View clinical trials related to Muscular Atrophy.
Filter by:The purpose of this trial is to evaluate safety and efficacy of intravenous delivery of EXG001-307 as a treatment of spinal muscular atrophy Type 1 (SMN1).
Study with the aim to see the effect on a space ground analogue , Hyper-Buoyancy Floatation (HBF) on lumbar column and the effect of a supplementary 50% of an axial load.
The group of children diagnosed with Spinal Muscular Atrophy (SMA) has serious restrictions on participation. SMA is a neuromuscular disease that leads to neuromusculoskeletal disorders that limit functional activities, sometimes making it impossible to sit down autonomously and to walk. Scientific evidence has highlighted the importance of implementing physiotherapy interventions in pediatrics that facilitate the integration and participation of children with reduced mobility in their natural environment through the use of different assisted mobility devices that allow the child to acquire a degree of independence and motivation according to their potential and needs. For some time, with the aim of offering independent movement opportunities for children with severe motor impairment, adapted electric cars have been used, as they are simple to use and easy for the child and family to incorporate into daily tasks within natural environments. These low-cost motorized devices can generate a very positive impact on the participation of children diagnosed with SMA type I from an early age, after training the family and/or the child himself, guaranteeing the maximum possible safety, comfort, motivation and autonomy. Due to the above, there is a need to carry out the research project defined below, to generate opportunities for the inclusion of children diagnosed with SMA type I through the use of low-cost electric cars that encourage their participation, motivation and quality of life.
Introduction： Muscle wasting is a serious complication that affects a large proportion of patients with heart failure (HF). Muscle wasting is a strong predictor of frailty and reduced survival in HF patients. Currently, standard treatments for slowing muscle loss in patients with HF are not available. The main intervention remains various types of physical activity programs. Telemonitoring is a promising strategy for improving heart failure outcomes by making it possible to monitor patients remotely. There are numerous examples of home-based exercise programs administered through telehealth services that have been beneficial for maintaining physical activity levels. These results highlight the potential utility of telehealth services for combatting sedentarism and muscle wasting among epidemic and post-epidemic phases. Objective： The purpose of this study is to evaluate the effect of a multi-component physical activity program based on home telemonitoring on patients with heart failure and muscle wasting. Methods： This study used an quasi-experimental study, two-group repeated measurement design. The experimental group received the Home-based exercise with telemonitoring and control group according to regular nursing care. Data were collected at baseline (T0), and post-tests will be conducted right after the intervention period (T1). Additionally, detraining effects will be measured 12 weeks after program cessation (T2) . Data were collected including demographic questionnaire, sarcopenia, cachexia assessment, clinical blood parameters from patient record, physical activity, loneliness, and quality of life. Scientific or Clinical Implication of the Expected Results： The study results can be used to design designated interventions and provide information for policymaking.
This is a double-blind, randomized phase 3 multicenter placebo-controlled study in at least 16 evaluable male participants diagnosed with MCT8 deficiency. Male participants, from 4 years of age (at randomization) and having demonstrated stable maintenance treatment with tiratricol, will be randomized to receive placebo or tiratricol for 30 days or until reaching rescue criterion (serum total triiodothyronine [T3] > upper limit of normal [ULN] of the participant's normal range, for a sample collected during the 30-day Randomized Treatment Period). The research hypothesis to be tested is that, for participants in the placebo group, removal of tiratricol will lead to an increase of serum total T3 concentration, measured by liquid chromatography with tandem mass spectrometry (LC/MS/MS), above the ULN and requirement of rescue treatment with tiratricol, compared to those who continue to receive tiratricol.
The primary objective of the study is to evaluate the safety and tolerability of single ascending dose of BIIB115 administered via intrathecal (IT) bolus injection to healthy male participants in Part A and multiple ascending doses of BIIB115 administered via IT bolus injection to pediatric Spinal Muscular Atrophy (SMA) participants previously treated with onasemnogene abeparvovec in Part B. The secondary objective of the study is to evaluate the pharmacokinetics (PK) of single-dose of BIIB115 administered via IT bolus injection to healthy male participants in Part A and multiple ascending doses of BIIB115 administered via IT bolus injection to pediatric SMA participants who previously received onasemnogene abeparvovec in Part B.
The aim of the study is to measure and compare acute systemic irisin, myostatin and decorin levels after a single session of blood flow restricted resistance exercise and resistance exercise without blood flow restriction in healthy, trained male participants aged 18-35 years. For this purpose, a total of 22 people will be included in the study. Participants will be randomly allocated to 2 exercise groups as resistance exercise with blood flow restriction (BFR-RE) and resistance exercise without blood flow restriction (HL-RE) and will be subjected to cross-over. In the HL-RE intervention, the exercise will be performed with a loading of 80% of 1 RM, with 4 sets x 7 repetitions, with 60 seconds of rest between sets. In the BFR-RE intervention, the exercise will be done as a set of 30 repetitions with a loading of 30% of 1 RM and an additional 3 sets x 15 repetitions, with 30 seconds rest between sets. Total exercise volumes were tried to be equalized and skeletal muscle hypertrophy was selected in accordance with exercise guidelines. In both groups, bilateral leg extension exercise will be performed using the leg extension machine for resistance exercise. In the blood flow restriction group (BFR-RE), the cuff will be placed in the proximal region of the thigh bilaterally, inflated to a pressure equivalent to 50% of the estimated arterial occlusion pressure (AOP), and leg extension exercise will be performed under this condition. In the BFR-RE group, the blood flow restriction time will be between 5-10 minutes. Exercise sessions will be conducted under supervision. Venous blood samples will be collected from the arm antecubital region of the participants just before the exercise session, immediately after the exercise, and 1 hour after the exercise. Plasma irisin, myostatin and decorin levels will be measured from the samples taken. It is well known that resistance exercise is important in maintaining and increasing muscle mass (hypertrophy). Studies have shown the involvement of certain myokines in skeletal muscle hypertrophy, although few studies have been conducted on the systemic response of myokines to BFR-RE that may play a potential role in hypertrophy. Therefore, the planned study aimed to reveal the similarities or differences in the systemic myokine response between BFR-RE and HL-RE.
The goal of this study is to compare quadriceps muscle activation in patients recovering from lower extremity injury, such as an ACL tear. Specifically, the main objectives are as follows: - Determine the effects of ankle positioning on quad muscle activation during straight leg raise exercises performed while the patient is lying on his or her back - Determine if there is a difference between ankle positioning on quad muscle activation during straight leg raise exercises performed while the patient is lying on his or her back, comparing healthy versus post-operative participants
The research project is aiming to examine the muscular adaptations to resistance training (RT), detraining (DT) and repeated RT (i.e. retraining). The research project will also examine differences in muscular adaptations between 20 weeks of continuous RT and 20 weeks of intermittent RT including a 10-week DT period in the middle of the training intervention. This is randomized controlled trial in which the research participants will be randomized into discontinuous and continuous groups (both n=~20). Both will be doing a 2-3-week familiarization and control period at the start. Then in the former there will be an initial strength training period (10-wks), a DT period (10-wks), and a second strength training (retraining) period (10-wks). The second group includes a 10-wk non-training control period (10-wks) followed by a RT period (20-wks). Participants will be young, healthy men and women (age 18-35, which 50% are females) with no systematic RT experience during the last 6 months. Measurements will be completed before and after each study period. Body composition will be measured via bioelectrical impedance analysis (BIA) and 3D body scans. Dynamic leg press and elbow flexion one repetition-maximum (1RM) will be used to test maximal strength. Anaerobic performance and strength endurance will be tested in elbow flexion and dynamic leg press using RM tests. Vastus lateralis (VL) and biceps brachii muscle cross-sectional area (CSA) will be assessed via ultrasound. Muscle biopsies of the VL muscle will be obtained to assess changes in muscle fiber morphology and factors regulating and associated with the hypertrophic processes and metabolism. Blood samples will be collected to analyze changes in metabolism and physiology. A rating of perceived exertion (RPE) during training will be collected after every exercise to ensure proper training intensity. Finally, nutrition and habitual physical activity will be assessed with 4-day diet diaries and physical activity questionnaires before the intervention and during each 10-week period.
In the last 10-15 years, a better understanding of the pathophysiology and molecular genetics of SMA has led to the emergence of previously unavailable pharmacological and genetic treatments.One of these new treatments, Nusinersen, targets SMN2, which is a slightly different copy of SMN1, and increases SMN protein levels. Preclinical studies have provided evidence that neuroprotection is strongly formed, with exercise significantly increasing motor neuron survival independent of SMN expression. In a limited number of clinical studies prior to Nusinersen treatment, it was reported that aerobic exercise training improved maximum oxygen uptake (VO2 max) without causing muscle damage, but still caused fatigue. The aim of this study is to determine the effect of aerobic exercise training on motor and respiratory functions, exercise capacity, fatigue and quality of life in SMA Type III patients who can walk and receive Nusinersen therapy. Twenty cases aged 10-50 years with genetically confirmed SMA diagnosis will be included in this study. The cases to be included in the study will be randomized into 2 groups as the training and control groups. In addition to the routine physiotherapy program, medium-intensity Aerobic Exercise Training will be given to the study group for 12 weeks. Before and 12 weeks after the training, the cases will be evaluated with the Six Minute Walking Test, Submaximal Exercise Test, SMN protein level, function and strength assessments, (FVC) value, fatigue and quality of life scales. In clinical trials, the supporting evidence for aerobic interventions in SMA is limited. Additional studies on aerobic intervention parameters (frequency, intensity and duration) are needed.The results of this study will determine the feasibility of aerobic exercise training and provide important guidance for the clinical management of SMA patients.