Recessive Dystrophic Epidermolysis BullosaSIMOCEBNCT04285294
Molecular Signatures of Cutaneous Squamous Cell Carcinoma During Recessive Dystrophic Epidermolysis Bullosa
An observational study of Recessive Dystrophic Epidermolysis Bullosa, run by Assistance Publique - Hôpitaux de Paris.
Trial summary
Recessive dystrophic epidermolysis bullosa (RDEB) is a hereditary skin disease characterized by cutaneous and mucosa fragility. Blister formations and erosions, resulting in chronic wounds and dystrophic scars, lead development of aggressive cutaneous squamous cell carcinoma (cSCC) in young subjects. Read more about this study
- Who is it for?
- Ages 18 years and older
- Conditions the study is for
- Recessive Dystrophic Epidermolysis Bullosa
- How many people
- 66 participants
- Where
- No sites listed yet
- Study dates
- Mar 2020to Dec 2023
- 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 years and older
- All sexes
- Adult, Older adult
Study population: RDEB patients with a SCC and non-RDEB patients with a SCC induced by ultraviolet radiation
Who may be able to join
- for RDEB patients with a SCC :
- aged older than 18 years old
- one or more SCC surgically treated
- signed genetic consent form
- for non-RDEB patients with a SCC induced by ultraviolet radiation :
- aged older than 18 years old
- one or more SCC induced by ultraviolet radiation
- signed genetic consent form
Who cannot join
- under protection by law (tutorship or curatorship)
- without health insurance coverage
Trial locations and contacts
No sites are listed yet.
Matthieu Resche-Rigon, Pr contact
About this study
Recessive dystrophic epidermolysis bullosa (RDEB) is a hereditary skin disease characterized by cutaneous and mucosa fragility. Blister formations and erosions, resulting in chronic wounds and dystrophic scars, lead development of aggressive cutaneous squamous cell carcinoma (cSCC) in young subjects. cSCC in RDEB patients are often recurrent and sometimes aggressive. Although fibrotic and inflammatory microenvironment plays an important role in the tumoral process, specific mechanisms in cSCC of RDEB patients are still unknown. Actually, the only treatment is a wide surgical excision with poor prognostic (80% of death after the first occurrence of cSCC).
The objective of the study is to describe the molecular signatures in the cSCC in RDEB patients
- 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.
| Study group | Role | What they receive |
|---|---|---|
| RDEB patients with a cSCC | Not described | |
| Non-RDEB patients with a SCC induced by ultraviolet radiation | Not described | |
| Healthy donors without RDEB nor SCC | Not described |
What is being measured
The primary outcome is the main question the study is designed to answer.
Characterize the molecular signatures in the cSCC and the peri-tumoral dystrophic area occurring in RDEB patients
Molecular signatures will be assessed by genomic, transcriptomic, and proteomic analyses
Measured: at inclusion
Secondary outcomes (2)
Comparison of molecular signature according to the aggressive evolution
Molecular signatures will be assessed by genomic, transcriptomic, proteomic analyses and will be compared between RDEB patients with different clinical outcomes (cSCC with an aggressive and metastatic evolution versus cSCC without aggressive evolution at 3 months).
Measured: at inclusion
Comparison of molecular signatures between cSCC from RDEB patients versus cSCC from non-RDEB patients (induced by ultraviolet radiation)
Molecular signatures will be assessed by genomic, transcriptomic, and proteomic analyses and will be compared between cSCC from RDEB patients versus cSCC from non-RDEB patients (induced by ultraviolet radiation)
Measured: at inclusion
Timeline
From the start of the study to its completion; the mark is today. Estimated dates are the sponsor’s plan.
- First posted26 Feb 2020
- Study startMar 2020 (estimated)
- Primary completionDec 2023 (estimated)
- Study completionDec 2023 (estimated)
Resources
- Official record on ClinicalTrials.govThe registry entry this page is built from, with its full change history.
- Other trials for Recessive Dystrophic Epidermolysis BullosaStudies 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
- Other
- Time perspective
- Other
- Enrolment
- 66 participants (estimated)
Sponsor and investigators
- Lead sponsor
- Assistance Publique - Hôpitaux de Paris
- Sponsor type
- University, hospital or other
- Responsible party
- Sponsor
IDs, dates and publications
- ClinicalTrials.gov ID
- NCT04285294
- Sponsor's protocol ID
- APHP190820
- Acronym
- SIMOCEB
- Status last verified
- Feb 2020
- First submitted
- 24 Feb 2020
- Last updated
- 26 Feb 2020
- Results posted
- No
- Data monitoring committee
- No
- FDA-regulated drug
- No
- FDA-regulated device
- No
Study at a glance
From the registry record, last updated by the sponsor on 26 Feb 2020. Recruitment status is as the sponsor reported it: confirm with the study team.
- Official title
- Molecular Signatures of Cutaneous Squamous Cell Carcinoma During Recessive Dystrophic Epidermolysis Bullosa
- ClinicalTrials.gov ID
- NCT04285294
- Acronym / sponsor ID
- SIMOCEB / APHP190820
- Study type
- Observational
- Recruitment status
- Unknown status
- Enrolment
- 66 participants (estimated)
- Lead sponsor
- Assistance Publique - Hôpitaux de ParisUniversity, hospital or other
- Study start
- Mar 2020 (estimated)
- Primary completion
- Dec 2023 (estimated)
- Study completion
- Dec 2023 (estimated)
- First posted
- 26 Feb 2020 (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.
Recessive dystrophic epidermolysis bullosa (RDEB) is a hereditary skin disease characterized by cutaneous and mucosa fragility. Blister formations and erosions, resulting in chronic wounds and dystrophic scars, lead development of aggressive cutaneous squamous cell carcinoma (cSCC) in young subjects. cSCC in RDEB patients are often recurrent and sometimes aggressive. Although fibrotic and inflammatory microenvironment plays an important role in the tumoral process, specific mechanisms in cSCC of RDEB patients are still unknown. Actually, the only treatment is a wide surgical excision with poor prognostic (80% of death after the first occurrence of cSCC).
The objective of the study is to describe the molecular signatures in the cSCC in RDEB patients
Study design
- Observational model
- Other
- Time perspective
- Other
- Number of arms
- 3
| Arm | Type | Receives |
|---|---|---|
| RDEB patients with a cSCC | – | – |
| Non-RDEB patients with a SCC induced by ultraviolet radiation | – | – |
| Healthy donors without RDEB nor SCC | – | – |
Who may qualify
- Ages
- 18 years and older
- Sex
- All
- Age groups
- Adult, Older adult
- Study population
- RDEB patients with a SCC and non-RDEB patients with a SCC induced by ultraviolet radiation
Key inclusion criteria (8 listed)
- for RDEB patients with a SCC :
- for non-RDEB patients with a SCC induced by ultraviolet radiation :
Key exclusion criteria (2 listed)
- under protection by law (tutorship or curatorship)
- without health insurance coverage
Eligibility is decided by the study team after screening; this overview does not establish that a patient qualifies. Full inclusion and exclusion criteria.
Primary outcome
Characterize the molecular signatures in the cSCC and the peri-tumoral dystrophic area occurring in RDEB patients
Time frame: at inclusion
Molecular signatures will be assessed by genomic, transcriptomic, and proteomic analyses
2 secondary outcomes
Comparison of molecular signature according to the aggressive evolution
Time frame: at inclusion
Comparison of molecular signatures between cSCC from RDEB patients versus cSCC from non-RDEB patients (induced by ultraviolet radiation)
Time frame: at inclusion
Results and safety
Results are not available in the source record.
Research links
- Official record on ClinicalTrials.govLast updated by the sponsor 26 Feb 2020.
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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.
Matthieu Resche-Rigon, Pr contact
Questions about this trial
What is this clinical trial studying?
An observational study of Recessive Dystrophic Epidermolysis Bullosa, run by Assistance Publique - Hôpitaux de Paris.
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 8 inclusion and 2 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 26 Feb 2020. Status can change between updates, so confirm with the study contact before making plans.
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 Emmanuel BOURRAT, Dr (+33 1 42 49 90 90, emmanuelle.bourrat@aphp.fr), or a site near the patient. AzTrials does not enrol participants or take referrals.
What does the primary outcome measure?
Characterize the molecular signatures in the cSCC and the peri-tumoral dystrophic area occurring in RDEB patients (time frame: at inclusion). 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 26 Feb 2020.