Clinical trials: Recessive Dystrophic Epidermolysis Bullosa
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
Open-Label Extension Study to Evaluate the Safety of SD-101 Cream in Participants With Epidermolysis Bullosa
The purpose of this study was to assess the continued safety of topical use of SD-101 cream (6%) in participants with Epidermolysis Bullosa (EB). Funding Source: FDA Office of Orphan Products Development
- Trial name
- NCT02090283
- Testing
- SD-101 dermal cream (6%)
- Where
- 7 sites in United States
- Volunteers
- 42 people
- Sponsor
- Scioderm, Inc.
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
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
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, 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.
Epidermolysis Bullosa Dystrophica, Recessive
Safety Study and Preliminary Efficacy of Infusion Haploidentical Mesenchymal Stem Cells Derived From Bone Marrow for Treating Recessive Dystrophic Epidermolysis Bullosa
Phase I / II pilot clinical trial, to evaluate the safety and preliminary efficacy of the systemic infusion of mesenchymal stem cells derived from bone marrow (BM-MSCs) from a haploidentical donor to improve the healing process…
- Trial name
- NCT04153630
- Testing
- mesenchymal stem cells derived from bone marrow (BM-MSCs)
- Where
- Spain
- Volunteers
- 9 people
- Sponsor
- Instituto de Investigación Hospital Universitario La Paz
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
Epidermolysis Bullosa Dystrophica
Study of the Blood and Skin Immunological Profile of Patients With Recessive Dystrophic Epidermolysis Bullosa: in Vivo Analysis and the Impact of Placental Stem Cells in Vitro
Patients with recessive dystrophic epidermolysis bullosa (RDEB) suffer from acute and chronic post-bullous wounds along with impaired skin healing.
- Trial name
- ISTRADEB (NCT06177353)
- Testing
- Sampling
- Where
- 2 sites in France
- Volunteers
- 30 people, healthy volunteers welcome
- Sponsor
- Assistance Publique - Hôpitaux de Paris
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
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
Epidermolysis Bullosa Dystrophica, Epidermolysis Bullosa
Treatment of Chronic and Non-Chronic Wounds in Patients With Recessive Dystrophic Epidermolysis Bullosa Using Helicoll Collagen Dressings Versus Standard of Care
The purpose of this study is to test the effectiveness of Helicoll (a collagen wound dressing) in treating chronic and non-chronic wounds of recessive dystrophic epidermolysis bullosa (RDEB) patients.
- Trial name
- NCT01716169
- Testing
- Helicoll, Standard of Care Dressings
- Where
- United States
- Volunteers
- 10 people
- Sponsor
- Stanford University
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