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NCT02907619
This study is an open-label extension to protocol B5161002 and will provide an assessment of the long term safety, efficacy, pharmacodynamics and pharmacokinetics of intravenous dosing of PF 06252616 in boys with Duchenne muscular dystrophy. Approximately 105 eligible subjects will be assigned to receive a monthly individualized maximum tolerated dose based on their tolerability profile/data from B5161002. This study will not contain a placebo comparator. Subjects will undergo safety evaluations (Laboratory, cardiac monitoring, physical exams, x-ray, MRI), functional capacity evaluations (4 stair climb, range of motion, strength testing, Northstar Ambulatory Assessment, upper limb functional testing, six minute walk test and pulmonary function tests) and pharmacokinetic testing.
NCT01731600
This trial is conducted globally. The aim of the trial is to investigate safety, efficacy and pharmacokinetics (the exposure of the trial drug in the body) of NNC 0129-0000-1003 (N8-GP) in children with severe haemophilia A who have undergone treatment with previous factor VIII (FVIII) products.
NCT02284334
The investigators are conducting a research study on nutrition in mitochondrial disease. The investigators are interested in the kind of carbohydrates that people eat. Carbohydrates are an important source of energy. Certain kinds of carbohydrates tend to raise blood sugar more in healthy people, and others tend to raise blood sugar less in healthy people. The investigators would like to know if by eating the "right" type of carbohydrate, people with mitochondrial disease can avoid high lactate levels, high blood sugar levels, and, later, low blood sugars. In this study, this question will be answered by finding out how people with mitochondrial disease respond to two different test meals containing different kinds of carbohydrates. Neither participants nor the investigators will know which kind of test meal participants are eating.
NCT03197532
Women of late to mid reproductive age may participate in a study using novel methodology to identify subtle changes in the reproductive endocrine axis via daily urine hormone analysis, which may have important implications for reproduction and may signal reproductive senescence.
NCT04636242
This is a prospective, randomized controlled trial to evaluation the ability of 5-aminolevulinic acid HCL topical solution photodynamic therapy to decrease the colonization of Cutibacterium acnes (C. acnes- a bacteria commonly found in the dermis of the skin surrounding the shoulder) in order to decrease postoperative joint infections. -Aminolevulinic acid (ALA) is a naturally occurring metabolite in the synthesis of pathway of cellular heme production. Adding ALA to bacteria encourages porphyrin production which serve as the immediate precursors to heme production. When these porphyrins are illuminated with blue light at an emission peak of 407-420nm, these metabolites become exothermic and cause internal destruction of the bacterial cells. This therapy does not cause any damage to the mammalian cells, which makes PDT safe for human skin treatment.
NCT04636125
Cutibacterium acnes (C. acnes) is an anaerobic aerotolerant bacteria commonly isolated during revision shoulder surgery. It is increasingly recognized as a pathogen, mainly in implant-related infections. As an anaerobe, it usually needs a prolonged culture incubation time of up to 14 days for growth and the association between implant surgery and C. acnes infection is not always obvious. Unfortunately, prolonged incubation also increases the risk of false positive cultures in isolating organisms that may exist as a result of contamination. Given high rates of positive C. acnes cultures in cases of both primary and revision shoulder surgery, the ramifications of positive C. acnes cultures for clinical decision making remains uncertain. The purpose of this study is to prospectively study the efficacy and side-effect profile of surgical treatment plus an oral antibiotic regiment for shoulder PJI with indolent organisms (C. acnes and CNS).
NCT02090426
This trial investigates the value created by the highly innovative Camden Coalition of Healthcare Providers' Care Management Program: Link2Care. The program targets "super-utilizers" of the health care system - specifically adults with 2 or more hospitalizations in the last six months 2 or more chronic conditions, and 5 or more outpatient medications - with intensive case management services. In particular, a team of nurses, social workers, community health workers and health coaches, supported by real-time data of healthcare utilization, perform home visits, accompany patients to doctor visits, and help patients enroll in social-service programs. This approach aims to improve the self-sufficiency of patients in navigating the healthcare and social-service systems and has the potential to reduce healthcare costs and improve patient health.
NCT03565211
The purpose of the study is to assess the safety of Progesterone Vaginal Ring (PVR) in women undergoing fresh embryo transfer (ART).
NCT00930059
The purpose of this study is to evaluate the effects of PF-04447943 compared to placebo on cognitive, behavioral and overall symptoms of Alzheimer's disease; evaluate the safety and tolerability of PF-0444793 compared to placebo; and determine the levels of PF-04447943 in the plasma over the course of the study.
NCT01799889
The primary objective of the study is to evaluate efficacy of entospletinib in participants with relapsed or refractory hematologic malignancies. Participants with the following relapsed or refractory hematologic malignancies will be enrolled into the study: relapsed or refractory chronic lymphocytic leukemia (CLL), mantle cell lymphoma (MCL), diffuse large B-cell lymphoma (DLBCL), follicular lymphoma (FL), or non-FL indolent non-Hodgkin lymphomas (iNHL; including lymphoplasmacytoid lymphoma/ Waldenström macroglobulinemia \[LPL/WM\], small lymphocytic lymphoma \[SLL\], or marginal zone lymphoma \[MZL\]).
NCT01922258
To compare the efficacy of flexible dosing of brexpiprazole with placebo in subjects with agitation associated with dementia of the Alzheimer's type
NCT00447499
The purpose of this study is to determine whether subjects with acromegaly (or their partners) are able to self administer Somatuline Autogel at home.
NCT00940602
This was a randomized, double-blind trial to evaluate deferasirox vs placebo in patients with myelodysplastic syndromes (low/int-1 risk) and transfusional iron overload .The trial was conducted in 17 countries, started in 2010 and ended in 2018.
NCT02260752
The overall goal of this project is to better enable patients with uterine fibroids (UF) to make informed decisions about treatment options by leveraging the highest possible evidence of healthcare quality. The foundation of this project will be a multi-site, prospective registry of a diverse group of women who have undergone either medical or surgical treatment for UF. Specifically, the investigators are interested in: comparing management options for symptom relief; comparing management options for preserving reproductive function; and comparing effectiveness among different subpopulations, including consideration of patient needs and preferences of treatment options.
NCT01446913
The goal of this study is to develop a novel study design to safely and ethically conduct a long-term randomized controlled trial among patients at high risk for both sleep apnea and cardiovascular events that will examine whether effective positive airway pressure(PAP) therapy reduces cardiovascular risk. Patients with transient ischemic attack(TIA) or stroke have a high prevalence of sleep apnea(60-80%), and they are at high risk of cardiovascular events(myocardial infarction, congestive heart failure, recurrent stroke, and cardiovascular death)in the first year post event, despite current prevent strategies. Therefore, the treatment of sleep apnea may represent a novel therapeutic target to reduce cardiovascular outcomes in this high risk population.
NCT02384096
The study is a prospective, on-label, multi-center, blinded, randomized controlled trial with a cross-over design to evaluate the effect of Precision Spectra™ SCS System's programming features and lead options.
NCT02847650
The purpose of this study is to evaluate the efficacy, safety and tolerability of PF-06649751 in Parkinson's disease patients at early stage of the disease.
NCT03828617
This is a Phase 3, randomized, double-blind study with a 4-arm parallel design. Adults 18 through 49 years of age with no history of pneumococcal vaccination will be randomized in a 2:2:2:1 ratio to receive a single dose of: 20vPnC Lot 1; 20vPnC Lot 2; 20vPnC Lot 3; or 13vPnC.
NCT03837457
The main objective of this clinical trial is to study the efficacy and safety of cobomarsen (also known as MRG-106) for the treatment of cutaneous T-cell lymphoma (CTCL), mycosis fungoides (MF) subtype in subjects who have confirmed disease progression following treatment with vorinostat in the SOLAR clinical study (MRG106-11-201). Cobomarsen is designed to inhibit the activity of a molecule called miR-155 that may be important to the growth and survival of MF cancer cells. The effects of treatment will be measured based on changes in skin lesion severity, disease-associated symptoms, and quality of life, as well as the length of time that the subject's disease remains stable or improved, without evidence of disease progression. The safety and tolerability of cobomarsen will be assessed based on the frequency and severity of observed side effects.
NCT03553550
The purpose of this research study is to learn more about changes in cell-free tumor DNA in blood samples, also known as a liquid biopsy, as they relate to treatment and response to treatment.