Lancet neurology
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Randomized Controlled Trial Multicenter Study
High-dose albumin treatment for acute ischaemic stroke (ALIAS) Part 2: a randomised, double-blind, phase 3, placebo-controlled trial.
In animal models of ischaemic stroke, 25% albumin reduced brain infarction and improved neurobehavioural outcome. In a pilot clinical trial, albumin doses as high as 2 g/kg were safely tolerated. We aimed to assess whether albumin given within 5 h of the onset of acute ischaemic stroke increased the proportion of patients with a favourable outcome. ⋯ National Institute of Neurological Disorders and Stroke, US National Institutes of Health; and Baxter Healthcare Corporation.
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Randomized Controlled Trial Multicenter Study
Dexpramipexole versus placebo for patients with amyotrophic lateral sclerosis (EMPOWER): a randomised, double-blind, phase 3 trial.
In a phase 2 study, dexpramipexole (25-150 mg twice daily) was well tolerated for up to 9 months and showed a significant benefit at the high dose in a combined assessment of function and mortality in patients with amyotrophic lateral sclerosis. We aimed to assess efficacy and safety of dexpramipexole in a phase 3 trial of patients with familial or sporadic disease. ⋯ Biogen Idec.
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Randomized Controlled Trial Multicenter Study Comparative Study
Pitolisant versus placebo or modafinil in patients with narcolepsy: a double-blind, randomised trial.
Narcolepsy is characterised by excessive daytime sleepiness (EDS) and cataplexy. Histamine neurons are crucial to maintain wakefulness. We assessed the safety and efficacy of pitolisant (previously called BF2.649), a selective histamine H3 receptor inverse agonist that activates these neurons, in patients with narcolepsy. ⋯ Bioprojet, France.
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Randomized Controlled Trial Multicenter Study
Simvastatin for cognitive deficits and behavioural problems in patients with neurofibromatosis type 1 (NF1-SIMCODA): a randomised, placebo-controlled trial.
Neurofibromatosis type 1 is a common genetic disorder characterised by neurocutaneous manifestations and cognitive and behavioural problems. Statins were shown to reduce analogous learning deficits in a mouse model of the disease, but a short-term trial in humans was inconclusive. We aimed to assess the use of simvastatin for the improvement of cognitive and behavioural deficits in children with neurofibromatosis type 1 for 12 months. ⋯ The Netherlands Organization for Health Research and Development (ZonMw), Research Foundation Flanders (FWO-Vlaanderen), Marguerite-Marie Delacroix Foundation, and the Dutch Neurofibromatosis Association (NFVN).
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Available treatment options for relapsing-remitting multiple sclerosis (MS) have expanded in recent years, and several injectable therapies are under development. In this Rapid Review, we summarise emerging injectable therapies for relapsing-remitting MS, and discuss pharmacological mechanisms, clinical trials, adverse events, and use in clinical practice. Many new potential treatments for MS are at an intermediate to advanced stage of development. ⋯ Ofatumumab is a monoclonal antibody that has shown efficacy in a small phase 2 trial. Animal models suggest that anti-LINGO1 antibody has remyelinating potential, and phase 2 trials of the antibody are underway. Further clarification of purported mechanisms of action and continued surveillance will be essential to establish the safety and clinical efficacy of these drugs in patients with relapsing-remitting MS.