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NCT03526159 · ClinicalTrials.gov registry record · Phase 1
Gentamicin for Junctional Epidermolysis Bullosa
A Phase 1 study of Junctional Epidermolysis Bullosa, sponsored by University of Southern California.
- Recruiting
- Registry status
- Phase 1
- Development phase
- 6
- Enrollment target
- 1
- Study location
NCT03526159 is a Phase 1 study of Junctional Epidermolysis Bullosa that is actively recruiting participants, run by University of Southern California. The registered enrollment target is 6 participants. The trial reports 1 study location across 1 state.
The verdict
NCT03526159, a Phase 1 study of Junctional Epidermolysis Bullosa, is actively recruiting participants, sponsored by University of Southern California.
- RECRUITING
- Registry status
- Phase 1
- Development phase
- 6 participants
- Enrollment target
- 1
- Study location
Study Summary
Herlitz junctional epidermolysis bullosa (H-JEB), an incurable, fatal, inherited skin disease, is caused by loss-of-function mutations in the LAMA3, LAMB3 or LAMC2 genes, resulting in loss of laminin 332 and poor epidermal-dermal adherence. Eighty percent of H-JEB patients have LAMB3 mutations and about 95% of these are nonsense mutations. The investigators recently demonstrated that gentamicin readily induced nonsense mutation readthrough and produced full-length laminin beta3 in several nonsense mutations tested. Importantly, the gentamicin-induced laminin beta3 restored laminin 332 assembly, secretion, and deposition into the dermal-epidermal junction (DEJ). Newly induced laminin 332 reversed abnormal H-JEB cellular phenotypes. Herein, the investigators propose the first clinical trial of gentamicin (by topical and intravenous administration) in JEB patients with nonsense mutations. The milestones will include restored laminin 332 and hemidesmosomes at the DEJ, improved wound closure, and the absence of significant gentamicin side effects.
Conditions Studied
Interventions
- DRUG Gentamicin Sulfate
Study Locations (1)
California
- University of Southern California - Los Angeles
Trial Details
| Field | Value |
|---|---|
| Enrollment Target | 6 participants |
| Start Date | 2018-06-01 |
| Est. Completion | 2020-08-31 |
| Phase | Phase 1 |
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Full Details on ClinicalTrials.gov ↗What the Registry Record Tells You About NCT03526159
The ClinicalTrials.gov registry entry for NCT03526159 describes a study currently listed as recruiting, categorized as Phase 1. The registered enrollment target is 6 participants, a figure that helps gauge the scale of data the investigators plan to collect. The listed sponsor is University of Southern California, which has 617 total studies on file at ClinicalTrials.gov.
The record links to 1 condition, with Junctional Epidermolysis Bullosa appearing as the primary indexed condition, and to 1 intervention - of which Gentamicin Sulfate is the first listed.
NCT03526159 reports 1 study location spanning 1 distinct geographic area - top geographies include California.
Frequently Asked Questions
What is clinical trial NCT03526159 about?
NCT03526159 is a clinical study titled "Gentamicin for Junctional Epidermolysis Bullosa". Herlitz junctional epidermolysis bullosa (H-JEB), an incurable, fatal, inherited skin disease, is caused by loss-of-function mutations in the LAMA3, LAMB3 or LAMC2 genes, resulting in loss of laminin 332 and poor epidermal-dermal adherence. Eighty percent of H-JEB patients have LAMB3 mutations and a...
What is the current status of trial NCT03526159?
This trial is currently recruiting. It is a Phase 1 study. The enrollment target is 6 participants. The study started on 2018-06-01. Estimated completion is 2020-08-31.
What conditions does trial NCT03526159 study?
This clinical trial studies the following conditions: Junctional Epidermolysis Bullosa.
What interventions are being tested in trial NCT03526159?
The interventions under investigation include: Gentamicin Sulfate (DRUG).
Who is sponsoring clinical trial NCT03526159?
This trial is sponsored by University of Southern California, which has 617 total clinical trials registered on ClinicalTrials.gov.
Where is trial NCT03526159 being conducted?
This trial has 1 study location across California. Contact the study sites directly through ClinicalTrials.gov for enrollment availability.
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