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



Status:Recruiting
Conditions:Skin and Soft Tissue Infections
Therapuetic Areas:Dermatology / Plastic Surgery
Healthy:No
Age Range:Any
Updated:2/7/2015
Start Date:February 2012
Contact:Haydar Frangoul, MD
Email:haydar.frangoul@vanderbilt.edu
Phone:615-936-6989

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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.


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.
We found this trial at
1
site
2201 West End Ave
Nashville, Tennessee 37232
(615) 322-7311
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