DrugLib.com — Drug Information Portal

Rx drug information, pharmaceutical research, clinical trials, news, and more

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

Information source: Vanderbilt University
ClinicalTrials.gov processed this data on August 23, 2015
Link to the current ClinicalTrials.gov record.

Condition(s) targeted: Dystrophic Epidermolysis Bullosa

Intervention: Granulocyte Colony Stimulating Factor (GCSF) (Drug)

Phase: N/A

Status: Completed

Sponsored by: Vanderbilt University

Official(s) and/or principal investigator(s):
Haydar Frangoul, MD, Principal Investigator, Affiliation: Vanderbilt University


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.

Clinical Details

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

Study design: Endpoint Classification: Efficacy Study, Intervention Model: Single Group Assignment, Masking: Open Label, Primary Purpose: Treatment

Primary outcome: Number of active blisters

Secondary outcome:

Surface area of nonhealing erosions

Overall symptomatology


Minimum age: N/A. Maximum age: N/A. Gender(s): Both.


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


- Female patients who are pregnant.

- Patients with active signs and symptoms of infection.

Locations and Contacts

Vanderbilt University, Nashville, Tennessee 37232, United States
Additional Information

Starting date: February 2012
Last updated: December 5, 2014

Page last updated: August 23, 2015

-- advertisement -- The American Red Cross
Home | About Us | Contact Us | Site usage policy | Privacy policy

All Rights reserved - Copyright DrugLib.com, 2006-2017