View clinical trials related to Atrophy.
Filter by:This clinical trial aims to test a new physio-therapeutic approach tailored to type 2 and 3 Spinal Muscular Atrophy patients, based on physical training in swimming-pool. This specific exercise should promote motor skills of trained patients, as we have observed in different mouse models. Patient's motor skills will be assessed using different scales including MFM and Hammersmith. This clinical trial attempts to develop a new non-invasive motor scale with sophisticated instruments. This scale will be useful in future clinical trials on SMA, given the lack of sensitivity of currently available scales. In addition, the study attempts to validate a questionnaire on post-exercise physical well-being.
The primary objective of this study is to examine the safety and tolerability of nusinersen (ISIS 396443) administered intrathecally to participants with Spinal Muscular Atrophy (SMA) who previously participated in ISIS 396443-CS2 (NCT01703988) or ISIS 396443-CS10 (NCT01780246). The secondary objective is to examine the plasma and cerebrospinal fluid (CSF) pharmacokinetic(s) (PK) of nusinersen administered intrathecally to participants with SMA who previously participated in ISIS 396443-CS2 or ISIS 396443-CS10.
Age-related macular degeneration (AMD) is the leading cause of irreversible blindness in industrial countries. In the late stages of the disease, neovascular changes or the development of geographic atrophy (GA) may induce severe visual loss. GA is characterized by the development of areas of outer retinal atrophy with continuous spread over time that is corresponded to an visual field defect for the patient. The pathogenesis is still incompletely understood. Despite the break-through in the treatment of neovascular AMD by intravitreally administrated vascular endothelial growths factor (VEGF) inhibitors, there is yet no treatment available to slow down or halt the disease process in GA. We and others have demonstrated that the total GA area progression shows large differences between patients. Potential factors influencing differential progression have been intensely studied: While neither systemic nor genetic factors have been shown to influence GA progression, ocular characteristics such as GA baseline size or phenotypic features of fundus autofluorescence (FAF) abnormalities have been identified as risk characteristics for increased GA progression. While these previous studies have mainly focused on the characterization of total GA area progression, topographic directional spread has not been analyzed and relevant predictive markers are yet unknown. There may be large differences in the local GA progression. The primary objective of this study is to identify specific characteristics, for the local GA progression. The knowledge of such risk factors may help to better understand the pathogenesis of GA. The identification of predictive markers will allow for better prognostic assessment of the individual disease process. The DSGA study is the extension trial of the FAM (Fundus Autofluorescence in Age-related Macular Degeneration) study (NCT00393692).
This non-drug, single center, 24-week, longitudinal study in ambulant spinal muscular atrophy (SMA) patients and in age- and gender-matched healthy volunteers will assess the detection of disease progression by magnetic resonance imaging (MRI) and the Muscle Function Measure (MFM) test. Each participant will be evaluated in three testing sessions: at baseline, at Week 12 and at Week 24. Both patients and volunteers will undergo MRI scans. Patients will additionally undergo testing of motor function and have blood samples taken for Survival of the Motor Neuron (SMN) genes, proteins and mRNA analysis.
Multiple system atrophy (MSA) is a disorder of the nervous system of unclear cause. In MSA there is degeneration (progressive loss) of nerve cells in several brain and spinal cord regions. The result is a variety of symptoms, from physical (parkinsonism, ataxia, incoordination, falls, slowness) to autonomic (fainting, bladder incontinence, sexual dysfunction) to sleep problems (dream enactment, sleep apnea). This research aims to help us better understand the patterns and timing of nerve degeneration relatively early in the disease, and how this affects symptoms and progression. For instance: 1. Does MSA affect certain nerves that stimulate heart pumping? If so, does the severity of loss of heart nerves affect disease progression and survival? 2. It is thought that MSA does not affect memory and thinking much, unlike other diseases (such as Parkinson's). Is this accurate? Is there loss of nerves that transmit acetylcholine (a neurochemical important in mental functioning)? 3. What can we learn about mood and sleep in MSA, through visualizing the serotonin system in the brain? How does this relate to symptoms that subjects report in these often underappreciated areas? To answer these and other questions, investigators will take images of specific nerves in the brain and heart using Positron Emission Tomography (PET) scans. Such imaging gives us information that cannot be obtained from MRIs and CT scans. We will measure the levels of several nerve cell types: serotonin, acetylcholine, and norepinephrine. Subjects will also have many standardized assessments including quality-of-life and symptom assessments, neurological examination, autonomic assessments, neuropsychological assessments, coordination tests, and even assessments of vision and sense of smell. By pooling these results from many MSA patients, and comparing with other diseases (such as Parkinson's disease) we hope to gain a better understanding of what is happening early in MSA. Such knowledge could be very valuable in future efforts to develop better therapies in this rare disease.
Critically ill, ventilator-treated patients rapidly loose much of their muscle mass and strength. This can attribute to prolonged admission, prolonged mechanical ventilation, increased mortality and might have a negative impact on the physical function, degree of independence and quality of life. The pathophysiological background for the loss of muscle mass as well as possible effective treatment is still not well established. In the NONSEDA-trial we randomise critically ill patients to non-sedation or sedation with a daily wake-up trial during mechanical ventilation in the intensive care unit (ICU). It has never been assessed whether non-sedation reduces the loss of muscle mass and strength. Aim: To assess the effects of non-sedation versus sedation with a daily wake-up trial on physical function after discharge from ICU. Hypothesis: that non-sedation during ventilator-treatment will improve the physical function after ICU-discharge, compared with standard treatment of sedation with a daily wake-up.
This study is intended to enable a possible transition to intramuscular (IM) or subcutaneous (SQ) administration for subsequent studies with GSK933776 by characterizing the safety, tolerability, PK and pharmacodynamic profiles, and immunogenicity of GSK933776 following IM and SQ administration in healthy volunteers. Such alternate routes of administration may provide more options in the selection of an efficacious dose for subsequent development in patients with geographic atrophy. There will be four treatment arms in the study and participants will be assigned to 1 of 4 possible treatment arms in a 1:1:1:1 ratio. The planned number of evaluable participants for this study is 24 with 6 participants completing all critical assessments in each of the four treatment arms. The total duration of participation from screening to follow-up for Treatment Arms A, B and D (single dose of GSK933776), will be approximately 113 days and total duration for Treatment Arm C (repeat dose of GSK933776) will be approximately 134 days.
The purpose of this study was to determine if BVS857 is safe, tolerable and increases thigh muscle thickness in patients with spinal bulbar and muscular atrophy (SBMA).
To evaluate the effectiveness of low molecular weight hyaluronic acid oral tablets for the treatment of atrophic vaginitis.
The purpose of this study is to confirm the efficacy of intravaginal prasterone (DHEA) on symptoms of vulvovaginal atrophy due to menopause and to collect further data on subjects exposed to intravaginal DHEA in order to meet the ICH E1 guideline requirements.