View clinical trials related to Syndrome.
Filter by:There is no specific treatment for core symptoms of PWS. Regarding behavioral and psychiatric symptoms (hyperphagia, imulsivity and self-mutilations), one of the only drug options consists in antipsychotics, that are not efficient and might be responsible for a worsening of the weight gain (major issue in PWS). An alternative therapeutic approach for behavioral disturbances has been suggested by some authors with topiramate (Epitomax®), an antiepileptic drug that can be used as a mood stabilizer and anti-impulsive. In addition, topiramate is used as a treatment for eating disorders because it induces loss of weight and appetite. This last effect might be useful in the case of SPW. Except for some clinical case reports, the investigators only found one open study for topiramate in SPW 8 patientssuggesting promising effects. There si however no placebo controlled study.. Objective: To evaluate the efficacy of topiramate (200 mg / d) on Eating disorders (E), self Mutilations (M), irritability and Impulsivity (I), metabolic status, and tolerance among of PWS patients. Methodology: This is a multicenter (out-patients in Toulouse, Reims, Nantes and Paris and in-patients in Hendaye) 8 weeks double-blind placebo controlled study . Subjects (n = 125 for 112 analyzable) all having PWS, aged 12 years-old and more should have any of the following symptoms: E, M and U (see above). All subjects will be randomly allocated into two groups one taking a placebo, the other taking topiramate (50mg / day initially, increasing up to 50mg per week 200mg / day). The population of analyzable patients in and out patient will be of equal size (n = 56). The inclusion period is two years.. Are excluded subjects with antipsychotic or mood stabilizer medication or topiramate. The primary endpoint will be the rate of responders, with response defined by obtaining a score of 1 or 2 on the CGI improvement after 8 weeks of treatment Other assessments, secondary endpoints : - Clinic: Weight / Size / Self-injury behavior (french Echelle des Conduites Auto et Hétéro Aggressives, ECAHA)) - Psychometric: C-SHARP and A-SHARP / Conners (Impulsivity) / Dickens (Eating behavior for PWS) - Organic: NFS, serum electrolytes, creatinine, ammonia plasma, serum bicarbonate, AST / ALT / GGT, ghrelin, fasting glucose, lipid profile and insulin, leptin, TG and HbA1c. - Side effects of topiramate: SAPS / SANS and BPRS (hallucinations), anxiety scales and laboratory tests.
This study evaluates the safety, tolerability and efficacy of Sarizotan in reducing respiratory abnormalities in Rett Syndrome in an initial double blind 24 week period followed by an open label treatment phase of up to 168 weeks (the latter for patients with no safety and tolerability issues).
This a Phase 2 randomized, open-label, two-stage study designed to investigate the antitumor activity of tipifarnib in approximately 36 eligible subjects with MDS who have no known curative treatment. Subjects will be randomized to receive tipifarnib orally with food according to one of 2 treatment regimens.
This study compared the administration of porcine surfactant (poractant alfa, Curosurf®) through a less invasive method (LISA), using a thin catheter, CHF 6440 (LISACATH®), during non-invasive ventilation (CPAP, NIPPV, BiPAP) with an approved conventional surfactant administration during invasive ventilation followed by rapid extubation in terms of short term and mid-term safety and efficacy in spontaneously breathing preterm neonates who have clinical signs of respiratory distress syndrome (RDS).
Primary purpose : mortality at Day 28 Secondary purposes : - Daily SOFA (Sequential Organ Failure Assessment) score lessening within Intensive Care Unit (ICU) - Duration of increasing doses of norepinephrine infusion to maintain target mean arterial pressure - Number of care-related infections within ICU
The purpose of this study is to evaluate the use of IRX5183 in 1) patients with relapsed and/or refractory AML and 2) patients with high-risk MDS or chronic myelomonocytic leukemia (CMML).
This is a Phase I-II open- label single-dose study in subjects with significant refractory Rheumatoid Arthritis (RA), relapsing Systemic Lupus Erythematosus (SLE) or Sharp's Syndrome (SS). This study will enroll a minimum of 20 subjects for RA, 20 subjects for SLE and 20 patients for SS. 6 week data of serum Tumor Necrosis Factor- alpha (TNFa), Interleukin- 6 (IL-6), C- Reactive Protein (CRP), Erythrocyte Sedimentation Rate (ESR), Cluster of Differentiation (CD)4 +CD25 + Forkhead box P3(Foxp3) + regulatory T cells, Disease Activity Score for 28 joints (DAS-28) score and pain score will be collected in all patients who are enrolled in the study for the RA group (Baseline and 6 weeks after). For the SLE group, Transforming Growth Factor- beta (TGF-β), TNFa, IL-6, Interleukin- 17 (IL-17), CD3+CD8-IL17A+ T helper-17 (Th17) cells, CD4+CD25+Foxp3+ regulatory T cells and the Systemic Lupus Erythematosus Quality of Life Questionnaire (SLEQoL) score will be collected in all the subjects of this group. SS group will undergo the assessments of RA and SLE. Prior to the stem cell treatment, the patient will be assessed for 6 weeks by all the previously mentioned markers. Then, patients will receive the infusion of stromal vascular fraction cells containing the adult adipose derived stem cells 'aADSC' (single intravenous dose). The disease- modifying anti-rheumatic drugs (DMARDs) or the standard SLE treatment will not be interrupted with the exception of systemic steroids (excluding minimal maintenance dose of one steroid) during the duration of the study. Follow up visits will take place at 6 weeks, 3 Months and 6 Months after the cell infusion. Safety will be monitored on an ongoing basis, and an interim safety review will be conducted by the Investigator(s) and Sponsor after the first 10 patients have been enrolled and treated in each group.
This pilot phase I/II trial studies the side effects and how well sirolimus and mycophenolate mofetil work in preventing graft versus host disease (GvHD) in patients with hematologic malignancies undergoing hematopoietic stem cell transplant (HSCT). Biological therapies, such as sirolimus and mycophenolate mofetil, use substances made from living organisms that may stimulate or suppress the immune system in different ways and stop tumor cells from growing. Giving sirolimus and mycophenolate mofetil after hematopoietic stem cell transplant may be better in preventing graft-versus-host disease.
Churg-Strauss syndrome is a rare autoimmune inflammatory disease affecting medium- and small-sized blood vessels, causing asthma, abnormalities of the blood, lung diseases, and neuropathy. The main cause of death in these patients is heart attack. Without therapy, the 5-year survival in patients with Churg-Strauss syndrome is 25%. Although with the 5-year survival is increased to 62% with the appropriate therapy, many patients remain refractory to therapy. The long term outcome of these patients remains grim. The aim of this research study is to determine if suppressing the immune system using a combination of high dose chemotherapy, antibodies, and radiation followed by stem cell transplant will abolish the 'bad' immune system and let the patient's body establish a new immune system that does not attack the blood vessels.
The objectives of this trial are to evaluate the safety, tolerability, maximum tolerated dose (MTD), pharmacokinetics and preliminary efficacy of volasertib in two dosing schedules of intravenous volasertib as monotherapy or in combination with azacitidine in patients with myelodysplastic syndrome (MDS) after hypomethylating agents (HMA) treatment failure.