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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…

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…

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…

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.

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…