A Phase 1/2 interventional study of Gentamicin in Recessive Dystrophic Epidermolysis Bullosa, sponsored by University of Southern California. Status unknown at 1 site in United States. Open to participants aged 7 Years and older. Per ClinicalTrials.gov, last updated 2022-11-03.
Sponsored by University of Southern California · Phase 1/2, Interventional, and Treatment
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Among 45 studies in Epidermolysis Bullosa Dystrophica with a site in United States — 12 open to participants now, 16 under way, 4 of them phase 3. Epidermolysis Bullosa Dystrophica · United States.
Among the 45 studies in Epidermolysis Bullosa Dystrophica with a site in United States, this one is the 15th largest by planned enrollment of the 32 interventional ones that give a count, is one of 4 that started in 2018, is one of 12 in phase 1/2, and was the 15th registered.
78 studies on the registry are indexed under Epidermolysis Bullosa Dystrophica; 21 are open to participants now.
Browse Epidermolysis Bullosa Dystrophica studies →12 of the 45 studies in Epidermolysis Bullosa Dystrophica with a site in United States are open now. Recruiting first, the newest first:
Xinnate AB · Sites in United States, France, Greece and 4 more countries
Ages 4+
M. Peter Marinkovich · Sites in United States
Ages 6+
Abeona Therapeutics, Inc · Sites in United States
Krystal Biotech, Inc. · Sites in United States
Ages 6 mo+
Chiesi Farmaceutici S.p.A. · Sites in United States, France, Germany and 3 more countries
A study can be open while its sites in United States have not opened yet or have stopped. Only those recruiting at a site in United States →
Of the 17 Epidermolysis Bullosa Dystrophica studies recruiting now, 13 (76%) set no upper age limit and 3 (18%) stop at 75 or younger; 15 take children under 18; 2 take healthy volunteers.
This study: 7 Years and older · all sexes · no healthy volunteers.
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45 studies; the count open now beside each. This study's row is marked.
A study in two phases is counted once above. Listed by phase, each with its combined phases: Phase 1 15 · Phase 2 18 · Phase 3 8
First in 2007; this study’s year is marked. 4 started in 2018.
By drug class, each counting the studies that test any of its medicines:
This study's planned enrollment of 9 is close to the median of 9 across 55 interventional studies indexed under Epidermolysis Bullosa Dystrophica.
Median enrollment 8 across the 32 interventional studies, 29 across the 13 observational. Of the 24 that completed or were terminated, 11 (46%) have results posted.
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29 of 45 have an industry lead sponsor. The 10 most frequent:
University of Southern California is the lead sponsor of 792 studies on the registry; 137 are open to participants now.
Of its 70 completed or terminated interventional studies of FDA-regulated products, 32 (46%) have results posted.
University of Southern California has 3 studies filed under Epidermolysis Bullosa Dystrophica — fewer than ten, so there is no comparison to draw.
4 of the studies in Epidermolysis Bullosa Dystrophica with a site in United States that are under way reach primary completion within a year, 2 of them phase 3. Phase 3 first, then by date:
| Study | Primary completion |
|---|---|
| NCT07016750Phase 3Krystal Biotech, Inc.A Study Comparing KB803 and Matched Placebo in Patients With Dystrophic Epidermolysis Bullosa | 2026-12 |
| NCT06892639Phase 3Castle Creek Biosciences, LLC.Evaluation of D-Fi for the Treatment of Wounds Due to DEB | 2027-09 |
| NCT06834035M. Peter MarinkovichTargeting Collagen VII Antibodies With IV IgG in Dystrophic Epidermolysis Bullosa | 2027-03 |
| NCT06594393Xinnate ABA Phase 2 Study of TCP-25 Gel in Patients With Epidermolysis Bullosa, STEP-study | 2027-06-30 |
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Among the 45 studies in Epidermolysis Bullosa Dystrophica with a site in United States, from their registry updates:
Nothing recorded among them since 2026-09-25.
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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 milestones will be increased C7 and AFs in the patients' DEJ, improved EB Disease Activity Scores, and absence of gentamicin side effects.
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Exclusion Criteria:
Underlined terms in the criteria open these too. Explained by clinicaltrials.gg, not part of the registry record. The study team decides who can take part.
Before you join, the study team explains what taking part involves — its purpose, what happens, the risks and possible benefits — and answers your questions, so you can decide freely. Consent is usually recorded on a form (the informed consent form, ICF) signed by you or, where the law allows, by someone legally authorised to decide for you.
You can keep asking questions during the study, you are told of new information that matters, and you can choose to leave at any time.
A biopsy takes a small piece of tissue to examine. “Archival tissue” is a sample already taken, for example at diagnosis, and kept by the hospital.
Whether a stored sample will do depends on when it was taken, how much there is and what the study needs. Ask whether the study accepts your stored sample or needs a new biopsy.
Informed consent
Before you join, the study team explains what taking part involves — its purpose, what happens, the risks and possible benefits — and answers your questions, so you can decide freely. Consent is usually recorded on a form (the informed consent form, ICF) signed by you or, where the law allows, by someone legally authorised to decide for you.
You can keep asking questions during the study, you are told of new information that matters, and you can choose to leave at any time.
Explained by clinicaltrials.gg, not part of the registry record. The study team decides who can take part.
Biopsy and archival tissue
A biopsy takes a small piece of tissue to examine. “Archival tissue” is a sample already taken, for example at diagnosis, and kept by the hospital.
Whether a stored sample will do depends on when it was taken, how much there is and what the study needs. Ask whether the study accepts your stored sample or needs a new biopsy.
Explained by clinicaltrials.gg, not part of the registry record. The study team decides who can take part.
Intravenous gentamicin (7.5 mgs/kg) daily for for either 14 days and then stopped or twice weekly for three months and then stopped.
Drug: Gentamicin
Short-term intravenous gentamicin therapy should have the advantage of treating all of the patient's multiple skin wounds simultaneously. Six patients (three adults and 3 children) will receive intravenous gentamicin (7.5 mgs/kg) daily for 14 days and then stopped. Three adult patients will receive intravenous gentamicin (7.5mg/kg) biweekly for three months and then stopped.
Also known as: Gentamicin Sulfate
Full-length type VII collagen expression
Increased expression of full-length type VII collagen as assessed by immunofluorescence
Time frame: 6 months
Generation of anchoring fibrils
Generation of new anchoring fibrils as assessed by immuno-electron microscopy
Time frame: 6 months
Absence of gentamicin side effects
Absence of gentamicin side effects, especially the detection of any ototoxicity or nephrotoxicity
Time frame: 6 months
Improved Disease Activity scores
Improved epidermolysis bullosa Disease Activity scores
Time frame: 6 months
Improved Quality of Life score
Improved Quality of Life score
Time frame: 6 months
Plan to share: No
No publications or documents are linked to this record.
Started Jul 2018 (actual). Primary completion is estimated for Dec 2023, 64 months after the start.
How these figures are worked out, and which studies are left out of them.
This study is status unknown, as verified in Nov 2022. You cannot join it, but the record below documents what was studied.
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Studies of the drug tested here: Gentamicins
Epidermolysis Bullosa Dystrophica →
University of Southern California →