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A multi-centre, double-blind, parallel-group, randomised controlled study to investigate efficacy, safety and tolerability of orally administered BI 409306 during a 12-week treatment period compared to placebo in patients with cognitive impairment due to Alzheimer's Disease.

The primary objective of this study is to assess efficacy and safety of BI 409306 at doses of 10 mg, 25 mg and 50 mg once daily, 25 mg twice daily compared to placebo over a 12-week treatment period in patients with the following criteria: mild…

A comparative phase2 study assessing the efficacy of triheptanoin, an anaplerotic therapy in Huntington's Disease (HD)

The primary objective of TRIHEP 3 is to evaluate the efficacy of triheptanoin in (i) increasing the short term energy response in the metabolic profile of the brain of early affected HD patients, as captured by 31P-MRS, and (ii) slowing atrophy in…

The effect of transcranial direct current stimulation on processing speed in patients after ischemic stroke

To evaluate the effects of tDCS on information processing speed in comparison to sham tDCS in patients after stroke with ascertained reduced processing speed. A proof of principle study.

2-STEP: A single-centre, phase II study to evaluate the safety, tolerability and pharmacokinetics of 2-Iminobiotin (2-IB) in neonates with gestational age of *36 weeks with moderate to severe perinatal asphyxia treated with therapeutic hypothermia

To explore the safety, tolerability and the pharmacokinetic profile of 2-IB when given on top of therapeutic hypothermia.

A Multi-Center, Open-Label Study Evaluating the Safety, Tolerability, and
Efficacy of Pridopidine in Patients with Huntington's Disease

Primary Objective: The primary objective of this study is to evaluate safety and tolerability of pridopidine in patients with HD.Secondary Objectives: The secondary objectives of the study are to assess the effects of long-term, open-label dosing…

A phase IIb, open-label study to assess the efficacy, safety, pharmacodynamics and pharmacokinetics of multiple subcutaneous doses of BMN 045 (previously known as PRO045) in subjects with Duchenne muscular dystrophy

Primary objective:To assess the efficacy of BMN 045 after 48 weeks treatment in ambulant subjects with Duchenne muscular dystrophy.Secondary objectives:To assess the safety and tolerability of BMN 045 after 48 weeks of treatment in all study…

An open-label multi-part first-in-human study of oral LMI070 in infants with Type 1 spinal muscular atrophy

Primary:The purpose of this study is to evaluate the safety, tolerability, orally administered LMI070 in patients with Type 1 SMA.pharmacokinetics (PK), pharmacodynamics (PD) and efficacy; and to estimate the Maximum Tolerated Dose (MTD) and optimal…

Grey matter microglial imaging with [18F]DPA-714 in progressive MS patients

The main objective of this study is to depict microglial activation behind a closed BBB in vivo and possibly establish an imaging marker of fast clinical progression, we will aim at visualizing microglial activity in vivo in cortical and hippocampal…

Care4Stroke+ program: Caregiver mediated exercises with e-health support for early supported discharge after acquired brain injury.

The primary aim of this study is to evaluate the feasibility, clinical effectiveness and cost effectiveness of a caregiver mediated exercises programme combined with e-health services (CARE4STROKE+) to improve self-reported health status and reduce…

Symptomatic treatment of vascular cognitive impairment

Primary Objective: To study the change on performance on executive function and memory function (as measured on Neurocart), after an active challenge with methylphenidate (monoaminergic) and galantamine (cholinergic), compared to placebo, in…

Metformin and sitagliptin in patients with impaired glucose tolerance and a recent TIA or minor ischemic stroke - safety and feasibility

We aim to compare the effectiveness, feasibility and safety of both metformin and sitagliptin in patients with TIA or minor ischemic stroke and impaired glucose tolerance. Furthermore, we will assess whether a slow increase in dose of metformin over…

Bumetanide in Autism Medication and Biomarker study

The primary objective of the proposed study is to investigate whether bumetanide therapy indeed reduces autistic symptomatology. Important secondary goals of this project are to determine whether bumetanide will improve specific behavioral,…

A Randomized, Two-Period, Double-Blind Placebo-Controlled and Open-Label, Multicenter Extension Study to Determine the Long-Term Safety and Tolerability of JNJ-54861911 in Subjects in the Early Alzheimer*s Disease Spectrum

The primary objective of this study is to evaluate the long-term safety and tolerability of JNJ-54861911 in subjects in the early AD spectrum that have completed a Phase 1b or Phase 2 clinical trial with JNJ 54861911 (e.g., Study 54861911ALZ2002),…

A Phase 2, Dose-Finding, Randomized, Parallel-Group, Double-Blind, Placebo-Controlled Study, Evaluating the Safety and Efficacy of Pridopidine 45 mg, 67.5 mg, 90 mg, and 112.5 mg Twice-Daily versus Placebo for Symptomatic Treatment in Patients with Huntington*s Disease

The primary objective of this study is to assess the efficacy of pridopidine 67.5 to 112.5 mg twice daily (bid) on motor impairment in patients with HD after 26 weeks of treatment using the Unified Huntington*s Disease Rating Scale (UHDRS) Total…

A placebo-controlled double blind randomized trial to investigate the efficacy and safety of the combination of Penicillin G / Hydrocortisone treatment in ALS patients (PHALS).

The objective is to compare the efficacy and safety of intravenous high-dose penicillin G and hydrocortisone versus placebo in combination with riluzole in the treatment of patients suffering from Amyotrophic Lateral Sclerosis (ALS).

A Multicenter, Multinational, Randomized, Double Blind, Placebo Controlled, Parallel Group Study to Evaluate the Efficacy and Safety of Laquinimod (0.5, 1.0 and 1.5 mg/day) as Treatment in Patients with Huntington's Disease

Primary Study Objective: The primary objective of this study is to assess the efficacy of laquinimod 0.5 mg and 1.0 mg qd in patients with HD after 12 months of treatment using the UHDRS-TMS.Secondary Study Objectives:• To assess the effect of…

A Phase I/II, open-label, dose escalating with 48-week treatment study to assess the safety and tolerability, pharmacokinetics, pharmacodynamics and efficacy of BMN 053 (previously knoen as PRO053) in subjects with Duchenne muscular dystrophy

Primary objective: To assess the efficacy of BMN 053 at recommended dosing regimen after 48 weeks treatment in ambulant subjects with Duchenne muscular dystrophy.Secondary objectives: - To assess the safety and tolerability of BMN 053 after single…

A randomized, double blind, double-dummy placebo controlled, 3-way cross-over study to determine the test-retest reliability of, and the effect of oral retigabine and riluzole on, peripheral motor nerve excitability measurements in patients with ALS.

Patients with ALS are routinely prescribed riluzole, the only registered drug for the treatment of ALS. This study will evaluate the concentration-effect relationship of riluzole (100 mg) and retigabine (300 mg) on axonal excitability measures. In…

Multicenter, open-label, single arm study to evaluate long-term safety, tolerability, and effectiveness of 10mg/kg olesoxime in patients with SMA

Main objective: The main objective of this open-label, single arm study is to further characterize the safety, tolerability and effectiveness profile of olesoxime in SMAThe primary safety objective for this study is as follows:To evaluate the safety…

A double-blind, randomized, placebo-controlled study of the efficacy, safety/tolerability, and pharmacokinetic profile of UCB0942 in adult patients with highly drug-resistant focal epilepsy

To assess the efficacy as well as the safety and tolerability of an investigational drug called UCB0942 that will be given in addition to the current epilepsy treatment of the patient.