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Syndrome clinical trials

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NCT ID: NCT01325740 Suspended - Fragile X Syndrome Clinical Trials

A Study to Assess the Tolerability of a Single Dose of STX107 in Adults With Fragile X Syndrome

Start date: May 2011
Phase: Phase 2
Study type: Interventional

The study will consist of a Screening period (up to 14 days), a Treatment period, and a Follow-Up period. Sixteen subjects will be enrolled into two sequential dose cohorts - 10 or 30 mg (or matching placebo) across four study centers.

NCT ID: NCT01325220 Completed - Fragile X Syndrome Clinical Trials

Efficacy and Safety Study of STX209 (Arbaclofen) for the Treatment of Social Withdrawal in Children With Fragile X Syndrome

Harbor-C
Start date: June 2011
Phase: Phase 3
Study type: Interventional

There will be four study periods: Screening (up to 14 days in length), the Treatment Period (8 weeks), the Withdrawal Period (22 days), and Follow-up Period (up to 31 days). Subjects will be randomized to receive either STX209 (5 mg twice daily [BID], 10 mg BID or 10 mg three times daily [TID]) or placebo. Efficacy,safety and tolerability assessments will be performed periodically

NCT ID: NCT01324960 Withdrawn - Clinical trials for Myelodysplastic Syndromes

Azacitidine With or Without Ceplene/Interleukin-2 in Patients With Higher Risk Myelodysplastic Syndromes

Start date: March 2011
Phase: Phase 1/Phase 2
Study type: Interventional

A phase I study of azacitidine with Ceplene/interleukin-2 will first evaluate the safety and tolerability of this regimen in patients with higher risk myelodysplastic syndromes (MDS) who achieved a hematological response after 6 cycles of azacitidine. After approval by an independent Data Safety Monitoring Board (DSMB), the phase I study will be followed by an open label randomized phase II study designed to characterize the efficacy, safety, and tolerability of the addition of Ceplene/interleukin-2 to azacytidine in patients with higher risk myelodysplastic syndrome (MDS) who achieved a hematological response after 6 cycles of azacitidine.

NCT ID: NCT01324895 Completed - Clinical trials for Small Intestinal Bacterial Overgrowth Syndrome (SIBO)

Retrospective Analysis of Treatment Outcomes in Patients With Bacterial Overgrowth Syndrome Diagnosed by D-Xylose Breath Testing

SIBO
Start date: August 2010
Phase: N/A
Study type: Observational

The objective of this study is to compare the efficacy of prokinetics versus antibiotics versus a combination of antibiotics plus prokinetics in the eradication of Small-Intestinal Bacterial Over-Growth Syndrome (SIBO) in those with and without a positive D-xylose Breath Test. Hypothesis: Patients with SIBO treated with a combination of prokinetics and in particular octreotide and antibiotics will have reduced recurrence rates of SIBO than either therapy given alone.

NCT ID: NCT01323218 Recruiting - Clinical trials for Obstructive Sleep Apnea-hypopnea Syndrome

Sleep Apnea Syndrome and Vitamin D

Start date: March 2011
Phase: Phase 2
Study type: Interventional

Obstructive sleep apnea/hypopnea syndrome (OSAHS) is a highly prevalent disorder with multiple comorbidities. OSAHS is characterized by repetitive episodes of airflow reduction (hypopnoea) or cessation (apnoea) due to upper airway collapse during sleep. Its major risk factor is obesity. However, its pathogenesis is complex and multifactorial. Reduced upper airway muscle tonus and/or unstable neuromuscular output seem to be involved in this collapsus. A normal vitamin D status is necessary for normal muscle function and neuromuscular output. As obesity is associated with a high rate of hypovitaminosis D, it appears of interest to evaluate the effect of vitamin D supplementation on OSAHS patients with vitamin D deficiency.

NCT ID: NCT01322165 Completed - Turner Syndrome Clinical Trials

National Registry of Genetically Triggered Thoracic Aortic Aneurysms and Cardiovascular Conditions

GenTAC
Start date: November 2007
Phase: N/A
Study type: Observational

The National Registry of Genetically Triggered Thoracic Aortic Aneurysms and Cardiovascular Conditions (GenTAC) was initiated in 2006 by the National Heart, Lung, and Blood Institute (NHLBI) and the National Institute of Arthritis and Musculoskeletal and Skin Diseases (NIAMS). GenTAC established a registry of 3706 patients with genetic conditions that may be related to thoracic aortic aneurysms and collected medical data and biologic samples. The study ended in September 2016. Data and samples are available from NHLBI and requests should be made to BioLINCC. See the NHLBI website for more information: https://www.nhlbi.nih.gov/research/resources/gentac/.

NCT ID: NCT01319851 Terminated - Sickle Cell Disease Clinical Trials

Alefacept and Allogeneic Hematopoietic Stem Cell Transplantation

Start date: September 2010
Phase: N/A
Study type: Interventional

Allogeneic blood and marrow transplantation remains the only viable cure for children who suffer from many serious non-malignant hematological diseases. Transplantation, however, carries a high risk of fatal complications. Much of the risk stems from the use of high dose radiation and chemotherapy for conditioning, the treatment administered just prior to transplant that eliminates the patients' marrow and immune system, effectively preventing rejection of the donors' cells. Attempts to make blood and marrow transplantation safer for children with non-malignant diseases by using lower doses of radiation and chemotherapy have largely failed because of a high rate of graft rejection. In many such cases, it is likely that the graft is rejected because the recipient is sensitized to proteins on donor cells, including bone marrow cells, by blood transfusions. The formation of memory immune cells is a hallmark of sensitization, and these memory cells are relatively insensitive to chemotherapy and radiation. Alefacept, a drug used to treat psoriasis, on the other hand, selectively depletes these cells. The investigators are conducting a pilot study to begin to determine whether incorporating alefacept into a low dose conditioning regimen can effectively mitigate sensitization and, thereby, prevent rejection of allogeneic blood and marrow transplants for multiply transfused children with non-malignant hematological diseases.

NCT ID: NCT01319344 Completed - Metabolic Syndrome Clinical Trials

Effect of Eplerenone on Endothelial Function in Metabolic Syndrome

MetSyn
Start date: September 2010
Phase: Phase 3
Study type: Interventional

Patients with the metabolic syndrome (MetSyn) are at increased risk for cardiovascular mortality and morbidity.This increased cardiovascular risk is attributed to metabolic dysregulations like impaired glucose tolerance or diabetes mellitus and dyslipidemia, abdominal obesity and arterial hypertension, which promote oxidative stress and inflammation with consecutive endothelial dysfunction causing an atherogenic environment. Aldosterone promoted end organ damage is mainly found in the cardiovascular system and the kidney. Inflammation and activation of different factors promotes fibroblast growth and matrix production resulting in myocardial fibrosis, vascular remodelling and renal fibrosis. MetSyn and aldosterone are cardiovascular risk factors and it is of crucial importance to note that there is a connection between MetSyn and aldosterone. Other cross sectional studies show a direct correlation of aldosterone levels and impaired glucose metabolism in patients with and without the MetSyn. Taken together, aldosterone influences essential parameters of the MetSyn. Coincidentally parameters of the MetSyn are stimulus for an increased aldosterone synthesis, i.e. visceral adipocytes. In large scale clinical trials - RALES, EPHESUS, 4E - inhibition of MR has proven to be beneficial in patients with congestive heart failure and post myocardial infarction and this result has been confirmed for diabetic patients, who are known to have an increased cardiovascular risk. There is only very limited data on the impact of MR inhibition on metabolic, endocrine, and inflammatory parameters in patients with MetSyn, who have not yet suffered from cardiovascular events.

NCT ID: NCT01319162 Completed - Clinical trials for Polycystic Ovary Syndrome

Prevalence of Polycystic Ovary Syndrome (PCOS) in Obese Premenopausal Women

Start date: March 30, 2011
Phase: N/A
Study type: Observational

Between 40% and 85% of women with Polycystic Ovary Syndrome (PCOS) are overweight or obese and obesity is closely linked to the development of PCOS. Although it is well established that obesity increases the severity of the clinical features of PCOS, data regarding the prevalence of PCOS in obese women and the change in body weight in women with PCOS over time are scares. In a prevalence study it was investigated whether obesity increases the risk of PCOS in the general population and they demonstrated that the prevalence rate of PCOS in underweight, normal-weight, overweight, and obese women were 8.2, 9.8, 9.9, and 9.0%, respectively, similar to that observed in the general population. These results suggest that the risk of PCOS is only minimally increased with obesity. On the other hand, in a Spanish prevalence study among overweight and obese subjects, they demonstrated a 28.3% prevalence of PCOS, which is markedly higher compared with the 5.5% prevalence of PCOS in lean women in Spain. First the investigators aim to estimate the prevalence/probability of PCOS among obese, premenopausal women (between 18 and 50 years) with no symptoms of classic menopausal symptoms in Sweden. Secondly, to elucidate whether women diagnosed with PCOS respond to standard weight reduction regime to the same extent as women without PCOS.

NCT ID: NCT01318356 Completed - Clinical trials for Fatigue Syndrome, Chronic

The Qure Study: Q-fever Fatigue Syndrome - Response to Treatment

Qure
Start date: April 2011
Phase: Phase 4
Study type: Interventional

The objective of this study is to assess the efficacy of two treatment strategies for fatigue and disabilities in QFS: long term treatment with doxycycline or cognitive behavioral therapy (CBT).