A Phase 2 interventional study of Granulocyte Colony Stimulating Factor (GCSF) in Dystrophic Epidermolysis Bullosa, sponsored by Vanderbilt University Medical Center. Completed at 1 site in United States. Per ClinicalTrials.gov, last updated 2017-06-23.
Sponsored by Vanderbilt University Medical Center · Phase 2, Interventional, and Treatment
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.
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.
126 studies on the registry are indexed under Epidermolysis Bullosa; 23 are open to participants now.
This study's enrollment of 7 is below the median of 11 across 95 interventional studies indexed under Epidermolysis Bullosa.
Browse Epidermolysis Bullosa studies →Vanderbilt University Medical Center is the lead sponsor of 824 studies on the registry; 164 are open to participants now.
Of its 122 completed or terminated interventional studies of FDA-regulated products, 91 (75%) have results posted.
Counted across the registry records on this site, refreshed daily.
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:
Exclusion Criteria:
GCSF 10mcg/kg/d subcutaneously (SQ) for 7 days
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
Percent change of active blisters and in total blister/erosion counts from baseline to 7 days
Time frame: 7 days
Surface Area of Nonhealing Erosions
Change in surface area of one or two nonhealing erosions
Time frame: 7 days
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.
Time frame: 28 days
| Milestone | Granulocyte Colony Stimulating Factor (GCSF) |
|---|---|
| Started | 7 |
| Completed | 7 |
| Not completed | 0 |
Percent change of active blisters and in total blister/erosion counts from baseline to 7 days
| percent change | Granulocyte Colony Stimulating Factor (GCSF) |
|---|---|
| Percent Change of Active Blisters and in Total Blister/Erosion Counts | -29.6 ± 30.3 |
Change in surface area of one or two nonhealing erosions
| percentage change | Granulocyte Colony Stimulating Factor (GCSF) |
|---|---|
| Surface Area of Nonhealing Erosions | -35.3 ± 100.8 |
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.
| Participants | Granulocyte Colony Stimulating Factor (GCSF) |
|---|---|
| Overall Improved Symptomatology | 6 |
Collected over 30 days. Non-serious events are listed at a 0% frequency threshold.
| Group | Deaths | Serious | Other |
|---|---|---|---|
| Granulocyte Colony Stimulating Factor (GCSF) | — | 0/7 (0%) | 0/7 (0%) |
| Age, Categorical(Participants) | Granulocyte Colony Stimulating Factor (GCSF) |
|---|---|
| <=18 years | 6 |
| Between 18 and 65 years | 1 |
| >=65 years | 0 |
| Sex: Female, Male(Participants) | Granulocyte Colony Stimulating Factor (GCSF) |
|---|---|
| Female | 4 |
| Male | 3 |
| Region of Enrollment(participants) | Granulocyte Colony Stimulating Factor (GCSF) |
|---|---|
| United States | 7 |
Plan to share: No
No publications or documents are linked to this record.
This study is completed, as verified in May 2017. You cannot join it, but the record below documents what was studied.
Get an email when the registry record changes — status, dates, results — or when someone posts here.
Sign in to followQuestions and observations about this study, from anyone following it. Not medical advice, and not a channel to the study team — their contact details are on the registry record.
Sign in to join the discussion. Reading takes no account; posting does. You choose a display name, and a pseudonym is the default.
Nothing here yet. If you are running this trial, taking part in it, or weighing whether to, this is the place to say so.
Vanderbilt University Medical Center