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

NCT ID: NCT01538862

Last Updated: 2017-06-23

Basic Information

• Recruitment Status: COMPLETED
• Clinical Phase: PHASE2
• Total Enrollment: 7 participants
• Study Classification: INTERVENTIONAL
• Study Start Date: 2012-02-29
• Study Completion Date: 2014-11-30

Brief 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

Study Design

• Allocation Method: NA
• Intervention Model: SINGLE_GROUP
• Primary Study Purpose: TREATMENT
• Blinding Strategy: NONE

Study Groups

Granulocyte Colony Stimulating Factor (GCSF)

GCSF 10mcg/kg/d subcutaneously (SQ) for 7 days

Group Type: EXPERIMENTAL

Granulocyte Colony Stimulating Factor (GCSF)

Intervention Type: DRUG

G-CSF 10mcg/kg/d SQ for 7 days

Interventions

Granulocyte Colony Stimulating Factor (GCSF)

G-CSF 10mcg/kg/d SQ for 7 days

Intervention Type: DRUG

Eligibility 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.

• Eligible Sex: ALL
• Accepts Healthy Volunteers: No

Sponsors

Vanderbilt University Medical Center

OTHER

Sponsor Role: lead

Responsible Party

Haydar Frangoul

Professor of Pediatrics

Responsibility Role: PRINCIPAL_INVESTIGATOR

Principal Investigators

Haydar Frangoul, MD

Role: PRINCIPAL_INVESTIGATOR

Vanderbilt University

Locations

Vanderbilt University

Nashville, Tennessee, United States

Countries

United States

Other Identifiers

VICCNCPED1210

Identifier Type: -

Identifier Source: org_study_id