View clinical trials related to Syndrome.
Filter by:The purpose of this study is to evaluate the efficacy and safety of imetelstat in transfusion-dependent participants with low or intermediate-1 risk myelodysplastic syndrome (MDS) that is relapsed/refractory to erythropoiesis-stimulating agent (ESA) treatment in Part 1 of the study and to compare the efficacy, in terms of red blood cell (RBC) transfusion independence (TI), of imetelstat to placebo in transfusion-dependent participants with low or intermediate-1 risk MDS that is relapsed/refractory to ESA treatment in Part 2 of the study. An Extension Phase has been included to allow continued treatment for those subjects who are benefitting from imetelstat and to continue to evaluate the long-term safety, overall survival (OS), and disease progression, including progression to acute myeloid leukemia (AML) in transfusion-dependent participants with low or immediate-1 risk MDS that is relapsed/refractory to ESA treatment.
In the clinical data, the changes of RIPK3 and FXR were monitored in the lung lavage fluid and blood from the patients. In vivo experiments to find high risk factors to induce AEC necrosis and further lead to ARDS evidence, can provide a more direct theoretical research foundation for the pathogenesis of ARDS.
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 overall objective of this project is to compare the three home-managed treatment regimens for PFPS: neuromuscular electrical stimulation (NMES), transcutaneous electrical nerve stimulation (TENS), and NMES combined with TENS to a standard home exercise program (HEP). Each of the three treatment arms will be supplemented by HEP and compared to a group receiving standard HEP alone. The central hypothesis is that the combination of NMES with TENS will show significantly greater improvements in muscle strength, mobility, pain, daily activity and quality of life (QOL) than HEP alone. The investigators are examining: 1) whether the three treatment regimens are significantly more efficacious than standard HEP alone in improving lower extremity muscle strength, physical activity, mobility, pain, and quality of life; 2) lower extremity muscle strength, physical activity, mobility, pain, and quality of life differ significantly across the 4 time periods; 3) is there an interaction between treatment and time in relation to lower extremity muscle strength, physical activity, mobility, pain, and quality of life.
This study aims to verify the hypothesis that patients with Vascular Ehlers Danlos syndrome (vEDS) should benefit of the blockade of angiotensin (Ang) II noxious effects on their vasculature affected by a defect in type III collagen in addition to the effects celiprolol. This randomized, double blind, placebo controlled trial compares the administration of the Ang II type I receptor blocker (ARB) - irbesartan- to placebo over a 2-year period in vEDS patients with the main objective to reduce the incidence of both symptomatic and asymptomatic vascular events.
This study evaluates the benefits of hydroxychloroquine on arterial function in antiphospholipid syndrome. Briefly, the patients will be randomized in two groups, one will receive hydroxychloroquine and standard treatment, the other will receive placebo in addition of standard treatment.
Background: Proteus syndrome (PS) is caused by a mutation in the AKT1 gene. This gene makes a protein that communicates with other proteins in the body to make cells grow. The AKT1 mutation changes chemical signals in the body and causes overgrowth. PS can be fatal. The drug MK-7075 reduces signals from the AKT1 protein. This may reduce or stabilize some of the overgrowth in people with PS. Researchers want to find the best dose of MK-7075 based on its effect on tissues in people with PS. Objective: To determine the safety, tolerability, and recommended dose of MK-7075 in people with PS. Eligibility: People ages 6 and older with PS Design: Participants will be screened with medical history, physical exam, and blood and urine tests. Participants will take MK-7075 by mouth once daily for up to 12 28-day cycles. Participants must stay near the NIH Clinical Center (CC) during the whole first cycle, for weekly visits to the CC. For cycle 2, they will have visits every 2 weeks. They will have 1 visit before cycles 3 and 4, and once before every other cycle for cycles 5 11. The final visit will be at the end of cycle 12. Visits may include: Small skin samples taken. ECG: Soft electrodes on the skin record heart signals. Echocardiogram: A small probe held to the chest takes pictures of the heart. MRI: Participants will lie in a machine that takes pictures of the body. Joint and mobility function tests. Participants will complete surveys by phone and in person. Participants will keep a daily medication and symptom diary. ...
The purpose of this study is evaluate if abatacept is effective and safe in decreasing the level of protein loss in the urine in patients with excessive loss of protein in the urine (nephrotic syndrome) due to either focal segmental glomerulosclerosis (FSGS) or minimal change disease (MCD). Candidates must have a prior kidney biopsy with either diagnosis. Another kidney biopsy will not be required as part of the study. Candidates must have failed or be intolerant of prior therapy for their kidney disease. The failed or intolerant therapy must include corticosteroids and at least one other drug. Candidates can be adults and children over the age of 6. Abatacept will be administered by venous infusion every 4 weeks.
The purpose of this study is to develop and validate a work-model in Primary Health Care for identifying patients with Sleep Apnea Syndrome, based on clinical variables and an ambulatory monitoring study.
The purpose of this study is to determine if the combination product (URG101) is safe and effective versus its individual components (Heparin Sodium and Lidocaine Hydrochloride) for the treatment of Interstitial Cystitis/Bladder Pain Syndrome. The study is randomized and double-blinded such that neither the subject nor doctor will know which drug or placebo is received.