Epidermolysis Bullosa Simplex Dowling MearaNCT01556308
Study of Inflammatory Mechanisms in Epidermolysis Bullosa Simplex- Dowling Meara
A study testing Taking of liquid and top of blisters in people with Epidermolysis Bullosa Simplex Dowling Meara, run by Centre Hospitalier Universitaire de Nice at 1 site in France.
Trial summary
Introduction: Epidermolysis Bullosa Simplex-Dowling-Meara (DM-EBS) is a rare genodermatosis characterized by spontaneous or post traumatic large cutaneous blisters. No curative treatment is actually available. Some data suggest a role of inflammation in the occurrence of blisters. Read more about this study
- Who is it for?
- Ages 1 year and older
- Conditions the study is for
- Epidermolysis Bullosa Simplex Dowling Meara
- What is being tested?
- Taking of liquid and top of blisters
- Type of treatment
- Surgery or procedure
- Could I receive a placebo?
- No, everyone receives an active treatment
- How many people
- 6 participants
- Where
- 1 siteFrance
- Study dates
- Mar 2012to Oct 2013
- Phase
- Interventional
- Are there results?
- None posted in the registry
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 1 year and older
- All sexes
- No healthy volunteers
- Child, Adult, Older adult
Who may be able to join
- male or female
- older than 1 year-old
- severe Dowling Meara epidermolysis bullosa (more than 2 blisters per day)
- systematic written informed consent
- child consent
- affiliated to french social security
Who cannot join
- younger than one year-old
- no consent
- non affiliated to french social security
- participation to another clinical trial within 3 months ago
- intercurrent disease justifying the withdrawal of the study
- own patient decision to stop the study, withdrawal of informed consent
Trial locations and contacts
1 site in 1 country.
About this study
Introduction: Epidermolysis Bullosa Simplex-Dowling-Meara (DM-EBS) is a rare genodermatosis characterized by spontaneous or post traumatic large cutaneous blisters. No curative treatment is actually available. Some data suggest a role of inflammation in the occurrence of blisters. The aim of this study is to study the epidermis inflammatory mechanisms in DM-EBS.
Material and methods: A first retrospective immunohistochemical study will be led on remainder skin biopsies of DM-EBS patients took for the diagnosis. A second clinical multicentric prospective study will be led on 8 patients older than 1 year with severe DM-EBS. After informed written consent, they will answer to a standardized questionnaire. In case of flare of the disease, the liquid and the top of the blisters will be took. Samples will be analyzed in the Pr Nicolas 851 INSERM unit. After centrifugation of the liquid blisters, the repartition of inflammatory cells will be evaluated by Fluorescence Activated Cell Sorting on the pellet. Markers of inflammation will be evaluated on the surnageant with Luminex® technical with a multiplex targeting cytokines and chemokines. An immuno-histochemic analysis in association with a quantitative PCR will be made on the top of the blisters. If unknown, genotypic study will be made.
Perspectives: A better comprehension of physiopathological mechanisms in DM-EBS could offer new therapeutic ways.
- Being tested
- Taking of liquid and top of blisters
- Keywords
- Dowling Meara, Epidermolysis bullosa, inflammation
- Medical subject headings
- Epidermolysis Bullosa, Inflammation
What participants receive
What participants receive. In a randomised study each person is assigned to one group by chance.
Taking of liquid and top of blistersProcedure or surgery
the liquid and the top of blisters will be took.
| Study group | Role | What they receive |
|---|---|---|
| DM-EBS | Experimental | Not described Receives: Procedure: Taking of liquid and top of blisters |
What is being measured
The primary outcome is the main question the study is designed to answer.
Markers of inflammation in epidermis in patients with severe DM-BS
Measured: Baseline
Secondary outcomes (1)
clinical characterization of DM-EBS Genotype-phenoty
Measured: Baseline
Timeline
From the start of the study to its completion; the mark is today. Estimated dates are the sponsor’s plan.
- First posted16 Mar 2012 (estimated)
- Study startMar 2012
- Primary completionMar 2012
- Study completionOct 2013
Resources
- Official record on ClinicalTrials.govThe registry entry this page is built from, with its full change history.
- Other trials for Epidermolysis Bullosa Simplex Dowling MearaStudies of the same condition, open and under way.
- More trials run by Centre Hospitalier Universitaire de NiceThe 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 N/A
- Primary purpose
- Basic science
- Allocation
- Not applicable
- Intervention model
- Single group
- Masking
- None (open label)
- Enrolment
- 6 participants (actual)
Sponsor and investigators
- Lead sponsor
- Centre Hospitalier Universitaire de Nice
- Sponsor type
- University, hospital or other
- Responsible party
- Sponsor
IDs, dates and publications
- ClinicalTrials.gov ID
- NCT01556308
- Sponsor's protocol ID
- 11-AOI-02
- Status last verified
- Mar 2012
- First submitted
- 15 Mar 2012
- Last updated
- 29 Jul 2014
- Results posted
- No
- Data monitoring committee
- No
Study at a glance
From the registry record, last updated by the sponsor on 29 Jul 2014. Recruitment status is as the sponsor reported it: confirm with the study team.
- Official title
- Study of Inflammatory Mechanisms in Epidermolysis Bullosa Simplex- Dowling Meara
- ClinicalTrials.gov ID
- NCT01556308
- Acronym / sponsor ID
- 11-AOI-02
- Keywords
- Dowling Meara, Epidermolysis bullosa, inflammation
- Study type
- Interventional
- Recruitment status
- Completed
- Enrolment
- 6 participants (actual)
- Lead sponsor
- Centre Hospitalier Universitaire de NiceUniversity, hospital or other
- Study start
- Mar 2012
- Primary completion
- Mar 2012 (actual)
- Study completion
- Oct 2013 (actual)
- First posted
- 16 Mar 2012 (estimated)
- Results
- Not posted in the source record
Clinical summary
The research question as the sponsor registered it. This describes what the study plans to test, not what it found.
Introduction: Epidermolysis Bullosa Simplex-Dowling-Meara (DM-EBS) is a rare genodermatosis characterized by spontaneous or post traumatic large cutaneous blisters. No curative treatment is actually available. Some data suggest a role of inflammation in the occurrence of blisters. The aim of this study is to study the epidermis inflammatory mechanisms in DM-EBS.
Material and methods: A first retrospective immunohistochemical study will be led on remainder skin biopsies of DM-EBS patients took for the diagnosis. A second clinical multicentric prospective study will be led on 8 patients older than 1 year with severe DM-EBS. After informed written consent, they will answer to a standardized questionnaire. In case of flare of the disease, the liquid and the top of the blisters will be took. Samples will be analyzed in the Pr Nicolas 851 INSERM unit. After centrifugation of the liquid blisters, the repartition of inflammatory cells will be evaluated by Fluorescence Activated Cell Sorting on the pellet. Markers of inflammation will be evaluated on the surnageant with Luminex® technical with a multiplex targeting cytokines and chemokines. An immuno-histochemic analysis in association with a quantitative PCR will be made on the top of the blisters. If unknown, genotypic study will be made.
Perspectives: A better comprehension of physiopathological mechanisms in DM-EBS could offer new therapeutic ways.
Study design
- Primary purpose
- Basic science
- Allocation
- Not applicable
- Intervention model
- Single group
- Masking
- None (open label)
- Number of arms
- 1
| Arm | Type | Receives |
|---|---|---|
| DM-EBS | Experimental | Procedure: Taking of liquid and top of blisters |
Who may qualify
- Ages
- 1 year and older
- Sex
- All
- Healthy volunteers
- Not accepted
- Age groups
- Child, Adult, Older adult
Key inclusion criteria (6 listed)
- male or female
- older than 1 year-old
- severe Dowling Meara epidermolysis bullosa (more than 2 blisters per day)
- systematic written informed consent
- child consent
Key exclusion criteria (6 listed)
- younger than one year-old
- no consent
- non affiliated to french social security
- participation to another clinical trial within 3 months ago
- intercurrent disease justifying the withdrawal of the study
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 |
|---|---|---|
| Taking of liquid and top of blistersthe liquid and the top of blisters will be took. | Procedure or surgery | DM-EBS |
Primary outcome
Markers of inflammation in epidermis in patients with severe DM-BS
Time frame: Baseline
1 secondary outcome
clinical characterization of DM-EBS Genotype-phenoty
Time frame: Baseline
Results and safety
The study is completed, but results are not available in the source record. That does not mean the treatment did or did not work.
Research links
- Official record on ClinicalTrials.govLast updated by the sponsor 29 Jul 2014.
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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.
The record lists no contact. Use the official record to reach the sponsor.
1 site in 1 country: see every site and its status.
Questions about this trial
What is this clinical trial studying?
A study testing Taking of liquid and top of blisters in people with Epidermolysis Bullosa Simplex Dowling Meara, run by Centre Hospitalier Universitaire de Nice at 1 site in France.
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 6 inclusion and 6 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 29 Jul 2014. 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 France. 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.
Are results available for this study?
The study is completed, but results are not available in the source record. That does not mean the treatment did or did not work.
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?
Markers of inflammation in epidermis in patients with severe DM-BS (time frame: Baseline). 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 29 Jul 2014.