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NCT Number: NCT01538862

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

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.

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Key information

Conditions

Dystrophic Epidermolysis Bullosa Collagen Diseases Congenital Abnormalities Congenital, Hereditary, and Neonatal Diseases and Abnormalities Connective Tissue Diseases Epidermolysis Bullosa Epidermolysis Bullosa Dystrophica Genetic Diseases, Inborn Skin Abnormalities Skin Diseases Skin Diseases, Genetic Skin Diseases, Vesiculobullous Skin and Connective Tissue Diseases

Sex eligibility

All sexes

Study type

Interventional

Phase

Phase 2

Primary location

Vanderbilt University

Nashville, Tennessee, 37232, United States

About this study

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.

Who can participate

Healthy volunteers accepted: No

Only the study team can determine whether someone qualifies for participation.

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:
  • transmission electron microscopy
  • immunofluorescence antigenic mapping and type VII collagen monoclonal antibody staining
  • 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.

Treatment and study plan

granulocyte colony stimulating factor (GCSF)

Drug

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

Primary outcomes

  1. 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 outcomes

  1. Surface Area of Nonhealing Erosions

    Time frame: 7 days

    Change in surface area of one or two nonhealing erosions

  2. 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.

Sponsors and collaborators

Lead sponsor

Vanderbilt University Medical Center

Other

Registry information

Important dates

Study start
2012
Primary completion
2014
Study completion
2014
First posted
Feb 24, 2012
Registry last updated
Jun 23, 2017

OpenTrials presents study information sourced from ClinicalTrials.gov. The official registry record should be consulted for the latest information.

View the official ClinicalTrials.gov record (opens in a new tab)

This listing is for discovery and informational purposes only. It is not medical advice, does not guarantee that a study is recruiting, and does not determine eligibility. Contact the study team and a qualified healthcare professional when considering participation.

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