Trial · NCT02698735
Gentamicin Therapy for Recessive Dystrophic Epidermolysis Bullosa (RDEB) Nonsense Mutation Patients
NCT02698735, Phase 1, Phase 2, Completed, University of Southern California; results first posted 2019-08-14
3 sourcestrials-2026-11-12-0.5.0
Cite this record
ConductScience Library. Gentamicin Therapy for Recessive Dystrophic Epidermolysis Bullosa (RDEB) Nonsense Mutation Patients. Trials, record NCT02698735. Release trials-2026-11-12-0.5.0, 12 Nov 2026. https://conductscience.org/library/trials/NCT02698735
On this page
Trial
As registered on ClinicalTrials.gov, in the graph's capture of the registry.
- Title
- Gentamicin Therapy for Recessive Dystrophic Epidermolysis Bullosa (RDEB) Nonsense Mutation Patients
- NCT number
- NCT02698735
- Status
- Completed
- Phases
- Phase 1, Phase 2
- Enrollment
- 5 participants, actual
- Lead sponsor
- University of Southern California
- Conditions
- Recessive Dystrophic Epidermolysis Bullosa
- Interventions
- Gentamicin, Placebo
- First posted
- 2016-03-04
- Last updated
- 2019-10-29
- Results first posted
- 2019-08-14
Source ClinicalTrials.gov
Registered outcomes2
Each outcome the trial registered, primary first, in ClinicalTrials.gov's words.
| List | Outcome | Time frame | Description |
|---|---|---|---|
| Primary | Restoration of Full-length Type VII Collagen as Assessed by Immunofluorescence. | 3 months | The expression of type VII collagen at the patients' dermal-epidermal junction was assessed by immunofluorescence (IF) using an antibody specific to type VII collagen. The expression was semi-quantitated using NIH Image J software. The IF expression of type VII collagen was assessed before treatment and at one and three months after treatment for each patient. All treated and untreated sites for all patients were also analyzed to determine statistical significance of treatment versus placebo for topical and intradermal administrations. At each assessment time point, type VII collagen expression was also measured in normal human skin. The expression of type VII collagen was then expressed as a percentage of the type VII collagen expressed in normal human skin. |
| Primary | Number of Participants With Anchoring Fibrils as Assessed by Immuno-electron Microscopy | 3 months | The expression of anchoring fibril structures at the patients' dermal-epidermal junction was assessed by immuno-electron microscopy (IEM) using an antibody specific to type VII collagen. The IEM expression of anchoring fibrils was assessed before treatment and at one and three months after treatment. At each assessment time point, anchoring fibrils were compared with normal human skin. Baseline pre-treatment and one and three month post-treatment sites were compared for the presence of anchoring fibrils after gentamicin treatment (or increase if anchoring fibrils were detected at baseline in patients). Comparisons were also made between placebo-treated and gentamicin-treated sites. |
Source ClinicalTrials.gov
Outcomes2
Each outcome the trial posted results for, in ClinicalTrials.gov's words.
| Type | Outcome | Time frame | Population | Units | Measured as |
|---|---|---|---|---|---|
| PRIMARY | Restoration of Full-length Type VII Collagen as Assessed by Immunofluorescence. | 3 months | Fluorescence Intensity (MFI) for C7 | MEAN | |
| PRIMARY | Number of Participants With Anchoring Fibrils as Assessed by Immuno-electron Microscopy | 3 months | Participants | COUNT_OF_PARTICIPANTS |
Source ClinicalTrials.gov
Results by group8
Each measurement the trial posted, group by group, in the words and numbers it posted.
| Outcome | Group | Category | Value | Spread | Units |
|---|---|---|---|---|---|
| Restoration of Full-length Type VII Collagen as Assessed by Immunofluorescence. | Topical Gentamicin Ointment | 5 | Standard Error 55.5 | Fluorescence Intensity (MFI) for C7 | |
| Restoration of Full-length Type VII Collagen as Assessed by Immunofluorescence. | Topical Placebo Ointment | 5 | Standard Error 3.5 | Fluorescence Intensity (MFI) for C7 | |
| Restoration of Full-length Type VII Collagen as Assessed by Immunofluorescence. | Intradermal Gentamicin Injection | 4 | Standard Error 56.2 | Fluorescence Intensity (MFI) for C7 | |
| Restoration of Full-length Type VII Collagen as Assessed by Immunofluorescence. | Intradermal Placebo Injection | 4 | Standard Error 2.2 | Fluorescence Intensity (MFI) for C7 | |
| Number of Participants With Anchoring Fibrils as Assessed by Immuno-electron Microscopy | Topical Gentamicin Ointment | 3 | Participants | ||
| Number of Participants With Anchoring Fibrils as Assessed by Immuno-electron Microscopy | Topical Placebo Ointment | 0 | Participants | ||
| Number of Participants With Anchoring Fibrils as Assessed by Immuno-electron Microscopy | Intradermal Gentamicin Injection | 3 | Participants | ||
| Number of Participants With Anchoring Fibrils as Assessed by Immuno-electron Microscopy | Intradermal Placebo Injection | 0 | Participants |
Source ClinicalTrials.gov
Participant flow3
How many started, completed or left each group, period by period.
| Period | Milestone | Group | Participants | Units counted |
|---|---|---|---|---|
| Overall Study | STARTED | Gentamicin Versus Placebo | 5 | 20 |
| Overall Study | COMPLETED | Gentamicin Versus Placebo | 5 | 18 |
| Overall Study | NOT COMPLETED | Gentamicin Versus Placebo | 0 | 2 |
Source ClinicalTrials.gov
Adverse events0
Each adverse event the trial posted, group by group: the participants affected out of those at risk.
None in this release.
Adverse event totals12
The totals the trial posted for each group.
| Total | Group | Affected | At risk |
|---|---|---|---|
| Total, all-cause mortality | Topical Gentamicin Ointment | 0 | 5 |
| Total, all-cause mortality | Topical Placebo Ointment | 0 | 5 |
| Total, all-cause mortality | Intradermal Gentamicin Injection | 0 | 4 |
| Total, all-cause mortality | Intradermal Placebo Injection | 0 | 4 |
| Total, other adverse events | Topical Gentamicin Ointment | 0 | 5 |
| Total, other adverse events | Topical Placebo Ointment | 0 | 5 |
| Total, other adverse events | Intradermal Gentamicin Injection | 0 | 4 |
| Total, other adverse events | Intradermal Placebo Injection | 0 | 4 |
| Total, serious adverse events | Topical Gentamicin Ointment | 0 | 5 |
| Total, serious adverse events | Topical Placebo Ointment | 0 | 5 |
1 to 10 of 12Source ClinicalTrials.gov
See all 12 adverse event totalsPapers1
The papers the trial lists on ClinicalTrials.gov.
| PMID | Listed as |
|---|---|
| 28691931 | DERIVED |
Source PubMed
Grants0
The grants NIH RePORTER links to the trial, newest funded first: NIH's, and some of AHRQ's, CDC's, FDA's and VA's. Each shows its title, principal investigators, organization and funder in its newest year.
None in this release.
Trial comparisons0
Comparisons of the trial's two groups, calculated by ConductScience from the counts it posted.
None in this release.
Tests0
The ConductCognition tests this trial's registered outcomes name, as ScienceGraph matched them in trial-tests-2026-11-12-0.2.1.
None in this release.
Data dates
The day each source was copied for this release.
Sources 3
- ClinicalTrials.gov's posted results, through CTTI's AACTFreely available. Acknowledgement requested
- ClinicalTrials.govNo license stated
- NIH RePORTER's clinical studies and project filesPublic domain
Each source's terms
- ClinicalTrials.gov's posted results, through CTTI's AACTClinicalTrials.gov data via Aggregate Analysis of ClinicalTrials.gov (AACT), CTTI. CTTI makes its materials freely available and requests acknowledgement. https://aact.ctti-clinicaltrials.org/documentation/299
- ClinicalTrials.govnone stated
- NIH RePORTER's clinical studies and project filesPublic domain (NIH RePORTER)
ClinicalTrials.gov's data carry an international copyright outside the United States: https://clinicaltrials.gov/about-site/terms-conditions.
For software
Read this trial from software.
The record and each of its lists are JSON, twenty rows a page. No key.
GET https://conductscience.org/api/trials/v1/NCT02698735
GET https://conductscience.org/api/trials/v1/NCT02698735/registered