
A Trial of Linaclotide 290 μg in Patients With Irritable Bowel Syndrome With Constipation (IBS-C)...
Irritable Bowel Syndrome Characterized by ConstipationTo evaluate the efficacy on abdominal symptoms (abdominal bloating, abdominal discomfort, and abdominal pain) and safety of linaclotide 290 μg administered orally to patients with IBS-C.

Effect of Curcumin on Gut Microbiota in IBS
Irritable Bowel SyndromeTo evaluate the effect of curcumin food supplement on gut microbiota of children with irritable bowel syndrome (IBS) and to review any correlation between the changes in the microbiota with symptoms.

AMO-01 to Treat Adolescents and Adults With Phelan-McDermid Syndrome (PMS) and Co-morbid Epilepsy...
Phelan-McDermid SyndromeEpilepsyThe purpose of this study is to investigate the safety, tolerability and efficacy of a single 6-hour intravenous infusion of AMO-01 to treat adolescents and adults with PMS and co-morbid epilepsy. Phelan-McDermid Syndrome (PMS) is a neurodevelopmental disorder characterized by a chromosomal deletion or mutation at 22q13.3 that contains the SHANK3/ProSAP2 gene. A key co-morbidity in PMS is the presence of epilepsy. Currently there are no approved treatments for PMS. Furthermore, there has been relatively little clinical study of pharmacological interventions for PMS. AMO-01 may provide benefit to PMS patients exhibiting behavioral abnormalities and seizures.

Effects of Exercise Trainings on Pain, Function and AHD in Patients With SPS
Subacromial Impingement SyndromeAbnormal reduction of the AHD has been considered as one of the possible mechanisms in the aetiology of subacromial pain syndrome. Maintenance of the AHD is crucial for prevention and rehabilitation of rotator cuff related disorders. The development of a rehabilitation treatment plan is based in part on the assessment of scapular motion and muscle deficits in patients with shoulder pain. Rehabilitation should be based on the identified impairments. The aim of this study is to investigate the effect of utilizing scapula retraction exercises with or without glenohumeral rotational exercises at gradual shoulder elevation angles into a scapular stabilization program on functionality, pain and AHD in patients with SPS and compare with health population.

Streptokinase Versus Unfractionated Heparin Nebulization in Severe ARDS
Acute Respiratory Distress SyndromeSevere Acute Respiratory SyndromeBackground: Intra-alveolar clotting and alveolar collapse in ARDS is due to alveolar capillaries epithelial and leakage. Subsequently, collapse induces hypoxemia that is resistant to recruitment (RM). Heparin and Streptokinase may prevent or dissolve intra-alveolar fibrin clot respectively helping alveolar re-expansion. We examined and compared the effect of nebulizing Heparin versus Streptokinase on reversing this pathology. Methods: Sixty severe ARDS (PaO2/FiO2<100) patients and failure of RM, prone position (PP) and neuromuscular block (NMB) were partially randomised into Group (I): (n=20) received nebulized Heparin 10000 IU/4h. Group (II): (n=20) received nebulized Streptokinase 250,000 IU/4h. Group (III): (n=20) received conservative management. Randomization to either Heparin or Streptokinase groups was applied to patients whom guardian accepted participation, while those who declined participation were followed-up as a control. The primary outcome was the change in PaO2/FiO2; the secondary outcomes included the change in compliance, plateau pressure, ventilation-off days, coagulation and ICU mortality.

The Effects of Muscle Training in Coronary Artery Patients With Metabolic Syndrome
Metabolic SyndromeCoronary Artery DiseaseThe aim of this study is to investigate the effects of peripheral and respiratory muscle training on respiratory muscle strength, respiratory functions, exercise capacity and quality of life in coronary artery patients with metabolic syndrome. Prospectively, randomly, double-blind, and controlled study. Stable coronary artery patients with metabolic syndrome will be included to the study from the Istanbul University Cardiology Institute clinic. Patients will be randomized into 3 groups; Neuromuscular Electrical Stimulation (NMES) and Peripheral Muscle Training Group (n: 20): 3 days per week for 6 weeks duration. Peripheral Muscle Training will be applied by elastic band and Proprioceptive Neuromuscular Facilitation three times per week for 6 weeks.Inspirator Muscle Training (IMT) and peripheral muscle training Group (n: 20): IMT will be applied 7 days per week, twice a day for 15 minutes. The program will continue for 6 weeks duration under weekly control of the investigator. Training intensity will set at 30% of the maximum inspiratory pressure.Peripheral Muscle Training will be applied by elastic band and Proprioceptive Neuromuscular Facilitation three times per week for 6 weeks. Peripheral Muscle Training Group (n: 20): Exercise will be applied by elastic band and Proprioceptive Neuromuscular Facilitation three times per week for 6 weeks.Parameters will be recorded before and after training. Evaluation parameters:Demographic and anthropometric measurements, respiratory function test,respiratory muscle strength,dyspnea, peripheral muscle strength, 6 minutes walking test, physical activity, quality of life, depression, sleep quality and laboratory evaluation.

Efficacy and Safety of HU007 Eye Drops in Patients With Dry Eye Syndrome
Dry Eye SyndromeA multicenter, Randomized, Double blind, Restasis®-controlled Non-inferiority, Moisview® Eye Drops-controlled Superiority, Phase III Study to Evaluate the Efficacy and Safety of HU007 Eye Drops in Patients with Dry Eye Syndrome

Targeting the Gut Microbiome for Prader-Willi Syndrome Treatment
Prader-Willi SyndromeThe gut microbiome has recently emerged as a major contributor to obesity, systemic inflammation, and metabolic disease. Furthermore, intestinal bacteria are crucial players in the gut-brain axis, regulating a broad range of central nervous system processes, from satiety mechanisms to anxiety and social behavior. Thus, targeting the microbiome is being actively investigated as a therapeutic strategy for a wide array of diseases, including obesity, anxiety, depression, and autism. Among all intestinal bacteria, Bifidobacterium animalis spp. lactis (BAL) has shown promise for obesity treatment in experimental animal models and human subjects, improving body composition and metabolic health, and reducing energy intake. Moreover, tryptophan metabolism, a crucial regulator of satiety mechanisms and anxiety, is a main target of BAL. Given that clinical manifestations of Prader-Willi syndrome (PWS) include hyperphagia, anxiety, altered body composition, and metabolic dysregulation, the aforementioned effects of BAL might prove highly beneficial for children with PWS. Here, the investigators will test this hypothesis by performing a randomized double-blinded placebo-controlled crossover clinical study to assess the effects of BAL supplementation on an array of clinical manifestations of PWS. Children with PWS will undergo a 3-month placebo/probiotic treatment period, a 3-month washout period, followed by a 3-month probiotic/placebo supplementation. Anthropometric, biochemical, and psychological data as well as biological samples will be obtained at the beginning of the study, and after each of the study periods, with a total of four time-points. Specifically, the investigators will determine body composition by DXA analysis; metabolic health by assessing glucose and lipid metabolic parameters as well as circulating hormonal and cytokine levels; thermoregulation by non-invasive thermal imaging; and hyperphagia and emotional and behavioral problems by applying parental-rated validated questionnaires.

A Phase 1 Study of AMV564 in Patients With Intermediate or High-Risk Myelodysplastic Syndromes
Myelodysplastic Syndrome (MDS)An open label, Phase 1, study of AMV564 as monotherapy to assess the safety and efficacy in patients with Myelodysplastic Syndromes

Conditioning Regimen for Allogeneic Hematopoietic Stem-Cell Transplantation
Bone Marrow Failure SyndromeThalassemia14 moreIn this study, the investigators test 2 dose levels of thiotepa (5 mg/kg and 10 mg/kg) added to the backbone of targeted reduced dose IV busulfan, fludarabine and rabbit anti-thymocyte globulin (rATG) to determine the minimum effective dose required for reliable engraftment for subjects undergoing hematopoietic stem cell transplantation for non-malignant disease.