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Efficacy of Granulocyte Colony Stimulating Factor (GCSF) In Patients With Dystrophic Epidermolysis Bullosa

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ClinicalTrials.gov Identifier: NCT01538862
Recruitment Status : Completed
First Posted : February 24, 2012
Results First Posted : April 25, 2017
Last Update Posted : June 23, 2017
Sponsor:
Information provided by (Responsible Party):
Haydar Frangoul, Vanderbilt University Medical Center

Study Type Interventional
Study Design Intervention Model: Single Group Assignment;   Masking: None (Open Label);   Primary Purpose: Treatment
Condition Dystrophic Epidermolysis Bullosa
Intervention Drug: Granulocyte Colony Stimulating Factor (GCSF)
Enrollment 7
Recruitment Details  
Pre-assignment Details  
Arm/Group Title Granulocyte Colony Stimulating Factor (GCSF)
Hide Arm/Group Description

GCSF 10mcg/kg/d SQ for 7 days

Granulocyte Colony Stimulating Factor (GCSF): G-CSF 10mcg/kg/d SQ for 7 days

Period Title: Overall Study
Started 7
Completed 7
Not Completed 0
Arm/Group Title Granulocyte Colony Stimulating Factor (GCSF)
Hide Arm/Group Description

GCSF 10mcg/kg/d SQ for 7 days

Granulocyte Colony Stimulating Factor (GCSF): G-CSF 10mcg/kg/d SQ for 7 days

Overall Number of Baseline Participants 7
Hide Baseline Analysis Population Description
[Not Specified]
Age, Categorical  
Measure Type: Count of Participants
Unit of measure:  Participants
Number Analyzed 7 participants
<=18 years
6
  85.7%
Between 18 and 65 years
1
  14.3%
>=65 years
0
   0.0%
Sex: Female, Male  
Measure Type: Count of Participants
Unit of measure:  Participants
Number Analyzed 7 participants
Female
4
  57.1%
Male
3
  42.9%
Region of Enrollment  
Measure Type: Number
Unit of measure:  Participants
United States Number Analyzed 7 participants
7
1.Primary Outcome
Title Percent Change of Active Blisters and in Total Blister/Erosion Counts
Hide Description Percent change of active blisters and in total blister/erosion counts from baseline to 7 days
Time Frame 7 days
Show Outcome Measure DataHide Outcome Measure Data
Hide Analysis Population Description
Time frame was originally entered as 30 days. This was not consistent with the protocol which listed a 7 day time frame for this Outcome
Arm/Group Title Granulocyte Colony Stimulating Factor (GCSF)
Hide Arm/Group Description:

GCSF 10mcg/kg/d subcutaneously for 7 days

Granulocyte Colony Stimulating Factor (GCSF): G-CSF 10mcg/kg/d subcutaneously for 7 days

Overall Number of Participants Analyzed 7
Mean (Standard Deviation)
Unit of Measure: percent change
-29.6  (30.3)
Show Statistical Analysis 1 Hide Statistical Analysis 1
Statistical Analysis Overview Comparison Group Selection Granulocyte Colony Stimulating Factor (GCSF)
Comments [Not Specified]
Type of Statistical Test Superiority
Comments [Not Specified]
Statistical Test of Hypothesis P-Value 0.82
Comments [Not Specified]
Method Regression, Linear
Comments [Not Specified]
2.Secondary Outcome
Title Surface Area of Nonhealing Erosions
Hide Description Change in surface area of one or two nonhealing erosions
Time Frame 7 days
Show Outcome Measure DataHide Outcome Measure Data
Hide Analysis Population Description
Time frame was originally entered as 30 days. This was not consistent with the protocol which listed a 7 day time frame for this Outcome
Arm/Group Title Granulocyte Colony Stimulating Factor (GCSF)
Hide Arm/Group Description:

GCSF 10mcg/kg/d subcutaneously for 7 days

Granulocyte Colony Stimulating Factor (GCSF): G-CSF 10mcg/kg/d subcutaneously for 7 days

Overall Number of Participants Analyzed 7
Mean (Standard Deviation)
Unit of Measure: percentage change
-35.3  (100.8)
3.Secondary Outcome
Title Overall Improved Symptomatology
Hide Description Overall clinical improvement in symptomatology and/or findings, as assessed by either the patient or parent. This would include decrease in the number and size of blister and erosions, decreased pain, improved comfort of the patient.
Time Frame 28 days
Show Outcome Measure DataHide Outcome Measure Data
Hide Analysis Population Description
[Not Specified]
Arm/Group Title Granulocyte Colony Stimulating Factor (GCSF)
Hide Arm/Group Description:

GCSF 10mcg/kg/d SQ for 7 days

Granulocyte Colony Stimulating Factor (GCSF): G-CSF 10mcg/kg/d SQ for 7 days

Overall Number of Participants Analyzed 7
Measure Type: Count of Participants
Unit of Measure: Participants
6
  85.7%
Time Frame 30 days
Adverse Event Reporting Description [Not Specified]
 
Arm/Group Title Granulocyte Colony Stimulating Factor (GCSF)
Hide Arm/Group Description

GCSF 10mcg/kg/d SQ for 7 days

Granulocyte Colony Stimulating Factor (GCSF): G-CSF 10mcg/kg/d SQ for 7 days

All-Cause Mortality
Granulocyte Colony Stimulating Factor (GCSF)
Affected / at Risk (%)
Total   --/-- 
Show Serious Adverse Events Hide Serious Adverse Events
Granulocyte Colony Stimulating Factor (GCSF)
Affected / at Risk (%)
Total   0/7 (0.00%) 
Show Other (Not Including Serious) Adverse Events Hide Other (Not Including Serious) Adverse Events
Frequency Threshold for Reporting Other Adverse Events 0%
Granulocyte Colony Stimulating Factor (GCSF)
Affected / at Risk (%)
Total   0/7 (0.00%) 
Certain Agreements
All Principal Investigators ARE employed by the organization sponsoring the study.
Results Point of Contact
Name/Title: Haydar Frangoul, MD, MS
Organization: Vanderbilt University
Phone: 615-342-7339
Responsible Party: Haydar Frangoul, Vanderbilt University Medical Center
ClinicalTrials.gov Identifier: NCT01538862     History of Changes
Other Study ID Numbers: VICCNCPED1210
First Submitted: February 20, 2012
First Posted: February 24, 2012
Results First Submitted: April 1, 2016
Results First Posted: April 25, 2017
Last Update Posted: June 23, 2017