Clinical trials: RDEB
Epidermolysis Bullosa (EB), Recessive Dystrophic Epidermolysis Bullosa (RDEB)
Phase 4 Master Protocol for Patients Prescribed Prademagene Zamikeracel for the Treatment of Wounds
The Master Protocol includes 3 studies (Study A, Study B, and Study C) that will evaluate pz-cel and related processes in the post-marketing setting.
- Conditions
- Epidermolysis Bullosa (EB), Recessive Dystrophic Epidermolysis Bullosa (RDEB), Dystrophic Epidermolysis Bullosa (DEB)
- Trial name
- NCT07717736
- Testing
- Non-conforming pz-cel surgical application to RDEB wounds, Additional biopsies, Pz-cel surgical application to DEB wounds
- Volunteers
- 100 people
- Sponsor
- Abeona Therapeutics, Inc
Epidermolysis Bullosa Dystrophica, Recessive
A Study of FCX-007 for Recessive Dystrophic Epidermolysis Bullosa (RDEB)
The purpose of this study is to evaluate the safety of FCX-007, evaluate Type VII collagen (COL7) expression and the presence of anchoring fibrils and to analyze wound healing as a result of FCX-007 administration in subjects…
- Trial name
- NCT02810951
- Testing
- FCX-007
- Where
- 2 sites in United States
- Volunteers
- 6 people
- Sponsor
- Castle Creek Biosciences, LLC.
Recessive Dystrophic Epidermolysis Bullosa
Safety and Effectiveness Study of Allogeneic Umbilical Cord Blood-derived Mesenchymal Stem Cell in Patients With RDEB
Previously, many studies have been conducted on mesenchymal stem cells derived from bone marrow or subcutaneous fat, but interest in cord blood-derived mesenchymal stem cell treatments has been increasing recently.
- Trial name
- NCT04520022
- Testing
- Human Umbilical Cord Blood-derived Mesenchymal Stem Cells
- Where
- South Korea
- Volunteers
- 5 people
- Sponsor
- Gangnam Severance Hospital
Recessive Dystrophic Epidermolysis Bullosa
Gentamicin Therapy for Recessive Dystrophic Epidermolysis Bullosa (RDEB) Nonsense Mutation Patients
Recessive dystrophic epidermolysis bullosa (RDEB) is an incurable, devastating, inherited skin disease for which there is only supportive care.
- Trial name
- NCT02698735
- Testing
- Gentamicin
- Volunteers
- 5 people
- Sponsor
- University of Southern California
RDEB, Recessive Dystrophic Epidermolysis Bullosa
GMEB-SASS: A Gene-Modified Skin Substitute for RDEB Treatment
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).
- Conditions
- RDEB, Recessive Dystrophic Epidermolysis Bullosa, Epidermolysis Bullosa Dystrophica, Recessive
- Trial name
- NCT07193134
- Testing
- Genetically Modified Epidermolysis Bullosa Self-Assembled Skin Substitute (GMEB-SASS)
- Where
- Canada
- Volunteers
- 9 people
- Sponsor
- CHU de Quebec-Universite Laval
Recessive Dystrophic Epidermolysis Bullosa
Safety Study of Gene-modified Autologous Fibroblasts in Recessive Dystrophic Epidermolysis Bullosa
Recessive dystrophic epidermolysis bullosa (RDEB) is a severe form of blistering skin disease caused by mutations in COL7A1 gene.
- Trial name
- NCT02493816
- Testing
- Gene-modified autologous fibroblasts
- Where
- United Kingdom
- Volunteers
- 5 people
- Sponsor
- King's College London
Epidermolysis Bullosa Dystrophica
Characteristics of Patients With Recessive Dystrophic Epidermolysis Bullosa
Recessive dystrophic epidermolysis bullosa (RDEB) is a disease caused by genetic mutations in the gene for type VII collagen. Patients with RDEB develop large, severely painful blisters and open wounds from minor trauma to their…
- Trial name
- NCT01019148
- Where
- United States
- Volunteers
- 70 people
- Sponsor
- Stanford University
Recessive Dystrophic Epidermolysis Bullosa
Rigosertib in Patients With Recessive Dystrophic Epidermolysis Bullosa Associated SCC
This pilot trial studies how rigsertib sodium works in treating patients with Recessive Dystrophic Epidermolysis bullosa (RDEB) with locally advanced Squamous Cell Carcinoma (SCC).
- Trial name
- NCT04177498
- Testing
- Rigosertib Sodium, Quality-of-Life Assessment
- Where
- United States
- Volunteers
- 3 people
- Sponsor
- Thomas Jefferson University
Epidermolysis Bullosa
Phase I Study of Isotretinoin in Patients With Recessive Dystrophic Epidermolysis Bullosa
OBJECTIVES: I. Determine the safety of isotretinoin in patients with recessive dystrophic epidermolysis bullosa.
- Trial name
- NCT00014729
- Testing
- isotretinoin
- Volunteers
- 20 people
- Sponsor
- University of North Carolina
Epidermolysis Bullosa, Recessive Dystrophic Epidermolysis Bullosa
A Phase 3b Study for the Treatment of Dystrophic Epidermolysis Bullosa (DEB) in New and Previously EB-101 Treated Patients
To evaluate and further characterize the safety of EB-101 (LZRSE-Col7A1 gene-corrected keratinocyte sheets with type VII collagen [C7] expression) for the treatment of large, chronic DEB wounds in new and previously EB-101…
- Conditions
- Epidermolysis Bullosa, Recessive Dystrophic Epidermolysis Bullosa, RDEB
- Trial name
- NCT05725018
- Testing
- EB-101 Surgical application of RDEB wounds
- Where
- 2 sites in United States
- Volunteers
- 12 people
- Sponsor
- Abeona Therapeutics, Inc
RDEB
A Long-Term Extension Study for Participants Previously Treated With EB-101 for the Treatment of RDEB
A Long-Term Extension Study for Participants Previously Treated with EB-101 for the Treatment of Recessive Dystrophic Epidermolysis Bullosa (RDEB)
- Trial name
- NCT05708677
- Testing
- EB-101
- Where
- 2 sites in United States
- Volunteers
- 22 people
- Sponsor
- Abeona Therapeutics, Inc
Recessive Dystrophic Epidermolysis Bullosa
A Long-Term Safety Follow-up Registry for Patients Who Received Treatment With Prademagene Zamikeracel for Recessive Dystrophic Epidermolysis Bullosa (RDEB) in the Post-Marketing Setting
The goal of this observational study is to evaluate the long-term safety and wound healing durability (up to 15 years) for participants with Recessive Dystrophic Epidermolysis Bullosa (RDEB) who were treated with pz-cel.
- Trial name
- NCT07700966
- Testing
- Prademagene Zamikeracel
- Where
- 4 sites in United States
- Volunteers
- 250 people
- Sponsor
- Abeona Therapeutics, Inc
Epidermolysis Bullosa Dystrophica, Epidermolysis Bullosa
Gene Transfer for Recessive Dystrophic Epidermolysis Bullosa
This trial will create a skin graft, which the investigators call "LEAES," using the patient's own skin cells that have been genetically engineered in the lab to express a missing protein called type VII collagen.
- Trial name
- NCT01263379
- Testing
- LZRSE-Col7A1 Engineered Autologous Epidermal Sheets
- Where
- United States
- Volunteers
- 12 people
- Sponsor
- Abeona Therapeutics, Inc
Recessive Dystrophic Epidermolysis Bullosa, Dysphagia
Treatment of Dysphagia in Patients With Dystrophic Epidermolysis Bullosa With Budesonide: a Retrospective Bicentric Study Nice - Paris St Louis / Necker (EBUDE Study)
Budesonide is an anti-inflammatory medication that works by decreasing the immune system's overreaction. In the treatment of eosinophilic esophagitis, especially with Jorveza or a preparation made in a pharmacy, it reduces the…
- Conditions
- Recessive Dystrophic Epidermolysis Bullosa, Dysphagia, Esophageal Stenosis
- Trial name
- EBUDE (NCT07684105)
- Testing
- The retrospective data collected will be entered into an eCRF and then analyzed centrally
- Where
- 3 sites in France
- Volunteers
- 15 people
- Sponsor
- Centre Hospitalier Universitaire de Nice
Dystrophic Epidermolysis Bullosa
A Phase I/II Study of KB103, a Topical HSV1-COL7, on DEB Patients
This study was conducted to assess the safety and efficacy of topical Beremagene Geperpavec (KB103, HSV1-COL7) on DEB patients.
- Trial name
- GEM-1 (NCT03536143)
- Testing
- Topical beremagene geperpavec
- Where
- United States
- Volunteers
- 12 people
- Sponsor
- Krystal Biotech, Inc.
Epidermolysis Bullosa Dystrophica, Squamous Cell Carcinoma
Rigosertib for RDEB-SCC
Epidermolysis bullosa (EB) is a heritable skin disease characterized by marked fragility of epithelialized tissue with blistering in skin and mucous membranes following the slightest mechanical trauma.
- Trial name
- NCT03786237
- Testing
- Rigosertib Oral Capsules / Rigosertib Intravenous
- Where
- Austria
- Volunteers
- 2 people
- Sponsor
- Prof. Johann Bauer
Epidermolysis Bullosa (EB), Epidermolysis Bullosa Acquisita
Targeting Collagen VII Antibodies in Bullous Diseases Using Efgartigimod IV (VYVGART)
The study objective is to see if IV Efgartigimod and Vyjuvek treatment in Recessive Dystrophic Epidermolysis Bullosa (RDEB) and IV Efgartigimod treatment in Epidermolysis Bullosa Acquisita (EBA) improves wound healing and affects…
- Conditions
- Epidermolysis Bullosa (EB), Epidermolysis Bullosa Acquisita, Recessive Dystrophic Epidermolysis Bullosa, Dystrophic Epidermolysis Bullosa
- Trial name
- NCT07011589
- Testing
- Efgartigimod
- Where
- United States
- Volunteers
- 18 people
- Sponsor
- M. Peter Marinkovich
Recessive Dystrophic Epidermolysis Bullosa
A Pilot Study to Evaluate a Temporary Skin Substitute (Spincare® Matrix) for Wound Healing in RDEB Patients
Recessive dystrophic epidermolysis bullosa (RDEB) is a subtype of epidermolysis bullosa (EB), an inherited skin condition that presents with blistering skin.
- Trial name
- RDEB (NCT05944250)
- Testing
- Spincare matrix
- Where
- United States
- Volunteers
- 6 people
- Sponsor
- Stanford University
Recessive Dystrophic Epidermolysis Bullosa
Molecular Signatures of Cutaneous Squamous Cell Carcinoma During Recessive Dystrophic Epidermolysis Bullosa
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…
- Trial name
- SIMOCEB (NCT04285294)
- Volunteers
- 66 people
- Sponsor
- Assistance Publique - Hôpitaux de Paris
Recessive Dystrophic Epidermolysis Bullosa
A Study of FCX-007 for Recessive Dystrophic Epidermolysis Bullosa
The purpose of this study is to determine whether administration of FCX-007 in addition to standard of care improves wound healing as compared to standard of care alone (control) in children, adolescents, and adults with…
- Trial name
- DEFI-RDEB (NCT04213261)
- Testing
- FCX-007 (dabocemagene autoficel; see below for FCX-007 description)
- Where
- 5 sites in United States
- Volunteers
- 6 people
- Sponsor
- Castle Creek Biosciences, LLC.