Active clinical trials for "Syndrome"

Autism Spectrum Disorders (ASD), with its core symptoms of communication and repetitive behaviors, is a serious neurodevelopmental disorder common in childhood and affects about 1% of children. So far, autism remains a clinical dilemma with no effective therapy. The most common chromsomal ability among ASD patients is 15q11-13q duplication syndrome(dup15q syndrome).Clinical phenotypes of dup15q syndrome include autism, mental retardation, epilepsy (usually refractory epilepsy, often manifested as infantile spasm), congenital heart disease, mild facial abnormalities, etc. UBE3A is one of the most important genes in the 15q11-q13 region.Biochemistry and molecular biology of the Chinese Academy of Sciences Hu Ronggui group found a new kind of autism in mechanisms and potential therapeutic targets - describe the ubiquitin ligase UBE3A protein and retinoic acid.Previous studies have shown that the basis of the relevant treatment measures can effectively relieve the mouse model of autism characteristics. Therefore, retinoic acid supplementation in the treatment of dup15q syndrome is a potential therapeutic target.

This study will evaluate whether a geriatric assessment can lead to better treatment outcomes in older patients (age 60+) with a myeloid malignancy including acute myeloid leukemia, ,myelodysplastic syndromes, myeloproliferative neoplasms, or related blood disorders who are going to receive chemotherapy or another treatment to prepare the body for an allogeneic hematopoietic stem cell transplant (allo-HCT). The geriatric assessment includes looking at patients' cognitive function (thinking processes), physical function, mobility (ability to move the body), mood, nutrition, and current medications to help decide the type of treatment they'll receive. Another purpose of this study is to see whether use of the geriatric assessment improves participants' quality of life. We will evaluate participants' quality of life through questionnaires.

The aim of this study is to investigate the effects of regular forest therapy under guidance of a licensed nature therapist on patients with metabolic syndrome and cardiovascular risks

This phase I trial is to find out the best dose, possible benefits and/or side effects of fostamatinib in treating patients with lower-risk myelodysplastic syndromes or chronic myelomonocytic leukemia who have failed therapy with hypomethylating agents. Fostamatinib may stop the growth of tumor cells by blocking some of the enzymes needed for cell growth.

To observe the efficacy and safety of Ruxolitinib and Etoposide combined with DDGP regimen ( cis-Platinum, Dexamethasone, Gemcitabine and Pegaspargase) in the first-line induction therapy of T cell lymphoma and NK/T cell lymphoma-associated hemophagocytic syndrome.

The investigators had observed that baricitinib was effective and safe in active pSS patients in a pilot study. So the investigators plan to conduct a multi-center, prospective, open-label, randomized study to compare the efficacy of baricitinib + hydroxychloroquine (HCQ) with HCQ alone in active pSS patients. The participants will be randomized (1:2) to receive HCQ (200mg twice a day) or baricitinib (4mg per day) + HCQ (200mg twice a day) until week 24. The primary endpoint is the ESSDAI and ESSPRI response (define as an improvement of ESSDAI at least three points, and ESSPRI at least one point or 15%) at 12 weeks. According to an expected response rate of 70% in baricitinib + HCQ group and 30% in HCQ group, the investigators will involve approximately 87 participants (29:58) with 20% drop out rate. The investigators will switch HCQ to baricitinib + HCQ if the participants has no response at 12 weeks. The investigators hypothesized that baricitinib was effective and safe in active pSS patients.

The purpose of this study is to evaluate the effect of olezarsen (formerly known as AKCEA-APOCIII-LRx) on the percent change in fasting triglycerides (TG) from baseline.

The ASET Japan Pilot study is a multicenter, single arm, open-label trial of single antiplatelet therapy with prasugrel for patients undergoing successful and optimal Percutaneous Coronary Intervention (PCI) for Chronic Coronary Syndrome (CCS) and Non-ST elevation Acute coronary syndrome (NSTE-ACS). The enrollment consists of two phases: i) 200 patients presenting with CCS; ii) 200 patients presenting with NSTE-ACS. The patients will be loaded with standard dual antiplatelet therapy according to local practice (usually aspirin 81 to 330 mg and clopidogrel 300 mg or prasugrel 20 mg or ticagrelor 180 mg, unless patient is on long-term therapy) prior to the PCI procedure. After PCI, if the results are considered to be satisfactory by the operator based on clinical (e.g. clinical status, ECG, etc.), angiographic and/or findings from intracoronary imaging, only then patients will be enrolled in the study and loaded with prasugrel 20 mg if the patients have not loaded prasugrel prior to PCI or have not taken a maintenance dose of prasugrel before the index PCI. Patients continued with prasugrel only (3.75 mg once a day) for three months in CCS patients and for 12 months in NSTE-ACS patients. Aspirin, clopidogrel, and ticagrelor will be discontinued just after the index procedure. i. CCS patients (phase 1): At the 3-months follow-up visit, prasugrel monotherapy will be replaced by aspirin monotherapy or dual-antiplatelet therapy according to local standard of care. Clinical follow-up with office visit will be performed at 3 months and telephone contacts at 1, and 4 months (final follow-up). ii. NSTE-ACS patients (phase 2): At the 12-months follow-up visit, prasugrel monotherapy will be replaced by aspirin monotherapy for an observational period of 1 month, followed by antiplatelet treatment according to local practice. Clinical follow-up with office visit will be performed at 1 and 12 months and telephone contacts at 3, 6, 9 and 13 months (final follow-up). All events will be adjudicated by an independent clinical events committee (CEC). An independent Data Safety and Monitoring Board (DSMB) will monitor the individual and collective safety of the patients in the study during enrolment of CCS patients and up to 3 months follow-up of CCS patients, and during enrollment of NSTE-ACS patients and up to 12 months follow-up of NSTE-ACS patients (timepoint for primary endpoint).

Previous research shows that gypenoside could keep blood lipid and blood sugar under control in an effective way. In this study, we intend to explore whether gypenosides have clinical improvement effects on metabolic diseases such as diabetes, hyperlipidemia, and non-alcoholic fatty liver and their cardiovascular protective potential. It is designed to recruit 150 patients with metabolic syndrome diagnosed who meet the criteria for enrollment, and randomly divide them into three groups. Gynostemma powder, with two different saponins content (12% and 8%) from Pingli County, Shaanxi Province, and spinach powder with almost no saponins were used to conduct the intervention on patients. Patients will be followed up regularly at the beginning of each month from the first enrollment until the third month. At each follow-up, data of patient's anthropometric indicators as well as clinical inspection indicators related to metabolism (blood routine, liver function, etc.) will be recorded. In addition, patient's blood, hair, urine, and stool samples will be collected to further explore the mechanism of diseases.

This study is a controlled trial of metformin in children with fragile X syndrome(FXS). The age of FXS children range from 2 to 16 years old. Participants will be randomized in a double-blind design to either drug or placebo for 6-month period. The primary objectives are to assess metformin in treatment of behavior problems, cognitive and language with fragile X syndrome.