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
ClinicalTrials.gov 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: Endpoint Classification: Efficacy Study
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 ] [ Designated as safety issue: No ]
    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 ] [ Designated as safety issue: No ]
    Reduction in surface area of one or two nonhealing erosions by at least 20%

  • Overall symptomatology [ Time Frame: 30 days ] [ Designated as safety issue: No ]
    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


Genders Eligible for Study:   Both
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 ClinicalTrials.gov 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
ClinicalTrials.gov Identifier: NCT01538862     History of Changes
Other Study ID Numbers: VICCNCPED1210 
Study First Received: February 20, 2012
Last Updated: December 5, 2014
Health Authority: United States: Institutional Review Board

Additional relevant MeSH terms:
Epidermolysis Bullosa
Epidermolysis Bullosa Dystrophica
Collagen Diseases
Congenital Abnormalities
Connective Tissue Diseases
Genetic Diseases, Inborn
Skin Abnormalities
Skin Diseases
Skin Diseases, Genetic
Skin Diseases, Vesiculobullous
Adjuvants, Immunologic
Immunologic Factors
Physiological Effects of Drugs

ClinicalTrials.gov processed this record on May 23, 2016