Medical Information Only. Always consult your healthcare provider before enrolling in any clinical trial.
NCT03012191 · ClinicalTrials.gov registry record · Phase 1
Gentamicin for RDEB
A Phase 1 study, sponsored by University of Southern California.
- Completed
- Registry status
- Phase 1
- Development phase
- 6
- Enrollment target
NCT03012191 is a Phase 1 study that has completed, run by University of Southern California. The registered enrollment target is 6 participants.
The verdict
NCT03012191, a Phase 1 study, has completed, sponsored by University of Southern California.
- COMPLETED
- Registry status
- Phase 1
- Development phase
- 6 participants
- Enrollment target
Study Summary
Recessive dystrophic epidermolysis bullosa (RDEB) is an incurable, devastating, inherited skin disease caused by mutations in the COL7A1 gene that encodes for type VII collagen (C7), the major component of anchoring fibrils (AFs), structures that mediate epidermal-dermal adherence. Thirty percent of RDEB patients have nonsense mutations. The investigators recently demonstrated in 5 such patients that intradermal and topical gentamicin induced "read-through" of their nonsense mutations and created robust and sustained new C7 and AFs at the dermal-epidermal junction (DEJ) of their skin and also stimulated wound closure and reduced new blister formation. No untoward side effects occurred. Herein, the investigators propose evaluating the safety and efficacy of intravenous gentamicin in these patients. In theory, this intravenous administration has the possibility of treating simultaneously all of the patients' skin wounds. The investigators also propose optimizing the concentration and manner of delivery of topical gentamicin. The unambiguous milestones will be increased C7 and AFs in the patients' DEJ, improved EB Disease Activity Scores, and absence of significant gentamicin side effects.
Interventions
- DRUG Gentamicin Sulfate
Trial Details
| Field | Value |
|---|---|
| Enrollment Target | 6 participants |
| Start Date | 2017-02-02 |
| Est. Completion | 2018-08-31 |
| Phase | Phase 1 |
Interested in This Trial?
Full Details on ClinicalTrials.gov ↗What the Registry Record Tells You About NCT03012191
The ClinicalTrials.gov registry entry for NCT03012191 describes a study currently listed as completed, 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 0 conditions, and to 1 intervention - of which Gentamicin Sulfate is the first listed.
NCT03012191 reports 0 study locations.
Frequently Asked Questions
What is clinical trial NCT03012191 about?
NCT03012191 is a clinical study titled "Gentamicin for RDEB". Recessive dystrophic epidermolysis bullosa (RDEB) is an incurable, devastating, inherited skin disease caused by mutations in the COL7A1 gene that encodes for type VII collagen (C7), the major component of anchoring fibrils (AFs), structures that mediate epidermal-dermal adherence. Thirty percent of...
What is the current status of trial NCT03012191?
This trial is currently completed. It is a Phase 1 study. The enrollment target is 6 participants. The study started on 2017-02-02. Estimated completion is 2018-08-31.
What interventions are being tested in trial NCT03012191?
The interventions under investigation include: Gentamicin Sulfate (DRUG).
Who is sponsoring clinical trial NCT03012191?
This trial is sponsored by University of Southern California, which has 617 total clinical trials registered on ClinicalTrials.gov.
Learn More About Clinical Trials
Explore more on PlainTrial
Read our methodology - how this data is sourced, computed, and verified.
Related
Every figure on PlainTrial is rendered directly from the ClinicalTrials.gov registry, no number is typed in by an editor. This page mirrors this trial's own ClinicalTrials.gov registry record, live from the dataset. See our editorial standards & corrections policy, the methodology behind these numbers, or report a data error.