GMEB-SASS: A Gene-Modified Skin Substitute for RDEB Treatment
A legfontosabb adatok
- Ki végzi
- CHU de Quebec-Universite Laval
- Hol
- Kanada1 helyszín, 1 országbanVálassz országot a helyszínekhez
- Azonosító
- Állapot
- Toboroz
- Fázis
- 1/2. fázis
- Résztvevők
- 9 fő, tervezett
- Kezdés
- 2026. jan. 7.
- Várható befejezés
- 2035. dec.
- A vizsgálat típusa
- Beavatkozással járó
- A vizsgált kezelések
- Genetically Modified Epidermolysis Bullosa Self-Assembled Skin Substitute (GMEB-SASS)
- A regiszteradat frissítve
- 2026. március 18.
Ki vehet részt
MI-összefoglaló
A vizsgálat saját részvételi feltételeiből. Gép olvasta ki az oldalon álló regiszterszövegből, ezért tévedhet. Hogy ki vehet részt, azt a lenti regiszterszöveg és a vizsgálat csapata dönti el.
The trial is looking for people with recessive dystrophic epidermolysis bullosa (RDEB) who have open or blistered skin wounds. They will get a lab-grown skin graft made from their own gene-modified skin cells.
- Életkor
- 7 years and older. The first 3 people must be 18 years or older, people 4 to 6 must be 12 years or older, and people 7 to 9 must be 7 years or older.
- Nem
- All sexes
- A betegség stádiuma
- A clinical diagnosis of RDEB. On the day of grafting, blistered or open skin areas on the trunk, arms or legs big enough for at least three 25 to 50 cm2 grafts. Able to have anesthesia.
- Korábbi kezelés
- No immunotherapy, including oral corticosteroids (Prednisolone > 1 mg/kg), for more than one week within 2 weeks before the study treatment. Nasal and skin preparations are allowed. No chemotherapy within 60 days before. No gene therapy, chemical or biological product that changes collagen 7 within the last 6 months.
- Biomarker vagy leletérték
- Confirmed disease-causing changes in both copies of the COL7A1 gene (biallelic pathogenic variant).
A részvétel fő kizáró okai
- Current or past squamous cell carcinoma (a skin cancer) at the site to be grafted
- Signs of an infection throughout the body
- Allergy to bovine (cow) proteins
- Pregnant or breastfeeding
- Active drug or alcohol addiction
A regiszter feltételei, szó szerint
Mind a 19 feltétel megnézéseKevesebb
Ki vehet részt
- Age; Learning phase:
- Subjects 1 to 3: Eighteen (18) years old or older
- Subjects 4 to 6: Twelve (12) years old or older
- Subjects 7 to 9: Seven (7) years old or older; Other Inclusion Criteria:
- Clinical diagnosis of recessive dystrophic epidermolysis bullosa (RDEB) with confirmed biallelic pathogenic variant in the COL7A1 gene
- Candidates - or their parents/caregivers if the candidates have limited comprehension, who are able to understand the study and to comply with the study procedures
- On the day of grafting, one or more blistered and/or erosive skin areas on the trunk and/or extremities large enough to graft at least three 25 to 50 cm2 GMEB-SASS grafts
- Ability to undergo anesthesia
Ki nem vehet részt
- Active drug or alcohol addiction
- Candidate who has received immunotherapy, including oral corticosteroids (Prednisolone > 1 mg/kg), for more than one week, within 2 weeks prior to the study intervention (initial biopsy and GMEB-SASS grafting) (intranasal and topical preparations are permitted)
- Candidate who has received chemotherapy within 60 days prior to the study intervention (initial biopsy and GMEB-SASS grafting)
- Candidate who has received, in the last 6 months prior to the study intervention (initial biopsy and GMEB-SASS grafting), any gene therapy, chemical or biological product modifying collagen 7 expression
- Any medical condition or illness that may impact study participation or compromise the safety of the participants, as per the investigator's judgment
- Evidence of systemic infection
- Current evidence or a history of non-metastatic or metastatic squamous cell carcinoma at the site to be grafted
- History of or known allergy to bovine proteins
- Any clinically significant abnormal laboratory values or abnormal findings identified during physical examination or through medical history that could compromise participant safety, as per the investigator's judgment
- Medical instability limiting the ability to travel to the investigative center
- Female candidate who are pregnant or breast-feeding
Ennek a vizsgálatnak nincs helyszíne itt: Egyesült Államok.
Egy országban van helyszíne. Válassz országot, hogy lásd a helyszíneket.
A vizsgálatról
Mit akar kideríteni a vizsgálat
This study is being done to find out if a new type of skin graft, called GMEB-SASS, is safe and effective for helping wounds heal in people with RDEB (Recessive Dystrophic Epidermolysis Bullosa).
A teljes eredeti leírásKevesebb
The GMEB-SASS graft contains two types of living skin cells: keratinocytes and fibroblasts. It is made in a laboratory using a small sample of the patient's own skin.
To help the patient's skin cells produce a missing protein called type VII collagen, scientists grow the patient's cells in the lab and use a virus-like tool (called a retroviral vector) to give the cells the correct instructions. This allows the cells to make the normal protein that is missing in people with RDEB.
The graft is designed to be permanent, and the goal is to improve wound healing by replacing damaged skin cells with healthy ones.
A vizsgálat csoportja
- GMEB-SASSA vizsgált kezelés
All patients will receive GMEB-SASS
Kezelés: Genetically Modified Epidermolysis Bullosa Self-Assembled Skin Substitute (GMEB-SASS)
Kérdések a vizsgálat csapatának
- Milyen gyakran kell bejárnom, és egy-egy alkalom mennyi ideig tart?
- Megtérítik az utazási költséget?
- Megtarthatom a saját orvosomat?
- Mi történik, ha ki akarok lépni?
Contact the study team: see the registry record → Contacts and Locations
Oldal nyomtatása az orvosomnakAdatok és források
- Regiszteradat
- Szerepel a ClinicalTrials.gov-on.
- Hivatalos cím
- Genetically Modified Epidermolysis Bullosa Self-Assembled Skin Substitute (GMEB-SASS) to Treat Patients Suffering From Recessive Dystrophic Epidermolysis Bullosa (RDEB)
- MI-összefoglaló
- A vizsgálat saját részvételi feltételeiből. Gépi kiolvasás: 2026. november 15.
- A regiszteradat frissítve
- 2026. március 18.