Phase 2
N=7
Efficacy of Granulocyte Colony Stimulating Factor (GCSF) In Patients With Dystrophic Epidermolysis Bullosa
Dystrophic Epidermolysis Bullosa
Bottom Line
View on ClinicalTrials.gov: NCT01538862 ↗Enrolled (actual)
7
Serious AEs
0.0%
Results posted
Apr 2017
Primary outcome: Primary: Percent Change of Active Blisters and in Total Blister/Erosion Counts — -29.6 percent change — p=0.82
Study Design & Population
- Study type
- Interventional
- Phase
- Phase 2
- Interventions
- Granulocyte Colony Stimulating Factor (GCSF) (Drug)
- Age
- Pediatric, Adult, Older Adult
- Sex
- All
- Sponsor
- Vanderbilt University Medical Center
- Primary completion
- Oct 2014
Outcome Measures
| Outcome | Result | p-value |
|---|---|---|
| PRIMARY Percent Change of Active Blisters and in Total Blister/Erosion Counts |
-29.6 | 0.82 |
| SECONDARY Surface Area of Nonhealing Erosions |
-35.3 | — |
| SECONDARY Overall Improved Symptomatology |
6 | — |
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.
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.
Data sourced from ClinicalTrials.gov (NCT01538862). Outcome figures and adverse-event rates are extracted automatically from the registry's posted results and are provided for clinician reference, not as a substitute for the primary publication.