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

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ClinicalTrials.gov Identifier: NCT01538862
Recruitment Status : Completed
First Posted : February 24, 2012
Results First Posted : April 25, 2017
Last Update Posted : June 23, 2017
Sponsor:
Information provided by (Responsible Party):
Haydar Frangoul, Vanderbilt University Medical Center

Tracking Information
First Submitted Date  ICMJE February 20, 2012
First Posted Date  ICMJE February 24, 2012
Results First Submitted Date  ICMJE April 1, 2016
Results First Posted Date  ICMJE April 25, 2017
Last Update Posted Date June 23, 2017
Study Start Date  ICMJE February 2012
Actual Primary Completion Date October 2014   (Final data collection date for primary outcome measure)
Current Primary Outcome Measures  ICMJE
 (submitted: March 13, 2017)
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
Original Primary Outcome Measures  ICMJE
 (submitted: February 23, 2012)
Number of active blisters [ Time Frame: 30 days ]
Reduction in active blisters and in total blister/erosion counts by at least 30%
Change History
Current Secondary Outcome Measures  ICMJE
 (submitted: May 30, 2017)
  • 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.
Original Secondary Outcome Measures  ICMJE
 (submitted: February 23, 2012)
  • Surface Area of Nonhealing Erosions [ Time Frame: 30 days ]
    Reduction in surface area of one or two nonhealing erosions by at least 20%
  • Overall symptomatology [ Time Frame: 30 days ]
    Overall clinical improvement in symptomatology and/or findings, as assessed by either the patient or parent, of at least 30%
Current Other Pre-specified Outcome Measures Not Provided
Original Other Pre-specified Outcome Measures Not Provided
 
Descriptive Information
Brief Title  ICMJE Efficacy of Granulocyte Colony Stimulating Factor (GCSF) In Patients With Dystrophic Epidermolysis Bullosa
Official Title  ICMJE Efficacy of Granulocyte Colony Stimulating Factor (GCSF) In Patients With Dystrophic Epidermolysis Bullosa
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.
Study Type  ICMJE Interventional
Study Phase  ICMJE Phase 2
Study Design  ICMJE Allocation: N/A
Intervention Model: Single Group Assignment
Masking: None (Open Label)
Primary Purpose: Treatment
Condition  ICMJE Dystrophic Epidermolysis Bullosa
Intervention  ICMJE Drug: Granulocyte Colony Stimulating Factor (GCSF)
G-CSF 10mcg/kg/d SQ for 7 days
Study Arms  ICMJE Experimental: Granulocyte Colony Stimulating Factor (GCSF)
GCSF 10mcg/kg/d subcutaneously (SQ) for 7 days
Intervention: Drug: Granulocyte Colony Stimulating Factor (GCSF)
Publications * Not Provided

*   Includes publications given by the data provider as well as publications identified by ClinicalTrials.gov Identifier (NCT Number) in Medline.
 
Recruitment Information
Recruitment Status  ICMJE Completed
Actual Enrollment  ICMJE
 (submitted: December 5, 2014)
7
Original Estimated Enrollment  ICMJE
 (submitted: February 23, 2012)
20
Actual Study Completion Date  ICMJE November 2014
Actual Primary Completion Date October 2014   (Final data collection date for primary outcome measure)
Eligibility Criteria  ICMJE

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.
Sex/Gender  ICMJE
Sexes Eligible for Study: All
Ages  ICMJE Child, Adult, Older Adult
Accepts Healthy Volunteers  ICMJE No
Contacts  ICMJE Contact information is only displayed when the study is recruiting subjects
Listed Location Countries  ICMJE United States
Removed Location Countries  
 
Administrative Information
NCT Number  ICMJE NCT01538862
Other Study ID Numbers  ICMJE VICCNCPED1210
Has Data Monitoring Committee No
U.S. FDA-regulated Product Not Provided
IPD Sharing Statement  ICMJE
Plan to Share IPD: No
Responsible Party Haydar Frangoul, Vanderbilt University Medical Center
Study Sponsor  ICMJE Vanderbilt University Medical Center
Collaborators  ICMJE Not Provided
Investigators  ICMJE
Principal Investigator: Haydar Frangoul, MD Vanderbilt University
PRS Account Vanderbilt University Medical Center
Verification Date May 2017

ICMJE     Data element required by the International Committee of Medical Journal Editors and the World Health Organization ICTRP