12 Clinical Trials for Various Conditions
The 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.
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.
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.
Herlitz junctional epidermolysis bullosa (H-JEB), an incurable, fatal, inherited skin disease, is caused by loss-of-function mutations in the LAMA3, LAMB3 or LAMC2 genes, resulting in loss of laminin 332 and poor epidermal-dermal adherence. Eighty percent of H-JEB patients have LAMB3 mutations and about 95% of these are nonsense mutations. The investigators recently demonstrated that gentamicin readily induced nonsense mutation readthrough and produced full-length laminin beta3 in several nonsense mutations tested. Importantly, the gentamicin-induced laminin beta3 restored laminin 332 assembly, secretion, and deposition into the dermal-epidermal junction (DEJ). Newly induced laminin 332 reversed abnormal H-JEB cellular phenotypes. Herein, the investigators propose the first clinical trial of gentamicin (by topical and intravenous administration) in JEB patients with nonsense mutations. The milestones will include restored laminin 332 and hemidesmosomes at the DEJ, improved wound closure, and the absence of significant gentamicin side effects.
Herlitz junctional epidermolysis bullosa (H-JEB), an incurable and fatal inherited skin disease, is caused by loss-of-function mutations in LAMA3, LAMB3 and LAMC2. These mutations result in diminished laminin 332 and epidermal-dermal adherence. 85% of JEB patients have nonsense mutations in LAMA3, LAMB3, or LAMC2, suggesting that H-JEB is a prime therapeutic target for nonsense suppression therapy. The investigators recently demonstrated in three patients that topical gentamicin created new and stable laminin 332 at the dermal-epidermal junction (DEJ), and also improved wound closure and skin quality. Furthermore, these preliminary studies showed that intravenous gentamicin also induced laminin 332 and transiently improved patients' clinical outcomes. No untoward side effects occurred. The investigators propose to optimize the intravenous gentamicin regimen including dosage and infusion schedules to enhance the therapeutic outcome. The milestones will be an increase of laminin 332 in the patients' DEJ, improvement in EB Disease Activity Scores, and no gentamicin-associated side effects.
The 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).
A pharmacokinetic (PK) study in 16-20 EB subjects to be allocated to two cohorts. Cohort 1 to include 8-10 subjects (ages 12 yrs and older); Cohort 2 to include 8-10 subjects (ages 6 months-11 yrs, inclusive). Cohort 2 only included subjects 4 yrs and older. Serial PK blood sampling collected on Days 1 and 10. Analyses were performed to determine the concentrations of diacerein and rhein.
This survey intends to collect information on key aspects of life with epidermolysis bullosa (EB), including diagnostic journey, treatment, management, daily living challenges, and overall psycho-social, socio-economic, academic and family impact. Objectives: * To understand the unmet needs for people living with EB in the US * To assess the differences/similarities in the management/treatment of EB patients (including wound care, symptom management and other issues) * To assess the EB patients' and caregivers' perceptions of current management/treatment * To assess the challenges and the burden of daily living with EB * To understand EB diagnostic journey (the time to diagnosis and by what type of healthcare provider) * To identify professional disciplines involved in the diagnosis and management of EB * To understand the psycho-social, socio-economic, academic, and family impact of EB
The study aimed to assess the long-term safety of topical use of Zorblisa (SD-101-6.0) in participants with Epidermolysis Bullosa (EB).
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).
The 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
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).