Clinical Trial: Efficacy of Granulocyte Colony Stimulating Factor (GCSF) In Patients With Dystrophic Epidermolysis Bullosa
Study Status: Completed
Recruit Status: Completed
Study Type: Interventional
Official Title: Efficacy of Granulocyte Colony Stimulating Factor (GCSF) In Patients With Dystrophic Epidermolysis Bullosa
Brief Summary: This is a feasibility study to see if Granulocyte Colony Stimulating Factor (GCSF) is effective as a treatment of Dystrophic Epidermolysis Bullosa (EB.) Patients will receive one course of treatment with the study drug. The course will be 7 days in length. After receiving GCSF, patients will be followed at 7 and 30 days following the discontinuation of the drug. Thirty day follow up can be done via telephone communication with the patient or family.
Detailed Summary:
Sponsor: Vanderbilt University Medical Center
Current Primary Outcome: Percent Change of Active Blisters and in Total Blister/Erosion Counts [ Time Frame: 7 days ]
Original Primary Outcome: Number of active blisters [ Time Frame: 30 days ]
Current Secondary Outcome:
- Surface Area of Nonhealing Erosions [ Time Frame: 7 days ]Change in surface area of one or two nonhealing erosions
- Overall Improved Symptomatology [ Time Frame: 28 days ]Overall clinical improvement in symptomatology and/or findings, as assessed by either the patient or parent
Original Secondary Outcome:
- Surface Area of Nonhealing Erosions [ Time Frame: 30 days ]Reduction in surface area of one or two nonhealing erosions by at least 20%
- Overall symptomatology [ Time Frame: 30 days ]Overall clinical improvement in symptomatology and/or findings, as assessed by either the patient or parent, of at least 30%
Information By: Vanderbilt University Medical Center
Dates:
Date Received: February 20, 2012
Date Started: February 2012
Date Completion:
Last Updated: March 13, 2017
Last Verified: March 2017