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
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.
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 erosionsOverall symptomatology
Eligibility
Minimum age: N/A.
Maximum age: N/A.
Gender(s): Both.
Criteria:
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.
Locations and Contacts
Vanderbilt University, Nashville, Tennessee 37232, United States
Additional Information
Starting date: February 2012
Last updated: December 5, 2014
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