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
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Condition or disease | Intervention/treatment | Phase |
---|---|---|
Dystrophic Epidermolysis Bullosa | Drug: Granulocyte Colony Stimulating Factor (GCSF) | Phase 2 |
Study Type : | Interventional (Clinical Trial) |
Actual Enrollment : | 7 participants |
Allocation: | N/A |
Intervention Model: | Single Group Assignment |
Masking: | None (Open Label) |
Primary Purpose: | Treatment |
Official Title: | Efficacy of Granulocyte Colony Stimulating Factor (GCSF) In Patients With Dystrophic Epidermolysis Bullosa |
Study Start Date : | February 2012 |
Actual Primary Completion Date : | October 2014 |
Actual Study Completion Date : | November 2014 |

Arm | Intervention/treatment |
---|---|
Experimental: Granulocyte Colony Stimulating Factor (GCSF)
GCSF 10mcg/kg/d subcutaneously (SQ) for 7 days
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Drug: Granulocyte Colony Stimulating Factor (GCSF)
G-CSF 10mcg/kg/d SQ for 7 days |
- 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
- 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.

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Ages Eligible for Study: | Child, Adult, Older Adult |
Sexes Eligible for Study: | All |
Accepts Healthy Volunteers: | No |
Inclusion Criteria:
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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.

To learn more about this study, you or your doctor may contact the study research staff using the contact information provided by the sponsor.
Please refer to this study by its ClinicalTrials.gov identifier (NCT number): NCT01538862
United States, Tennessee | |
Vanderbilt University | |
Nashville, Tennessee, United States, 37232 |
Principal Investigator: | Haydar Frangoul, MD | Vanderbilt University |
Responsible Party: | Haydar Frangoul, Professor of Pediatrics, Vanderbilt University Medical Center |
ClinicalTrials.gov Identifier: | NCT01538862 |
Other Study ID Numbers: |
VICCNCPED1210 |
First Posted: | February 24, 2012 Key Record Dates |
Results First Posted: | April 25, 2017 |
Last Update Posted: | June 23, 2017 |
Last Verified: | May 2017 |
Individual Participant Data (IPD) Sharing Statement: | |
Plan to Share IPD: | No |
Epidermolysis Bullosa Epidermolysis Bullosa Dystrophica Skin Abnormalities Congenital Abnormalities Skin Diseases, Genetic Genetic Diseases, Inborn Skin Diseases Skin Diseases, Vesiculobullous |
Collagen Diseases Connective Tissue Diseases Sargramostim Lenograstim Immunologic Factors Physiological Effects of Drugs Adjuvants, Immunologic |