Gentamicin for RDEB

Plain-language summary

This study examines Gentamicin Sulfate treatment in patients with recessive dystrophic epidermolysis bullosa (RDEB) who carry a nonsense mutation in the COL7A1 gene. RDEB is an inherited disease that causes the skin to blister easily. It enrolled patients whose skin showed an absence or decrease of the C7 protein. The study was carried out in Phase 1/Phase 2 and has been completed.

The summary above is a plain-language rendering of the official record. The original English title is shown for reference.

Trial details

Phase

PHASE1/PHASE2

Status

Completed

Sponsor

University of Southern California

Intervention

Gentamicin Sulfate

Start date

2/2/2017

Estimated completion

8/31/2018

Countries

United States

Related genes

Locations

• Los Angeles, United States

Eligibility criteria

Criteria are reproduced from the ClinicalTrials.gov record in the original English. Only the trial team can determine eligibility.

Inclusion Criteria:

(i) RDEB patients with a nonsense mutation in COL7A1 in either one or two alleles (ii) An absence or decrease in C7 expression at their DEJ when compared to that of normal human skin.

Exclusion Criteria:

(i) Pre-existing renal or auditory impairment (ii) Allergies to aminoglycosides or sulfate compounds (iii) Pregnancy (iv) Exposure to gentamicin within the past 6 weeks.