View clinical trials related to Epidermolysis Bullosa.
Filter by:This study will collect genetic and clinical information of junctional epidermolysis bullosa (JEB) patients. Computer analysis will be performed on genetic mutations found in these patients and this will be correlated with their clinical characteristics.
The overall purpose of this study is to address whether topical gentamicin therapy is an effective and feasible treatment. Specifically, we will investigate the effect of non-intensive treatment (once daily or every other day) on skin protein expression, as well as quantify the effect on wound healing in patients with EB caused by PSC (part A). Furthermore, we will address in vitro whether gentamicin restores protein expression of genes affected by SSM in fibroblasts derived from skin biopsies obtained from patients with EB caused by SSM (part B). If these in vitro experiments yield positive results, the patients donating the cells will be offered to enter part A of this study. The overall duration of part A in this study is planned to be 18 weeks per patients and consists of a 6 weeks treatment period followed by a 12 week follow up period. Each patient will attend 3 study visits: at week 0, week 6 and week 18. All patients will be included within a time period of 12 months. The overall duration of part B will be up to 8 weeks per patients of which 4-7 weeks are spent to prepare fibroblasts obtained from skin biopsies. Then 5 days of in vitro intervention and subsequent analysis follows. Altogether, the duration of the GENTELBULL study will be 78 weeks or less.
Epidermolysis bullosa (EB) is rare, devastating, and currently incurable genetic blistering disorders characterized by fragility of skin and mucosal membranes. Therapeutic options for EB are limited. Acute and chronic wounds cause pain, itching and infection, altering quality of life and impair wound healing. In absence of a cure, wound care is paramount to alleviate suffering. Anecdotal experience suggest that CBD application alleviates the pain and itching and improves wound healing by controlling the inflammatory process. We propose to undertake a phase II/III study exploring the safety, tolerability and efficacy of topical application of 3% Cannabidiol cream (CBD) on acute and chronic wounds affecting patients with EB. We are doing this trial to determine safety and tolerability of topical CBD cream in a cohort of RDEB patients with chronic wounds (phase II trial), and to evaluate the efficacy and safety of CBD cream in EB with acute and chronic wounds in promoting wound healing, decrease blister formation, pain, itching and improve overall quality of life (phase III trial)
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.
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. In the case of cord blood as a source, the isolation of mesenchymal stem cells is easier than bone marrow or fat tissue, and cord blood-derived mesenchymal stem cells have an advantage as a treatment because they have faster population doubling time. To date, no clinical research on the treatment of patients using cord blood-derived mesenchymal stem cells has been reported in the literature, but there have already been registered at clinicaltrials.gov and currently being conducted overseas. In this study, we will study the safety and effectiveness of RDEB patient treatment using cord blood-derived mesenchymal stem cells with these advantages.
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.
Recessive dystrophic epidermolysis bullosa (RDEB) is a hereditary skin disease characterized by cutaneous and mucosa fragility. Blister formations and erosions, resulting in chronic wounds and dystrophic scars, lead development of aggressive cutaneous squamous cell carcinoma (cSCC) in young subjects. cSCC in RDEB patients are often recurrent and sometimes aggressive. Although fibrotic and inflammatory microenvironment plays an important role in the tumoral process, specific mechanisms in cSCC of RDEB patients are still unknown. Actually, the only treatment is a wide surgical excision with poor prognostic (80% of death after the first occurrence of cSCC). The objective of the study is to describe the molecular signatures in the cSCC in RDEB patients
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).
Hereditary Epidermolysis Bullosa (EBH) are rare dermatologic diseases characterized by cutaneous and mucosa fragility. Oral manifestations of few small cohort have been published. The main objective of this multicentric cohort study first in Europe was to report the oral status of these patients that were consulted in the MRDRC of this disease in Nice (France), Toulouse (France) and Louvain (Belgium). Then a correlation between the oral characteristics and the EBH type will be made, in order to facilitate the management of patient care and the prevention program that can be established to improve their oral health.
This study is a non-interventional, observational study that will evaluate the natural history of wounds in patients with Dystrophic Epidermolysis Bullosa (DEB) for inclusion into the Krystal Biotech Phase III protocol of B-VEC (previously KB103). Wound recurrence and wound size will be evaluated for up to four months.