Dystrophic Epidermolysis BullosaPhase 2NCT01538862
Efficacy of Granulocyte Colony Stimulating Factor (GCSF) In Patients With Dystrophic Epidermolysis Bullosa
A Phase 2 study testing Granulocyte Colony Stimulating Factor (GCSF) in people with Dystrophic Epidermolysis Bullosa, run by Vanderbilt University Medical Center at 1 site in United States.
Trial 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. Read more about this study
- Who is it for?
- Any age
- Conditions the study is for
- Dystrophic Epidermolysis Bullosa
- What is being tested?
- Granulocyte Colony Stimulating Factor (GCSF)
- Type of treatment
- Injection or infusion
- Could I receive a placebo?
- No, everyone receives an active treatment
- How many people
- 7 participants
- Where
- 1 siteUnited States
- Study dates
- Feb 2012to Nov 2014
- Phase
- Phase 2
- Are there results?
- Yes, posted in the registrySummary in the doctors' view
Key requirements
Who can take part, as listed by the sponsor. Only the study team can confirm whether you are eligible, so take this list to your doctor.
- Ages Any age
- All sexes
- No healthy volunteers
- Child, Adult, Older adult
Who may be able to join
- 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
Who cannot join
- 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.
Trial locations and contacts
1 site in 1 country.
About this study
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.
Read the 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.
- Conditions
- Dystrophic Epidermolysis Bullosa
- Being tested
- Granulocyte Colony Stimulating Factor (GCSF)
- Medical subject headings
- Epidermolysis Bullosa Dystrophica
What participants receive
What participants receive. In a randomised study each person is assigned to one group by chance.
Granulocyte Colony Stimulating Factor (GCSF)Drug
G-CSF 10mcg/kg/d SQ for 7 days
| Study group | Role | What they receive |
|---|---|---|
| Granulocyte Colony Stimulating Factor (GCSF) | Experimental | GCSF 10mcg/kg/d subcutaneously (SQ) for 7 days Receives: Drug: Granulocyte Colony Stimulating Factor (GCSF) |
What is being measured
The primary outcome is the main question the study is designed to answer.
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
Measured: 7 days
Secondary outcomes (2)
Surface Area of Nonhealing Erosions
Change in surface area of one or two nonhealing erosions
Measured: 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.
Measured: 28 days
Timeline
From the start of the study to its completion; the mark is today. Estimated dates are the sponsor’s plan.
- First posted24 Feb 2012 (estimated)
- Study startFeb 2012
- Primary completionOct 2014
- Study completionNov 2014
Resources
- Official record on ClinicalTrials.govThe registry entry this page is built from, with its full change history.
- Posted resultsParticipant flow, outcome measures and adverse events reported by the sponsor.
- Other trials for Dystrophic Epidermolysis BullosaStudies of the same condition, open and under way.
- More trials run by Vanderbilt University Medical CenterThe sponsor's other registered studies.
- What the trial phases meanHow a treatment moves from first tests in people to approval.
More details
For doctors and researchers: how the study is designed, who runs it, and its registry IDs.
Study design
- Study type
- Interventional
- Phase
- Phase 2
- Primary purpose
- Treatment
- Allocation
- Not applicable
- Intervention model
- Single group
- Masking
- None (open label)
- Enrolment
- 7 participants (actual)
Sponsor and investigators
- Lead sponsor
- Vanderbilt University Medical Center
- Sponsor type
- University, hospital or other
- Responsible party
- Principal investigator, Haydar Frangoul, Vanderbilt University Medical Center
- Investigators
- Haydar Frangoul, MD, Principal investigator, Vanderbilt University
IDs, dates and publications
- ClinicalTrials.gov ID
- NCT01538862
- Sponsor's protocol ID
- VICCNCPED1210
- Status last verified
- May 2017
- First submitted
- 20 Feb 2012
- Last updated
- 23 Jun 2017
- Results posted
- Yes, on ClinicalTrials.gov
- Data monitoring committee
- No
- Individual data sharing
- No
Study at a glance
From the registry record, last updated by the sponsor on 23 Jun 2017. Recruitment status is as the sponsor reported it: confirm with the study team.
- Official title
- Efficacy of Granulocyte Colony Stimulating Factor (GCSF) In Patients With Dystrophic Epidermolysis Bullosa
- ClinicalTrials.gov ID
- NCT01538862
- Acronym / sponsor ID
- VICCNCPED1210
- Conditions
- Dystrophic Epidermolysis Bullosa
- Phase
- Phase 2
- Study type
- Interventional
- Recruitment status
- Completed
- Enrolment
- 7 participants (actual)
- Lead sponsor
- Vanderbilt University Medical CenterUniversity, hospital or other
- Study start
- Feb 2012
- Primary completion
- Oct 2014 (actual)
- Study completion
- Nov 2014 (actual)
- First posted
- 24 Feb 2012 (estimated)
- Results
- Posted 25 Apr 2017
Clinical summary
The research question as the sponsor registered it. This describes what the study plans to test, not what it found.
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.
Study design
- Primary purpose
- Treatment
- Allocation
- Not applicable
- Intervention model
- Single group
- Masking
- None (open label)
- Number of arms
- 1
| Arm | Type | Receives |
|---|---|---|
| Granulocyte Colony Stimulating Factor (GCSF) | Experimental | Drug: Granulocyte Colony Stimulating Factor (GCSF)GCSF 10mcg/kg/d subcutaneously (SQ) for 7 days |
Who may qualify
- Sex
- All
- Healthy volunteers
- Not accepted
- Age groups
- Child, Adult, Older adult
Key inclusion criteria (4 listed)
- 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:
Key exclusion criteria (3 listed)
- 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.
Eligibility is decided by the study team after screening; this overview does not establish that a patient qualifies. Full inclusion and exclusion criteria.
Interventions
| Intervention | Type | Arms |
|---|---|---|
| Granulocyte Colony Stimulating Factor (GCSF)G-CSF 10mcg/kg/d SQ for 7 days | Drug | Granulocyte Colony Stimulating Factor (GCSF) |
Primary outcome
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
2 secondary outcomes
Surface Area of Nonhealing Erosions
Time frame: 7 days
Overall Improved Symptomatology
Time frame: 28 days
Results and safety
Loading the posted results from ClinicalTrials.gov…
Research links
- Official record on ClinicalTrials.govLast updated by the sponsor 23 Jun 2017.
- Posted resultsParticipant flow, baseline, all outcome measures and adverse events.
- Individual participant dataSharing planned: No
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Referral and contact
AzTrials does not enrol participants or take referrals. Contact the study team or a site listed below; they decide who can take part. This study is not recruiting at the moment.
Haydar Frangoul, MD principal investigator
Vanderbilt University
1 site in 1 country: see every site and its status.
Questions about this trial
What is this clinical trial studying?
A Phase 2 study testing Granulocyte Colony Stimulating Factor (GCSF) in people with Dystrophic Epidermolysis Bullosa, run by Vanderbilt University Medical Center at 1 site in United States.
The full registered description is in the About this study section and the official record.
Who may be eligible to take part?
The sponsor lists 4 inclusion and 3 exclusion criteria. Eligibility is decided by the study team after screening, so read the full criteria and contact a listed site.
Is this clinical trial currently recruiting?
The record says: finished, as of the sponsor's last update on 23 Jun 2017. Status can change between updates, so confirm with the study contact before making plans.
Where is the study taking place?
The record lists 1 site in United States. Whether a site is taking part right now should be confirmed with that site. See every site.
What are the possible benefits and risks?
They depend on the study and the treatment, and no study can promise a benefit. The study team explains the known risks during informed consent, before anyone joins; the record itself does not list them.
What does this phase mean?
Phase 2 studies give the treatment to a larger group to see whether it has the intended effect and to keep checking safety.
Not every study follows the same path, and some studies (observational studies, device studies) have no phase.
Are results available for this study?
Yes. Results are posted in the registry (first posted 25 Apr 2017). A summary is in the For doctors view, and the full results are on ClinicalTrials.gov.
How can a doctor refer a patient or contact the research team?
The record lists no contact. The official record names the sponsor, who can be asked directly. AzTrials does not enrol participants or take referrals.
What does the primary outcome measure?
Percent Change of Active Blisters and in Total Blister/Erosion Counts (time frame: 7 days). It is what the study was designed to measure, not a finding.
Where can I read the original study information?
Does a listing mean the treatment is approved or proven to work?
No. A listing means the study is registered. It does not show that the treatment is safe, works, or is approved by a regulator.
How often is this information updated?
AzTrials copies changed records from ClinicalTrials.gov every night and reloads the whole registry every week. The sponsor last updated this record on 23 Jun 2017.