A Study of Soticlestat as an Add-on Therapy in Children and Young Adults With Dravet Syndrome
Dravet Syndrome (DS)The main aim of the study is to learn if soticlestat, when given as an add-on therapy, reduces the number of convulsive seizures in children and young adults with DS. Participants will receive their standard antiseizure therapy, plus either a tablet of soticlestat or placebo for 16 weeks. A placebo looks just like soticlestat but will not have any medicine in it. Participants may continue treatment in an extension study, based on the extension study's entry criteria. Those that want to stop treatment will have a gradual dose reduction during 1 week and then be followed up for 2 weeks.
A Study Evaluating the Efficacy, Safety, Pharmacokinetics and Pharmacodynamics of Crovalimab in...
Atypical Hemolytic Uremic SyndromeThis study aims to evaluate the efficacy and safety of crovalimab in pediatric participants with aHUS.
Human Umbilical Cord Blood (RegeneCyte) Infusion in Patients With Post-COVID Syndrome
Long COVIDPost-COVID Syndrome1 moreRegeneCyte (HPC, Cord Blood, hUCB) for treatment in patients with post-COVID.
A Study With Imlifidase in Anti-GBM Disease
Anti-Glomerular Basement Membrane DiseaseAnti-Glomerular Basement Membrane Antibody Disease2 moreAn open-label, controlled, randomised, multi-centre Phase 3 trial evaluating renal function in patients with severe anti-GBM disease comparing imlifidase and standard of care (SoC) with SoC alone. All patients will remain in the trial for 24 months.
Effects of Low- Load Strengthening Using Different Exercises Modes During Blood Flow Restriction...
Shoulder Impingement SyndromePurpose of the study: This study compares effects of isometric, concentric and eccentric exercises for shoulder muscles by using low load blood flow restriction training on pain, strength and function in patients with SIS.
Investigating the Impact of JASPER Behavioral Therapy in Children With Down Syndrome
Down SyndromeIn this study, investigators will study the impact of a 1:1 caregiver coaching intervention using the JASPER (Joint Attention, Symbolic Play, Engagement, Regulation) behavioral therapy curriculum compared to a psychoeducational curriculum that will be provided to caregivers for self-directed learning. Investigators want to determine the impact of both interventions on the child's development and behavior, and caregiver implementation of strategies.
Prednisolone and Vitamin B1/6/12 in Patients With Post-Covid-Syndrome
Post-COVID-19 SyndromeThis is a multicenter, randomized, placebo controlled, double-blind phase III trial with four parallel groups studying studying the feasibility of RCT in primary care as well as the effectiveness of treatment with prednisolone and/or vitamin B1/6/12 for PC19S.
Wheat-free Diet in the Treatment of Sjogren's Syndrome
Sjogren's SyndromeNon-celiac Gluten SensitivityRecent data show that some foods can increase intestinal mucosa permeability and immune activation of subjects with gastrointestinal (GI) symptoms. Wheat seems the most frequent food which activates this inflammatory response and can cause both GI and extra-intestinal symptoms. Patients suffering from wheat-related troubles, in absence of celiac disease diagnosis, can suffer from non-celiac wheat sensitivity (NCWS) and our previous studies showed that about 25% of them are also affected by autoimmune diseases (AD). A gluten-free diet (GFD) can influence inflammatory pattern of AD, including Sjogren's syndrome (SS). Thus, the investigators would enquire if SS patients may also suffer from NCWS and how a wheat-free diet (WFD) modifies their clinical features, and inflammatory and cytokine pattern. The investigators will also assess how wheat reintroduction, by an open challenge, modifies their clinical parameters, intestinal permeability, and both local and systemic inflammatory response.
Personalized Dietary Intervention Based on Microbiome Analysis vs FODMAP Diet for Irritable Bowel...
Irritable Bowel SyndromeIrritable Bowel Syndrome With Diarrhea2 moreirritable bowel syndrome (IBS) is a well-known risk factor for diseases of the anal region (hemorrhoids, anal fissures, etc.) and large intestine (colon cancer, diverticular disease). Untreatable IBS disease not only impairs the patient's quality of life but also reduces the success of surgical treatment in these diseases. Today, only one-third of IBS patients achieve successful results with the treatment methods and diets used routinely. Balancing the composition of the gut microbiota may yield satisfactory results in this patient group. With the results of our study, we aim to reveal the effect of microbiome analysis and personalized diet on symptoms and its place in treatment in patients with IBS disease. Patients applying to Gastroenterology outpatient clinics in respective centers due to IBS will be randomized into two groups. The patients in the control group will follow the FODMAP diet protocol for IBS; In the study group, a personalized diet will be applied according to the gut microbiota. No nutritional supplements will be used in either group. Changes in defecation habits and quality of life scale of both groups will be evaluated at the end of 6 weeks and the results will be compared.
Ebastine Versus Mebeverine in IBS Patients
IBS - Irritable Bowel SyndromeIBSMulticenter randomized controlled clinical trial comparing ebastine and mebeverine as treatment of irritable bowel syndrome Trial rationale To perform a randomized superiority trial comparing the clinical efficacy of ebastine and mebeverine To evaluate the impact of treatment with ebastine compared to mebeverine on quality of life and quality-adjusted life years Primary objective To provide further evidence of the superiority of histamine 1 receptor antagonism as novel treatment for patients with non-constipated IBS, as compared to mebeverine, one of the spasmolytics currently used as first line treatment of IBS. Secondary objective(s) To provide evidence that the histamine 1 receptor antagonist ebastine is more effective in reducing abdominal pain compared to the commonly used antispasmodic mebeverine