Active clinical trials for "Syndrome"

The study aims to test the safety of Repetitive Magnetic Stimulation (RMS) treatment for dry eye disease. Patients are asked to undergo a one-time treatment with Epitech Ocular Magnetic Stimulation Device on one eye in the first stage and both eyes sequentially in the second stage of the study. Changes are monitored for over a study period of 3 months.

This research is being done to determine whether a home-based behavioral therapy can be successfully used to treat tics in children with Tourette syndrome (TS). Behavioral therapy administered by a trained therapist has been shown to be helpful in reducing tics in children with Tourette syndrome. In this study, the investigators want to determine whether an instructional video can provide parents of children with TS with the tools necessary to successfully use behavioral therapy at home. The investigators also want to determine the effectiveness of home-based behavioral therapy as compared to improvement achieved when the therapy is provided by a trained therapist.

The purpose of this study is to determine if an investigational treatment (teduglutide) is safe and effective in Japanese children (age 4 months through 15 years of age) with SBS who are dependent on parenteral support. This study will also evaluate how teduglutide moves through the body (pharmacokinetics) and how it affects the body (pharmacodynamics).

The objective of this study is to compare the safety and efficacy of RGN-259 Ophthalmic Solutions to placebo for the treatment of the signs and symptoms of dry eye.

The purpose of this study is to determine the maximum tolerated dose (MTD) of ARGX-110 and/or the recommended Phase II dose (RP2D) in combination with a standard dose of azacytidine (AZA) in Phase 1; and to evaluate efficacy of ARGX-110 when administered at a RP2D level established in Phase I in combination with a standard dose of AZA (proof-of concept) by evaluating overall response rate (ORR) in Phase 2.

Analysis of data from the recently completed NIH Child Comprehensive Behavioral Intervention for Tics (CBIT) study found a manualized behavioral treatment approach strongly superior to psychoeducation/supportive therapy for reducing tic severity in 9-16 year-old youths with TS or other Chronic Tic Disorders. Buoyed by the success of the NIH study, the research group now seeks to extend and disseminate the CBIT treatment through the systematic adaptation of the CBIT protocol for use across a broader range of ages and treatment settings. The goal of the this project is to develop a downward extension of the CBIT therapist guide and parent workbook for use in 4-8 year old children with chronic tics. The revised CBIT-JR manual/workbook will be pilot tested in five children at each of the three study sites (UCLA, UWM, Weill Cornell) in order to provide initial data regarding treatment feasibility and acceptability as well as our ability to implement the new intervention, along with relevant quality control procedures, consistently across sites. These pilot data will then be used to seek R01 support for a larger controlled multisite trial examining the efficacy of CBIT-JR. Although arguably more complex than a single-site design, we have opted for a multsite study in order: 1) to take advantage of the established productive collaborative relationship and collective expertise in childhood tic disorders and psychosocial treatment development across our three sites, 2) to collect the proposed feasibility data in a much shorter period of time than otherwise possible, and as noted above 3) to demonstrate the cross-site portability of the treatment - which will be necessary if we are to obtain subsequent funding for a larger-scale efficacy trial.

Schnitzler's Syndrome (SchS) is a late-onset multifactorial autoinflammatory disease characterized by chronic urticarial skin lesions and a monoclonal gammopathy usually belonging to the immunoglobulin M (IgM) or IgG class. Symptoms associated with SchS are recurrent fever attacks, bone and muscle pain, arthralgia or arthritis, fatigue and lymphadenopathy. SchS is a rare disease with approximately 300 cases reported in the literature. The nature of SchS is chronic without known reports about spontaneous remissions. Disease onset occurs around the age of 50. About 15% of patients eventually develop a lymphoproliferative disorder, most often Waldenström's macroglobulinemia. The pathogenesis of SchS is still not well defined. Functional ex vivo studies showed excessive cytokine production (IL-1ß, IL-6 and IL-18) of peripheral blood monocytes (PBMCs) in SchS as compared to healthy controls. In addition to excessive IL-6 secretion from PBMCs IL-6 has repeatedly been reported to be elevated in the serum of SchS patients too. As IL-6 plays a major role in the development of multiple myeloma, IL-6 may also be associated with the formation of lymphoproliferative disorders in SchS. Until now, there is no approved standard therapy available for the treatment of SchS. Non-steroidal anti-inflammatory drugs (NSAIDs), corticosteroids, and other immunosuppressive agents have been reported to provide variable relief from symptoms of bone pain and arthralgia. Case reports and small studies about successful treatment of SchS with anti-IL-1 blockers (anakinra, rilonacept and canakinumab) accumulate. However, there have been complete and partial treatment failures to anti-IL-1 blockade in SchS. In these patients, anti-IL-6 treatment (tocilizumab [TCZ]) demonstrated to be very effective in reducing the clinical symptoms and inflammation markers in SchS. TCZ treatment has proved to be very effective, well-tolerated and safe in other acquired autoinflammatory disorders, systemic juvenile idiopathic arthritis (sJIA) and adult-onset Still's disease that share many clinical features (rash, fever, joint involvement, lymphadenopathy, fatigue) and excessive cytokine production with SchS. The study consists of a run-in baseline period of 1-4 weeks followed by an open-label 20-week TCZ treatment phase with weekly s.c. injections (TCZ 162mg), followed by an optional study extension up to a total of 1 year with ongoing once weekly TCZ 162mg injections and a 4 week period of follow-up.

The primary objective of this study is to assess the efficacy of filgotinib, lanraplenib, and tirabrutinib in adults with active Sjogren's Syndrome (SjS).

Many patients with exhaustion disorders continue to have significant difficulties with attention and memory function which reduce their work ability. This randomized study investigates whether the addition of a 12-week period of specific cognitive training or physical fitness training could further enhance cognitive function in patients with exhaustion disorder participating in a multimodal rehabilitation program.

The primary objective of the study is to compare the efficacy of an intervention with high-intensity interval, low volume training (HIIT-LV) or continuous aerobic exercise (CAE) on insulin resistance, insulin sensitivity and percentage of pancreatic β-cell function in adults with metabolic syndrome (MS). The secondary objective is to compare the efficacy of an intervention with HIIT-LV or CAE on glycosylated hemoglobin, mass and muscle fibre type composition of right thigh and plasma levels of musclin and apelin in adults with MS. The investigators hypothesized that HIIT-LV is more effective in decreasing insulin resistance and glycosylated hemoglobin and plasma concentrations of musclin and increasing plasma concentrations of apelin, and both mass and muscle fibre type I percentage in thigh, than CAE.