This site became the new ClinicalTrials.gov on June 19th. Learn more.
Show more
ClinicalTrials.gov Menu IMPORTANT: Listing of a study on this site does not reflect endorsement by the National Institutes of Health. Talk with a trusted healthcare professional before volunteering for a study. Read more...
ClinicalTrials.gov Menu IMPORTANT: Talk with a trusted healthcare professional before volunteering for a study. Read more...
ClinicalTrials.gov Menu
Give us feedback

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

This study has been completed.
Sponsor:
Information provided by (Responsible Party):
Haydar Frangoul, Vanderbilt University Medical Center
ClinicalTrials.gov Identifier:
NCT01538862
First received: February 20, 2012
Last updated: May 30, 2017
Last verified: May 2017
  Purpose
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.

Condition Intervention Phase
Dystrophic Epidermolysis Bullosa Drug: Granulocyte Colony Stimulating Factor (GCSF) Phase 2

Study Type: Interventional
Study Design: Intervention Model: Single Group Assignment
Masking: None (Open Label)
Primary Purpose: Treatment
Official Title: Efficacy of Granulocyte Colony Stimulating Factor (GCSF) In Patients With Dystrophic Epidermolysis Bullosa

Resource links provided by NLM:


Further study details as provided by Haydar Frangoul, Vanderbilt University Medical Center:

Primary Outcome Measures:
  • 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


Secondary Outcome Measures:
  • Surface Area of Nonhealing Erosions [ Time Frame: 7 days ]
    Change in surface area of one or two nonhealing erosions

  • 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.


Enrollment: 7
Study Start Date: February 2012
Study Completion Date: November 2014
Primary Completion Date: October 2014 (Final data collection date for primary outcome measure)
Arms Assigned Interventions
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

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.
  Eligibility

Ages Eligible for Study:   Child, Adult, Senior
Sexes Eligible for Study:   All
Accepts Healthy Volunteers:   No
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.
  Contacts and Locations
Choosing to participate in a study is an important personal decision. Talk with your doctor and family members or friends about deciding to join a study. To learn more about this study, you or your doctor may contact the study research staff using the Contacts provided below. For general information, see Learn About Clinical Studies.

Please refer to this study by its ClinicalTrials.gov identifier: NCT01538862

Locations
United States, Tennessee
Vanderbilt University
Nashville, Tennessee, United States, 37232
Sponsors and Collaborators
Vanderbilt University Medical Center
Investigators
Principal Investigator: Haydar Frangoul, MD Vanderbilt University
  More Information

Responsible Party: Haydar Frangoul, Professor of Pediatrics, Vanderbilt University Medical Center
ClinicalTrials.gov Identifier: NCT01538862     History of Changes
Other Study ID Numbers: VICCNCPED1210
Study First Received: February 20, 2012
Results First Received: April 1, 2016
Last Updated: May 30, 2017
Individual Participant Data (IPD) Sharing Statement:
Plan to Share IPD: No

Additional relevant MeSH terms:
Epidermolysis Bullosa
Epidermolysis Bullosa Dystrophica
Skin Abnormalities
Congenital Abnormalities
Skin Diseases, Genetic
Genetic Diseases, Inborn
Skin Diseases
Skin Diseases, Vesiculobullous
Collagen Diseases
Connective Tissue Diseases
Lenograstim
Adjuvants, Immunologic
Immunologic Factors
Physiological Effects of Drugs

ClinicalTrials.gov processed this record on August 22, 2017