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Register:	ClinicalTrials.gov
Last refreshed on:	14 November 2022
Main ID:	NCT04140786
Date of registration:	23/10/2019
Prospective Registration:	Yes
Primary sponsor:	University of Southern California
Public title:	Optimizing IV Gentamicin in JEB
Scientific title:	Optimization of Intravenous Gentamicin Treatment to Restore Functional Laminin 332 in JEB Patients With Nonsense Mutations
Date of first enrolment:	October 31, 2019
Target sample size:	6
Recruitment status:	Recruiting
URL:	https://clinicaltrials.gov/show/NCT04140786
Study type:	Interventional
Study design:	Allocation: Non-Randomized. Intervention model: Single Group Assignment. Primary purpose: Treatment. Masking: None (Open Label).
Phase:	Phase 1/Phase 2

Countries of recruitment
United States

Contacts

Name:	Mei Chen, PhD
Address:
Telephone:
Email:
Affiliation:	University of Southern California

Name:	David Woodley, MD
Address:
Telephone:	323-865-0956
Email:	dwoodley@med.usc.edu
Affiliation:

Name:	David T Woodley, MD
Address:
Telephone:	(323) 442 0084
Email:	dwoodley@med.usc.edu
Affiliation:

Key inclusion & exclusion criteria

Inclusion Criteria:

- JEB patients with nonsense mutations in LAMB3 or LAMA3 in either one or two alleles

- Immunofluorescence (IF) analysis showing absence or decreased laminin 332 expression
at their DEJ compared with normal skin.

Exclusion Criteria:

- Pre-existing known auditory impairment.

- Pre-existing known renal impairment.

- Pre-existing known allergies to aminoglycosides or sulfate compounds.

- Pregnancy.

- Recent exposure to systemic gentamicin within the past 6 weeks.

- Current use of any medications with known potential ototoxicity or nephrotoxicity.

Age minimum: 30 Days
Age maximum: N/A
Gender: All

Health Condition(s) or Problem(s) studied

Junctional Epidermolysis Bullosa

Intervention(s)

Drug: Gentamicin Sulfate, Injectable

Primary Outcome(s)

Laminin 332 Expression in Skin [Time Frame: 3 months]

Safety (Nephrotoxicity) [Time Frame: 3 months]

Safety (Autoimmune Response) [Time Frame: 3 months]

Safety (Ototoxicity) [Time Frame: 3 months]

Secondary Outcome(s)

Wound Healing [Time Frame: 3 months]

Epidermolysis Bullosa Disease and Activity and Scarring Index (EBDASI) [Time Frame: 3 months]

Secondary ID(s)

HS-19-00760

Source(s) of Monetary Support

Please refer to primary and secondary sponsors

Secondary Sponsor(s)

Ethics review

Results

Results available:
Date Posted:
Date Completed:
URL:

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