Active clinical trials for "Syndrome"

The primary objective of the trial is to confirm the efficacy of glepaglutide in reducing parenteral support volume in patients with short bowel syndrome. Glepaglutide is the International Nonproprietary Name and USAN for ZP1848.

The primary objective of this study is to determine the safety and tolerability of combined abaloparatide and bevacizumab in patients with Myelodysplastic Syndromes (MDS). A secondary objective is to determine the response to treatment (based on bone marrow and peripheral blood findings). A tertiary objective is to determine the impact of therapy on health-related quality of life (HRQOL) and patient-reported outcomes (PRO). A quaternary (scientific) objective is to determine the impact of treatment on both hematopoietic and stromal cell populations within the bone marrow of MDS patients.

This is a global, multicenter, randomized, double-blind, stratified, vehicle-controlled study of the efficacy and safety of Patidegib Topical Gel, 2%, applied topically twice daily to the face of adult participants with Gorlin syndrome. Participants will be required to apply the investigational product for 12 months. The primary endpoint is a comparison between the two treatment arms of the number of new BCCs that develop over the 12 month period.

The investigator's recent pilot study of vagus nerve stimulation (VNS) from a surgically implanted medical device to control the excess eating behaviour characteristic of Prader-Willi Syndrome (PWS) found that it was safe and acceptable. In addition, there were unanticipated marked improvements in rates of problem behaviours, such as emotional disturbances and verbal and physical outbursts. These observations indicated the need for a trial specifically focusing on the effects of VNS on problem behaviour and also that the use of VNS might be extended to include people with other neurodevelopmental disorders, such as autism spectrum conditions (ASC). The primary aims of this study are: a) to investigate whether VNS, now given by an external medical device, is associated with a significant reduction in the number and severity of maladaptive behaviours in adults with PWS; and b) to undertake a pilot study that includes others with a different neurodevelopmental syndrome who have histories of similar behaviours. The study will be a single case cross-over design with 4 to 6 months baseline phase and a similar period of active treatment. The study cannot be blind as the stimulation is apparent but the participants will wear the device initially for four hours a day, at times convenient to them, with it switched off in the baseline phase and activated, according to standard protocols, in the treatment phase. Six adults with PWS and six with a different neurodevelopmental disorder with histories of significant problem behaviours will be included initially, with a view to extending if the analysis indicates a likely effect. Behaviours will be operationally defined and measured over time using participant and informant diaries with additional secondary outcome measures. Before and during the treatment phases autonomic nervous system and brain biomarkers will be assessed using ambulatory monitoring of heart rate variability and fMRI brain scans.

A novel intervention for targeting the treatment of Intraoperative Floppy Iris Syndrome.

The purpose of this study is to evaluate whether SkQ1 ophthalmic solutions are safe and effective compared to placebo for the treatment of the signs and symptoms of dry eye syndrome.

This is a phase 2, multicenter, double-blind, placebo control, randomized study to evaluate the efficacy and safety of orally administered SA001 compared to placebo in patients with Dry Eye Syndrome. The clinical trial consists of a wash-out period of 14 days, a treatment period of 12 weeks, and a follow-up period of 1 week after administration of the Investigational Product. If the subject voluntarily signs the informed consent form(ICF), the investigator conducts screening tests and check medical history to evaluate the subject's suitability. As a result of the screening test, eligible subjects should stop using the prior medication for dry eye syndrome during the 14 days of observation period, and if necessary, subjects can use rescue drug(artificial tears) for the first 11 days, and then discontinue all eye drops including rescue drug(artificial tears) for 3 days. And all of these subjects will be randomized in a 1:1:1:1 ratio to receive 3 different doses of investigational product (SA001 or placebo) everyday for 12 weeks. During the treatment period, If necessary, subjects can use the rescue drug (artificial tears), and the number of administration of rescue drug is limited to 3 times a day, and when used, the administration time should be recorded in the subject's diary. Subjects should visit to the study site on 2, 4, 8 and 12 weeks after starting dosing investigational product. Efficacy evaluation results are collected from both eyes, and the primary evaluation variable is analyzed using the test results collected from 'Worse eye' (the eye with the worse keratoconjunctival staining result among both eyes). Worse eye will be determined at the baseline visit and, if the results of both eyes are the same, the test result of the left eye is used.

Related to urological and gynecological systems, patients with Bladdeer Pain Syndrome (BPS) often present pain, pressure and chronic discomfort in suprapubic and perineal area associated with low urinary tract symptoms. This condition can generates a negative impact in cognitive, social, behavioral and sexual quality of life. As Bladder Pain Syndrome originates from different causes and includes the genitourinary, gastrointestinal, musculoskeletal and neuropsychological systems, a multidisciplinary approach is needed with doctors,physiotherapists, psychologists and others. Physiotherapeutic interventions are recommended as a conservative treatment for patients with BPS. The physiotherapeutic interventions include the use of Biofeedback to relax the pelvic floor muscles and manual therapy (myofascial trigger points release) to decrease muscle tension. Transcutaneous electrostimulation (TENS) is used to decrease the pain and postural exercises to improve the pelvic mobility. In this study, our hypothesis was that women with BPS presented musculoskeletal dysfunction, and we tested a different physiotherapy approach that was not being used. The reason for that understanding was the presence of refractory urinary and pain symptoms notwithstanding the physiotherapy conventional treatment, such as manual therapy and biofeedback. To test our hypothesis, we decided to add either TENS or postural exercises to the conventional treatment. The objective of this study was to verify the effects of biofeedback (BF) and manual therapy (MT) associated with transcutaneous electrical nerve stimulation (TENS) or postural exercises (PE) in the treatment of bladder pain syndrome (BPS) in women regarding pain and urinary symptoms.

Prader-Willi syndrome (PWS) is the most common syndromic cause of obesity. Individuals with PWS characteristically experience excessive weight gain and severe hyperphagia with food compulsivity in early childhood, which often leads to the onset of obesity and metabolic complications. The pathogenesis of hyperphagia and progressive weight gain in PWS is far from being understood, and thus efficacious interventions are still under development. Emerging evidence indicates an important etiological contribution of dysbiotic gut microbiota in the hyperphagia, obesity and metabolic abnormalities associated with PWS, implicating a potentially effective target for appetite control and alleviation of obesity in PWS. This study aims to evaluate whether dietary fibers can improve hyperphagia and metabolic profile in children with PWS, and further will determine if these improvements correlate with dietary-fiber-induced changes of the gut microbiota. Twenty children with PWS (age 5-17 years) will receive 3-week fiber or placebo treatment and 3-week alternate treatment with a 4-week washout period in between. A validated PWS-specific hyperphagia questionnaire will be used to assess the severity of hyperphagia in participants. Fasting blood and fecal samples will be collected for the analyses of appetite-related hormones, metabolic biomarkers, bacterial composition and gut metabolites. This study should provide potential new approaches for effective non-pharmacologic treatment of excessive weight gain and hyperphagia associated with PWS to improve overall health and quality of life in affected patients.

Sjogren's Syndrome (SS) is an autoimune disease of unknown etiology characterized by lymphocytic infiltration of the exocrine glands and other organs. patients usually presents with xerophthalmia, xerostomia, fatigue and other symptoms. Fatigue has often been reported as the biggest problem and the most difficult symptom patients have to deal with. Fatigue management in pSS is difficult. However, in other diseases such as Parkinson disease, post-polio syndrome and multiple sclerosis the use of Transcranial Direct Current Stimulation (tDCS) has recently been studied and has shown effectiveness. The overarching objective of this study is to examine the effect of a tDCS protocol in patients with pSS.