Clinical trials: Recessive Dystrophic Epidermolysis Bullosa
Epidermolysis Bullosa
Phase I Study of Isotretinoin in Patients With Recessive Dystrophic Epidermolysis Bullosa
OBJECTIVES: I. Determine the safety of isotretinoin in patients with recessive dystrophic epidermolysis bullosa.
- Trial name
- NCT00014729
- Testing
- isotretinoin
- Volunteers
- 20 people
- Sponsor
- University of North Carolina
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
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
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
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
Recessive Dystrophic Epidermolysis Bullosa
Allogeneic ABCB5-positive Stem Cells for Treatment of Epidermolysis Bullosa
The aim of this clinical trial is to investigate the efficacy (by monitoring overall improvement of EB symptoms) and safety (by monitoring adverse events) of three doses of allo-APZ2-EB administered intravenously to patients with…
- Trial name
- NCT03529877
- Testing
- allo-APZ2-EB
- Where
- 6 sites in United States, Austria and 3 more
- Volunteers
- 16 people
- Sponsor
- RHEACELL GmbH & Co. KG
Epidermolysis Bullosa
Survey to Identify Burdens and Unmet Needs of Patients With Epidermolysis Bullosa
This survey intends to collect information on key aspects of life with epidermolysis bullosa (EB), including diagnostic journey, treatment, management, daily living challenges, and overall psycho-social, socio-economic, academic…
- Trial name
- NCT03158662
- Testing
- Survey
- Where
- United States
- Volunteers
- 150 people
- Sponsor
- Amicus Therapeutics
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
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
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
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, 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
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, 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
Characteristics of Adult Patients With Recessive Dystrophic Epidermolysis Bullosa
Recessive dystrophic epidermolysis bullosa (RDEB) is a severe inherited blistering disease caused by the absence of type VII collagen.
- Trial name
- NCT00904163
- Where
- United States
- Volunteers
- 13 people
- Sponsor
- Stanford University
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
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
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
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
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.