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NCT ID: NCT01557400 Completed - Clinical trials for Duchenne Muscular Dystrophy

Study of Ataluren for Previously Treated Participants With Nonsense Mutation Duchenne/Becker Muscular Dystrophy (nmDBMD) in Europe, Israel, Australia, and Canada

Start date: May 20, 2012
Phase: Phase 3
Study type: Interventional

Duchenne/Becker muscular dystrophy (DBMD) is a genetic disorder that develops in boys. It is caused by a mutation in the gene for dystrophin, a protein that is important for maintaining normal muscle structure and function. Loss of dystrophin causes muscle fragility that leads to weakness and loss of walking ability during childhood and teenage years. A specific type of mutation, called a nonsense (premature stop codon) mutation, is the cause of DBMD in approximately 10-15% of boys with the disease. Ataluren is an orally delivered, investigational drug that has the potential to overcome the effects of the nonsense mutation. This study comprises a Phase 3, open-label study of ataluren in participants with nmDBMD who previously received ataluren at an Investigator site in a prior PTC-sponsored clinical study. A separate open-label study (PTC124-GD-016-DMD; NCT01247207) is being conducted for nmDBMD participants who previously received ataluren at an Investigator site in the United States (US).

NCT ID: NCT01556399 Completed - Duodenal Diseases Clinical Trials

Endoscopic Characteristics of Duodenal and Ampullary Lesions

DUO/AMP-LST
Start date: November 2011
Phase:
Study type: Observational

The purpose is to investigate whether polyps that look different at endoscopy, have formed via different mutations and have different risks of turning into cancer.

NCT ID: NCT01554592 Completed - Heart Failure Clinical Trials

Polypharmacy in the Heart Failure Patient: Are All Prescribed Drug Classes Required? Statin Withdrawal Study

Start date: March 2012
Phase: N/A
Study type: Interventional

Heart failure (cardiomyopathy) is a chronic condition in which the heart fails to function as a pump to move blood around the body. This sets up a complex physiologic response to compensate, which include activation of many hormonal mechanisms which result in fluid accumulation. In recent years, medications to block the hormonal response to heart failure are given as standard drugs, and these include ACE inhibitors and beta blockers. Mortality is reduced with these medications, as well as symptoms improved. Other medications are also used in heart failure, for which a clear-cut benefit has not been demonstrated. Statins, also called HMG CoA reductase inhibitors, are used to reduce cholesterol levels and can help to prevent heart failure by preventing heart attacks. They have been used in heart failure that is not caused by heart attacks in the belief that they had "pleiotropic" effects, meaning that they had beneficial effects in heart failure separate from the reduction in cholesterol. However large trials in heart failure have demonstrated that statins do not increase survival compared with placebo. There is no evidence to recommend their routine use in established heart failure caused by either heart attacks or genetics. The investigators propose that the use of statins in heart failure is unnecessary and could be stopped. The importance of finding evidence to cease unproven medications in heart failure cannot be understated. Patients with heart failure take an average of six prescription medications each day. Each medication has side effects and the interactions of all the drugs together are unknown. Statins are the commonest reason for side effects in patients with heart failure, causing muscle pains and gastrointestinal upset. In this study, the investigators plan to withdraw statins from patients with stable heart failure in a closely monitored environment and watch for the effect of this on heart failure and on how they feel generally.

NCT ID: NCT01554280 Completed - Clinical trials for Constriction, Pathological

Fully Coated, Removable, Self-expanding Oesophageal Stents for Preventing Strictures Following Complete Barretts Excision.

CBE-001-SEOS
Start date: January 2012
Phase: N/A
Study type: Interventional

The purpose of this study is to investigate whether preventative placement of a removable oesophageal stent reduces the rate of scar tissue, or stricture formation after removing the precancerous or early cancerous Barrett's mucosa by Endoscopic Mucosal Resection (EMR). The stent will be placed 10-14 days after initial EMR. The stent will then be removed 8 weeks later by repeat Endoscopy. Patients will be followed up weekly following insertion of the oesophageal stent.

NCT ID: NCT01553149 Completed - Clinical trials for Neurofibromatosis Type 1

Low-Dose or High-Dose Lenalidomide in Treating Younger Patients With Recurrent, Refractory, or Progressive Pilocytic Astrocytoma or Optic Pathway Glioma

Start date: July 5, 2012
Phase: Phase 2
Study type: Interventional

This randomized phase II trial studies how well low-dose lenalidomide works compared with high-dose lenalidomide in treating younger patients with juvenile pilocytic astrocytomas or optic nerve pathway gliomas that have come back (recurrent), have not responded to treatment (refractory), or are growing, spreading, or getting worse (progressive). Lenalidomide is classified as an immunomodulatory drug as it boosts the immune system. It has other potential anti-tumor effects, for example, it may stop the growth of tumor cells by blocking blood flow to the tumor. It is not yet known whether low-dose lenalidomide is more or less effective than high-dose lenalidomide in treating patients with juvenile pilocytic astrocytomas or optic nerve pathway gliomas.

NCT ID: NCT01551160 Completed - Clinical trials for Hospital Rapid Response Team

Impact of a Communication and Team-working Intervention on Performance and Effectiveness of a Medical Emergency Team

IMPACT
Start date: July 2014
Phase: N/A
Study type: Interventional

Patients in hospital can have unexpected clinical emergencies. When this occurs the Medical Emergency Team (MET) are called with the intention of resolving the problem. Previous investigations have found that patients who have more than one call during their admission have worse outcomes than patients who only have one call. But it has not been established why. The aim of this research will be to examine these repeated calls and why patients subject to them go on to have worse outcomes. A predictive model will be developed to identify potential sources of risk. One potential source is poor communication between health care providers. An intervention to improve communication around MET calls may provide benefit to patients and improve outcomes.

NCT ID: NCT01550484 Completed - Parkinson's Disease Clinical Trials

A Trial of 18F-AV-133 Positron Emission Tomography (PET) Imaging to Differentiate Subjects With Parkinson's Disease (PD) From Other Movement Disorders

Start date: April 2012
Phase: Phase 2/Phase 3
Study type: Interventional

The purpose of this study is to determine whether 18F-AV-133 PET scans can be used to differentiate subjects with Parkinson's Disease from other movement disorders.

NCT ID: NCT01549938 Completed - Clinical trials for Vitamin D Deficiency

Cholecalciferol Intervention to Prevent Respiratory Infections Study

CIPRIS
Start date: May 2012
Phase: Phase 2
Study type: Interventional

This is a feasibility double-blind randomised controlled trial in 32 participants. It evaluates the feasibility of a full trial which will examine the efficacy of weekly supplementation of cholecalciferol (vitamin D3) relative to placebo on the subsequent frequency and severity of objectively-verified symptomatic acute respiratory tract infection, overall and as a proportion of detected colonisations of the upper respiratory tract by 9 of the most common aetiologic viral pathogens.

NCT ID: NCT01545453 Completed - Asthma Clinical Trials

A Study of Lebrikizumab in Patients With Uncontrolled Asthma Who Are on Inhaled Corticosteroids and A Second Controller Medication (VERSE)

Start date: March 2012
Phase: Phase 2
Study type: Interventional

This randomized, multicenter, double-blind, placebo-controlled, parallel-group study will assess the efficacy and safety of lebrikizumab in patients with asthma whose disease remains uncontrolled despite daily therapy with an inhaled corticosteroid and a second controller medication. Patients will be randomized in a 1:1:1:1 ratio to receive double-blind treatment with subcutaneous lebrikizumab ("highest", "middle", "lowest" dose) or placebo every 4 weeks for 52 weeks, in addition to their standard-of-care therapy. This will be followed by a 52-week double-blind active treatment extension. The anticipated time on study treatment is up to 104 weeks. There will be a safety follow-up of 24 weeks after the last dose of study drug for all patients.

NCT ID: NCT01545440 Completed - Asthma Clinical Trials

A Study of Lebrikizumab in Patients Whose Asthma is Uncontrolled With Inhaled Corticosteroids and A Second Controller Medication (LUTE)

Start date: March 2012
Phase: Phase 2
Study type: Interventional

This randomized, multicenter, double-blind, placebo-controlled, parallel-group study will assess the efficacy and safety of lebrikizumab in patients with asthma whose disease remains uncontrolled despite daily therapy with an inhaled corticosteroid and a second controller medication. Patients will be randomized in a 1:1:1:1 ratio to receive double-blind treatment with subcutaneous lebrikizumab ("highest", "middle", "lowest" dose) or placebo every 4 weeks for 52 weeks, in addition to their standard-of-care therapy. This will be followed by a 52-week double-blind active treatment extension. The anticipated time on study treatment is up to 104 weeks. There will be a safety follow-up of 24 weeks after the last dose of study drug for all patients.