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

May 30, 2017 updated by: Haydar Frangoul, Vanderbilt University Medical Center
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.

Study Overview

Status

Completed

Conditions

Intervention / Treatment

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.

Study Type

Interventional

Enrollment (Actual)

7

Phase

  • Phase 2

Contacts and Locations

This section provides the contact details for those conducting the study, and information on where this study is being conducted.

Study Locations

  • United States
    • Tennessee
      • Nashville, Tennessee, United States, 37232
        • Vanderbilt University

Participation Criteria

Researchers look for people who fit a certain description, called eligibility criteria. Some examples of these criteria are a person's general health condition or prior treatments.

Eligibility Criteria

Ages Eligible for Study

  • ADULT
  • OLDER_ADULT
  • CHILD

Accepts Healthy Volunteers

No

Genders Eligible for Study

All

Description

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 Plan

This section provides details of the study plan, including how the study is designed and what the study is measuring.

How is the study designed?

Design Details

  • Primary Purpose: TREATMENT
  • Allocation: NA
  • Interventional Model: SINGLE_GROUP
  • Masking: NONE

Arms and Interventions

Participant Group / Arm
Intervention / Treatment
EXPERIMENTAL: Granulocyte Colony Stimulating Factor (GCSF)
GCSF 10mcg/kg/d subcutaneously (SQ) for 7 days
Drug: Granulocyte Colony Stimulating Factor (GCSF)
G-CSF 10mcg/kg/d SQ for 7 days

What is the study measuring?

Primary Outcome Measures

Outcome Measure
Measure Description
Time Frame
Percent Change of Active Blisters and in Total Blister/Erosion Counts
Time Frame: 7 days
Percent change of active blisters and in total blister/erosion counts from baseline to 7 days
7 days

Secondary Outcome Measures

Outcome Measure
Measure Description
Time Frame
Surface Area of Nonhealing Erosions
Time Frame: 7 days
Change in surface area of one or two nonhealing erosions
7 days
Overall Improved Symptomatology
Time Frame: 28 days
Overall clinical improvement in symptomatology and/or findings, as assessed by either the patient or parent. This would include decrease in the number and size of blister and erosions, decreased pain, improved comfort of the patient.
28 days

Collaborators and Investigators

This is where you will find people and organizations involved with this study.

Sponsor

Vanderbilt University Medical Center

Investigators

  • Principal Investigator: Haydar Frangoul, MD, Vanderbilt University

Study record dates

These dates track the progress of study record and summary results submissions to ClinicalTrials.gov. Study records and reported results are reviewed by the National Library of Medicine (NLM) to make sure they meet specific quality control standards before being posted on the public website.

Study Major Dates

Study Start

February 1, 2012

Primary Completion (ACTUAL)

October 1, 2014

Study Completion (ACTUAL)

November 1, 2014

Study Registration Dates

First Submitted

February 20, 2012

First Submitted That Met QC Criteria

February 23, 2012

First Posted (ESTIMATE)

February 24, 2012

Study Record Updates

Last Update Posted (ACTUAL)

June 23, 2017

Last Update Submitted That Met QC Criteria

May 30, 2017

Last Verified

May 1, 2017

More Information

Terms related to this study

Additional Relevant MeSH Terms

Other Study ID Numbers

  • VICCNCPED1210

Plan for Individual participant data (IPD)

Plan to Share Individual Participant Data (IPD)?

NO

This information was retrieved directly from the website clinicaltrials.gov without any changes. If you have any requests to change, remove or update your study details, please contact register@clinicaltrials.gov. As soon as a change is implemented on clinicaltrials.gov, this will be updated automatically on our website as well.

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