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

Primary Purpose

Dystrophic Epidermolysis Bullosa

Status
Completed
Phase
Phase 2
Locations
United States
Study Type
Interventional
Intervention
Granulocyte Colony Stimulating Factor (GCSF)
Sponsored by
Vanderbilt University Medical Center
About
Eligibility
Locations
Arms
Outcomes
Full info

About this trial

This is an interventional treatment trial for Dystrophic Epidermolysis Bullosa

Eligibility Criteria

undefined - undefined (Child, Adult, Older Adult)All SexesDoes not accept healthy volunteers

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.

Sites / Locations

  • Vanderbilt University

Arms of the Study

Arm 1

Arm Type

Experimental

Arm Label

Granulocyte Colony Stimulating Factor (GCSF)

Arm Description

GCSF 10mcg/kg/d subcutaneously (SQ) for 7 days

Outcomes

Primary Outcome Measures

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

Secondary Outcome Measures

Surface Area of Nonhealing Erosions
Change in surface area of one or two nonhealing erosions
Overall Improved Symptomatology
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.

Full Information

First Posted
February 20, 2012
Last Updated
May 30, 2017
Sponsor
Vanderbilt University Medical Center
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1. Study Identification

Unique Protocol Identification Number
NCT01538862
Brief Title
Efficacy of Granulocyte Colony Stimulating Factor (GCSF) In Patients With Dystrophic Epidermolysis Bullosa
Official Title
Efficacy of Granulocyte Colony Stimulating Factor (GCSF) In Patients With Dystrophic Epidermolysis Bullosa
Study Type
Interventional

2. Study Status

Record Verification Date
May 2017
Overall Recruitment Status
Completed
Study Start Date
February 2012 (undefined)
Primary Completion Date
October 2014 (Actual)
Study Completion Date
November 2014 (Actual)

3. Sponsor/Collaborators

Responsible Party, by Official Title
Principal Investigator
Name of the Sponsor
Vanderbilt University Medical Center

4. Oversight

Data Monitoring Committee
No

5. Study Description

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.

6. Conditions and Keywords

Primary Disease or Condition Being Studied in the Trial, or the Focus of the Study
Dystrophic Epidermolysis Bullosa

7. Study Design

Primary Purpose
Treatment
Study Phase
Phase 2
Interventional Study Model
Single Group Assignment
Masking
None (Open Label)
Allocation
N/A
Enrollment
7 (Actual)

8. Arms, Groups, and Interventions

Arm Title
Granulocyte Colony Stimulating Factor (GCSF)
Arm Type
Experimental
Arm Description
GCSF 10mcg/kg/d subcutaneously (SQ) for 7 days
Intervention Type
Drug
Intervention Name(s)
Granulocyte Colony Stimulating Factor (GCSF)
Intervention Description
G-CSF 10mcg/kg/d SQ for 7 days
Primary Outcome Measure Information:
Title
Percent Change of Active Blisters and in Total Blister/Erosion Counts
Description
Percent change of active blisters and in total blister/erosion counts from baseline to 7 days
Time Frame
7 days
Secondary Outcome Measure Information:
Title
Surface Area of Nonhealing Erosions
Description
Change in surface area of one or two nonhealing erosions
Time Frame
7 days
Title
Overall Improved Symptomatology
Description
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.
Time Frame
28 days

10. Eligibility

Sex
All
Accepts Healthy Volunteers
No
Eligibility 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: 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.
Overall Study Officials:
First Name & Middle Initial & Last Name & Degree
Haydar Frangoul, MD
Organizational Affiliation
Vanderbilt University
Official's Role
Principal Investigator
Facility Information:
Facility Name
Vanderbilt University
City
Nashville
State/Province
Tennessee
ZIP/Postal Code
37232
Country
United States

12. IPD Sharing Statement

Plan to Share IPD
No

Learn more about this trial

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

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