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Filter by:Wolfram syndrome is a rare genetic disorder characterized by juvenile-onset diabetes mellitus, diabetes insipidus, optic nerve atrophy, hearing loss, and neurodegeneration. The purpose of this study is to assess the safety and tolerability of dantrolene sodium in patients with Wolfram syndrome. In addition, we will assess the efficacy of dantrolene sodium on the cardinal manifestations of Wolfram syndrome, including visual acuity, remaining beta cell functions, and neurological functions. There is a screening period up to 56 days, a 6-month treatment period with an optional extension phase up to 24 months, and a 4-week safety follow-up period. Study assessments include medical & medication history, physical exams, neurological exams, eye exams, endocrine exams, vital signs, height, weight, electrocardiograms, blood and urine tests, pregnancy test if applicable, and questionnaires.
This is a multicenter, double-blind, parallel-group, placebo-controlled, study to assess the efficacy, safety, and pharmacokinetics of ZX008 when used as adjunctive therapy in pediatric and young adult subjects with Dravet syndrome. Subjects who qualify for the study will be randomized (1:1:1) in a double-blind manner to receive 1 of 2 doses of ZX008 or placebo. All subjects will be titrated to their randomized dose over a 14-day Titration Period. Following titration, subjects will continue treatment at their randomly assigned dose over a 12-week Maintenance Period. Total treatment time from the beginning of the Titration Period through the end of the Maintenance Period is 14 weeks.
Treatment Phase I and II Primary Objective: To evaluate the efficacy and safety of FCM (750 mg dose x 2) for treatment of Restless Legs Syndrome (RLS) in patients with iron-deficiency anemia (IDA). Long-Term Extension Phase III Primary Objective: To evaluate the duration of effect of prior FCM treatment and to determine the effectiveness of further iron repletion with FCM when RLS symptoms worsen or reoccur.
The present study was designed to evaluate, in premature babies with RDS breathing spontaneously, the efficacy of combined treatment with nasal continuous positive airway pressure (CPAP) and aerosolized surfactant. The first objective of investigators is to assess the safety of surfactant nebulization in this clinical situation, and to find out whether treatment with aerosolized surfactant would reduce the need for mechanical ventilation. And other aim suggest that aerosolized dates compared with dates of INSURE (intubation-surfactant-extubation) and minimally invasive surfactant therapy (MIST) method.
This is an international, multicenter, open-label, long-term safety study of ZX008 in subjects with Dravet syndrome.
The investigators will conduct a cross-sectional study to discovery the distribution of IBS subtypes who would like to seek TCM treatment and their TCM patterns. At the same time, the investigators will also collect the serum, urine and stool samples of the participants to explore the host-gut microbiota metabolic interactions associated with IBS subtypes.
The purpose of this study is to evaluate the cutaneous toxicity and treatment response associated with administering concurrent TSEB and brentuximab vedotin in patients with mycosis fungoides or Sézary Syndrome.
To evaluate the efficacy and safety of TCM decoction Chang'an I Recipe in the treatment of IBS-D. A multicenter, randomized, double-blind, placebo-controlled clinical trial was designed. Patients were applied for central random number and were given corresponding treatment according to inclusion sequences.The treatment group was administered the Chang'an I Recipe, 150ml/bag, 3 times/day; while the control group was administered the placebo, 150ml/bag, 3 times/day. Both courses of treatment were 8 weeks.
Recent studies have found that up to 45 % of patients with type 1 diabetes have metabolic syndrome, a cluster of conditions (abdominal obesity, hypertension, dyslipidemia, insulin resistance) that increase the cardiovascular risk. The investigators have observed in previous studies a strong association between the adherence to a Mediterranean diet and the prevalence of metabolic syndrome on patients with metabolic syndrome. However, no intervention has been realized on this population and the current recommendations (low fat diet) differ from the Mediterranean diet. A nutritional intervention on the principles of the Mediterranean diet could therefore play a role in the treatment of the metabolic syndrome in Type 1 diabetes patients. The main objective of this study is to examine the impact of a nutritional intervention on the waist circumference after 6 months of an intervention group (Mediterranean diet) versus a control group (low fat diet).
1. To test the effectiveness of the theory-based interventions (text+visual and text-only BCT-based interventions) against usual care in changing patients' intentions to phone ambulance immediately with symptoms of ACS ≥ 15 minutes duration. 2. To determine the most effective mode of delivery by comparing the text+visual BCT-based intervention with text-only BCT-based intervention. 3. To investigate any unintended consequences of the intervention on intentions to phone an ambulance for non-life-threatening symptoms.