NCT01538862

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

• Status: Completed
• Phase: Phase 2
• Study type: Interventional
• Enrollment: 7 (actual)
• Sponsor: Vanderbilt University Medical Center · Academic / Other
• Sex: All
• Age: —
• Healthy volunteers: Not accepted

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.

Detailed description

Each patient will be given 10 micrograms per kilogram per day of G-CSF subcutaneously for 6 consecutive days. On day 7 each patient will be seen and evaluated in the same manner as on day 0. Patients or their parents (if children are too young to reliably respond themselves) will also be asked to rate the following via a visual analog scale of 1-9- oral pain, pruritus, oral pain, swallowing, and overall sense of well-being. A telephone follow-up will be conducted on all patients 28 days after G-CSF so as to evaluate if the effect noted on day 7 was sustained.

Conditions

• Dystrophic Epidermolysis Bullosa

Interventions

Type	Name	Description
DRUG	Granulocyte Colony Stimulating Factor (GCSF)	G-CSF 10mcg/kg/d SQ for 7 days

Timeline

• Start date: 2012-02-01
• Primary completion: 2014-10-01
• Completion: 2014-11-01
• First posted: 2012-02-24
• Last updated: 2017-06-23
• Results posted: 2017-04-25

Locations

1 site across 1 country: United States