Active clinical trials for "Syndrome"

The primary objective of this study was to evaluate the efficacy of GWP42003-P as adjunctive treatment in reducing the number of drop seizures when compared with placebo in participants with Lennox-Gastaut syndrome (LGS).

Polycystic ovary syndrome (PCOS) is a syndrome which includes elevated androgen levels, irregular menstrual cycles and insulin resistance. Standard treatments, which include weight loss and medications to improve insulin secretion are only partly successful, and may require that young women take medications for decades. The study investigators have been evaluating the effects of specific diets on insulin resistance in healthy volunteers and subjects with type 2 diabetes, and have found that subjects with insulin resistance seem to respond particularly well to these diet regimens. Volunteers with PCOS are being asked to participate to see if following these diets can help regularize your menstrual cycles. The results of this study may help improve fertility treatments for women with PCOS.

The purpose of this study is to evaluate the feasibility and effectiveness of acupuncture for the treatment of phantom limb syndrome in lower limb amputees and pilot an acupuncture protocol. The study will be a comparative effectiveness study comparing acupuncture and standard care versus standard care alone and will be a randomised controlled trial using a mixed methods approach. The study hypothesis is a course of acupuncture will reduce the symptoms of phantom limb syndrome in lower limb amputees. The study will be based the Amputee Rehabilitation Unit, London. Twenty lower limb amputees with phantom limb syndrome will be randomly assigned to either receive usual care or usual care plus acupuncture. Acupuncture intervention will include 8 treatments over 4 weeks and treatment will involve using both body and auricular acupuncture points. Usual care will include physiotherapy, occupational therapy, pharmacological intervention and other interventions as deemed appropriate by clinical staff. An eleven point numerical rating scale will be the primary outcome measure in this study. Secondary outcome measures will include the Short Form McGill Pain Questionnaire 2, EQ-5D-5L, Hospital Anxiety and Depression Scale, Perceived Stress Scale, Insomnia Severity Index, Participant Global Impression of Change and information will also be obtained on rescue medication. A cheek swab will also be taken to explore potential subgroups of responders in relation to the genetic polymorphism of the glucocorticoid receptor. Outcomes will be recorded weekly for the duration of the study and one and three months post completion of the study. In order to identify protocol acceptability and acceptability of outcome measures five participants will be interviewed post completion of the study. Data will also inform feasibility such as recruitment rate, completion rate and reasons for dropout. Data from this study will guide the development of a full randomised controlled trial.

The goals of the project is to evaluate the effects of Heliox therapy on obstructive sleep apnea syndrome (OSAS).

The purpose of this study is to determine the safety and tolerability [maximum tolerated dose (MTD) within planned dosing range] of intravenous (IV) infusions of TPI 287 administered once every 3 weeks for 9 weeks (for a total of 4 infusions) in patients with primary four repeat tauopathies (4RT), corticobasal syndrome (CBS; also called corticobasal degeneration, CBD) or progressive supranuclear palsy (PSP).

Fecal Microbial Transplantation in Treatment of Irritable Bowel Syndrome; a Double Blinded Placebo Controlled Trial - the REFIT project Irritable bowel syndrome (IBS) is a condition characterized by variable complaints like diarrhoea, bloating and abdominal pain, and may result in considerably reduced quality of life and increased sickness absence. The prevalence of IBS in the general population may be as high as 15 per cent depending on the diagnostic criteria used. The pathophysiology of IBS is poorly understood and theories of visceral hypersensitivity, micro-inflammation and other less well founded theories have been stated. So far, no measurement or test can affirm diagnosis, but exclusion of organic diseases in combination with a typical symptom pattern according to the Rome classification can set the diagnosis. Earlier non-controlled case reports have shown a convincing effect of fecal microbial transplantation (FMT) in IBS patients. However, no placebo controlled trial has been performed in this condition. Changes in the gut microbiome may be an important factor in IBS pathogenesis. Microbiome analysis has revealed changes in microbiome composition that may trigger changes in visceral sensibility and pain perception. The fecal microbial transplantation (FMT) procedure has been used primarily to treat clostridium difficile infections. Few minor side effects have been reported. Hypothesis: IBS is caused by an imbalance of the gut microbiome that may be reset by transplanting a microbiome sample from a healthy donor. Aim of study: To test the clinical effect of FMT in patients with IBS To describe the fecal microbiome in IBS patients To describe changes in the fecal microbiome of IBS patients following FMT The REFIT study will perform a randomized placebo-controlled double blinded trial of FMT on IBS according to the Rome 3 criteria. A study group of 60 IBS (diarrhoea variant) patients will recruited from general practice and allocated to active (30) or placebo (30) by block randomization. Donors will be 15 healthy volunteers with no risk behaviour and a pre-screening for communicable diseases will be performed. Outcome measures will be clinical assessment by IBS-severity scoring system (IBS-SSS) at 0, 1, 3, 6, and 12 months. Microbiome analysis will be performed by metagenomic sequencing (University of Tromsø) at 0, 3, and 12 months for patients, and at inclusion for donors.

The investigators will conduct a pilot study investigating the effectiveness of a computerized response inhibition training program at reducing the response inhibition difficulties often seen in children with Williams syndrome ages 10-17. The investigators hypothesize that after completing the training program, children with Williams syndrome will show improvement on computerized measures of response inhibition and on parent measures of impulsivity.

In the management of acute coronary syndromes with ST-segment elevation (STEMI), early analgesia reduces the effects of hyperadrenalism which increases the size of myocardial infarction. In order to reduce pain intensity, the recommendations advocate emergency use of morphine. In STEMI patients, other analgesic treatments could provide analgesia that is at least as effective as morphine. The equimolar oxygen/nitrous oxide mixture (MEOPA) is widely used in emergency medicine and has minor secondary effects that are very rapidly reversible when inhalation is discontinued. Used in association with paracetamol, it could be an at least equally effective alternative to the use of morphine.

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 purpose of this study is to examine the safety and efficacy of Allopregnanolone as a possible treatment for symptoms of Fragile X-associated Tremor/Ataxia Syndrome (FXTAS).