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Gentamicin for Junctional Epidermolysis Bullosa

StatusRecruiting
PhasePhase 1/2
Started
View on ClinicalTrials.gov ↗
Record status
This record was last updated April 7, 2020 (before its estimated July 30, 2020 completion). Its status may not reflect the trial's current state.

Record History

  1. Primary completion date: Jun 30, 2020 to Jul 30, 2020
    • Study completion date: Jul 31, 2020 to Aug 31, 2020
    v4
  2. Trial status: Not Yet Recruiting to Recruiting
    • Start date changed from estimate of Jul 1, 2018 to Jun 1, 2018
    • Site details revised
    v3
  3. Start date: Jun 1, 2018 to Jul 1, 2018
    • Primary completion date: May 31, 2020 to Jun 30, 2020
    v2
  4. Study First Submitted
  • Stopped: an ongoing trial was terminated, suspended or withdrawn.
  • Notable: the trial’s plan changed such as a big move in its timeline or size, a new primary endpoint, sponsor or phase, or recruiting opened or resumed.
  • Minor: routine progress and housekeeping, such as sites added or enrollment closed.
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.
Trial Details
NCT Number NCT03526159
Lead Sponsor University of Southern California
Conditions
(from sponsor)		 Junctional Epidermolysis Bullosa
Related terms Epidermolysis Bullosa, Junctional
Enrollment 6 participants
Start Date
Primary Completion (estimated)
Study Completion (estimated)
Updated on ClinicalTrials.gov