Using gentamicin to treat junctional epidermolysis bullosa
Official title: A Pilot Study of the Restoration of Functional Laminin 332 in JEB Patients With Nonsense Mutations After Topical and Intravenous Gentamicin Treatment
Run by University of Southern California · NCT03526159 · Data updated 2026-11-12
This study is testing if a medication called gentamicin can help improve skin healing and condition in people with a rare skin disorder caused by specific gene mutations.
Quick facts
| Phase | Phase1; Phase2 |
|---|---|
| Study type | Interventional |
| Sex | All |
| Enrollment | 6 (estimated) |
| Sponsor | University of Southern California Academic / other |
| Locations | 1 site (Los Angeles, California) |
| Status | Recruiting |
| Started | June 1, 2018 |
| Main results expected | July 30, 2020 |
| Study ends | August 31, 2020 |
| Registry record updated | April 7, 2020 |
| Trial ID | NCT03526159 on ClinicalTrials.gov |
Is this trial a fit for you?
✓Likely a good fit
Ideal candidates are patients with junctional epidermolysis bullosa who have nonsense mutations in the LAMB3 gene.
✕Probably not a fit
Patients without nonsense mutations in the LAMB3 gene or those with pre-existing conditions like auditory or renal impairment may not benefit from this study.
A plain-English reading of the study's rules. Only the study team can confirm whether you qualify.
What this trial studies
This clinical trial investigates the use of gentamicin, administered both topically and intravenously, to treat patients with Herlitz junctional epidermolysis bullosa (H-JEB) caused by nonsense mutations in the LAMB3 gene. The study aims to restore the production of laminin beta3, which is crucial for skin integrity, and assess its effects on wound healing and skin adherence. A total of six subjects, including both adults and children, will be monitored for improvements in skin condition and healing during follow-up visits.
Why it matters
Potential benefit: If successful, this treatment could significantly improve skin integrity and healing in patients with junctional epidermolysis bullosa.
How similar studies have gone: While this approach is novel in the context of junctional epidermolysis bullosa, previous studies have shown promise in using gentamicin for nonsense mutation readthrough in other conditions.
Who can join: the full rules
Show full inclusion / exclusion criteria
Inclusion Criteria: 1\. JEB patients with nonsense mutations in the LAMB3 gene in either one or two alleles. Exclusion Criteria: 1. JEB patients who do not have nonsense mutations in the LAMB3 gene in either allele. 2. Pre-existing known auditory impairment. 3. Pre-existing known renal impairment. 4. Pre-existing known allergies to aminoglycosides or sulfate compounds. 5. Pregnancy.
Where it's running
-
University of Southern California
Los Angeles, California, United States Recruiting
Study contacts
- Study coordinator: Mei Chen, Ph.D.
- Email: chenm@usc.edu
- Phone: 3238650621
Similar studies
Other studies for the same condition or treatment. Recruiting studies are listed first.
-
Energy-F703EB Cream for Epidermolysis Bullosa: a skin cream test in adults
This phase 2 study tests ENERGI-F703EB Cream versus a matching vehicle to see if it is safe and helps small EB skin lesions in adults.
-
Oleogel-S10 gel for healing skin wounds in Japanese patients with epidermolysis bullosa.
We will try Oleogel‑S10 gel to see if it closes or shrinks wounds in Japanese children and adults with junctional or dystrophic epidermolysis bullosa.
-
Topical TCP-25 gel to help heal wounds in epidermolysis bullosa
This trial tests whether applying TCP-25 gel to matched skin wounds helps improve healing in children and adults with dystrophic or junctional epidermolysis bullosa.
How to take part
- Review the eligibility criteria above with your treating physician.
- Check the official listing on ClinicalTrials.gov for the latest contact details and recruitment status.
- Contact the study coordinator or principal investigator to ask for pre-screening. Pre-screening is free and never obligates you to enroll.