View clinical trials related to Neuromuscular Diseases.
Filter by:A study looking at the effect of pioglitazone in skeletal muscle of patients with sporadic inclusion body myositis (sIBM).
HOPE-2 is a double-blind clinical trial evaluating the safety and efficacy of a cell therapy called CAP-1002 in study participants with Duchenne muscular dystrophy (DMD). Non-ambulatory and ambulatory boys and young men who meet eligibility criteria will be randomly assigned to receive either CAP-1002 or placebo every 3 months for a total of 4 doses during a 12-month period.
The Electronic Medical Records and Genomics (eMERGE) Network is in its third phase and during this time is enrolling and sequencing 25,000 individuals on a custom sequencing panel of clinically relevant, actionable genes. The genetic results will be returned to participants and outcomes tracked through the electronic health records.
To determine if a prolonged high altitude simulation test (HAST) lasting two hours, identifies more patients at risk of respiratory failure than the standard HAST lasting 20 minutes, in patients with neuromuscular disease (NMD) and severe pulmonary restriction. To evaluate the safety of supplemental oxygen administered in those with a positive HAST in the NMD population.
Monocenter study to determine and evaluate the optimal amount of humidification doses delivered by the On-Demand humidification system in neuromuscular patients requiring home mechanical ventilation. The patient's perception will be monitored with subjective as well as with objective measurements of the optimum comfortable on-demand humidification doses.
Background Mechanical assistance cough for patients with neuromuscular disorders (NMD) are known, but there is no adaptation model established. Currently is performed subjectively by the physiotherapist following the pressures recommended in the current literature as optimal (±40CmH2O). To check is these pressures are the most optimal pressures to achieve the better peak cough flow (PCF) monitoring cough assist (Mechanical insuffllation-exsuflattion MI-E) maneuvers and analyzing pressure- flow / time curves Adults with MI-E criteria (PCFbaseline <160 l /min). A pneumotachograph, PeakAnalysis software and nasobucal mask were used to monitor and analyze flow/time curves. Protocol included 9 PCF values in each patient: 1 baseline, 4 related with inspiratory pressure in sequential increase of 10 cmH2O (10 to 40 cmH2O) and 4 adding expiratory pressures (±10 to ±40cmH2O)
The aim of this study is to evaluate if a intervention with hippotherapy will improve spasticity for children ages 3-14 who have cerebral palsy. The hip aductors spasticity will be measured using the Modified Ashworth Scale (MAS). The intervention will be performed in addition to traditional treatment.
Objective: To prove that initiation of chronic ventilatory support at home, in patients with chronic hypercapnic respiratory failure due to neuromuscular disease (NMD) or thoracic cage problem is not inferior compared to initiation in a hospital based setting. In addition we believe that the start at home is cheaper compared to an in-hospital start. Hypothesis: Initiation of chronic ventilatory support at home is effective, safe and cost effective compared to a hospital-based initiation. Study design: A nationwide non-inferiority multi-center randomized parallel active control study. Study population: Patients with chronic respiratory insufficiency due to a neuromuscular disease (NMD) or thoracic cage problem who are referred for chronic ventilator support. Intervention: The start of HMV at home Standard intervention to be compared to: The start of HMV is normally in a clinical setting as recommended in the national guideline. Outcome measures: Primary: PaCO2. Secondary: Health related quality of life; lung function; nocturnal transcutaneous carbon dioxide assessment and saturation, and costs Sample size calculation/data analysis: This is a non-inferiority trial based on PaCO2 as primary outcome. A difference in favor of the hospital care group of smaller than 0.5 kPa will be labeled as non-inferior. To retain 72 evaluable patients, and allow for drop-outs, we will include 96 patients in total. Cost-effectiveness analysis: A cost analysis will be conducted alongside the clinical trial. Costs of the initiation of HMV at home and in the hospital will be estimated form a societal perspective over a time horizon of 6 months. Time schedule: After an initial phase of 6 months recruitment will start and will take 24 months. Thirty-six months after the start of the study the last assessments will be done and analysis and writing of the papers will start. After 42 months the study will end.
This study is a long-term study of ataluren in participants with nonsense mutation Duchenne muscular dystrophy.
The CENTAUR trial was a 2:1 (active:placebo) randomized, double-blind, placebo-controlled Phase II trial to evaluate the safety and efficacy of AMX0035 for the treatment of ALS.