View clinical trials related to Syndrome.
Filter by:Study WCMC IST-CTI-MDS evaluates the safety and tolerability of tosedostat in adult patients with pathologically confirmed MDS (< 20% blasts in bone marrow, peripheral blood, or both) by World Health Organization (WHO) classification after failure of hypomethylating agent-based therapy.
The aim of this study is to assess the suitability of selected scales (floor/ceiling effects, variability, test-retest reliability) to measure cognitive function in children with Down syndrome over 6 months, and to evaluate the influence of covariates such as age, gender or language on these neurocognitive scales.
The objective of the study is to investigate whether prophylactic treatment with melatonin has an effect on depressive symptoms. Secondarily melatonin's effect on anxiety, sleep and circadian disturbances will be investigated. The MEDACIS trial is a randomised, placebo-controlled, double-blinded multicenter trial investigating the effect of 25 mg exogenous melatonin (intervention group) against placebo (control group) and the study is designed as a parallel group superiority trial.
Rare diseases frequently affect women of childbearing age. Pregnancy in these women has become less rare, but remains associated with high levels of complications. One obstacle to their optimal management during pregnancy is that there are no prospective studies of pregnancy during rare diseases and several connective tissue diseases. As a consequence, the management of these pregnancies is non-standardised in terms of treatment, monitoring (frequency of consultations, laboratory tests and ultrasound), and organisation of care. Moreover, although these women (all diseases combined) are frequently exposed to medications potentially incompatible with pregnancy, little is known about the frequency of these exposures and especially their consequences to mother and child. For these reasons, researchers and clinicians from different specialties created an interdisciplinary research group on pregnancy and rare diseases (GR2), intended to improve the management of these patients' pregnancies. Using a single computer server, the investigators plan to set up a large prospective study of pregnancies in patients with rare diseases: various forms of myositis, lupus, antiphospholipid syndrome, Sjogren syndrome, scleroderma, and inflammatory rheumatic diseases. The investigators objective is to analyse the complications of pregnancies in women with rare diseases and then to improve their management and their quality of life.
Subjects with confirmed diagnosis of Irritable Bowel Syndrome will be blinded and randomly assigned to one of two groups, the low FODMAP diet or the conventional IBS diet group. They will be referred to do three hydrogen breath tests prior to diet education. Upon completion, subjects will attend dietitian consultations at Week 0, 6 and 10 for diet assessment, education and reinforcement. At Week 4 and 8, dietitians will telephone patients to check on diet compliance and answer any diet question. Two sets of questionnaires, the IBS Global Improvement Scale and the IBSQOL will be given to patients to complete before each dietitian visit at week 0, 6 and 10. Two sample t-test and Chi square test will be used to test the difference between the two groups and the association between breath tests results and responds to the two diets.
The 3q29 deletion syndrome is caused by a deletion of a small part of human chromosome 3, and the duplication syndrome is caused by a duplication of this same small region. The purpose of this study is to understand the medical and behavioral consequences of these syndromes.
Indication Treatment of pediatric subjects with newly diagnosed advanced myelodysplastic syndrome (MDS) or juvenile myelomonocytic leukemia (JMML) prior to hematopoietic stem cell transplantation (HSCT). Objectives Primary Objective The primary objective is to assess the treatment effect on response rate (MDS: either complete remission [CR], partial remission [PR], or marrow CR; JMML: either clinical complete remission [cCR] or clinical partial remission [cPR]); at Cycle 3 Day 28 (each cycle is 28 days) and to compare against standard therapy using a matched-pairs analysis of historical data. Secondary Objective The secondary objective is to further evaluate safety, efficacy, pharmacokinetics (PK), and pharmacodynamics (PD) of azacitidine in this subject population. Study Design This is a prospective, open-label, Phase 2 study consisting of 2 parallel experimental arms, one for each disease group: MDS and JMML. Each arm is designed based on Simon's Optimal 2 stage study design. The sample size has been calculated to allow evaluation of the response rate at 28 day-Cycle 3 Day 28 in each of the 2 disease groups. Each of the experimental arms will also individually be compared against a historical control arm using data retrospectively collected from the European Working Group of MDS in childhood (EWOG-MDS) registry by means of a matched-pairs analysis; matched for predefined subject baseline characteristics defined before any results from this study are known post Stage 1. If matched pair is not viable then other methodologies will be explored to evaluate and compare response rates reported in literature and also in registry database Twenty subjects with MDS and 35 JMML subjects evaluable for the primary endpoint (ie, subjects that receive at least 1 dose of investigational product [IP]) will be enrolled at approximately 45 centers in Europe. Each experimental arm has 1 interim analysis planned (at the end of Stage 1). If, during Stage 1 evaluation, less than 2 subjects are observed with a CR, PR, or marrow CR after 3 months of azacitidine in the first 9 subjects with MDS, then enrollment will be stopped. Similarly, if less than 3 subjects are observed with a cPR or cCR after 3 months of azacitidine in the first 18 subjects with JMML, then enrollment will be stopped.
The study is designed to investigate the natural course of Primary snoring in 1-2 years or more and the different effect of drug and surgical treatment applied in children with obstructive sleep apnea (OSAS) by comparing the polysomnography(PSG) and sleep questionaires in 6 months after treatment.
This phase I trial studies the side effects and best dose of total bone marrow and lymphoid irradiation when given together with chemotherapy before donor stem cell transplant in treating patients with myelodysplastic syndrome or acute leukemia. Total marrow and lymphoid irradiation is a type of radiation therapy that targets bone marrow and blood, where the cancer is, instead of applying radiation to the whole body. Stem cell transplants use high doses of chemotherapy and radiation therapy, such as total marrow and lymphoid irradiation, to kill cancer cells, but these treatments kill normal cells as well. After chemotherapy, healthy cells from a donor are given to the patient to help the patient grow new blood cells.
Significant clinical improvements of ME/CFS symptoms were observed in two patients with long-standing ME/CFS who received adjuvant chemotherapy including cyclophosphamide for breast cancer, also in one ME/CFS patient who received chemotherapy including iphosphamide for Hodgkin lymphoma. Three pilot ME/CFS patients were thereafter treated with six intravenous infusions four weeks apart, in two of these with a significant clinical response. The hypothesis is that a subset of ME/CFS patients have an activated immune system, and that ME/CFS symptoms may be alleviated by treatment with cyclophosphamide as intravenous pulse infusions four weeks apart, six infusions in total. The purpose of the present study is to treat ME/CFS patients with cyclophosphamide as intravenous pulse infusions four weeks apart, six infusions in total. The effects on ME/CFS symptoms and tolerability/side effects during 12 months follow-up will be registered, and additional tests will be performed to objectively register changes in physical ability during follow-up. Studies to investigate possible large vessel endothelial dysfunction and skin microvascular dysfunction will be performed before start of intervention and during follow-up.