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 ]

Percent change of active blisters and in total blister/erosion counts from baseline to 7 days


Original Primary Outcome: Number of active blisters [ Time Frame: 30 days ]

Reduction in active blisters and in total blister/erosion counts by at least 30%


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