Efficacy of Granulocyte Colony Stimulating Factor (GCSF) In Patients With Dystrophic Epidermolysis Bullosa
1 other identifier
interventional
7
1 country
1
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.
Trial Health
Trial Health Score
Automated assessment based on enrollment pace, timeline, and geographic reach
participants targeted
Target at below P25 for phase_2
Started Feb 2012
1 active site
Health score is calculated from publicly available data and should be used for screening purposes only.
Trial Relationships
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Study Timeline
Key milestones and dates
Study Start
First participant enrolled
February 1, 2012
CompletedFirst Submitted
Initial submission to the registry
February 20, 2012
CompletedFirst Posted
Study publicly available on registry
February 24, 2012
CompletedPrimary Completion
Last participant's last visit for primary outcome
October 1, 2014
CompletedStudy Completion
Last participant's last visit for all outcomes
November 1, 2014
CompletedResults Posted
Study results publicly available
April 25, 2017
CompletedJune 23, 2017
May 1, 2017
2.7 years
February 20, 2012
April 1, 2016
May 30, 2017
Conditions
Outcome Measures
Primary Outcomes (1)
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
7 days
Secondary Outcomes (2)
Surface Area of Nonhealing Erosions
7 days
Overall Improved Symptomatology
28 days
Study Arms (1)
Granulocyte Colony Stimulating Factor (GCSF)
EXPERIMENTALGCSF 10mcg/kg/d subcutaneously (SQ) for 7 days
Interventions
G-CSF 10mcg/kg/d SQ for 7 days
Eligibility Criteria
You may qualify if:
- 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
You may not qualify if:
- 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.
Contact the study team to confirm eligibility.
Sponsors & Collaborators
Study Sites (1)
Vanderbilt University
Nashville, Tennessee, 37232, United States
MeSH Terms
Conditions
Interventions
Condition Hierarchy (Ancestors)
Intervention Hierarchy (Ancestors)
Results Point of Contact
- Title
- Haydar Frangoul, MD, MS
- Organization
- Vanderbilt University
Study Officials
- PRINCIPAL INVESTIGATOR
Haydar Frangoul, MD
Vanderbilt University
Publication Agreements
- PI is Sponsor Employee
- Yes
Study Design
- Study Type
- interventional
- Phase
- phase 2
- Allocation
- NA
- Masking
- NONE
- Purpose
- TREATMENT
- Intervention Model
- SINGLE GROUP
- Sponsor Type
- OTHER
- Responsible Party
- PRINCIPAL INVESTIGATOR
- PI Title
- Professor of Pediatrics
Study Record Dates
First Submitted
February 20, 2012
First Posted
February 24, 2012
Study Start
February 1, 2012
Primary Completion
October 1, 2014
Study Completion
November 1, 2014
Last Updated
June 23, 2017
Results First Posted
April 25, 2017
Record last verified: 2017-05
Data Sharing
- IPD Sharing
- Will not share