Active clinical trials for "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.

The aim was to assess the efficacy and safety of SD-101-6.0 cream versus Placebo (SD-101-0.0) cream in the treatment of skin lesions in participants with Epidermolysis Bullosa. Funding Source - United States Food and Drug Administration (FDA) Office of Orphan Products Development (OOPD).

This is a single-institution, phase II study to determine the event-free survival at 1 year post allogeneic transplant and serial mesenchymal stem cell (MSC) infusions from a related donor (HLA identical, mismatched or haploidentical) or matched unrelated donor for the biochemical correction of severe epidermolysis bullosa (EB).

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. The corrected cells will be transplanted back to the patient.

Dystrophic epidermolysis bullosa hereditaria are genodermatosis responsible for formation of cutaneous bullous lesion arising spontaneously or after mechanical trauma. These lesions are due to mutation on gene COL7A1 coding for collagen VII. There is no treatment available. Cares are consisting to dress lesions and to protect the skin. The investigators have recently observed on patients having residual expression of collagen VII that phenotype severity is modulated by activation degree of dermic metalloproteinase. The investigators have also observed that epigallocatechin-3-gallate (Polyphenon E®) could be regulated this activity. The primary purpose of this study is to assessing the efficacity of Polyphenon E to decrease the number of cutaneous bullosa after four month of treatment. The primary outcome measure is the rate of patient presenting a decrease of 20% or more of the number of cutaneous bullosa. Secondary outcomes are: severity of mucosa impairment, affected cutaneous surface, the average duration of cicatrisation and treatment tolerance. This study foresees the inclusion of 22 patients older than 2 years old in 5 centers. When patients are included, they will be randomized and receive the treatment (or placebo) for 4 months.

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)

The term epidermolysis bullosa (EB) is used to describe a group of genetic skin diseases associated with skin weakness, blisters, and chronic wounds. "Revertant mosaicism" means that there are two genetically different populations of cells due to spontaneous mutations. Some EB patients have normal, non-fragile skin patches which may be areas of revertant mosaicism. In the revertant areas, the proteins function normally, like non-EB skin. In this study, we plan to culture cells from the revertant areas and graft them on to the wounded areas.

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 hereditary dystrophic epidermolysis bullosa are genodermatosis responsible of a poor adhesion of the epidermis to the dermis pulling a large mucocutaneous fragility and recurrent spontaneous or posttraumatic bullous detachment. They are caused by mutations in the COL7A1 gene encoding for the collagen VII. No curative treatment is avaible. The main cause of patients death is the development of squamous cell carcinoma, sometimes multiple and paticularly aggressive in repeated healing part. The photodynamic therapy (PDT) is one of technical reference of multiple actinic keratoses lesions for adults, which are also pre-epithelioma lesions. The PDT is well tolerated even by the elderly and requires only a single session. The main objective of this study is to determine the efficiency of the photodynamic therapy in the treatment of epidermic dysplasies for patients affected by dystrophic epidermolysis bullosa (DEB). The secondary objectives are to evaluate the tolerance of this treatment in terms of pain and healing, and to evaluate the contribution of confocal microscopy in the diagnosis of epidermal dysplasia for patients affected by hereditary dystrophic epidermolysis bullosa. The main evaluation criterion is the cutaneous biopsy before and after (M2) a PDT session of an epidermal dysplasia area. The secondary criteria are the evaluation of the pain during the PDT session and the healing of the cutaneous lesion at M0, M2 and M4 (lesion area and healing time) and correlation histology / MC. Each patient with a suspicious lesion will be biopsied. In case of agreement for this protocol, there will be 1 PDT session followed by a consultation of control at 2 and 4 months after the end of treatment.

This is a pilot study designed to see if HP802-247, an investigational treatment with living human skin cells, helps to heal blisters or wounds in subjects with Dystrophic Epidermolysis Bullosa (DEB). The durability of the skin in healed wounds treated with HP802-247 will also be assessed.