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

• Status: Completed
• Trial end date: 2014-11-01
• Target enrollment: 0
• Participant gender: All

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.

• Phase: Phase 2
• Accepts Healthy Volunteers?: No

Details

• Lead Sponsor: Vanderbilt University; Vanderbilt University Medical Center
• Treatments: Lenograstim; Sargramostim

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.