Clinical trials: RDEB
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.
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
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 Study of PTR-01 in Recessive Dystrophic Epidermolysis Bullosa
Protocol PTR-01-002 is a 3-part Phase 2, open-label study of PTR-01. While new patients will be enrolled, priority will be given to patients that satisfactorily completed study PTR-01-001.
- Trial name
- NCT04599881
- Testing
- PTR-01
- Where
- 2 sites in United States
- Volunteers
- 6 people
- Sponsor
- Phoenix Tissue Repair, Inc.
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
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
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
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.
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.
Recessive Dystrophic Epidermolysis Bullosa
Mesenchymal Stromal Cells in Adults With Recessive Dystrophic Epidermolysis Bullosa
To assess whether intravenously administered third-party bone marrow-derived mesenchymal stromal cells (MSCs) are safe and have an impact on disease severity in RDEB
- Trial name
- ADSTEM (NCT02323789)
- Testing
- Mesenchymal stromal cells
- Where
- United Kingdom
- Volunteers
- 10 people
- Sponsor
- King's College London
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
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
Study of Immune Tolerance and Capacity for Wound Healing of Patients With Recessive Dystrophic Epidermolysis Bullosa (RDEB)
Recessive Dystrophic Epidermolysis Bullosa (RDEB) is one of the most severe rare inherited skin disorders affecting children and adults.
- Trial name
- RDEB (NCT01874769)
- Testing
- Blood collection, Skin biopsies
- Where
- 3 sites in France, United Kingdom
- Volunteers
- 30 people
- Sponsor
- Institut National de la Santé Et de la Recherche Médicale, France
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
Epidermolysis Bullosa Dystrophica, Recessive, Epidermolysis Bullosa Dystrophica, Dominant
A Double-blind, Randomized, Intra-subject Placebo-controlled, Multicenter, Multiple Dose Study, Evaluating Safety, Proof of Mechanism, Preliminary Efficacy and Systemic Exposure in Subjects With Confirmed DDEB or RDEB Diagnosis With One or More Pathogenic Mutations in Exon 73 in the COL7A1 Gene
A double-blind, randomized, intra-subject placebo-controlled, multicenter, multiple dose study, evaluating safety, proof of mechanism, preliminary efficacy and systemic exposure in subjects with confirmed DDEB or RDEB diagnosis…
- Trial name
- NCT03605069
- Testing
- QR-313
- Where
- 6 sites in United States, France and 1 more
- Volunteers
- 2 people
- Sponsor
- Phoenicis Therapeutics
Recessive Dystrophic Epidermolysis Bullosa
Extension Study to PTR-01-002 (A Study in Recessive Dystrophic Epidermolysis Bullosa (RDEB) Patients Previously Treated With PTR-01)
A sub-set of patients who participated in PTR-01-002 will be enrolled in an open-label study, if they meet the study eligibility criteria.
- Trial name
- NCT05143190
- Testing
- PTR-01
- Where
- 2 sites in United States
- Volunteers
- 2 people
- Sponsor
- Phoenix Tissue Repair, Inc.
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