Clinical Trial to Assess Safety and Efficacy of Autologous Cultured Epidermal Grafts Containing...
Recessive Dystrophic Epidermolysis BullosaProspective 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 COL7A1 complementary DNA (cDNA) for restoration of the epidermis in patients with recessive dystrophic epidermolysis bullosa. The purpose of this study is to demonstrate the safety and efficacy after one or more treatments with genetically corrected cultured epidermal autograft (Hologene 7) in patients suffering of recessive dystrophic epidermolysis bullosa (RDEB) with COL7A1 mutation.
Evaluation of the Safety and Efficacy Study of RGN-137 Topical Gel for Junctional and Dystrophic...
Junctional Epidermolysis BullosaDystrophic Epidermolysis BullosaThe objective of this study is to compare the efficacy and safety of RGN-137 topical gel with that of placebo gel for treatment of junctional epidermolysis bullosa (JEB) or dystrophic epidermolysis bullosa (DEB).
Allogeneic Stem Cell Transplantation (ALLOSCT) in Recessive Dystrophic Epidermolysis Bullosa (RDEB)...
Epidermolysis BullosaReduced 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. To determine the event-free survival (EFS) and overall survival (OS) following RIC consisting of busulfan/fludarabine/alemtuzumab (BFA) and AlloSCT in selected patients with RDEB.
A Phase 2 Study on Effect of Thymosin Beta 4 on Wound Healing in Patients With Epidermolysis Bullosa...
Epidermolysis BullosaThe purpose of this study is to investigate a treatment to enhance the healing of acute and chronic nonhealing cutaneous wounds, such as the erosions experienced by patients with Epidermolysis Bullosa (EB), by the known activity of thymosin beta 4 (Tβ4). Funding Source - FDA Office of Orphan Product Development (OOPD).
A Double-blind, Randomized, Intra-subject Placebo-controlled, Multicenter, Multiple Dose Study,...
Epidermolysis Bullosa DystrophicaRecessive2 moreA 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 Study of FCX-007 for Recessive Dystrophic Epidermolysis Bullosa (RDEB)
Epidermolysis Bullosa DystrophicaRecessiveThe 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 with recessive dystrophic epidermolysis bullosa (RDEB). Funding Source- FDA OOPD
A Observational Study to Evaluate Apligraf(R) in Nonhealing Wounds of Subjects With Epidermolysis...
Epidermolysis BullosaWound HealingEpidermolysis Bullosa (EB) is a very rare disease, with a severe impact on the life of the patient and the caregiver. Epidermolysis Bullosa (EB) comprises a group of genetically determined skin fragility disorders characterized by blistering of the skin and mucosae following mild mechanical trauma. There is no specific proven treatment for any form of EB, and the mainstay of clinical management is based on protection and avoidance of provoking factors. Chronic nonhealing erosions and ulcers have been treated with conventional split-thickness skin grafts. Alternatively some patients may benefit from the use of autologous or allogeneic cultured keratinocyte grafts.
Open-Label Extension Study to Evaluate the Safety of SD-101 Cream in Participants With Epidermolysis...
Epidermolysis BullosaThe 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
Safety and Efficacy of Diacerein 1% Ointment for Subjects With Epidermolysis Bullosa Simplex (EBS)...
Epidermolysis Bullosa SimplexEpidermolysis bullosa simplex (EBS) is a rare genetic skin disease characterized by fragility of the skin and mucous membranes resulting in painful blisters and erosions after minor trauma. The purpose of this study is to compare the efficacy of diacerein 1% ointment to vehicle ointment when applied once-daily for 8 weeks in subjects with EBS.
Safety and Efficacy of Apligraf in Nonhealing Wounds of Subjects With Junctional or Dystrophic Epidermolysis...
Epidermolysis BullosaJunctional1 moreThe purpose of this study is to evaluate the use of Apligraf for the treatment of nonhealing wounds in subjects with dystrophic or junctional epidermolysis bullosa. Apligraf will be evaluated for efficacy and safety compared to a conventional nonadherent dressing. A matched-pair design will be used to evaluate Apligraf treatment versus conventional treatment in 68 study pairs.