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Sleep problems in children with Angelman syndrome: the effect of a behavioral treatment.

Primary Objective:To evaluate the effect and sustainability over time of behavioural treatment for sleep problems in children with AS on the actual hours of sleep and sleep behaviour of the children.Secondary Objectives:To evaluate the effect and…

Dietary treatment of children with Angelman Syndrome and refractory epilepsy

Primary objective: - 50% seizure reduction or more at 3 months of dietary treatment .Secondary objectives: -level of ketosis on KD and MAD.-number of patients maintaining >50% seizure reduction on MAD.-number of withdrawals (not able to…

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…

APOLLO: A Phase 3 Multicenter, Multinational, Randomized, Double-blind, Placebo-controlled Study to Evaluate the Efficacy and Safety of Patisiran (ALN-TTR02) in Transthyretin (TTR)-Mediated Polyneuropathy (Familial Amyloidotic Polyneuropathy-FAP)

This study aims to demonstrate the clinical efficacy of patisiran and to establish the safety of chronic dosing in ATTR patients with FAP.The primary objective of the study is to determine the efficacy of patisiran by evaluating the difference…

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, Double-Blind, Placebo-Controlled, Dose-Escalating, Phase 2a Safety, Tolerability, and Pharmacodynamic Study of Two Doses of an Histone Deacetylase Inhibitor (FRM-0334) in Subjects with Prodromal to Moderate Frontotemporal Dementia with Granulin Mutation

2.1 Primary Objectives• Evaluate the safety and tolerability of 2 fixed doses of FRM-0334 (300 and 500 mg daily in 2 sequential periods) over 28 days in subjects with prodromal to moderate FTD-GRN• Assess the PD effects of FRM-0334 on the change…

A Phase 3, Randomized, Double-Blind, Placebo-Controlled Safety and Efficacy Study of Dimebon in Patients with Mild-to-Moderate Huntington Disease

To determine the safety and efficacy of Dimebon in patients with mild-to-moderate Huntington Disease

LEARN 2 MOVE 16-24;Effectiveness of an age specific intervention to stimulate mobility related activity and physical fitness of adolescents with spastic cerebral palsy.

The aim of the research is to evaluate the efficacy and working mechanisms of the "active lifestyle" intervention, which is directed to strategies to prevent low levels of daily activities and poor fitness in adolescents and young adults…

Utrecht Spina Bifida And Graded Exercise (USAGE) Study part 2: Does training in ambulatory children with SB increase levels of physical fitness, energy cost of locomotion and daily physical activity?

Primary objective:To evaluate the effects of a training program on physical fitness, energy cost of locomotion and daily physical activity in ambulatory children and adolescents with Spina Bifida Secondary objectives: (1) evaluate physical fitness (…

A Phase 1a Double-Blind, Randomized, Placebo-Controlled, Single and Multiple Ascending Dose Study of the Safety, Pharmacokinetics, and Pharmacodynamics of ABX-002 in Healthy Adult Subjects

Part 1 Single Ascending Dose (SAD)- To evaluate the safety and tolerability of a single oral dose of prodrug, ABX-002, in healthy adult subjectsPart 2 Multiple Ascending Dose (MAD)-To evaluate the safety and tolerability of once daily oral doses of…

HELIOS-A: A Phase 3 Global, Randomized, Open-label Study to Evaluate the Efficacy and Safety of ALN-TTRSC02 in Patients with Hereditary Transthyretin Amyloidosis (hATTR Amyloidosis)

This study has been transitioned to CTIS with ID 2023-508365-33-00 check the CTIS register for the current data. To determine the efficacy of ALNTTRSC02 in patients with hATTR amyloidosis by evaluating the effect on neurologic impairment. To…

Respiratory muscle training in patients with Spinal Muscular Atrophy

The aim of this study is to assess the feasibility and efficacy of respiratory muscle training (RMT) in patients with SMA and respiratory muscle weakness.

A DOUBLE-BLIND, PLACEBO-CONTROLLED, STUDY TO EVALUATE THE EFFICACY AND SAFETY OF 24 WEEKS TREATMENT WITH REN001 IN PATIENTS WITH PRIMARY MITOCHONDRIAL MYOPATHY (PMM)

Main Objective:To evaluate the efficacy of REN001 in subjects with PMM treated for 24 weeks, assessed by the effect on exercise endurance.Secondary Objective:To evaluate the efficacy of REN001 in subjects with PMM treated for 24 weeks, assessed by…

A double-blind, randomized, placebo-controlled trial of adjunctive ganaxolone treatment in female children with protocadherin 19 (PCDH19)-related epilepsy followed by long-term open-label treatment

Primary: To assess the efficacy of GNX compared with PBO, as adjunctive therapy for the treatment of primary seizure types in children with genetically-confirmed PCDH19-related epilepsy during the 17-week double-blind (DB) phase. Secondary: • To…

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).

Oxytocin treatment in neonates and infants aged from 0 to 3 months with prader-willi syndrome: a study of the safety and efficacy on oral and social skills and, feeding behavior of intranasal administrations of oxytocin vs. placebo (phase iii clinical trial)

The primary objective is to demonstrate the superiority versus placebo of a 4 weeks intranasal OT administration on oral skills assessed by the Neonatal Oral-Motor Assessment Scale (NOMAS) in infants with PWS aged less than or equal to 3 months at…

A Mindfulness-Based Stress Reduction programme in at-risk mutation carriers of genetic frontotemporal dementia

The present study aims to investigate whether a standardized 8-week group MBSR treatment can significantly reduce symptoms of stress, anxiety and depression, and improve quality of life in at-risk mutation carriers for FTD. The primary objective is…

The effect of Lamotrigine on cognitive deficits associated with Neurofibromatosis type 1: a phase II randomized controlled multi-centre trial (NF1-EXCEL)

The objective of this proposal is to determine the effect of Lamotrigine on cognitive functioning and neurophysiology in adolescents with NF1.

International, multi-center, randomized, double-blind, placebo-controlled phase III study assessing in parallel groups the efficacy and safety of 2 doses of PXT3003 in patients with Charcot-Marie-Tooth Disease type 1A treated 15 months

Primary objective:To assess the efficacy of PXT3003 compared to Placebo on the disability measured by the ONLS score in CMT1A patients treated for 15 months.Secondary objectives:- To assess the efficacy of PXT3003 compared to Placebo on clinical and…