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

Dystrophic Epidermolysis Bullosa Clinical Trial

Official title:

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

Clinical Trial Details — Status: Completed

Administrative data

• NCT number: NCT01538862
• Other study ID #: VICCNCPED1210
• Status: Completed
• Phase: N/A
• First received: February 20, 2012
• Last updated: December 5, 2014
• Start date: February 2012
• Est. completion date: November 2014

Study information

• Verified date: December 2014
• Source: Vanderbilt University
• Contact: n/a
• Is FDA regulated: No
• Health authority: United States: Institutional Review Board
• Study type: Interventional

Clinical Trial 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.

Recruitment information / eligibility

• Status: Completed
• Enrollment: 7
• Est. completion date: November 2014
• Est. primary completion date: October 2014
• Accepts healthy volunteers: No
• Gender: Both
• Age group: N/A and older

Eligibility

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.

Study Design

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

Related Conditions & MeSH terms

• Dystrophic Epidermolysis Bullosa
• Epidermolysis Bullosa
• Epidermolysis Bullosa Dystrophica

Intervention

Drug:
• Granulocyte Colony Stimulating Factor (GCSF)
G-CSF 10mcg/kg/d SQ for 7 days

Locations

Country	Name	City	State
United States	Vanderbilt University	Nashville	Tennessee

Sponsors (1)

Lead Sponsor	Collaborator
Vanderbilt University

Country where clinical trial is conducted

United States, 

Outcome

Type	Measure	Description	Time frame	Safety issue
Primary	Number of active blisters	Reduction in active blisters and in total blister/erosion counts by at least 30%	30 days	No
Secondary	Surface area of nonhealing erosions	Reduction in surface area of one or two nonhealing erosions by at least 20%	30 days	No
Secondary	Overall symptomatology	Overall clinical improvement in symptomatology and/or findings, as assessed by either the patient or parent, of at least 30%	30 days	No