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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-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 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…

Effectiveness of Eye Movement Desensitization and Reprocessing on Post Traumatic Stress Disorder in persons with mild intellectual disability and psychosis: A multiple baseline study

The main purpose of this study is to find out whether EMDR is a safe and effective treatment to reduce the symptoms of PTSD in people with mild intellectual disability and borderline intellectual functioning. In addition, it is expected to reduce…

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…

SPACE trial
SMA and Pyridostigmine in Adults and Children; Efficacy trial
Phase II, mono-center, doubleblind, placebo-controlled, crossover trial to assess efficacy of pyridostigmine in patients with spinal muscular atrophy types 2, 3 and 4

to investigate the effect and efficacy of pyridostigmine on muscle strength and fatiquabillity in patients with SMA.

Tiratricol treatment of children with Monocarboxylate Transporter 8 deficiency: Triac Trial II

This study has been transitioned to CTIS with ID 2024-516123-13-00 check the CTIS register for the current data. Part I:Primary Objective: Evaluate the effects of tiratricol on neurodevelopment in young MCT8 deficiency patients, as measured by the…

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…

AN OPEN-LABEL, MULTI-CENTRE STUDY TO EVALUATE THE LONG-TERM SAFETY AND TOLERABILITY OF REN001 IN SUBJECTS WITH PRIMARY MITOCHONDRIAL MYOPATHY (PMM)

Primary Objective:To evaluate long-term safety and tolerability of REN001 in subjects with PMM.Secondary Objective:To evaluate subjects with mtDNA-PMM who are receiving long-term treatment with REN001 in terms of PMM associated symptoms, exercise…

A randomized open-label Phase 2a study to assess the pharmacokinetics and pharmacodynamic parameters of PXL770 after 12 weeks of treatment in male subjects with adrenomyeloneuropathy (AMN)

The primary objective of the study is to assess the pharmacokinetics (PK) of PXL770 in AMN subjects at the dose of 500mg once daily (OD) and 250mg twice daily (BID) after 4 weeks of treatment.

Effects of sodium lactate infusion in patients with glucose transporter 1 deficiency syndrome (GLUT1DS)

The purpose of this explorative study is to investigate whether treatment with lactate has any positive effect on the symptoms of GLUT1DS, especially the drug-resistant epilepsy.Primary Objective: - To assess changes in EEG during and shortly after…

The TRAIN study: Trametinib in neurofibromatosis type 1 related symptomatic plexiform neurofibromas

Primary objective: Response to trametinib treatment defined as a tumor volume decreases from baseline of >=20%, monitored by using volumetric MRI analysis. Secondary objectives are: patient reported outcomes of pain and disability and quality…

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.