Trial · NCT03012191
Gentamicin for RDEB
NCT03012191, Phase 1, Phase 2, Completed, University of Southern California; results first posted 2019-07-23
3 sourcestrials-2026-11-12-0.5.0
Cite this record
ConductScience Library. Gentamicin for RDEB. Trials, record NCT03012191. Release trials-2026-11-12-0.5.0, 12 Nov 2026. https://conductscience.org/library/trials/NCT03012191
On this page
Trial
As registered on ClinicalTrials.gov, in the graph's capture of the registry.
- Title
- Gentamicin for RDEB
- NCT number
- NCT03012191
- Status
- Completed
- Phases
- Phase 1, Phase 2
- Enrollment
- 6 participants, actual
- Lead sponsor
- University of Southern California
- Conditions
- Recessive Dystrophic Epidermolysis Bullosa
- Interventions
- Gentamicin Sulfate
- First posted
- 2017-01-06
- Last updated
- 2019-07-23
- Results first posted
- 2019-07-23
- Limitations and caveats
- Small number of subjects analyzed due to rarity of the disease and experimental plan.
Source ClinicalTrials.gov
Registered outcomes4
Each outcome the trial registered, primary first, in ClinicalTrials.gov's words.
| List | Outcome | Time frame | Description |
|---|---|---|---|
| Primary | Increased Expression of Full-length Type VII Collagen as Assessed by Immunofluorescence | 6 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. 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 New or Increased Numbers of Anchoring Fibrils as Assessed by Immuno-electron Microscopy | 6 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). |
| Primary | Number of Participants With Absence of Gentamicin Side Effects Especially the Detection of Any Ototoxicity or Nephrotoxicity | 6 months | Prolonged exposure to systemic gentamicin is associated with ototoxicity and nephrotoxicity. Specific tests (creatinine clearance and gold-tone audiometry) are performed throughout the study in order to detect any drug-specific adverse events as a result of systemic exposure to gentamicin. Additionally, we test patient skin and serum throughout the study to look for increase of autoantibodies to C7. Since some of these patients may have never had C7 expressed in their bodies, gentamicin-induced C7 may cause in auto-immune response. |
| Secondary | Improved EBDASI Scores | 6 months | EBDASI - Epidermolysis Bullosa Disease Activity and Scarring Index Scale - Minimum (0) to Maximum (506) A lower score indicates a better outcome (less disease activity and/or damage) A clinical tool for evaluating the disease activity and damage associated with bullous patients. To be administered by a licensed dermatologist. |
Source ClinicalTrials.gov
Outcomes4
Each outcome the trial posted results for, in ClinicalTrials.gov's words.
| Type | Outcome | Time frame | Population | Units | Measured as |
|---|---|---|---|---|---|
| PRIMARY | Increased Expression of Full-length Type VII Collagen as Assessed by Immunofluorescence | 6 months | C7 Expression (percent of normal skin) | MEAN | |
| PRIMARY | Number of Participants With New or Increased Numbers of Anchoring Fibrils as Assessed by Immuno-electron Microscopy | 6 months | Participants | COUNT_OF_PARTICIPANTS | |
| PRIMARY | Number of Participants With Absence of Gentamicin Side Effects Especially the Detection of Any Ototoxicity or Nephrotoxicity | 6 months | Participants | COUNT_OF_PARTICIPANTS | |
| SECONDARY | Improved EBDASI Scores | 6 months | EBDASI is a system-wide disease activity score used for participants in the Intravenous Gentamicin phase of the study. Participants receiving Topical and Intradermal Gentamicin were not administered the EBDASI. | Scores on a scale | MEAN |
Source ClinicalTrials.gov
Results by group10
Each measurement the trial posted, group by group, in the words and numbers it posted.
| Outcome | Group | Category | Value | Spread | Units |
|---|---|---|---|---|---|
| Increased Expression of Full-length Type VII Collagen as Assessed by Immunofluorescence | Topical Gentamicin | 31.8 | Full Range 13.7 to 54.0 | C7 Expression (percent of normal skin) | |
| Increased Expression of Full-length Type VII Collagen as Assessed by Immunofluorescence | Intravenous Gentamicin | 28.4 | Full Range 4.0 to 59.0 | C7 Expression (percent of normal skin) | |
| Increased Expression of Full-length Type VII Collagen as Assessed by Immunofluorescence | Intradermal Gentamicin | 65.6 | Full Range 2 to 134 | C7 Expression (percent of normal skin) | |
| Number of Participants With New or Increased Numbers of Anchoring Fibrils as Assessed by Immuno-electron Microscopy | Topical Gentamicin | 1 | Participants | ||
| Number of Participants With New or Increased Numbers of Anchoring Fibrils as Assessed by Immuno-electron Microscopy | Intravenous Gentamicin | 2 | Participants | ||
| Number of Participants With New or Increased Numbers of Anchoring Fibrils as Assessed by Immuno-electron Microscopy | Intradermal Gentamicin | 1 | Participants | ||
| Number of Participants With Absence of Gentamicin Side Effects Especially the Detection of Any Ototoxicity or Nephrotoxicity | Topical Gentamicin | 2 | Participants | ||
| Number of Participants With Absence of Gentamicin Side Effects Especially the Detection of Any Ototoxicity or Nephrotoxicity | Intravenous Gentamicin | 2 | Participants | ||
| Number of Participants With Absence of Gentamicin Side Effects Especially the Detection of Any Ototoxicity or Nephrotoxicity | Intradermal Gentamicin | 2 | Participants | ||
| Improved EBDASI Scores | Intravenous Gentamicin | 171.3 | Full Range 161 to 187 | Scores on a scale |
Source ClinicalTrials.gov
Participant flow9
How many started, completed or left each group, period by period.
| Period | Milestone | Group | Participants | Units counted |
|---|---|---|---|---|
| Overall Study | STARTED | Topical Gentamicin | 2 | |
| Overall Study | STARTED | Intravenous Gentamicin | 2 | |
| Overall Study | STARTED | Intradermal Gentamicin | 2 | |
| Overall Study | COMPLETED | Topical Gentamicin | 2 | |
| Overall Study | COMPLETED | Intravenous Gentamicin | 2 | |
| Overall Study | COMPLETED | Intradermal Gentamicin | 2 | |
| Overall Study | NOT COMPLETED | Topical Gentamicin | 0 | |
| Overall Study | NOT COMPLETED | Intravenous Gentamicin | 0 | |
| Overall Study | NOT COMPLETED | Intradermal Gentamicin | 0 |
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 totals9
The totals the trial posted for each group.
| Total | Group | Affected | At risk |
|---|---|---|---|
| Total, all-cause mortality | Topical Gentamicin | 0 | 2 |
| Total, all-cause mortality | Intravenous Gentamicin | 0 | 2 |
| Total, all-cause mortality | Intradermal Gentamicin | 0 | 2 |
| Total, other adverse events | Topical Gentamicin | 0 | 2 |
| Total, other adverse events | Intravenous Gentamicin | 0 | 2 |
| Total, other adverse events | Intradermal Gentamicin | 0 | 2 |
| Total, serious adverse events | Topical Gentamicin | 0 | 2 |
| Total, serious adverse events | Intravenous Gentamicin | 0 | 2 |
| Total, serious adverse events | Intradermal Gentamicin | 0 | 2 |
Source ClinicalTrials.gov
Papers0
The papers the trial lists on ClinicalTrials.gov.
None in this release.
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/NCT03012191
GET https://conductscience.org/api/trials/v1/NCT03012191/registered