Active clinical trials for "Epidermolysis Bullosa"

A phase II, comparative, open label, prospective, multicentre clinical trial where each patient will undergo two procedures; implant of a patch of cultured chimeric skin (experimental therapy) in a half of the skin lesion and an occlusive non-adherent dressing (control) in the other half for 12 months of follow-up in two Spanish centres.

OBJECTIVES: I. Determine the safety of isotretinoin in patients with recessive dystrophic epidermolysis bullosa.

Hereditary epidermolysis bullosa (EBH) is a rare, orphan disease characterized by skin and mucous membrane fragility. The latest scientific data show that the proposed treatments are still in the experimental stage and that no curative treatment is available. The repercussions of this chronic disease, with neonatal onset, are major. Epidermolysis bullosa requires multidisciplinary medical management, nursing care, psychological and social care. Skin care involves preventing and treating chronic wounds and identifying their complications. The very great cutaneous-mucous fragility makes these treatments painful, long and complex, the caring hand itself being able to cause new wounds. Analgesics of different levels are not effective enough during treatment. Along with counseling and education, nursing takes a central role in multi-professional accompaniment interventions to support and relieve families. Parents became home caregivers out of necessity, and developed specific skills in epidermolysis bullosa, their child and dressings. They have great and demanding expectations of caregivers facing this rare disease, for which they are not trained in their degree course. Despite the severe nature of the disease, few studies have been carried out on the impact and psychosocial consequences on patients and their families, yet there is an expressed need for support. The burden on parents is heavy, assessed by specific scales, but to date there are no studies examining the impact of epidermolysis bullosa care on caregiver stress.

The purpose of this study was to assess whether the topical use of SD-101 cream (3% or 6%) was effective in treating wounds in participants with Simplex, Recessive Dystrophic, or Junctional non-Herlitz Epidermolysis Bullosa (EB).

The primary objective of this study is to evaluate the long term safety and tolerability of diacerein 1% ointment for 2 treatment cycles in subjects with EBS that previously participated in the CCP-020-301 or the CCP-020-101 studies.

This study was conducted to assess the safety and efficacy of topical Beremagene Geperpavec (KB103, HSV1-COL7) on DEB patients.

The purpose of this study is to determine whether topical application of Ropivacaine is effective for treating refractory pain during dressing changes and so improve quality of life of patients (newborn, child, adolescent or adults under 21) suffering from hereditary epidermal epidermolysis bullosa.

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 (AFs), structures that mediate epidermal-dermal adherence. Thirty percent of RDEB patients have nonsense mutations. The investigators recently demonstrated in 5 such patients that intradermal and topical gentamicin induced "read-through" of their nonsense mutations and created robust and sustained new C7 and AFs at the dermal-epidermal junction (DEJ) of their skin and also stimulated wound closure and reduced new blister formation. No untoward side effects occurred. Herein, the investigators propose evaluating the safety and efficacy of intravenous gentamicin in these patients. In theory, this intravenous administration has the possibility of treating simultaneously all of the patients' skin wounds. The investigators also propose optimizing the concentration and manner of delivery of topical gentamicin. The unambiguous milestones will be increased C7 and AFs in the patients' DEJ, improved EB Disease Activity Scores, and absence of significant gentamicin side effects.

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).

The purpose of this study is to assess the efficacy of trimethoprim in promoting wound healing and decreasing blister formation in patients with Epidermolysis Bullosa.