Clinical trials: DEB - Dystrophic Epidermolysis Bullosa
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
Epidermolysis Bullosa, Recessive Dystrophic Epidermolysis Bullosa
Phase 3, Open-label Clinical Trial of EB-101 for the Treatment of Recessive Dystrophic Epidermolysis Bullosa (RDEB)
The purpose of this trial is to evaluate safety and efficacy of surgical application of EB-101 (autologous, gene-corrected keratinocyte sheets) as a treatment of recessive dystrophic epidermolysis bullosa (RDEB).
- Trial name
- NCT04227106
- Testing
- EB-101
- Where
- 2 sites in United States
- Volunteers
- 11 people
- Sponsor
- Abeona Therapeutics, Inc
Recessive Dystrophic Epidermolysis Bullosa
Clinical Trial to Assess Safety and Efficacy of Autologous Cultured Epidermal Grafts Containing Epidermal Stem Cells Genetically Modified in Patients With RDEB.
Prospective open-label, uncontrolled clinical study to assess the safety and efficacy of autologous cultured epidermal grafts containing epidermal stem cells genetically modified with the aid of a gamma-retroviral vector carrying…
- Trial name
- HOLOGENE7 (NCT02984085)
- Testing
- Genetically corrected cultured epidermal autograft (ATMP)
- Where
- Austria
- Volunteers
- 3 people
- Sponsor
- Holostem s.r.l.
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
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
Dystrophic Epidermolysis Bullosa
Efficacy of Granulocyte Colony Stimulating Factor (GCSF) In Patients With Dystrophic Epidermolysis Bullosa
This is a feasibility study to see if Granulocyte Colony Stimulating Factor (GCSF) is effective as a treatment of Dystrophic Epidermolysis Bullosa (EB). Patients will receive one course of treatment with the study drug.
- Trial name
- NCT01538862
- Testing
- Granulocyte Colony Stimulating Factor (GCSF)
- Where
- United States
- Volunteers
- 7 people
- Sponsor
- Vanderbilt University Medical Center
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
Allogeneic Stem Cell Transplantation (ALLOSCT) in Recessive Dystrophic Epidermolysis Bullosa (RDEB)
Reduced Intensity Conditioning (RIC) and Allogeneic Stem Cell Transplantation (AlloSCT) from family-related donors and unrelated cord blood (UCB) donors will be safe and well tolerated in selected patients with RDEB.
- Trial name
- RDEB (NCT00881556)
- Testing
- Palifermin, Fludarabine, Busulfan
- Where
- 3 sites in United States
- Volunteers
- 3 people
- Sponsor
- Columbia University
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 (EB), Epidermolysis Bullosa Simplex
To Evaluate the Pharmacokinetic of Diacerein and Rhein After Maximum Use in Patients With Epidermolysis Bullosa (EB)
A pharmacokinetic (PK) study in 16-20 EB subjects to be allocated to two cohorts. Cohort 1 to include 8-10 subjects (ages 12 yrs and older); Cohort 2 to include 8-10 subjects (ages 6 months-11 yrs, inclusive).
- Conditions
- Epidermolysis Bullosa (EB), Epidermolysis Bullosa Simplex, Dystrophic Epidermolysis Bullosa, Junctional Epidermolysis Bullosa
- Trial name
- NCT03472287
- Testing
- Diacerein 1% Ointment
- Where
- 3 sites in United States
- Volunteers
- 11 people
- Sponsor
- Castle Creek Pharmaceuticals, LLC
Dystrophic Epidermolysis Bullosa
Study to Evaluate the Safety of ALLO-ASC-DFU in the Subjects With Dystrophic Epidermolysis Bullosa
This is a phase I/II open-label study to evaluate the efficacy and safety of ALLO-ASC-DFU in patients with Dystrophic Epidermolysis Bullosa.
- Trial name
- NCT02579369
- Testing
- Allogeneic mesenchymal stem cells, Polyurethene Film
- Where
- South Korea
- Volunteers
- 2 people
- Sponsor
- Anterogen Co., Ltd.
Epidermolysis Bullosa, Junctional, Epidermolysis Bullosa Dystrophica
Safety and Efficacy of Apligraf in Nonhealing Wounds of Subjects With Junctional or Dystrophic Epidermolysis Bullosa (EB)
The purpose of this study is to evaluate the use of Apligraf for the treatment of nonhealing wounds in subjects with dystrophic or junctional epidermolysis bullosa.
- Trial name
- NCT00587223
- Testing
- Apligraf, Standard dressing regimen
- Where
- 5 sites in United States
- Volunteers
- 1 people
- Sponsor
- Organogenesis
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
Intravenous Gentamicin Therapy for Recessive Dystrophic Epidermolysis Bullosa (RDEB)
Recessive dystrophic epidermolysis bullosa (RDEB) is an incurable, devastating, inherited skin disease caused by mutations in the COL7A1 gene that encodes for type VII collagen (C7), the major component of anchoring fibrils…
- Trial name
- NCT03392909
- Testing
- Gentamicin
- Where
- United States
- Volunteers
- 9 people
- Sponsor
- University of Southern California
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.
Squamous Cell Carcinoma
Impact of COL7A1 Gene Therapy on SCC Recurrence in RDEB Skin
The study objective is to see if BVEC induced C7 expression in Recessive Dystrophic Epidermolysis Bullosa (RDEB) skin following Squamous cell carcinoma (SCC) excision will normalize the invasive tumor microenvironment and reduce…
- Trial name
- NCT06731933
- Testing
- BVEC
- Where
- 2 sites in United States, Italy
- Volunteers
- 16 people
- Sponsor
- Stanford University
Dystrophic Epidermolysis Bullosa, DEB - Dystrophic Epidermolysis Bullosa
The Natural History of Wounds in Patients With Dystrophic Epidermolysis Bullosa (DEB)
This study is a non-interventional, observational study that will evaluate the natural history of wounds in patients with Dystrophic Epidermolysis Bullosa (DEB) for inclusion into the Krystal Biotech Phase III protocol of B-VEC…
- Trial name
- NCT04214002
- Where
- United States
- Sponsor
- Krystal Biotech, Inc.
Dystrophic Epidermolysis Bullosa, Recessive Dystrophic Epidermolysis Bullosa
Ph 3 Efficacy and Safety of B-VEC for the Treatment of DEB
To determine whether administration of topical B-VEC improves wound healing as compared to placebo, and to evaluate durability, repeat dosing (Primary Endpoint) and further obtain safety and tolerability data.
- Conditions
- Dystrophic Epidermolysis Bullosa, Recessive Dystrophic Epidermolysis Bullosa, Dominant Dystrophic Epidermolysis Bullosa
- Trial name
- GEM-3 (NCT04491604)
- Testing
- Topical Beremagene Geperpavec
- Where
- 3 sites in United States
- Volunteers
- 31 people
- Sponsor
- Krystal Biotech, Inc.
Dystrophic Epidermolysis Bullosa, Recessive Dystrophic Epidermolysis Bullosa
Long-Term Follow-up Protocol
The main objective of this prospective, observational, long-term follow-up (LTFU) study is to evaluate the long-term safety profile of the gene therapy products evaluated by Krystal Biotech, Inc.
- Conditions
- Dystrophic Epidermolysis Bullosa, Recessive Dystrophic Epidermolysis Bullosa, Dominant Dystrophic Epidermolysis Bullosa
- Trial name
- NCT04917887
- Where
- 3 sites in United States
- Volunteers
- 50 people
- Sponsor
- Krystal Biotech, Inc.