Epidermolysis BullosaPhase 3NCT01340235
Treatment of Dowling Maera Type of Epidermolysis Bullosa Simplex by Oral Erythromycin
A Phase 3 study testing Oral erythromycin in people with Epidermolysis Bullosa, run by Centre Hospitalier Universitaire de Nice at 4 sites in France.
Trial summary
Dowling Meara type of epidermolysis bullosa simplex (EBS-DM) is a rare genodermatosis due to keratin 5 and 14 mutation, characterized by skin fragility and spontaneous or post traumatic blisters. Neonatal period and infancy are critical since this autonomic dominant affection usually improves with age. Read more about this study
- Who is it for?
- Ages 6 months to 8 years
- Conditions the study is for
- Epidermolysis Bullosa
- What is being tested?
- Oral erythromycin
- Type of treatment
- Pill or oral medicine
- Could I receive a placebo?
- No, everyone receives an active treatment
- How many people
- 8 participants
- Where
- 4 sitesFrance
- Study dates
- Jun 2011to Jun 2012
- Phase
- Phase 3
- 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 6 months to 8 years
- All sexes
- No healthy volunteers
- Child
Who may be able to join
- Severe Dowling Meara EBS patients (2 or more new blisters a day)
- signature of informed consent
- Patient of 2 sexes
- Age from 6 months to 8 years. From this age we consider that the patient will less need this treatment or can take cyclines.
- Systematic Obtaining of the consent lit(enlightened) by the relatives(parents) of the child, after information about the objectives and the constraints of the study.
- Agreement of the minor
- Patient member to the Social Security
Who cannot join
- Patient allergic to the erythromycin
- Patient presenting an intolerance to the fructose, a syndrome of malabsorption some glucose and some galactose or a deficit sucrase-isomaltase
- Renal and\or hepatic Insufficiency
- Patient taking a medicine against indicated or misadvised in association with the erythromycin
Trial locations and contacts
4 sites in 1 country.
France4 sites, 4 recruiting
- Recruiting
CHU de Dijon
Dijon
Pierre Vabres, PU-PH, pierre.vabres@chu-dijon.fr
- Not yet recruiting
Hôpital Saint Eloi
Montpellier
Didier Bessis, PH
- Recruiting
CHU de Nice - Hôpital de Cimiez
Nice, 06000
Christine Chiaverini, PH, 0033 4 92 03 61 07, chiaverini.c@chu-nice.fr
Vanina Oliveri, ARC, 0033 4 92 03 42 54, oliveri.v@chu-nice.fr
- Recruiting
Hôpital Purpan
Toulouse
Juliette MAZEREEUW, PU-PH, 00 33 5 67 77 81 41, mazereeuw-hautier.j@chu-toulouse.fr
About this study
Dowling Meara type of epidermolysis bullosa simplex (EBS-DM) is a rare genodermatosis due to keratin 5 and 14 mutation, characterized by skin fragility and spontaneous or post traumatic blisters. Neonatal period and infancy are critical since this autonomic dominant affection usually improves with age. Cyclins seem to be efficient in some cases of EBS but are prohibited in children younger than 8 years old. Erythromycin can be a good alternative in this population due to its antibacterial and anti-inflammatory potential.
The aim of this study is the evaluation of the efficiency of oral erythromycin to decrease the number of cutaneous blisters in severe EBS-DM patients from 6 months to 8 years old after 3 months of treatment.
Read more
Primary end point is the number of patients with decrease of blisters' number of at least 20% after 3 months of treatment by oral erythromycin.
It is a preliminary study on 8 patients. Treatment is oral erythromycin twice a day during 3 months. Follow up for each patient is 5 months. The duration of the study is 1 year.
- Conditions
- Epidermolysis Bullosa
- Being tested
- Oral erythromycin
- Medical subject headings
- Epidermolysis Bullosa
What participants receive
What participants receive. In a randomised study each person is assigned to one group by chance.
Oral erythromycinDrug
Severe Dowling Meara EBS patients from 6 months to 8 years old
| Study group | Role | What they receive |
|---|---|---|
| Oral erythromycin | Experimental | Oral erythromycin Receives: Drug: Oral erythromycin |
What is being measured
The primary outcome is the main question the study is designed to answer.
number of patients with decrease of blisters' number of at least 20% after 3 months of treatment by oral erythromycin
Principal end point is evaluated at inclusion and after one month of treatment, 3 months of treatment and 2 months after the end of the treatment
Measured: at 3 months of treatment
Secondary outcomes (3)
Secondary end points are : effect of 3 months of oral erythromycin on - Global tolerance of treatment.
For each patient and globally, the nature, the frequency and the severity of the various unwanted effects will be described on the duration of the study.
Measured: at 3 months of treatment
Secondary end points are : effect of 3 months of oral erythromycin on - Involved area
These criteria will be analyzed in comparison with the values to the inclusion (M0). We shall try to estimate the obstinacy of an effect 2 months after the end of the treatment.
Measured: at 3 months of treatment
Secondary end points are : effect of 3 months of oral erythromycin on - pruritus,
These criteria will be analyzed in comparison with the values to the inclusion (M0). We shall try to estimate the obstinacy of an effect 2 months after the end of the treatment.
Measured: at 3 months of treatment
Timeline
From the start of the study to its completion; the mark is today. Estimated dates are the sponsor’s plan.
- First posted22 Apr 2011 (estimated)
- Study startJun 2011
- Primary completionDec 2011 (estimated)
- Study completionJun 2012 (estimated)
Resources
- Official record on ClinicalTrials.govThe registry entry this page is built from, with its full change history.
- Other trials for Epidermolysis BullosaStudies 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 3
- Primary purpose
- Treatment
- Allocation
- Not applicable
- Intervention model
- Single group
- Masking
- None (open label)
- Enrolment
- 8 participants (estimated)
Sponsor and investigators
- Lead sponsor
- Centre Hospitalier Universitaire de Nice
- Sponsor type
- University, hospital or other
- Responsible party
- Principal investigator, Del Cont Delphine, Centre Hospitalier Universitaire de Nice
- Investigators
- Christine Chiaverini, PH, Principal investigator, Centre Hospitalier Universitaire de Nice
IDs, dates and publications
- ClinicalTrials.gov ID
- NCT01340235
- Sponsor's protocol ID
- 10-PP-19
- Status last verified
- Oct 2011
- First submitted
- 11 Apr 2011
- Last updated
- 14 Oct 2011
- Results posted
- No
- Data monitoring committee
- No
Study at a glance
From the registry record, last updated by the sponsor on 14 Oct 2011. Recruitment status is as the sponsor reported it: confirm with the study team.
- Official title
- Treatment of Dowling Maera Type of Epidermolysis Bullosa Simplex by Oral Erythromycin
- ClinicalTrials.gov ID
- NCT01340235
- Acronym / sponsor ID
- 10-PP-19
- Conditions
- Epidermolysis Bullosa
- Phase
- Phase 3
- Study type
- Interventional
- Recruitment status
- Unknown status
- Enrolment
- 8 participants (estimated)
- Lead sponsor
- Centre Hospitalier Universitaire de NiceUniversity, hospital or other
- Study start
- Jun 2011
- Primary completion
- Dec 2011 (estimated)
- Study completion
- Jun 2012 (estimated)
- First posted
- 22 Apr 2011 (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.
Dowling Meara type of epidermolysis bullosa simplex (EBS-DM) is a rare genodermatosis due to keratin 5 and 14 mutation, characterized by skin fragility and spontaneous or post traumatic blisters. Neonatal period and infancy are critical since this autonomic dominant affection usually improves with age. Cyclins seem to be efficient in some cases of EBS but are prohibited in children younger than 8 years old. Erythromycin can be a good alternative in this population due to its antibacterial and anti-inflammatory potential.
The aim of this study is the evaluation of the efficiency of oral erythromycin to decrease the number of cutaneous blisters in severe EBS-DM patients from 6 months to 8 years old after 3 months of treatment.
Primary end point is the number of patients with decrease of blisters' number of at least 20% after 3 months of treatment by oral erythromycin.
It is a preliminary study on 8 patients. Treatment is oral erythromycin twice a day during 3 months. Follow up for each patient is 5 months. The duration of the study is 1 year.
Study design
- Primary purpose
- Treatment
- Allocation
- Not applicable
- Intervention model
- Single group
- Masking
- None (open label)
- Number of arms
- 1
| Arm | Type | Receives |
|---|---|---|
| Oral erythromycin | Experimental | Drug: Oral erythromycinOral erythromycin |
Who may qualify
- Ages
- 6 months to 8 years
- Sex
- All
- Healthy volunteers
- Not accepted
- Age groups
- Child
Key inclusion criteria (7 listed)
- Severe Dowling Meara EBS patients (2 or more new blisters a day)
- signature of informed consent
- Patient of 2 sexes
- Age from 6 months to 8 years. From this age we consider that the patient will less need this treatment or can take cyclines.
- Systematic Obtaining of the consent lit(enlightened) by the relatives(parents) of the child, after information about the objectives and the constraints of the study.
Key exclusion criteria (4 listed)
- Patient allergic to the erythromycin
- Patient presenting an intolerance to the fructose, a syndrome of malabsorption some glucose and some galactose or a deficit sucrase-isomaltase
- Renal and\or hepatic Insufficiency
- Patient taking a medicine against indicated or misadvised in association with the erythromycin
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 |
|---|---|---|
| Oral erythromycinSevere Dowling Meara EBS patients from 6 months to 8 years old | Drug | Oral erythromycin |
Primary outcome
number of patients with decrease of blisters' number of at least 20% after 3 months of treatment by oral erythromycin
Time frame: at 3 months of treatment
Principal end point is evaluated at inclusion and after one month of treatment, 3 months of treatment and 2 months after the end of the treatment
3 secondary outcomes
Secondary end points are : effect of 3 months of oral erythromycin on - Global tolerance of treatment.
Time frame: at 3 months of treatment
Secondary end points are : effect of 3 months of oral erythromycin on - Involved area
Time frame: at 3 months of treatment
Secondary end points are : effect of 3 months of oral erythromycin on - pruritus,
Time frame: at 3 months of treatment
Results and safety
Results are not available in the source record.
Research links
- Official record on ClinicalTrials.govLast updated by the sponsor 14 Oct 2011.
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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.
Christine Chiaverini, PH principal investigator
Centre Hospitalier Universitaire de Nice
4 sites in 1 country: see every site and its status.
Questions about this trial
What is this clinical trial studying?
A Phase 3 study testing Oral erythromycin in people with Epidermolysis Bullosa, run by Centre Hospitalier Universitaire de Nice at 4 sites 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 7 inclusion and 4 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: status not confirmed, as of the sponsor's last update on 14 Oct 2011. Status can change between updates, so confirm with the study contact before making plans.
Where is the study taking place?
The record lists 4 sites 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.
What does this phase mean?
Phase 3 studies compare the treatment with the current standard treatment or a placebo in a large group, to confirm how well it works and watch for side effects.
Not every study follows the same path, and some studies (observational studies, device studies) have no phase.
Are results available for this study?
Results are not available in the source record.
How can a doctor refer a patient or contact the research team?
Each site lists its own contact: choose the nearest one under Trial locations. AzTrials does not enrol participants or take referrals.
What does the primary outcome measure?
number of patients with decrease of blisters' number of at least 20% after 3 months of treatment by oral erythromycin (time frame: at 3 months of treatment). 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 14 Oct 2011.