View clinical trials related to Syndrome.
Filter by:The REVEAL Adult Study is a multi-center, Phase 1/2 open-label, dose-escalation study of TSHA-102, an investigational gene therapy, in adult females with Rett syndrome. The safety, tolerability, and preliminary efficacy of two dose levels will be evaluated. The study duration is estimated to be up to 63 months.
The purpose of this paper is to analysis of therapeutic effect and immunological mechanism of low-dose IL-2 combined with rapamycin in the treatment of Sjogren's syndrome
This is a prospective, multicenter, non-randomized, controlled intervention clinical study.Patients with severe fever with thrombocytopenia syndrome who have been clinically diagnosed and met the study inclusion criteria will be included in the study for analysis. All patients with SFTS will be assigned to different groups according to the ratio of 1:3, including the non-intervention group (conventional treatment group) and the related drug intervention group. Non-intervention group:patients received conventional treatment during hospitalization. Intervention group: Part A group: Patients received methylprednisolone 1-2mg/kg/d（or other glucocorticoid equivalent to methylprednisolone 1-2mg/kg/d) + intravenous immunoglobulin (IVIG) 0.2g-0.4g/kg/d for a total of 3-5 days. If the disease progressed after treatment, the patients was given the dose of rescue therapy (methylprednisolone > 2mg/kg/d or other glucocorticoid equivalent to methylprednisolone > 2mg/kg/d + IVIG 0.4g/kg/d) for another 3-5 days. Part B group: Patients received tocilizumab 4mg/kg once. Part C group: Patients received low molecular weight heparin 100U/kg, qd or q12h IH for 4-7 days. If the platelet count is less than 30 × 10^9/L, the low molecular weight heparin should be discontinued. All patients received conventional treatment. All patients were followed up from the end of treatment to day 28 after completion of treatment.
The ASC - Autism Pilot Study is a single center randomized open dose titrating phase I clinical intervention pilot trial with the aim of investigating safety and treatment effect of an allogeneic adipose tissue derived mesenchymal stromal cell product (C2C_ASC) in children with autism spectrum disorder (ASD) and gastrointestinal symptoms.
This phase II trial tests whether decitabine and cedazuridine (ASTX727) in combination with venetoclax work better than ASTX727 alone at decreasing symptoms of bone marrow cancer in patients with chronic myelomonocytic leukemia (CMML), myelodysplastic syndrome/myeloproliferative neoplasm (MDS/MPN) with excess blasts. Blasts are immature blood cells. Decitabine is in a class of medications called hypomethylation agents. It works by helping the bone marrow produce normal blood cells and by killing abnormal cells in the bone marrow. Cobimetinib is used in patients whose cancer has a mutated (changed) form of a gene called BRAF. It is in a class of medications called kinase inhibitors. It works by blocking the action of an abnormal protein that signals cancer cells to multiply. This helps slow or stop the spread of cancer cells. Venetoclax is in a class of medications called B-cell lymphoma-2 (BCL-2) inhibitors. It may stop the growth of cancer cells by blocking Bcl-2, a protein needed for cancer cell survival. The combination of ASTX727 and venetoclax may be more effective in reducing the cancer signs and symptoms in patients with CMML, or MDS/MPN with excess blasts.
This is a parallel-group randomised clinical trial: Primary purpose: To analyse the clinical changes produced by two different physiotherapy treatments (Intratissue Percutaneous Electrolysis and Dry Needling) for myofascial trigger points in the infraspinatus muscle in subjects with non-specific shoulder pain. Hypothesis: A physiotherapy treatment including Intratissue Percutaneous Electrolysis therapy present greater benefits in terms of pain reduction and increased mobility and functionality rather than Dry Needling treatment in subjects with non-specific shoulder pain. The intervention consisted of 3 treatment sessions, different according to the group, once a week. Seven evaluation points were performed, two pre-intervention evaluations one week apart and after the second one the first treatment session was performed. The third and fourth assessments were prior to the second and third treatment sessions, one week apart. The fifth, sixth and seventh assessments were conducted one week, one month and two months after the last treatment session in each group.
Irritable bowel syndrome (IBS) is a common functional gastrointestinal disease. Evidence suggests that the concentration of serum VD is decreased in IBS patients, particularly in IBS-D. After giving a supplementation of VD, some symptoms of these patients were relieved to a certain degree. However, the specific mechanism still remains unclear.
Children with frequently relapsing nephrotic syndrome (FRNS) are exposed to prolonged courses of steroids and other immunosuppressant medications. Given the adverse side effect profiles and variable efficacy of these medications, there is an urgent need to identify novel and safe therapies to treat nephrotic syndrome in children. Stimulation of the vagus nerve, which can be activated non invasively by transcutaneous auricular vagus nerve stimulation (taVNS), has immunomodulatory effects mediated by the inflammatory reflex and spleen. taVNS has become a therapy of interest for treating chronic immune mediated illnesses. The aims of the study are (1) To determine the feasibility of protocol implementation and tolerability of taVNS in the treatment of nephrotic syndrome in children (2) To establish proof-of-concept and generate statistical estimates of variance parameters and effect sizes for treatment response outcomes in children with nephrotic syndrome randomized to taVNS therapy compared with sham therapy (3) To investigate the effects of taVNS on inflammatory markers in children with nephrotic syndrome.
In developed countries, mortality rates in pediatric intensive care units (PICUs) are around 4% and most children admitted to these units survive. However, some pediatric survivors experience long-term morbidity (cognitive, psychological and/or physical impairment) associated with their PICU stay and there is increasing awareness of the onset of post-intensive care syndromes (PICS) like in adults. However comprehensive descriptive data are still lacking regarding PICS in pediatrics (PICS-p). The aim of this study is to describe (in nature and frequency) the alterations in health defined by the WHO of children who have passed through the PICU and constitute a possible PICS-p. In order to do this, we will perform a prospective cohort study in Robert-Debré University Hospital including the PICU. We will include children with an unplanned hospitalization for more than 72 hours for acute complication of sickle cell disease (such as acute chest syndrome or vaso-occlusive crisis), acute asthma or sepsis and aged from 3 to 17 years. We plan to include 40 patients admitted to the PICU as well as 40 controls admitted to the general pediatrics unit or the pneumology unit without PICU admission, matched on diagnosis, age range and period. The primary endpoint will be the prevalence of children that had been admitted to PICU and reporting cognitive, psychological, physical and social impairments measured by questionnaire and medical record data collection on the day before discharge and at the routine post-hospitalization visit. Secondary objectives will be to study the risk factors for PICS-p, to compare alterations in cognitive, psychological, physical and social domains in children with the same diagnosis and age not admitted to the PICU during their hospitalization. In order to this, we will measure the association with the PICU stay characteristics, parental experience and social characteristics of families. We will also report the prevalence of children not admitted in intensive care and reporting cognitive, psychological, physical and social impairments measured by questionnaire and medical record data collection on the day before discharge and at the routine post-hospitalization visit. Patients and their parents will be given questionnaires the day before discharge and during the first follow-up consultation between 2 and 4 months after hospital discharge. Questionnaires will include the Pediatric Symptom Checklist long version (Assessment of cognitive, psychological and social domains - 35 items scored from 0 to 2) reported by parents for children under 8 years and by the patients for children older than 8 years. older), the physical items of Pediatric Quality of Life scale (8 items scored from 0 to 4) and a parent self-questionnaire (including relationship to child, annual income, household composition, understanding and use of the French language, highest diploma of mother and father, social support (""How many people can you really count on when you need help?"")) and a parental mental health self-assessment (PHQ-8). The analyses will be descriptive (description of the nature and frequency of alterations) and comparative (between children who have or have not been in PICU). Univariate tests will be performed to identify possible risk factors for post intensive care syndrome. Statistics will be carried out on SAS 9.4 software.
The study seeks primarily to determine whether modulation of systemic and testicular sex steroids balance by aromatase inhibitors will positively affect the metabolic health and spermatogenesis of men with Klinefelter syndrome (KFS) as compared to the current state of the art for each issue. Secondary objectives of this study are (i) to unravel the heterogeneity of the reproductive and metabolic phenotype of men with KFS by performing a multi-omic analysis in a large cohort at baseline; (ii) to evaluate the efficacy of semaglutide-induced weight loss to achieve metabolic and reproductive benefit in men with Klinefelter syndrome as compared to standard testosterone replacement; (ii) to assess whether addition of hCG to aromatase inhibitors further increases intratesticular testosterone and promotes spermatogenesis in men with KFS.