Theta Burst Stimulation to Improve Inhibitory Motor Physiology in Tourette Syndrome
Tourette SyndromeTourette Syndrome in Children3 moreDecades of Tourette Syndrome (TS) neuroimaging research has revealed abnormal cortical and subcortical motor system network, hypothesized to result from maladaptive plasticity. Repetitive transcranial magnetic stimulation ([r]TMS) is a promising technology that utilizes the concept of neuroplasticity to modulate brain circuits. TMS modulation has the distinct advantage in terms of its non-invasive nature. Furthermore, unique stimulation paradigms such as intermittent theta-burst repetitive TMS (iTBS) allows for short stimulation time (<3 min). Using a sham-controlled protocol, the investigators propose modulating pre-SMA output using iTBS, based on our prior data of abnormal pre-SMA-mediated motor system regulation. hypothesize pre-SMA modulation results in increased pre-SMA-mediated motor inhibition. Enhancing these inhibitory measures with pre-SMA-iTBS provides the basis for improving inhibitory function in TS patients, leading to our long-term goal of neuro-stimulation to achieve clinical tic reduction.
Perfusion Estimation For Optimal Treatment Strategy in Chronic Coronary Syndrome
Chronic Coronary SyndromeMyocardial Ischemia2 moreWe will establish a cohort of 570 symptomatic chronic coronary syndrome patients undergoing 15O-water PET and assess their symptoms through repeated questionnaires. Two hundred patients with abnormal perfusion will be randomized to immediate or delayed referral to invasive coronary angiography with concomitant optimization of guideline-directed medical therapy with repeated 15O-water PET and questionnaires at 3 and 6 months. The primary objective is to compare the potential benefit of early invasive coronary angiography (ICA) versus guideline directed medical therapy (GDMT) on symptomatic relief defined as freedom of angina after 3 months following a positive [15O]H2O cardiac PET/CT in patients with symptomatic chronic coronary syndrome.
A Study of BPN14770 in Male Adults (Aged 18 to 45) With Fragile X Syndrome
Fragile X SyndromeA Randomized, Double-blind, Placebo-controlled, Parallel Group Study of BPN14770 in Male Adults (Aged 18 to 45) with Fragile X Syndrome
Randomized Study of Conditioning of Fludarabine Combined With Single or Dual Alkylating Agents in...
Acute Myeloid LeukemiaMyelodysplastic SyndromesFludarabine and busulfan becomes standard conditioning regimen for adult patients with acute myeloid leukemia (AML) or myelodysplasia syndrome (MDS). The overall relapse rate is 15~20%. More recently, the investigators demonstrated that conditioning regimen with dual alkylating agents consistent of fludarabine, busulfan and melphalan achieved a low incidence of relapse (<10%). This multiple-center randomize study is aim to compare the transplantation outcome in adult patients with AML/MDS undergoing allo-HSCT with conditioning regimen of Flu-Bu4 vs. FLu-Bu-Mel.
Investigation of Treating Chronic Fatigue Syndrome After COVID With Pharmacotherapy (Pregabalin)...
Chronic Fatigue SyndromeCOVID-191 moreThis is a single-center, prospective, randomized, double-blind (pharmacotherapy), placebo-controlled, and comprehensive rehabilitation phase II clinical trial to determine the usefulness of pregabalin in a new indication (post-COVID chronic fatigue syndrome). Patients will be randomized in a 1:1:1:1 ratio to pregabalin (75-300 mg daily in two divided doses), comprehensive rehabilitation with a placebo drug, comprehensive rehabilitation with pregabalin (75-300 mg in two divided doses), or placebo (two divided doses) for 6 months (177-187 days). There will be 4 outpatient visits to the research center and 12 telephone consultations. The procedures and assessments performed as part of the study are listed in the study schedule. It is planned to include 132 patients in the study, which, assuming a 10% level of non-completion of the program, will result in the examination of 120 patients (30 in each arm). Patients will be recruited during an outpatient medical consultation with a general practitioner or neurologist, psychiatrist, psychologist or other specialists, as well as with the use of information materials in the form of leaflets and advertisements on the Internet.
Impact of Bright Light Therapy on Prader-Willi Syndrome
Prader-Willi SyndromeExcessive Daytime Sleepiness4 moreThis is a placebo controlled clinical trial to assess the utility of light therapy as a sufficient treatment for excessive daytime sleepiness in patients with Prader-Willi Syndrome
Pharmacologic Effect for Female Overactive Bladder Syndrome: Mirabegron Versus Solifenacin
Overactive Bladder SyndromeTo assess the impact of mirabegron versus solifenacin on psychological distress, sexual function, bladder wall thickness and bladder blood flow.
Extracorporeal Cytokine Adsorption as Additive Treatment of CAR-T Associated Cytokine Release Syndrome...
Cytokine Release SyndromeCAR-TPatients with severe CAR-T cell associated cytokine release syndrome (CRS) (defined as vasopressor dependent) will be treated with standard of care (SOC) + cytokine adsorption (6hourly for 24 hrs). Primary endpoint is the change in plasma IL-6 between 0 and 24 hrs.
Do Biomarkers Predict Response to a Pediatric Chronic Pain Symptom Management Program?
Irritable Bowel SyndromeFunctional Abdominal Pain Syndrome4 moreThe purpose of this study is find out if we can use simple tests (biomarkers) to tell us if a specific child would benefit most from CBT or from the low FODMAPs diet.
Study of Modified Atkins Diet in Kabuki Syndrome
Kabuki SyndromeAnimal models of Kabuki syndrome have showed a reversal of the cognitive phenotype with ketogenic diet. Modified Atkins diet is safer and easier tolerated than full ketogenic diet and still has the histone deacetylase inhibition believed to be responsible for the cognitive improvement. This study aims to examine a small number of adults with Kabuki syndrome before and after 12 weeks on a modified Atkins diet to determine if there is any cognitive improvement and if the diet can be tolerated.