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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…

Efficacy, safety and tolerability of multiple doses of oral cebranopadol in subjects with moderate to severe chronic pain due to diabetic peripheral neuropathy

To assess the analgesic efficacy, safety, and tolerability of once daily orally administered cebranopadol in a total of 3 fixed doses (100 µg, 300 µg, and 600 µg cebranopadol) compared to placebo in subjects with moderate to severe chronic pain due…

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 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…

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…

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…

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 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…

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…

BLUE LIGHT THERAPY FOR SLEEP IMPAIRMENT IN PARKINSON*S DISEASE - Pilot Study -

1. Primary Objective:The primary objective is to evaluate the efficacy of Propeaq light therapy glasses with integrated blue LED lights on sleep disorders in patients with PD using the PSQI. 2. Secondary Objectives: The secondary objective of the…

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.

The Park-in-Shape study: a randomized controlled trial evaluating the effects of exercise on motor and non-motor symptoms in Parkinson*s disease.

Objectives: To evaluate whether aerobic exercise leads to clinically relevant improvements in 1) motor and 2) non-motor symptoms as well as quality of life and physical fitness.

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.

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…

The Effect of a Nutritional Intervention on brain Glucose Metabolism in early Alzheimer*s disease

To provide further insight into Souvenaid*s hypothesised mode of action, the current NL-ENIGMA study explores brain glucose metabolism in mild cognitive impairment (MCI) and mild dementia due to AD, using Positron Emission Tomography with 18F-FDG-…

A Phase 2a Randomized, Double-blind, Placebo-Controlled, Parallel-Group, Multi-center Study Investigating the Safety and Tolerability of JNJ-54861911 in Subjects in the Early (Predementia) Alzheimer*s Disease Spectrum

This safety study in subjects in the early (predementia) AD spectrum is performed to investigate primarily the safety and tolerability of JNJ-54861911 during 6 months of treatment.

A phase 2, double-blind, randomized, placebo-controlled pharmacokinetic trial in two parallel groups to investigate possible drug- drug interactions between stiripentol or valproate and GWP42003-P in patients with epilepsy.

Primary Objective: To determine whether GWP42003-P affects the pharmacokinetic (PK) profile of stiripentol (STP) or valproate (VPA).Secondary Objective: To assess the safety and tolerability of GWP42003-P in the presence of STP or VPA.To assess…

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 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),…