Active clinical trials for "Epidermolysis Bullosa"

This pilot trial studies how rigsertib sodium works in treating patients with Recessive Dystrophic Epidermolysis bullosa (RDEB) with locally advanced Squamous Cell Carcinoma (SCC). Rigosertib may selectively target Epidermolysis bullosa (EB) cancer cells while leaving normal EB cells unaffected.

The aim of this clinical trial is to investigate the safety and efficacy of allo-APZ2-OTS administered intravenously to subjects with recessive dystrophic epidermolysis bullosa (RDEB) compared to placebo. An additional baseline-controlled open-label arm will be included to investigate the safety and efficacy of allo-APZ2-OTS administered intravenously to subjects with JEB and to RDEB subjects < 1 year.

In this pilot study, APR-TD011 antimicrobial wound cleansing spray will be given to all enrolled patients with junctional EB (JEB) or dystrophic epidermolysis bullosa (DEB) with Staphylococcus aureus or Pseudomonas aeruginosa culture-positive wounds. The primary aim will be to evaluate the change in skin microbiome (S. aureus, P. aeruginosa, commensal organisms) before, during, after treatment. Subjects who are colonized by S. aureus or pseudomonas will be treated for 8 weeks, will stop the spray and return at 12 weeks (4 weeks without the spray), and then will be able to use the spray as desired in a 6-month period of open-label use, with further feedback collected.

The purpose of this study is to determine whether administration of FCX-007 in addition to standard of care improves wound healing as compared to standard of care alone (control) in children, adolescents, and adults with Recessive Dystrophic Epidermolysis Bullosa. Funding Source - FDA OOPD

This phase I/II clinical trial aims to treat 3 adult subjects with Recessive Dystrophic Epidermolysis Bullosa, expressing residual C7 levels, by genetically corrected autologous skin equivalent grafts on selected areas (up to 300 cm2).

Recessive dystrophic epidermolysis bullosa (RDEB) patients' quality of life is severely affected by neuropathic pain and itch, which have recently been demonstrated to be secondary to skin small fiber neuropathy. To date, there is no evidence on what the best agent is to control these symptoms. Based on the anecdotal data and safety profile, the investigators believe that pregabalin is a therapeutic agent that will be effective and safe in this population. The investigators propose to conduct a blinded study, using pregabalin versus placebo in which each patient serves as its own control (cross-over design). This is a feasibility study that will provide preliminary data on efficacy and safety of pregabalin in RDEB patients with neuropathic pain and itch and gather much needed data (dosage, titration schedule, outcome measures, etc) to inform the design of a larger cohort, controlled, multicenter trial.

Single patient study. Patient diagnosed with dystrophic epidermolysis bullosa presenting chronic open wounds that are not responding to dressings, topical preparations (antimicrobials, antibiotics) and systemic agents (anti-inflammatory antibacterials). The Self-Assembled Skin Substitutes will be used to cover wounds.

Inherited Epidermolysis Bullosa (EB) is a disorder that causes skin fragility and blistering in skin and mucous membranes, including the mouth. Recurrent oral blisters and ulcer result in oral pain and discomfort. Dentoxol® is a mouthrinse that has anti-inflammatory, antimicrobial and analgesic effects. It has significant potential to reduce EB related oral symptoms. This study includes people living with Inherited Epidermolysis Bullosa aged 6 and above; and is aimed at determining the efficacy of two different dose regimens of Dentoxol mouthrinse in reducing oral symptoms.

The study will compare gene expression differences between blistered and non-blistered skin from individuals with all subtypes of EB, as well as normal skin from non-EB subjects. State of the art computational analysis will be performed to help identify new drugs that might help all EB wound healing and reduce pain. Researchers will focus on drugs that have already been approved for treatment of other dermatologic or non-dermatologic diseases, and therefore be repurposed for treatment of EB. Drug development is a very expensive process taking decades for execution. Drug repurposing on the other hand, significantly reduces the cost and shortens the amount of time that is needed to bring effective treatments to clinical use. To date, there is no specific treatment targeting the physiology and immunologic response in EB patients during wound healing. Market availability of repurposed medications will provide all EB patients rapid access to treatments, thus improving their quality of life.

The proposed Phase 2/3 trial with double-blind and open-label extension phases is an international, multicenter study designed to assess the efficacy and safety of diacerein 1% ointment in patients with generalized EBS.