Clinical trials: RDEB
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.
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, 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.
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
A Phase 1/2 Trial of PTR-01 in Adult Patients With Recessive Dystrophic Epidermolysis Bullosa (RDEB)
Protocol PTR-01-001 is a Phase 1/2 study of PTR-01. The study is divided into an up to 4-week Screening Period, a 10-week Treatment Period and an 8-week Follow-up Period.
- Trial name
- PTR-01-001 (NCT03752905)
- Testing
- PTR-01, Normal saline
- Where
- 3 sites in United States
- Volunteers
- 12 people
- Sponsor
- Phoenix Tissue Repair, Inc.
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
Pain, Neuropathic, Itch
Pregabalin Treatment for RDEB Pain and Itch
Recessive dystrophic epidermolysis bullosa (RDEB) patients' quality of life is severely affected by neuropathic pain and itch, which have recently been demonstrated to be secondary to skin small fiber neuropathy.
- Conditions
- Pain, Neuropathic, Itch, Epidermolysis Bullosa
- Trial name
- NCT03928093
- Testing
- Pregabalin
- Where
- Canada
- Volunteers
- 1 people
- Sponsor
- The Hospital for Sick Children
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
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.
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, Recessive, Ulcer
A Comparative Study of the Healing of Chronic Ulcers of Recessive Epidermolysis Bullosa : Dressing vs Amniotic Membrane
Skin wounds of Recessive Epidermolysis Bullosa Dystrophica (REBD) involve pain, superinfection, protein-losing, inflammation, and joint contractures are the bed of squamous cell carcinoma.
- Trial name
- MABUL (NCT02286427)
- Testing
- Amniotic Membrane
- Where
- France
- Volunteers
- 22 people
- Sponsor
- Assistance Publique - Hôpitaux de Paris
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 (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
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 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
Gentamicin for 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
- NCT03012191
- Testing
- Gentamicin Sulfate
- Where
- United States
- Volunteers
- 6 people
- Sponsor
- University of Southern California
Dystrophic Epidermolysis Bullosa, DEB - Dystrophic Epidermolysis Bullosa
A Long-term Treatment With B-VEC for Dystrophic Epidermolysis Bullosa
This is a 112-week (approximately two-year) open-label extension study of Beremagene Geperpavec (B-VEC), for participants aged 2 months and older, who have been diagnosed with Dystrophic Epidermolysis Bullosa (DEB).
- Conditions
- Dystrophic Epidermolysis Bullosa, DEB - Dystrophic Epidermolysis Bullosa, Recessive Dystrophic Epidermolysis Bullosa, Dominant Dystrophic Epidermolysis Bullosa
- Trial name
- NCT04917874
- Testing
- Open Label Topical Beremagene Geperpavec (B-VEC)
- Where
- 6 sites in United States
- Volunteers
- 47 people
- Sponsor
- Krystal Biotech, Inc.
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
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
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.