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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…

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…

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 Clinical Study of Two Doses of a Selective p38 MAP Kinase Inhibitor, VX-745, to Evaluate the Effects of 12-Week Oral Twice-Daily Dosing on Amyloid Plaque Load as Assessed by Quantitative Dynamic 11C-PiB Positive Emission Tomography (PET) Amyloid Scanning

The primary objective is to assess the effects on amyloid plaque burden of administration of VX-745 for 12-weeks, as assessed by Dynamic 11C-PiB (Carbon-11 labeled Pittsburgh Compound B) PET Amyloid Scanning in patients with Mild Cogntitive…

Thyroid hormone analog therapy of patients with severe psychomotor retardation caused by mutations in the MCT8 thyroid hormone transporter: The Triac Trial.

1. Normalization of the abnormal serum TH parameters and thereby improving the clinical condition of the AHDS patients2. Observation of changes in cognitive and motor function.

A 4-Year Open-Label Extension (OLE) Phase of the Double-Blind, Placebo-Controlled, Dose-Escalation, Parallel-Group Study of E2007 as an Adjunctive Therapy in Patients With Refractory Partial Seizures

To evaluate the safety of E2007 given as adjunctive, long-term treatment in patients with refractory partial onset seizures with or without secondary generalization that completed the E2007-A001-206 or the E2007-G000-208 studies (revised per…

Pharmacokinetics and safety of Meropenem in infants below 90 days of age (inclusive) with probable and confirmed meningitis: a European multicenter phase I- II trial

Primairy objectivesTo study the pharmacokinetics (plasma and cerebrospinal fluid) of meropenem in infants <= 90 days of postnatal age with probable or confirmed bacterial meningitis (BM); To describe the safety profile of meropenem in the…

Clinical and pharmacological feasibility study with 2B3-101 in patients with breast cancer
and leptomeningeal metastases

Primary: To determine the response of 2B3-101 treatment as single agent or in combination with trastuzumab in patients with LM from breast cancer using the LM response scoreSecundary: - To determine the safety profile in patients with LM treated…

Dexmedetomidine in awake implantation of neuromodulative systems.

The purpose of this study is to observe the usability of Dexmedetomidine and to determine the overall satisfaction of the patient.

Treatment of apomorphine-induced skin reactions: a pilot study

The primary objective of this pilot study is to determine the effectiveness of four treatment options for local skin reactions induced by continuous subcutaneous apomorphine infusion, as compared to no treatment.The primary endpoint is the measured…

Can bilateral functional electrical stimulation (FES) of the calf muscles improve ankle push-off during gait in people with Multiple Sclerosis? A proof of principle study

Aim of this proof of principle study is to develop a reliable and safe application of bilateral FES of the calf muscles to facilitate insufficient push-off in patients with MS and to assess the preliminary effects of FES on net ankle power…

An open label exploratory dose finding and pharmacokinetic clinical trial of bumetanide for the treatment of NEonatal seizure using Medication Off-patent (NEMO).

The aim of the trial is to obtain data on the optimal dose, feasibility and pharmacokinetics of bumetanide when given as an add-on treatment for seizures in full term babies with hypoxic ischemic encephalopathy (HIE). Bumetanide will be given in a…

Early psychological intervention in stroke patients independent in activities of daily living to reduce symptoms of anxiety and depression of influence on long-term functional outcome.

Primary: to determine whether an early psychological intervention poststroke in order to reduce symptoms of anxiety and depression, is feasible.Secondary: To evaluate if an early individual psychological intervention in patients independent in…

An open-label study to evaluate the long-term safety and tolerability of AFQ056 in adolescent patients with Fragile X Syndrome

PrimaryTo evaluate the long-term safety and tolerability of AFQ056 in adolescent patients with FXS as assessed by:* Incidence and severity of adverse events and serious adverse events* Changes in vital signs, laboratory assessments, and ECGs*…

The effect of cervical transcutaneous electrical neurostimulation on cerebral blood flow velocities, cerebral vasospasm and ischemic deficit in secondary cerebral vasospasm after subarachnoid haemorrhage

To find out if patients with secondary cerebral vasospasm after SAH can benefit from cervical TENS.