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A Double-Blind, Placebo-Controlled, Randomized, Parallel Group, 12-Month Safety and Efficacy Trial of Leuco-methylthioninium bis(hydromethanesulfonate) in Subjects with Behavioral Variant Frontotemporal Dementia (bvFTD)

Primary:To demonstrate the efficacy of LMTM as assessed by the change from Baseline on:* Addenbrooke*s Cognitive Examination Revised (ACE-R)* Symptomatic effect as reflected by the Functional Activities Questionnaire (FAQ)* Disease-modifying effect…

Phase 3b Open-Label, Multicenter, Safety Study of BIIB037 (aducanumab) in Subjects With Alzheimer*s Disease Who Had Previously Participated in the Aducanumab Studies 221AD103, 221AD301, 221AD302 and 221AD205

Primary objective:To evaluate the safety and tolerability of aducanumab over 100 weeks of treatment after a wash-out period imposed by discontinuation of feeder studies in participants who had previously received aducanumab (i.e., previously treated…

Open label extension study for patients with early Alzheimer's Disease (AD) enrolled in study ANAVEX2-73-AD-004

Primary objective:To continue assessing the safety and tolerability of ANAVEX2-73.Safety and Tolerability Measures:* Physical examination* Vital signs (heart rate, respiratory rate, systolic blood pressure [SBP], diastolic blood pressure [DBP],…

An open-label, single-arm, multicenter study of intracerebral administration of adeno-associated viral vectors serotype rh.10 carrying the human N-sulfoglycosamine sulfohydrolase (SGSH) cDNA for the treatment of mucopolysaccharidosis type IIIA (MPS IIIA)

Primary: To assess the efficacy of intracerebral delivery of AAVrh.10SGSH gene therapy (LYS-SAF302) in improving or stabilizing the neurodevelopmental status of MPS IIIA patients after 24 months (main cohort), compared to the expected evolution…

A Phase 2b/3, Double-Blind, Randomised, Placebo-Controlled 48-week Safety and Efficacy trial of ANAVEX2-73 for the Treatment of Early Alzheimer*s Disease (AD).

Primary Objectives:* Change from baseline to week 48 in cognition according to the Alzheimer Disease Assessment Scale-Cognition (ADAS-Cog) compared to placebo.* Changes from baseline to week 48 in ability to perform daily activities according to the…

A Phase 3, multicenter, randomized, double-blind, placebo-controlled study to assess the efficacy, safety, and tolerability of AVP-786 (deudextromethorphan hydrobromide [d6-DM]/quinidine sulfate [Q]) for the treatment of agitation in patients with dementia of the Alzheimer*s type.

The primary objective is to:* Evaluate the efficacy, safety, and tolerability of AVP-786 compared to placebo for thetreatment of agitation in patients with dementia of the Alzheimer*s typeThe secondary objectives are to:* Evaluate the effects of AVP…

A Phase 3, multi-center, randomized, double-blind, parallel-group, placebo-controlled clinical trial to evaluate the efficacy and safety of sodium oligomannate (GV-971) in treatment of mild to moderate Alzheimer's disease (GREEN MEMORY: GREen Valley 971 EvaluatioN Memory)

To assess the efficacy of GV-971 compared with placebo on cognition and global function in participants with mild to moderate Alzheimer's disease (AD).

Randomized, controlled growth hormone study in children and adolescents with a Prader-Willi-like phenotype, followed by a two year open-label growth hormone study - effects on body composition, growth, psychosocial development and quality of life

To investigate the effects of GH treatment on body composition, psychosocial development, psychomotor development, growth, glucose metabolism, serum lipids, cognition, behaviour, bone mineral density, quality of life and safety parameters in…

A Phase 3 Multicenter, Randomized, Double-Blind, Placebo-Controlled, Parallel-Group Study to Evaluate the Efficacy and Safety of Aducanumab (BIIB037) in Subjects with Early Alzheimer's Disease

The objectives are to evaluate the long-term safety and tolerability profile of aducanumab in subjects with early AD, and to evaluate the long-term efficacy of aducanumab treatment as measured by clinical, radiological and additional assessments…

The Sustained Aeration of Infant Lungs Study

In this research study we compare two approaches that can be used to give breathing support to prematurely born babies. Both techniques are currently used in different countries over the world. However, at this moment we don*t know which methods is…