Clinical trials: RDEB, active
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
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
Junctional Epidermolysis Bullosa, Epidermolysis Bullosa (EB)
Pilot Study of ELK-003 Eye Drops for Treating Ocular Manifestations of Epidermolysis Bullosa
This study consists of two phases: an Observational Phase to evaluate the natural history of ocular manifestations in subjects with Dystrophic and Junctional Epidermolysis Bullosa, followed by a Treatment Phase to assess the…
- Conditions
- Junctional Epidermolysis Bullosa, Epidermolysis Bullosa (EB), Dystrophic Epidermolysis Bullosa, Recessive Dystrophic Epidermolysis Bullosa
- Trial name
- GOTAS-ELK-EB (NCT06713434)
- Testing
- ELK-003 (Standarized Amniotic Fluid Secretome Eye Drops)
- Where
- Chile
- Volunteers
- 20 people
- Sponsor
- Fundación DEBRA Chile, Niños Piel de Cristal
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
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
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
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
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
Epidermolysis Bullosa Dystrophica, Recessive
Ex Vivo Gene Therapy Clinical Trial for RDEB Using Genetically Corrected Autologous Skin Equivalent Grafts
This phase I/II clinical trial aims to treat 3 adult subjects with Recessive Dystrophic Epidermolysis Bullosa, expressing residual C7 levels, by genetically corrected autologous skin equivalent grafts on selected areas (up to 300…
- Trial name
- EBGraft (NCT04186650)
- Testing
- COL7A1-SIN retroviral vector engineered autologous tissue-engineered skin
- Where
- France
- Volunteers
- 3 people
- Sponsor
- Institut National de la Santé Et de la Recherche Médicale, France
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
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.
Dystrophic Epidermolysis Bullosa, DEB - Dystrophic Epidermolysis Bullosa
A Study Comparing KB803 and Matched Placebo in Patients With Dystrophic Epidermolysis Bullosa
KB803-EYE-01 is a Phase 3 double-blind, randomized, placebo-controlled, crossover study to evaluate the safety and efficacy of KB803 versus matched placebo in pediatric and adult subjects with recurrent corneal abrasions due to…
- Conditions
- Dystrophic Epidermolysis Bullosa, DEB - Dystrophic Epidermolysis Bullosa, Recessive Dystrophic Epidermolysis Bullosa, Dominant Dystrophic Epidermolysis Bullosa
- Trial name
- IOLITE (NCT07016750)
- Testing
- KB803
- Where
- 5 sites in United States
- Volunteers
- 16 people
- Sponsor
- Krystal Biotech, Inc.
Epidermolysis Bullosa Dystrophica
Self-Assembled Skin Substitute for the Treatment of Epidermolysis Bullosa
Single patient study. Patient diagnosed with dystrophic epidermolysis bullosa presenting chronic open wounds that are not responding to dressings, topical preparations (antimicrobials, antibiotics) and systemic agents…
- Trial name
- SASS (NCT04171661)
- Testing
- SASS
- Where
- Canada
- Volunteers
- 1 people
- Sponsor
- CHU de Quebec-Universite Laval
Dystrophic Epidermolysis Bullosa
MSC EVs in Dystrophic Epidermolysis Bullosa
INVESTIGATIONAL PRODUCT: AGLE-102 is an allogeneic extracellular vesicle (EV) product derived from normal donor mesenchymal stem cells (MSCs).
- Trial name
- NCT04173650
- Testing
- AGLE-102
- Where
- 3 sites in United States
- Volunteers
- 8 people
- Sponsor
- Aegle Therapeutics
Epidermolysis Bullosa Dystrophica
Artificial Intelligence Patient App for RDEB SCCs
In this study, an artificial intelligence model to detect squamous cell carcinomas (SCC) on photos of recessive dystrophic epidermolysis bullosa (RDEB) skin is developed.
- Trial name
- NCT05843994
- Testing
- online survey
- Where
- United States
- Volunteers
- 20 people
- Sponsor
- Northwestern University
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
Epidermolysis Bullosa, Epidermolysis Bullosa Acquisita
Targeting Collagen VII Antibodies With IV IgG in Dystrophic Epidermolysis Bullosa
The study objective is to see if IV IgG treatment in Recessive Dystrophic Epidermolysis Bullosa (RDEB) skin in conjunction with VYJUVEK treatment improves wound healing and affects the levels of C7 and HSV-1 antibody levels in…
- Conditions
- Epidermolysis Bullosa, Epidermolysis Bullosa Acquisita, Dystrophic Epidermolysis Bullosa, Recessive Dystrophic Epidermolysis Bullosa
- Trial name
- IV IgG (NCT06834035)
- Testing
- Immunoglobulin G
- Where
- United States
- Volunteers
- 8 people
- Sponsor
- M. Peter Marinkovich
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.