Trial record 4 of 15 for:    "Epidermolysis Bullosa Dystrophica" OR "dystrophic epidermolysis bullosa"

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

This study is currently recruiting participants.
Verified July 2013 by Vanderbilt University
Sponsor:
Information provided by (Responsible Party):
Haydar Frangoul, Vanderbilt University
ClinicalTrials.gov Identifier:
NCT01538862
First received: February 20, 2012
Last updated: July 19, 2013
Last verified: July 2013
  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
Dystrophic Epidermolysis Bullosa
Drug: Granulocyte Colony Stimulating Factor (GCSF)

Study Type: Interventional
Study Design: Endpoint Classification: Efficacy Study
Intervention Model: Single Group Assignment
Masking: 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 Vanderbilt University:

Primary Outcome Measures:
  • Number of active blisters [ Time Frame: 30 days ] [ Designated as safety issue: No ]
    Reduction in active blisters and in total blister/erosion counts by at least 30%


Secondary Outcome Measures:
  • Surface area of nonhealing erosions [ Time Frame: 30 days ] [ Designated as safety issue: No ]
    Reduction in surface area of one or two nonhealing erosions by at least 20%

  • Overall symptomatology [ Time Frame: 30 days ] [ Designated as safety issue: No ]
    Overall clinical improvement in symptomatology and/or findings, as assessed by either the patient or parent, of at least 30%


Estimated Enrollment: 20
Study Start Date: February 2012
Estimated Primary Completion Date: March 2014 (Final data collection date for primary outcome measure)
Arms Assigned Interventions
Experimental: GCSF Drug: Granulocyte Colony Stimulating Factor (GCSF)
G-CSF 10mcg/kg/d SQ for 7 days

  Eligibility

Genders Eligible for Study:   Both
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
Please refer to this study by its ClinicalTrials.gov identifier: NCT01538862

Contacts
Contact: Haydar Frangoul, MD 615-936-6989 haydar.frangoul@vanderbilt.edu

Locations
United States, Tennessee
Vanderbilt University Recruiting
Nashville, Tennessee, United States, 37232
Contact: Haydar Frangoul, MD    615-936-6989    haydar.frangoul@vanderbilt.edu   
Principal Investigator: Haydar Frangoul, MD         
Sponsors and Collaborators
Vanderbilt University
Investigators
Principal Investigator: Haydar Frangoul, MD Vanderbilt University
  More Information

No publications provided

Responsible Party: Haydar Frangoul, Professor of Pediatrics, Vanderbilt University
ClinicalTrials.gov Identifier: NCT01538862     History of Changes
Other Study ID Numbers: VICCNCPED1210
Study First Received: February 20, 2012
Last Updated: July 19, 2013
Health Authority: United States: Institutional Review Board

Additional relevant MeSH terms:
Epidermolysis Bullosa Dystrophica
Epidermolysis Bullosa
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
Pharmacologic Actions

ClinicalTrials.gov processed this record on April 20, 2014