View clinical trials related to Syndrome.
Filter by:This multi-center, randomized, double-blind, 3-arm, parallel-group, placebo-controlled study will evaluate the efficacy and safety of RG1662 in adults and adolescents with Down syndrome. Subjects will be randomized to receive RG1662 either at low or high dose or placebo orally twice daily for 26 weeks.
To evaluate safety, tolerability and efficacy of cysteamine bitartrate delayed-release capsules (RP103) administered up to 1.3 g/m²/day in two divided doses, every 12 hours, for up to 6 months in patients with inherited mitochondrial disease.
This study aimed to investigate the effects of nut consumption on metabolic parameters and biomarkers related to inflammation, oxidative stress, and endothelial function in Korean adults with metabolic syndrome.
The purpose of this study is to learn the impact stress may have on weight management and emotional eating.
Primary Objective: To test the hypothesis that 6 months treatment with glatiramer acetate (GA) decreases epileptiform activity in young girls with Rett syndrome. Primary Safety Objective:To evaluate the safety and tolerability of 6 months treatment with GA in these patients. Secondary Objectives: 1. To test the hypothesis that 6 months treatment with glatiramer acetate (GA) improves respiratory dysfunction. 2. To evaluate the effect of GA treatment on general behaviour communication, hand stereotyping, feeding, sleep and other autonomic symptoms: gastrointestinal and cardiac. 3. To assess the effect of GA treatment on bodily development. Primary Endpoint:Improvement of epileptiform activity as recorded in a 24-hours EEG. Primary Safety Endpoint:Frequency and severity of treatment-related AEs (including safety lab parameters). Secondary Endpoints: 1. Improvement in the scoring of breath holds and hyperventilation, as measured with non-invasive respiratory inductance plethysmography (NoxT3 device) and parents' diaries. 2. Changes in general behaviour, communication, feeding and motor skills as assessed by the investigator (based on Kerr and Naidu validated severity scores) and recorded in parents' diary. 3. Decrease in seizure frequency as reported in parents' diary. 4. Improvement in sleep schedule as recorded in a sleep diary. 5. Change in height and weight. Population:Ten girls, 6 to 15 years old, diagnosed with Rett syndrome (RTT) Study Design:This is a single - center, exploratory, open-label, study in 10 girls diagnosed with RTT. The study will consist of four parts: Screening and baseline assessments, initial and final dose-setting period, treatment period and end-of study follow-up. Investigational Product:Glatiramer Acetate (Copaxone® , Teva Pharmaceutical Industries Ltd.) Sample Size Consideration: The planned sample size of 10 patients was considered adequate by the investigator for this phase I exploratory proof-of-concept study. The study is not expected to show statistical significance or statistical power, only a trend for the study endpoints. Each patient will serve as her own control. Duration of Study: Approximately 8 months per patient (including up to 2 weeks pre-treatment assessment, 6 months initial dose and treatment periods and end-of study visit). Overall study duration: the study is expected to be completed within 12 months (dependent on rate of recruitment).
Comparison of the efficacy and safety of T2762 and Vismed® in the treatment of moderate to severe Dry Eye Syndrome
The main purpose of this clinical trial is to determine the safety and effects of the study drug, sorafenib, in adults diagnosed with hepatopulmonary syndrome (HPS). The study will evaluate how well the drug is tolerated and its effect on the level of oxygen in the blood and the function of the lung vessels.
This is a single-site, open-label, phase II trial of C7, a food supplement or medical food, for the development of treatment outcome measures for glucose transporter type I deficiency (G1D). The primary outcome measures are: 1. Safety and tolerability of C7 as measured by changes in comprehensive blood work, including lipid and free fatty acid panels, self-reported side effects and clinical exam; 2. Changes in brain metabolic rate by MRI and EEG measurements during C7 treatment; and 3. Maintenance of ketosis in G1D patients on ketogenic diet, as measured by serial ketone levels during treatment initiation.
Investigate whether the course of an isolated nocturnal alveolar hypoventilation can predict the future occurrence of obesity-hypoventilation syndrome (OHS) in obese subjects
The study will examine the feasibility of Direct Dorsal Column Stimulation using existing Stimulation Leads (Ad-Tech). Secondary endpoints will include pain relief, somatosensory evoked potentials (SSEPs), stimulator settings required, improvement in quality of life and use of pain medications.