Trial of Sirolimus for Cognitive Impairment in Sturge-Weber Syndrome
The purpose of this research study is to gain a preliminary understanding of the safety of sirolimus in Sturge-Weber syndrome (SWS) and determine best outcomes to be used to assess the utility of sirolimus for the treatment of cognitive impairments related to Sturge-Weber syndrome.
UK Baby Study Using a Baby Wash and Lotion Regimen
The skin of a newborn has a sensitive skin barrier relative to an older child and an adult. Newborn's skin, for example, is extremely vulnerable to damage by environmental agents such as harsh detergents, some topical oils, and other irritant chemicals. Evolving perspectives on barrier dysfunction in newborn babies has led to the idea that there may be a window of opportunity in the first few months of a newborn's life to change the environmental agents that their skin is exposed to in order to maximize skin health. These environmental changes could involve the use of optimally formulated wash products and emollients, as well as the ...
Use of a Probiotic Supplement to Prevent Asthma in Infants
The goal of the study is to understand the mechanisms of how antigen presentation affects the developing immune system and subsequently affects susceptibility to, or protects against, asthma development. This randomized controlled study will test the effectiveness of daily supplementation of Lactobacillus GG for the first 6 months of life on the early immunological development of asthma.
Using Topical Sirolimus 2% for Patients With Epidermolysis Bullous Simplex (EBS) Study
Epidermolysis bullosa (EB) simplex is a rare orphan disease caused by a mutation in DNA leading to abnormal dominant keratins in the skin. Patients with EB simplex develop lifelong painful thick soles on their feet, and current standard of care is supportive. This pilot study will target the dominant mutant keratin proteins in the skin to ameliorate the severity of EB simplex. The purpose is to improve the function of EB simplex feet with an application of topical sirolimus, 2%. The investigators plan on inhibiting the mTOR pathway to down regulate the translation of defective keratin proteins and work through anti proliferative ...
Vaccine Combining Multiple Class I Peptides and Montanide ISA 51VG With Escalating Doses of Anti-PD-1 Antibody Nivolumab or Ipilimumab With Nivolumab For Patients With Resected Stages IIIC/ IV Melanoma
The purpose of this study is to test the side effects of an investigational vaccine with an immune booster, or 2 different boosters together. Investigators also want to find out its effects on the immune system and whether it will decrease the chance that melanoma will return.
What Are the Benefits and Harms of Risk Stratified Screening in the NHS Breast Screening Programme: Study Protocol
This study aims to identify key benefits and harms of integrating risk stratification (the BC-Predict intervention) into the NHS Breast Screening Programme. A non-randomised fully counterbalanced study design will be used, whereby women from screening sites will be offered usual NHS Breast Screening Programme or BC-Predict for an eight month period, followed by a cross-over point where women at each site will be offered the other invention during an eight month period.
Abrocitinib Expanded Access Protocol in Adolescents and Adults With Moderate to Severe Atopic Dermatitis
This is a multi-center, expanded access protocol to provide access to the investigational product, abrocitinib, to adolescent and adult patients with moderate to severe atopic dermatitis who have inadequate treatment options with available and approved medicated topical and systemic therapies and who are otherwise ineligible for participation in clinical studies with abrocitinib.
Clinical Trial on the Effect of Autologous Oral Mucosal Epithelial Sheet Transplantation
To investigate the effect of ocular surface reconstruction and assess the safety in cultivated oral mucosal epithelial cell sheet transplantation (COMET) regarding patients with cicatricial change of ocular surface.
Compassionate Use Program BRAF Mutation-positive Patients in the Adjuvant Treatment of Melanoma After Surgical Resection
The purpose of this Treatment Plan is to allow access to trametinib and dabrafenib for eligible high-risk BRAF mutation-positive patients in the adjuvant treatment of melanoma after surgical resection. The patient's Treating Physician should follow the suggested treatment guidelines and comply with all local health authority regulations.
Compassionate Use Program to Access Trametinib and Dabrafenib for Patients With BRAFmutation-positive Metastatic Melanoma
The purpose of this Cohort Treatment Plan is to allow access to trametinib (monotherapy or in combination) and dabrafenib (monotherapy or in combination) for eligible patients diagnosed with metastatic melanoma BRAF mutation-positive.
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