Intravenous Gentamicin Therapy for Recessive Dystrophic Epidermolysis Bullosa (RDEB)
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|ClinicalTrials.gov Identifier: NCT03392909|
Recruitment Status : Unknown
Verified April 2020 by David Woodley, University of Southern California.
Recruitment status was: Recruiting
First Posted : January 8, 2018
Last Update Posted : November 4, 2020
|Condition or disease||Intervention/treatment||Phase|
|Recessive Dystrophic Epidermolysis Bullosa||Drug: Gentamicin||Phase 1 Phase 2|
|Study Type :||Interventional (Clinical Trial)|
|Estimated Enrollment :||9 participants|
|Intervention Model:||Single Group Assignment|
|Masking:||None (Open Label)|
|Official Title:||Restoration of Full-Length Type VII Collagen in RDEB Patients With Nonsense Mutations After Intravenous Gentamicin Treatment|
|Actual Study Start Date :||July 5, 2018|
|Estimated Primary Completion Date :||June 2021|
|Estimated Study Completion Date :||July 2021|
Experimental: Intravenous Gentamicin
Intravenous gentamicin (7.5 mgs/kg) daily for for either 14 days and then stopped or twice weekly for three months and then stopped.
Short-term intravenous gentamicin therapy should have the advantage of treating all of the patient's multiple skin wounds simultaneously. Six patients (three adults and 3 children) will receive intravenous gentamicin (7.5 mgs/kg) daily for 14 days and then stopped. Three adult patients will receive intravenous gentamicin (7.5mg/kg) biweekly for three months and then stopped.
Other Name: Gentamicin Sulfate
- Full-length type VII collagen expression [ Time Frame: 6 months ]Increased expression of full-length type VII collagen as assessed by immunofluorescence
- Generation of anchoring fibrils [ Time Frame: 6 months ]Generation of new anchoring fibrils as assessed by immuno-electron microscopy
- Absence of gentamicin side effects [ Time Frame: 6 months ]Absence of gentamicin side effects, especially the detection of any ototoxicity or nephrotoxicity
- Improved Disease Activity scores [ Time Frame: 6 months ]Improved epidermolysis bullosa Disease Activity scores
- Improved Quality of Life score [ Time Frame: 6 months ]Improved Quality of Life score
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): NCT03392909
|Contact: David T Woodley, MDfirstname.lastname@example.org|
|Contact: Mei Chen, Ph.Demail@example.com|
|Principal Investigator:||David T. Woodley, MD||Professor, University of Southern California|
|Principal Investigator:||Mei Chen, Ph.D||Professor, University of Southern California|