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

The safety and scientific validity of this study is the responsibility of the study sponsor and investigators. Listing a study does not mean it has been evaluated by the U.S. Federal Government. Read our disclaimer for details. Identifier: NCT01538862
Recruitment Status : Completed
First Posted : February 24, 2012
Results First Posted : April 25, 2017
Last Update Posted : June 23, 2017
Information provided by (Responsible Party):
Haydar Frangoul, Vanderbilt University Medical Center

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.

Condition or disease Intervention/treatment Phase
Dystrophic Epidermolysis Bullosa Drug: Granulocyte Colony Stimulating Factor (GCSF) Phase 2

Detailed Description:
Each patient will be given 10 micrograms per kilogram per day of G-CSF subcutaneously for 6 consecutive days. On day 7 each patient will be seen and evaluated in the same manner as on day 0. Patients or their parents (if children are too young to reliably respond themselves) will also be asked to rate the following via a visual analog scale of 1-9- oral pain, pruritus, oral pain, swallowing, and overall sense of well-being. A telephone follow-up will be conducted on all patients 28 days after G-CSF so as to evaluate if the effect noted on day 7 was sustained.

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Study Type : Interventional  (Clinical Trial)
Actual Enrollment : 7 participants
Allocation: N/A
Intervention Model: Single Group Assignment
Masking: None (Open Label)
Primary Purpose: Treatment
Official Title: Efficacy of Granulocyte Colony Stimulating Factor (GCSF) In Patients With Dystrophic Epidermolysis Bullosa
Study Start Date : February 2012
Actual Primary Completion Date : October 2014
Actual Study Completion Date : November 2014

Arm Intervention/treatment
Experimental: Granulocyte Colony Stimulating Factor (GCSF)
GCSF 10mcg/kg/d subcutaneously (SQ) for 7 days
Drug: Granulocyte Colony Stimulating Factor (GCSF)
G-CSF 10mcg/kg/d SQ for 7 days

Primary Outcome Measures :
  1. 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

Secondary Outcome Measures :
  1. Surface Area of Nonhealing Erosions [ Time Frame: 7 days ]
    Change in surface area of one or two nonhealing erosions

  2. Overall Improved Symptomatology [ Time Frame: 28 days ]
    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.

Information from the National Library of Medicine

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Ages Eligible for Study:   Child, Adult, Older Adult
Sexes Eligible for Study:   All
Accepts Healthy Volunteers:   No

Inclusion Criteria:

  • Each patient must have the diagnosis of severe generalized recessive dystrophic EB (formerly known as Hallopeau-Siemens RDEB) confirmed by clinical criteria and either of the following:

    1. transmission electron microscopy
    2. immunofluorescence antigenic mapping and type VII collagen monoclonal antibody staining
    3. COL7A1 mutational analysis

Exclusion Criteria:

  • The patient must not have a history of squamous cell carcinoma or any internal malignancy.
  • Female patients who are pregnant.
  • Patients with active signs and symptoms of infection.

Information from the National Library of Medicine

To learn more about this study, you or your doctor may contact the study research staff using the contact information provided by the sponsor.

Please refer to this study by its identifier (NCT number): NCT01538862

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United States, Tennessee
Vanderbilt University
Nashville, Tennessee, United States, 37232
Sponsors and Collaborators
Vanderbilt University Medical Center
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Principal Investigator: Haydar Frangoul, MD Vanderbilt University
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Responsible Party: Haydar Frangoul, Professor of Pediatrics, Vanderbilt University Medical Center Identifier: NCT01538862    
Other Study ID Numbers: VICCNCPED1210
First Posted: February 24, 2012    Key Record Dates
Results First Posted: April 25, 2017
Last Update Posted: June 23, 2017
Last Verified: May 2017
Individual Participant Data (IPD) Sharing Statement:
Plan to Share IPD: No
Additional relevant MeSH terms:
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Epidermolysis Bullosa
Epidermolysis Bullosa Dystrophica
Skin Abnormalities
Congenital Abnormalities
Skin Diseases, Genetic
Genetic Diseases, Inborn
Skin Diseases
Skin Diseases, Vesiculobullous
Collagen Diseases
Connective Tissue Diseases
Immunologic Factors
Physiological Effects of Drugs
Adjuvants, Immunologic