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Multicenter, Randomized, Double-Blind, Placebo-Controlled, Parallel-Group, Phase 3 Study to Evaluate the Efficacy and Safety of OV101 in Pediatric Individuals With Angelman Syndrome (NEPTUNE)

All efficacy study objectives will be assessed in the pediatric AS study population of subjects who are 4 to 12 years old. All safety study objectives will be assessed in the pediatric AS study population of subjects who are 2 to 12 years old.…

An Open-Label Study to Evaluate the Long-Term Safety, Tolerability, and Efficacy of OV101 in Individuals with Angelman Syndrome (ELARA)

The primary objective of this study is to evaluate the long-term safety and tolerability of OV101 in individuals with AS assessed by the incidence and severity of AEs and SAEs in subjects who are at least 2 years old.The secondary objectives of this…

Ethanol submandibular duct ligation for drooling in children with neurodisabilities

the primary aim of this study is to evaluate the safety (adverse events and complaints) of submandibular duct ligation right after intraductal ethanol infusion into the submandibular salivary gland (Ethanol two-duct ligation [E-2DL] in order to…

A randomised, double-blind, controlled trial to evaluate the effects of a nutritional product on brain integrity in preterm infants

>Primary objective:During the intervention period (from randomisation until and including Term Equivalent Age [TEA]): To investigate the effect of the test product vs. the control product given to preterm infants born at 24+0 to <30+0…

A Phase 3, Randomized, Double-Blind, Placebo-Controlled, Parallel Arm, Multicenter Study Evaluating the Efficacy and Safety of Pridopidine in Patients with Early Stage of Huntington Disease

The purpose of this Phase 3 study is to further evaluate the effect of pridopidine 45*mg BID on functional capacity, as well as motor and behavioral features of HD in early-stage participants (TFC*713).

The effect of walking with and without music on executive functions and gait in children aged 8 to16 years old and adults over 40 years old with mild cerebral palsy

The main objective of this study is to investigate the acute beneficial effect of moderate inten-sive exercise (defined as walking) on neurocognitive functioning in healthy children and adults with mild CP, GMFCS I and II and if this effect is…

Dolphin CONTINUE: A nutritional intervention aimed at improving neurodevelopmental outcome in infants at risk for or with early life brain injury

The main objective is to study the effect of the nutritional intervention versus the control product on white matter development (as assessed using tract based spatial statistics of fractional anisotropy) using a DTI scan at the corrected age of…

Effect of ALlopurinol in addition to hypothermia for hypoxic-ischemic Brain Injury on Neurocognitive Outcome - a blinded randomized placebo controlled parallel group multicenter trial for superiority (Phase III)

This study has been transitioned to CTIS with ID 2024-511322-31-00 check the CTIS register for the current data. To evaluate whether in newborns with asphyxia and early clinical signs of hypoxic ischemic encephalopathy, early postnatal allopurinol…

Functional electrical stimulation of the ankle dorsiflexors during walking in children with unilateral spastic cerebral palsy: a randomized crossover intervention study.

The primary objective of this study is to evaluate the effect of FES on participation level: achieving of individual goals, using the goal attainment scale (GAS)), and the Canadian Occupational Performance Measure (COPM) for the long term. Secondary…

A Proof-of-Concept Study to Explore the Potential Efficacy of Deferiprone in Patients With
Pelizaeus-Merzbacher disease (PMD)

This study has been transitioned to CTIS with ID 2024-511968-81-00 check the CTIS register for the current data. Studying the effect of deferiprone on the motor function of patients with Pelizaeus-Merzbacher disease.

Phase 3, Open-Label, Single-Arm, Single-Dose Gene Replacement Therapy Clinical Trial for Patients with Spinal Muscular Atrophy Type 1 with One or Two SMN2 Copies Delivering AVXS-101 by Intravenous Infusion

Primary* Determine efficacy by demonstrating achievement of developmental milestone of sitting without support up to 18 months of age as defined by WHO Motor Developmental Milestones[22].Secondary* Determine efficacy based on survival at 14 months…

The effects of body weight support, guidance, and gait speed on the patterning of muscle activity in the Lokomat, in children with Cerebral Palsy.

This study has two objectives: (1)To establish the effects of (a) body weight support (b) treadmill speed, and (c) the level of movement support provided by the Lokomat (so called *guidance*), on the neuromuscular control of the lower extremities in…

Intrathecal baclofen treatment in dystonic cerebral palsy: a randomized clinical trial

Objectives: The primary aim of this study is to provide evidence for the effect of ITB treatment on the level of activities in dystonic CP patients.

Responding or not responding to training;
Prediction of balance Rehabilitation Outcome from structural and functional brain networks
in Cerebral Palsy

First, it will be investigated which is the best diagnostic tool for imbalance in CP. Second, it will be determined whether balance training can promote balance control in CP, and whether the improvements will be maintained after a follow-up period…