The ointment vehicle (same as used to formulate gentamicin) and vehicle solution for injection.
Other Name: Vehicle
- Restoration of Full-length Type VII Collagen as Assessed by Immunofluorescence. [ Time Frame: 3 months ]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.
- Number of Participants With Anchoring Fibrils as Assessed by Immuno-electron Microscopy [ Time Frame: 3 months ]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.
To learn more about this study, you or your doctor may contact the study research staff using the contact information provided by the sponsor.
Please refer to this study by its ClinicalTrials.gov identifier (NCT number): NCT02698735
|Principal Investigator:||David Woodley, MD||University of Southern California Department of Dermatology|