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

This study has been completed.
Information provided by (Responsible Party):
Haydar Frangoul, Vanderbilt University Identifier:
First received: February 20, 2012
Last updated: December 5, 2014
Last verified: December 2014
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 Intervention
Dystrophic Epidermolysis Bullosa
Drug: Granulocyte Colony Stimulating Factor (GCSF)

Study Type: Interventional
Study Design: Intervention Model: Single Group Assignment
Masking: Open Label
Primary Purpose: Treatment
Official Title: Efficacy of Granulocyte Colony Stimulating Factor (GCSF) In Patients With Dystrophic Epidermolysis Bullosa

Resource links provided by NLM:

Further study details as provided by Vanderbilt University:

Primary Outcome Measures:
  • Number of active blisters [ Time Frame: 30 days ]
    Reduction in active blisters and in total blister/erosion counts by at least 30%

Secondary Outcome Measures:
  • 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%

Enrollment: 7
Study Start Date: February 2012
Study Completion Date: November 2014
Primary Completion Date: October 2014 (Final data collection date for primary outcome measure)
Arms Assigned Interventions
Experimental: GCSF Drug: Granulocyte Colony Stimulating Factor (GCSF)
G-CSF 10mcg/kg/d SQ for 7 days


Ages Eligible for Study:   Child, Adult, Senior
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.
  Contacts and Locations
Choosing to participate in a study is an important personal decision. Talk with your doctor and family members or friends about deciding to join a study. To learn more about this study, you or your doctor may contact the study research staff using the Contacts provided below. For general information, see Learn About Clinical Studies.

Please refer to this study by its identifier: NCT01538862

United States, Tennessee
Vanderbilt University
Nashville, Tennessee, United States, 37232
Sponsors and Collaborators
Vanderbilt University
Principal Investigator: Haydar Frangoul, MD Vanderbilt University
  More Information

Responsible Party: Haydar Frangoul, Professor of Pediatrics, Vanderbilt University Identifier: NCT01538862     History of Changes
Other Study ID Numbers: VICCNCPED1210
Study First Received: February 20, 2012
Last Updated: December 5, 2014

Additional relevant MeSH terms:
Epidermolysis Bullosa Dystrophica
Epidermolysis Bullosa
Skin Abnormalities
Congenital Abnormalities
Skin Diseases, Genetic
Genetic Diseases, Inborn
Collagen Diseases
Connective Tissue Diseases
Skin Diseases
Skin Diseases, Vesiculobullous
Adjuvants, Immunologic
Immunologic Factors
Physiological Effects of Drugs processed this record on April 24, 2017