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Main
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Note: This record shows only 22 elements of the WHO Trial Registration Data Set. To view changes that have been made to the source record, or for additional information about this trial, click on the URL below to go to the source record in the primary register. |
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Register:
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ClinicalTrials.gov |
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Last refreshed on:
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12 December 2020 |
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Main ID: |
NCT03526159 |
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Date of registration:
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17/04/2018 |
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Prospective Registration:
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Yes |
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Primary sponsor: |
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Public title:
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Gentamicin for Junctional Epidermolysis Bullosa
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Scientific title:
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A Pilot Study of the Restoration of Functional Laminin 332 in JEB Patients With Nonsense Mutations After Topical and Intravenous Gentamicin Treatment |
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Date of first enrolment:
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June 1, 2018 |
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Target sample size:
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6 |
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Recruitment status: |
Recruiting |
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URL:
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https://clinicaltrials.gov/show/NCT03526159 |
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Study type:
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Interventional |
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Study design:
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Allocation: N/A. Intervention model: Single Group Assignment. Primary purpose: Treatment. Masking: None (Open Label).
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Phase:
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Phase 1/Phase 2
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Countries of recruitment
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United States
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Contacts
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Name:
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David Woodley, MD |
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Address:
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Telephone:
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323-865-0956 |
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Email:
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dwoodley@usc.edu |
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Affiliation:
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Name:
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Mei Chen, Ph.D. |
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Address:
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Telephone:
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3238650621 |
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Email:
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chenm@usc.edu |
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Affiliation:
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Key inclusion & exclusion criteria
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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.
Age minimum:
N/A
Age maximum:
N/A
Gender:
All
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Health Condition(s) or Problem(s) studied
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Junctional Epidermolysis Bullosa
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Intervention(s)
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Drug: Gentamicin Sulfate
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Primary Outcome(s)
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Generation of new hemidesmosomes as assessed by electron microscopy.
[Time Frame: 3 months]
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Incidence of Treatment-Emergent Adverse Events
[Time Frame: 3 months]
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Increased laminin beta 3 / laminin 332 expression as assessed by immunofluorescence.
[Time Frame: 3 months]
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Secondary Outcome(s)
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Reduction in blistering
[Time Frame: 3 months]
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Improved wound closure.
[Time Frame: 3 months]
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Secondary ID(s)
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HS-18-00290
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Source(s) of Monetary Support
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Please refer to primary and secondary sponsors
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Results
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Results available:
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Date Posted:
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Date Completed:
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URL:
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