Epidermolysis Bullosa DystrophicaISTRADEBNCT06177353
Study of the Blood and Skin Immunological Profile of Patients With Recessive Dystrophic Epidermolysis Bullosa: in Vivo Analysis and the Impact of Placental Stem Cells in Vitro
An observational study of Epidermolysis Bullosa Dystrophica, run by Assistance Publique - Hôpitaux de Paris at 2 sites in France.
Trial summary
Patients with recessive dystrophic epidermolysis bullosa (RDEB) suffer from acute and chronic post-bullous wounds along with impaired skin healing. These issues are attributed not only to mucocutaneous fragility and abnormal healing directly related to quantitative and/or qualitative constitutional abnormalities of… Read more about this study
- Who is it for?
- Ages 18 to 80 yearsHealthy volunteers can take part
- Conditions the study is for
- Epidermolysis Bullosa Dystrophica
- What is being tested?
- Sampling
- How many people
- 30 participants
- Where
- 2 sitesFrance
- Study dates
- 15 Apr 2024to 15 Apr 2025
- Phase
- Observational
- 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 18 to 80 years
- All sexes
- Healthy volunteers accepted
- Adult, Older adult
Study population: Patients with recessive dystrophic epidermolysis bullosa and healthy control patients
Who may be able to join
- EBDR patients :
- Patients aged 18 to 80 years old
- Clinically, histologically, and/or genetically confirmed intermediate, reversed or generalized, moderate to severe EBDR
- Healthy controls :
- Adults aged 18 to 80 years old
- PBMC healthy donors: subjects who have donated blood to the EFS according to the indication criteria who have consented to the use of their samples for research purposes.
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- Healthy skin biopsy donors: subjects undergoing abdominoplasty scheduled in plastic surgery and who have given their consent for the collection of a skin biopsy from post-operative abdominoplasty skin remnants.
- Healthy donors of bandages soiled with exudates from cutaneous wounds: subject consulting a plastic surgery department as part of their usual post-operative follow-up, who have given their consent for the collection of one of their dressings during their usual during their usual renewal.
- For all subjects :
- Free, informed, written consent, signed by the person and the investigator no later than the day of inclusion and before any examination carried out as part of the study.
- Person affiliated or benefiting from a social security scheme
Who cannot join
- EBDR patients :
- EBH with no definite diagnosis or other than EBDR intermediate, reversed or generalized
- Systemic anti-inflammatory or immunosuppressive therapy for less than one month
- Refusal of skin biopsy
- Healthy controls :
- Acute or chronic systemic or cutaneous inflammatory disease at the time of sampling
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- Current immunosuppressive anti-inflammatory treatment in the month prior to sampling
- For all subjects:
- Persons under guardianship or curatorship, or deprived of their liberty by judicial or administrative decision
- Patients receiving State Medical Aid
- Pregnant or breast-feeding women
Trial locations and contacts
2 sites in 1 country.
France2 sites, 2 recruiting
- Recruiting
Hopital Saint Louis - Service de chirurgie plastique reconstructrice et esthétique et traitement chirurgical des brûlés
Paris
Maurice Mimoun, MD
- Recruiting
Hopital Saint Louis - service dermatologie
Paris
Emmanuelle Bourrat, MD
About this study
Official title: Etude du Profil Immunologique Sanguin et cutané Des Patients Atteints d'épidermolyse Bulleuse Dystrophique récessive : Analyses in Vivo et Impact Des Cellules Souches Placentaires in Vitro
Patients with recessive dystrophic epidermolysis bullosa (RDEB) suffer from acute and chronic post-bullous wounds along with impaired skin healing. These issues are attributed not only to mucocutaneous fragility and abnormal healing directly related to quantitative and/or qualitative constitutional abnormalities of collagen VII but also to a contingent cutaneous and systemic inflammatory component. This inflammatory aspect contributes to the perpetuation of skin lesions and delayed healing. Our primary objective is to define the systemic immunological/inflammatory signature of patients with RDEB with an aim to develop a strategy that involves using stem cells with high immunomodulatory/anti-inflammatory capacity such as allogeneic placental stem cells (WJ-MSCs and trophoblasts).
- Conditions
- Epidermolysis Bullosa Dystrophica
- Being tested
- Sampling
- 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.
SamplingOther
Blood sampling Skin biopsy Collection of soiled bandages
| Study group | Role | What they receive |
|---|---|---|
| EBDR patients | Not described Receives: Other: Sampling | |
| Healthy control | Not described Receives: Other: Sampling |
What is being measured
The primary outcome is the main question the study is designed to answer.
Analysis of the cellular (PBMC) and cytokine (serum) immunological signature in the peripheral blood of EBDR subjects and healthy controls.
Measured: Up to 1 year
Secondary outcomes (3)
Cellular, cytokine, and lipid immunological signatures of immune cells
Immune cells are purified from skin biopsies and soiled dressings of EBDR patients and healthy controls. Individual data will be correlated to the severity grade of EBD.
Measured: Up to 1 year
Immunogenicity and impact of WJ-MSCs and trophoblasts
Immunogenicity and impact of WJ-MSCs and trophoblasts on cellular, cytokine, and lipid immunological signatures, activation, and proliferation of peripheral or cutaneous immune cells from EBDR patients and healthy controls, using in-vitro models.
Measured: Up to 1 year
Immunogenicity and impact of EV/Ex
Immunogenicity and impact of EV/Ex on cellular, cytokine, and lipid immunological signatures, activation, and proliferation of peripheral or cutaneous immune cells of EBDR patients and healthy controls, using in-vitro models.
Measured: Up to 1 year
Timeline
From the start of the study to its completion; the mark is today. Estimated dates are the sponsor’s plan.
- First posted20 Dec 2023
- Study start15 Apr 2024
- Primary completion15 Apr 2025 (estimated)
- Study completion15 Apr 2025 (estimated)
Resources
- Official record on ClinicalTrials.govThe registry entry this page is built from, with its full change history.
- Other trials for Epidermolysis Bullosa DystrophicaStudies of the same condition, open and under way.
- More trials run by Assistance Publique - Hôpitaux de ParisThe 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
- Observational
- Observational model
- Cohort
- Time perspective
- Prospective
- Enrolment
- 30 participants (estimated)
Sponsor and investigators
- Lead sponsor
- Assistance Publique - Hôpitaux de Paris
- Sponsor type
- University, hospital or other
- Responsible party
- Sponsor
- Investigators
- Reem Al-Daccak, Dr, Study director, Institut National de la Santé Et de la Recherche Médicale, France
IDs, dates and publications
- ClinicalTrials.gov ID
- NCT06177353
- Sponsor's protocol ID
- APHP230933
- Acronym
- ISTRADEB
- Status last verified
- Jul 2024
- First submitted
- 11 Dec 2023
- Last updated
- 17 Jul 2024
- Results posted
- No
- Data monitoring committee
- No
- FDA-regulated drug
- No
- FDA-regulated device
- No
- Individual data sharing
- Undecided
Study at a glance
From the registry record, last updated by the sponsor on 17 Jul 2024. Recruitment status is as the sponsor reported it: confirm with the study team.
- Official title
- Etude du Profil Immunologique Sanguin et cutané Des Patients Atteints d'épidermolyse Bulleuse Dystrophique récessive : Analyses in Vivo et Impact Des Cellules Souches Placentaires in Vitro
- ClinicalTrials.gov ID
- NCT06177353
- Acronym / sponsor ID
- ISTRADEB / APHP230933
- Conditions
- Epidermolysis Bullosa Dystrophica
- Study type
- Observational
- Recruitment status
- Unknown status
- Enrolment
- 30 participants (estimated)
- Lead sponsor
- Assistance Publique - Hôpitaux de ParisUniversity, hospital or other
- Study start
- 15 Apr 2024 (actual)
- Primary completion
- 15 Apr 2025 (estimated)
- Study completion
- 15 Apr 2025 (estimated)
- First posted
- 20 Dec 2023 (actual)
- 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.
Patients with recessive dystrophic epidermolysis bullosa (RDEB) suffer from acute and chronic post-bullous wounds along with impaired skin healing. These issues are attributed not only to mucocutaneous fragility and abnormal healing directly related to quantitative and/or qualitative constitutional abnormalities of collagen VII but also to a contingent cutaneous and systemic inflammatory component. This inflammatory aspect contributes to the perpetuation of skin lesions and delayed healing. Our primary objective is to define the systemic immunological/inflammatory signature of patients with RDEB with an aim to develop a strategy that involves using stem cells with high immunomodulatory/anti-inflammatory capacity such as allogeneic placental stem cells (WJ-MSCs and trophoblasts).
Study design
- Observational model
- Cohort
- Time perspective
- Prospective
- Number of arms
- 2
| Arm | Type | Receives |
|---|---|---|
| EBDR patients | – | Other: Sampling |
| Healthy control | – | Other: Sampling |
Who may qualify
- Ages
- 18 to 80 years
- Sex
- All
- Healthy volunteers
- Accepted
- Age groups
- Adult, Older adult
- Study population
- Patients with recessive dystrophic epidermolysis bullosa and healthy control patients
Key inclusion criteria (11 listed)
- Patients aged 18 to 80 years old
- Clinically, histologically, and/or genetically confirmed intermediate, reversed or generalized, moderate to severe EBDR
- Adults aged 18 to 80 years old
- PBMC healthy donors: subjects who have donated blood to the EFS according to the indication criteria who have consented to the use of their samples for research purposes.
- Healthy skin biopsy donors: subjects undergoing abdominoplasty scheduled in plastic surgery and who have given their consent for the collection of a skin biopsy from post-operative abdominoplasty skin remnants.
Key exclusion criteria (11 listed)
- EBH with no definite diagnosis or other than EBDR intermediate, reversed or generalized
- Systemic anti-inflammatory or immunosuppressive therapy for less than one month
- Refusal of skin biopsy
- Acute or chronic systemic or cutaneous inflammatory disease at the time of sampling
- Current immunosuppressive anti-inflammatory treatment in the month prior to sampling
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 |
|---|---|---|
| SamplingBlood sampling Skin biopsy Collection of soiled bandages | Other | EBDR patients, Healthy control |
Primary outcome
Analysis of the cellular (PBMC) and cytokine (serum) immunological signature in the peripheral blood of EBDR subjects and healthy controls.
Time frame: Up to 1 year
3 secondary outcomes
Cellular, cytokine, and lipid immunological signatures of immune cells
Time frame: Up to 1 year
Immunogenicity and impact of WJ-MSCs and trophoblasts
Time frame: Up to 1 year
Immunogenicity and impact of EV/Ex
Time frame: Up to 1 year
Results and safety
Results are not available in the source record.
Research links
- Official record on ClinicalTrials.govLast updated by the sponsor 17 Jul 2024.
- Individual participant dataSharing planned: Undecided
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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.
Reem Al-Daccak, Dr study director
Institut National de la Santé Et de la Recherche Médicale, France
2 sites in 1 country: see every site and its status.
Questions about this trial
What is this clinical trial studying?
An observational study of Epidermolysis Bullosa Dystrophica, run by Assistance Publique - Hôpitaux de Paris at 2 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 11 inclusion and 11 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 17 Jul 2024. Status can change between updates, so confirm with the study contact before making plans.
Where is the study taking place?
The record lists 2 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.
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?
Contact Emmannuelle Bourrat, Pr (+33142499090, emmanuelle.bourrat@aphp.fr), or a site near the patient. AzTrials does not enrol participants or take referrals.
What does the primary outcome measure?
Analysis of the cellular (PBMC) and cytokine (serum) immunological signature in the peripheral blood of EBDR subjects and healthy controls. (time frame: Up to 1 year). 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 17 Jul 2024.