90 results
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…
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…
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…
To explore the safety, tolerability and the pharmacokinetic profile of 2-IB when given on top of therapeutic hypothermia.
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…
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.
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…
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…
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…
The purpose of this study is to determine if SAR153191 150 mg once a week (qw) SC (subcutaneous) administration is safe and effective in reducing the recurrence and symptoms of ankylosing spondylitis over a long period of time (up to 5 years).
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…
To determine the instantaneous effects of cerebellar tDCS during PFT on posturographical and neurophysiological parameters in stroke patients and healthy subjects.
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.
The main goal of this study is the pharmacokinetic evaluation of a 30 mg and a 60 mg pulmonary administered levodopa with 2% l-leucine dry powder dose. The results gained with this study which will be used for further optimalisation and dose…
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…
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…
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…
Natalizumab (Tysabri®) for the treatment of anti-Hu associated paraneoplastic neurological syndromes
The primary objective of the study is the functional improvement with one point or more on the modified Rankin scale after the 12th week of natalizumab (compared to baseline).
This study will investigate whether bone marrow-derived allogeneic MSCs, as administered by the nasal route, can induce the formation of neuronal tissue and restore brain function in neonates who suffered from perinatal arterial ischemic stroke (…
The aim of this study is to explore whether subjectively experienced SB can be objectively assessed using computerized vision based analysis of motor performance upon awakening. Secondarily, patient experiences and data quality of the assessment…