En undersøgelse for at vurdere forekomsten af hudkræft hos patienter med epidermolyse bullosa, der modtager Filsuvez (FOSteR)
En langsigtet ikke-interventionel undersøgelse for at vurdere forekomsten af hudkræftsygdomme hos patienter med dystrofisk og Junctional Epidermolysis Bullosa, der modtager behandling med Filsuvez
In patients with epidermolysis bullosa (EB), collagen does not form properly, so their skin is very fragile and blisters easily. Such patients are also at greatly increased risk of developing skin cancers. Filsuvez is a topical gel used to promote healing of skin lesions in patients with certain types of EB. In this observational study, patients with either dystrophic EB (DEB) or junctional EB (JEB) will receive standard of care treatment, whether Filsuvez or something else, and will be followed for a minimum of 3 years. The main purpose is to see if the use of Filsuvez affects the likelihood of developing skin malignancies in these patient populations.
Studieoversigt
Status
Intervention / Behandling
Detaljeret beskrivelse
Some forms of EB are associated with a greatly increased incidence of aggressive skin and mucosal squamous cell carcinoma (SCC), as well as increased incidence of other skin malignancies including basal cell carcinoma (BCC) and malignant melanoma (MM). This long-term non-interventional study will assess all types of skin malignancies (SCC, BCC, and MM) in DEB and JEB patients. It is an observational study of real-world treatment practices, in which patients will receive standard of care therapy whatever that may entail. Data will be obtained from clinical study centers in the European Union (EU), United Kingdom (UK) and the United States (US). Participants will be followed for a minimum of 3 years, and information about the development and nature of skin malignancies will be collected over this time period both from patients who are taking and those who are not taking Filsuvez.
Undersøgelsestype
Tilmelding (Anslået)
Kontakter og lokationer
Studiekontakt
- Navn: Chiesi Clinical Trials
- Telefonnummer: +3905212791
- E-mail: clinicaltrials_info@chiesi.com
Studiesteder
-
-
-
London, Det Forenede Kongerige
- Rekruttering
- Guy's and St Thomas' NHS Foundation Trust
-
Kontakt:
- Jemima Mellerio
- Telefonnummer: 44 20 7188 6399
- E-mail: jemima.mellerio@gstt.nhs.uk
-
Ledende efterforsker:
- Jemima Mellerio
-
-
-
-
California
-
Palo Alto, California, Forenede Stater, 94304
- Ikke rekrutterer endnu
- Lucile Packard Children's Hospital
-
Kontakt:
- Joyce Teng, MD
- Telefonnummer: 650-724-9627
- E-mail: jteng3@stanford.edu
-
-
Colorado
-
Aurora, Colorado, Forenede Stater, 80045
- Ikke rekrutterer endnu
- UCHealth / Children's Hospital Colorado
-
Kontakt:
- Emily Gorell, DO
- Telefonnummer: 707-319-9269
- E-mail: emily.gorell@cuanschutz.edu
-
-
Florida
-
Gainesville, Florida, Forenede Stater, 32606
- Ikke rekrutterer endnu
- UF College of Medicine
-
Kontakt:
- Michael Lavery, MD
- Telefonnummer: 352-594-1547
- E-mail: m.lavery@dermatology.med.ufl.edu
-
-
Ohio
-
Cincinnati, Ohio, Forenede Stater, 45229
- Ikke rekrutterer endnu
- Cincinnati Children's
-
Kontakt:
- Kalyani Marathe
- Telefonnummer: 513-636-2009
- E-mail: Kalyani.Marathe@cchmc.org
-
-
Pennsylvania
-
Philadelphia, Pennsylvania, Forenede Stater, 19104
- Ikke rekrutterer endnu
- Childrens Hospital of Philadelphia
-
Kontakt:
- Marissa Perman-Laskin, MD
- Telefonnummer: 215-590-1930
- E-mail: permanm@chop.edu
-
-
South Carolina
-
Charleston, South Carolina, Forenede Stater, 29425
- Ikke rekrutterer endnu
- Medical University of South Carolina
-
Kontakt:
- Lara Wine Lee, MD
- Telefonnummer: 843-792-3021
- E-mail: winelee@musc.edu
-
-
-
-
-
Toulouse, Frankrig
- Rekruttering
- CHU de Toulouse, Hôpital Larrey
-
Kontakt:
- Juliette Mazereeuw Hautier
- Telefonnummer: 33 5 67 77 18 76
- E-mail: mazereeuw-hautier.j@chu-toulouse.fr
-
Ledende efterforsker:
- Juliette Mazereeuw Hautier
-
-
-
-
-
Thessaloniki, Grækenland
- Rekruttering
- Hospital of Skin and Venereal Diseases of Thessaloniki
-
Kontakt:
- Dimitra Kiritsi
- Telefonnummer: 0030-6983375771
- E-mail: dimkyritsi@auth.gr
-
Ledende efterforsker:
- Dimitra Kiritsi
-
-
-
-
-
Barcelona, Spanien
- Rekruttering
- Hospital Clinic, Barcelona
-
Kontakt:
- Josep Riera Monroig
- Telefonnummer: 93 227 54 00
- E-mail: jriera@clinic.cat
-
Ledende efterforsker:
- Josep Riera Monroig
-
Barcelona, Spanien
- Rekruttering
- Hospital San Juan de Dios (Barcelona)
-
Kontakt:
- Asuncion Vicente
- E-mail: asuncion.vicente@sjd.es
-
Ledende efterforsker:
- Asuncion Vicente
-
-
-
-
-
Freiburg im Breisgau, Tyskland, 79104
- Rekruttering
- UMC Freiburg EB Centre
-
Kontakt:
- Cristina Has, MD
- Telefonnummer: 4976127069920
- E-mail: cristina.has@uniklinik-freiburg.de
-
-
Deltagelseskriterier
Berettigelseskriterier
Aldre berettiget til at studere
- Barn
- Voksen
- Ældre voksen
Tager imod sunde frivillige
Køn, der er berettiget til at studere
Prøveudtagningsmetode
Studiebefolkning
Patients with dystrophic EB or junctional EB, whether they are receiving Filsuvez, receiving another treatment for EB, or not receiving any treatment.
Beskrivelse
Inclusion Criteria:
- Patients with a confirmed diagnosis of dystrophic EB or junctional EB
Exclusion Criteria:
- None
Studieplan
Hvordan er undersøgelsen tilrettelagt?
Design detaljer
- Observationsmodeller: COHORT
- Tidsperspektiver: PROSPECTIVE
Kohorter og interventioner
Gruppe / kohorte |
Intervention / Behandling |
|---|---|
|
Patients with dystrophic or junctional EB being treated with Filsuvez
Patients in this cohort will be receiving treatment with Filsuvez for their EB. |
Topical gel
Andre navne:
|
|
Patients with dystrophic or junctional EB not being treated with Filsuvez
Patients in this cohort will be receiving treatment other than Filsuvez or no treatment at all for their EB. |
Hvad måler undersøgelsen?
Primære resultatmål
Resultatmål |
Foranstaltningsbeskrivelse |
Tidsramme |
|---|---|---|
|
Incidence of first skin malignancy during follow-up in EB patients receiving Filsuvez
Tidsramme: Minimum 3 years
|
Patients being treated with Filsuvez will be followed for occurrence of skin malignancies from the date of study enrolment until either the date of discontinuation (withdrawal of consent, withdrawal from the site or registry, physician decision, death, or lost to follow-up) or termination of the study |
Minimum 3 years
|
Sekundære resultatmål
Resultatmål |
Foranstaltningsbeskrivelse |
Tidsramme |
|---|---|---|
|
Incidence of first skin malignancy during follow-up in EB patients not receiving Filsuvez
Tidsramme: Minimum 3 years
|
Patients with EB who are not being treated with Filsuvez will be followed for occurrence of skin malignancies from the date of study enrolment until either the date of discontinuation (withdrawal of consent, withdrawal from the site or registry, physician decision, death, or lost to follow-up) or termination of the study |
Minimum 3 years
|
Samarbejdspartnere og efterforskere
Sponsor
Datoer for undersøgelser
Studer store datoer
Studiestart (Faktiske)
Primær færdiggørelse (Anslået)
Studieafslutning (Anslået)
Datoer for studieregistrering
Først indsendt
Først indsendt, der opfyldte QC-kriterier
Først opslået (Faktiske)
Opdateringer af undersøgelsesjournaler
Sidste opdatering sendt (Faktiske)
Sidste opdatering indsendt, der opfyldte kvalitetskontrolkriterier
Sidst verificeret
Mere information
Begreber relateret til denne undersøgelse
Nøgleord
Yderligere relevante MeSH-vilkår
- Genetiske sygdomme, medfødte
- Bindevævssygdomme
- Hudsygdomme
- Medfødte abnormiteter
- Hudsygdomme, genetisk
- Hudabnormiteter
- Hudsygdomme, vesikulobuløse
- Kollagensygdomme
- Medfødte, arvelige og neonatale sygdomme og abnormiteter
- Hud- og bindevævssygdomme
- Epidermolyse Bullosa
- Epidermolysis Bullosa Dystrophica
- Epidermolysis Bullosa, Junctional
Andre undersøgelses-id-numre
- CLI-FILSVAA1-01
Lægemiddel- og udstyrsoplysninger, undersøgelsesdokumenter
Studerer et amerikansk FDA-reguleret lægemiddelprodukt
Studerer et amerikansk FDA-reguleret enhedsprodukt
Disse oplysninger blev hentet direkte fra webstedet clinicaltrials.gov uden ændringer. Hvis du har nogen anmodninger om at ændre, fjerne eller opdatere dine undersøgelsesoplysninger, bedes du kontakte register@clinicaltrials.gov. Så snart en ændring er implementeret på clinicaltrials.gov, vil denne også blive opdateret automatisk på vores hjemmeside .