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Gentamicin for RDEB

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ClinicalTrials.gov Identifier: NCT03012191
Recruitment Status : Completed
First Posted : January 6, 2017
Results First Posted : July 23, 2019
Last Update Posted : July 23, 2019
Sponsor:
Information provided by (Responsible Party):
David Woodley, University of Southern California

Study Type Interventional
Study Design Intervention Model: Single Group Assignment;   Masking: None (Open Label);   Primary Purpose: Treatment
Condition Recessive Dystrophic Epidermolysis Bullosa
Intervention Drug: Gentamicin Sulfate
Enrollment 6
Recruitment Details  
Pre-assignment Details  
Arm/Group Title Topical Gentamicin Intravenous Gentamicin Intradermal Gentamicin
Hide Arm/Group Description 0.5% Gentamicin ointment IV gentamicin is prepared to 7.5 mg/kg body weight and administered over a 30 minutes. 0.5% Gentamicin ointment applied after microneedle roller assistance
Period Title: Overall Study
Started 2 2 2
Completed 2 2 2
Not Completed 0 0 0
Arm/Group Title Topical Gentamicin Intravenous Gentamicin Intradermal Gentamicin Total
Hide Arm/Group Description 0.5% Gentamicin ointment IV gentamicin is prepared to 7.5 mg/kg body weight and administered over a 30 minutes. 0.5% Gentamicin ointment applied after microneedle roller assistance Total of all reporting groups
Overall Number of Baseline Participants 2 2 2 6
Hide Baseline Analysis Population Description
[Not Specified]
Age, Categorical  
Measure Type: Count of Participants
Unit of measure:  Participants
Number Analyzed 2 participants 2 participants 2 participants 6 participants
<=18 years
2
 100.0%
0
   0.0%
1
  50.0%
3
  50.0%
Between 18 and 65 years
0
   0.0%
2
 100.0%
1
  50.0%
3
  50.0%
>=65 years
0
   0.0%
0
   0.0%
0
   0.0%
0
   0.0%
Age, Continuous  
Mean (Full Range)
Unit of measure:  Years
Number Analyzed 2 participants 2 participants 2 participants 6 participants
9
(1 to 17)
28.5
(27 to 30)
22
(17 to 27)
19.8
(1 to 30)
Sex: Female, Male  
Measure Type: Count of Participants
Unit of measure:  Participants
Number Analyzed 2 participants 2 participants 2 participants 6 participants
Female
0
   0.0%
1
  50.0%
1
  50.0%
2
  33.3%
Male
2
 100.0%
1
  50.0%
1
  50.0%
4
  66.7%
Race (NIH/OMB)  
Measure Type: Count of Participants
Unit of measure:  Participants
Number Analyzed 2 participants 2 participants 2 participants 6 participants
American Indian or Alaska Native
0
   0.0%
0
   0.0%
0
   0.0%
0
   0.0%
Asian
0
   0.0%
0
   0.0%
0
   0.0%
0
   0.0%
Native Hawaiian or Other Pacific Islander
0
   0.0%
0
   0.0%
0
   0.0%
0
   0.0%
Black or African American
0
   0.0%
0
   0.0%
0
   0.0%
0
   0.0%
White
2
 100.0%
2
 100.0%
2
 100.0%
6
 100.0%
More than one race
0
   0.0%
0
   0.0%
0
   0.0%
0
   0.0%
Unknown or Not Reported
0
   0.0%
0
   0.0%
0
   0.0%
0
   0.0%
Region of Enrollment  
Measure Type: Number
Unit of measure:  Participants
United States Number Analyzed 2 participants 2 participants 2 participants 6 participants
2 2 2 6
Expression of C7 in Skin  
Mean (Full Range)
Unit of measure:  C7 Expression (percent of normal skin)
Number Analyzed 2 participants 2 participants 2 participants 6 participants
0
(0 to 0)
6.8
(2 to 11.6)
5.8
(0 to 11.6)
4.2
(0 to 11.6)
Presence of Anchoring Fibrils in Skin  
Measure Type: Count of Participants
Unit of measure:  Participants
Number Analyzed 2 participants 2 participants 2 participants 6 participants
0
   0.0%
1
  50.0%
1
  50.0%
2
  33.3%
1.Primary Outcome
Title Increased Expression of Full-length Type VII Collagen as Assessed by Immunofluorescence
Hide Description 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.
Time Frame 6 months
Hide Outcome Measure Data
Hide Analysis Population Description
[Not Specified]
Arm/Group Title Topical Gentamicin Intravenous Gentamicin Intradermal Gentamicin
Hide Arm/Group Description:
0.5% Gentamicin ointment
IV gentamicin is prepared to 7.5 mg/kg body weight and administered over a 30 minutes.
0.5% Gentamicin ointment applied after microneedle roller assistance
Overall Number of Participants Analyzed 2 2 2
Mean (Full Range)
Unit of Measure: C7 Expression (percent of normal skin)
31.8
(13.7 to 54.0)
28.4
(4.0 to 59.0)
65.6
(2 to 134)
2.Primary Outcome
Title Number of Participants With New or Increased Numbers of Anchoring Fibrils as Assessed by Immuno-electron Microscopy
Hide Description 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).
Time Frame 6 months
Hide Outcome Measure Data
Hide Analysis Population Description
[Not Specified]
Arm/Group Title Topical Gentamicin Intravenous Gentamicin Intradermal Gentamicin
Hide Arm/Group Description:
0.5% Gentamicin ointment
IV gentamicin is prepared to 7.5 mg/kg body weight and administered over a 30 minutes.
0.5% Gentamicin ointment applied after microneedle roller assistance
Overall Number of Participants Analyzed 2 2 2
Measure Type: Count of Participants
Unit of Measure: Participants
1
  50.0%
2
 100.0%
1
  50.0%
3.Primary Outcome
Title Number of Participants With Absence of Gentamicin Side Effects Especially the Detection of Any Ototoxicity or Nephrotoxicity
Hide Description 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.
Time Frame 6 months
Hide Outcome Measure Data
Hide Analysis Population Description
[Not Specified]
Arm/Group Title Topical Gentamicin Intravenous Gentamicin Intradermal Gentamicin
Hide Arm/Group Description:
0.5% Gentamicin ointment
IV gentamicin is prepared to 7.5 mg/kg body weight and administered over a 30 minutes.
0.5% Gentamicin ointment applied after microneedle roller assistance
Overall Number of Participants Analyzed 2 2 2
Measure Type: Count of Participants
Unit of Measure: Participants
2
 100.0%
2
 100.0%
2
 100.0%
4.Secondary Outcome
Title Improved EBDASI Scores
Hide Description 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.
Time Frame 6 months
Hide Outcome Measure Data
Hide Analysis Population Description
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.
Arm/Group Title Intravenous Gentamicin
Hide Arm/Group Description:
IV gentamicin is prepared to 7.5 mg/kg body weight and administered over a 30 minutes.
Overall Number of Participants Analyzed 2
Mean (Full Range)
Unit of Measure: Scores on a scale
171.3
(161 to 187)
Time Frame 6 months
Adverse Event Reporting Description [Not Specified]
 
Arm/Group Title Topical Gentamicin Intravenous Gentamicin Intradermal Gentamicin
Hide Arm/Group Description 0.5% Gentamicin ointment IV gentamicin is prepared to 7.5 mg/kg body weight and administered over a 30 minutes. 0.5% Gentamicin ointment applied after microneedle roller assistance
All-Cause Mortality
Topical Gentamicin Intravenous Gentamicin Intradermal Gentamicin
Affected / at Risk (%) Affected / at Risk (%) Affected / at Risk (%)
Total   0/2 (0.00%)   0/2 (0.00%)   0/2 (0.00%) 
Hide Serious Adverse Events
Topical Gentamicin Intravenous Gentamicin Intradermal Gentamicin
Affected / at Risk (%) Affected / at Risk (%) Affected / at Risk (%)
Total   0/2 (0.00%)   0/2 (0.00%)   0/2 (0.00%) 
Hide Other (Not Including Serious) Adverse Events
Frequency Threshold for Reporting Other Adverse Events 0%
Topical Gentamicin Intravenous Gentamicin Intradermal Gentamicin
Affected / at Risk (%) Affected / at Risk (%) Affected / at Risk (%)
Total   0/2 (0.00%)   0/2 (0.00%)   0/2 (0.00%) 
Small number of subjects analyzed due to rarity of the disease and experimental plan.
Certain Agreements
Principal Investigators are NOT employed by the organization sponsoring the study.
There is NOT an agreement between Principal Investigators and the Sponsor (or its agents) that restricts the PI's rights to discuss or publish trial results after the trial is completed.
Results Point of Contact
Layout table for Results Point of Contact information
Name/Title: Dr. David Woodley
Organization: University of Southern California Department of Dermatology
Phone: (323)442-0084
EMail: dwoodley@med.usc.edu
Layout table for additonal information
Responsible Party: David Woodley, University of Southern California
ClinicalTrials.gov Identifier: NCT03012191    
Other Study ID Numbers: HS-16-00788
First Submitted: January 4, 2017
First Posted: January 6, 2017
Results First Submitted: June 3, 2019
Results First Posted: July 23, 2019
Last Update Posted: July 23, 2019