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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: Recruiting Phase 1 study of Junctional Epidermolysis Bullosa, sponsored by University of Southern California.
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, below the 60-participant average among 29,502 other Phase 1 trials with a reported enrollment target (90% lower). The trial reports 1 study location across 1 state. According to ClinicalTrials.gov, the official US trial registry.
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.
Primary Outcome
New or increased staining of the target protein, laminin 332, in sections of skin biopsies obtained during follow-up visits in comparison with baseline biopsies. Five micron cryosections will be probed with three different antibodies against laminin 332. Patient samples along with normal control samples will be compared. Mean fluorescence intensity will be calculated for each sample and antibody using ImageJ software. Percent expression relative to normal human skin (set to 100%) will be calcula
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 |
What NCT03526159 shows while recruiting
NCT03526159 is an interventional study that assigns participants to a tested intervention. The registry caps enrollment at 6 participants, a relatively small participant target, below the 60-participant average among 29,502 other Phase 1 trials with a reported enrollment target (90% lower).
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 a single indexed study location in 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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Where NCT03526159's enrollment target sits among peer trials
6 1975th of 2000 higher than 12 of 2,000 other Phase 1 trials
participants (enrollment target), bucketed by value
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