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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…

The Effects of Transcutaneous Vagus Nerve Stimulation on the Pupil Diameter, Heart Rate and Heart Rate Variability in Epilepsy Patients and Healthy Subjects.

Primary objective: The main objective of this pilot study is to gain more insight into the effects of tVNS: to assess if the ABVN and consecutively the NTS are really stimulated by tVNS. Therefore this study investigates the effects of tVNS on the…

The effect of additional foot support with or without seat surface forward tilting on upper extremity function in children with spastic cerebral palsy*

To evaluate the effect of additional foot support during sitting and the effect of forward tilting of the seat surface on 1) postural adjustments during reaching and 2) the quality of reaching movement in school age children with CP.

HORIZON-Plus: An Open-Label Extension of the HORIZON Protocol (DIM20) Evaluating the Safety of Dimebon (Latrepirdine) in Subjects with Huntington Disease

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

Clinical study of the effect of PEEP ventilation on right ventricular afterload in pediatric patients after cardiac surgery.

The objective of the study is to investigate the effect of PEEP ventilation with or without a recruitment maneuver on right ventricular afterload in infants undergoing cardiac surgery.

Botulinum toxin for treating calf muscle spasticity in Hereditary Spastic Paraparesis (HSP): functional effects on dynamic balance and gait

To show that an individually tailored treatment of the calf muscles with BTX-A promotes the balance capacity and mobility of HSP patients by an effective reduction of spasticity while preserving muscle strength. This explorative study uses a pre-…

Robotic toys within occupational therapy for children with developmental disabilities.

Objective:This study has as its aim to examine the short-term effect of a robotic-facilitated play therapy within occupational therapy on the level of playfulness of children with developmental disabilities aged 2-16 years.

*Utrecht Spina Bifida And Graded Exercise (USAGE) Study: physical activity, physical fitness and development of an exercise program to improve daily activities and participation in ambulatory children with Spina Bifida*. Part 1: Development of protocols to measure physical fitness in ambulatory children with Spina Bifida

To develop valid and reproducible protocols to measure cardiorespiratory fitness (VO2peak and energy cost of locomotion) in ambulatory children with SB. These protocols will allow further studies regarding physical fitness in these children.

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 Phase 3 Study of Lenti-D Drug Product After Myeloablative Conditioning Using Busulfan and Fludarabine in Subjects <=17 Years of Age With Cerebral Adrenoleukodystrophy (CALD)

To evaluate the efficacy and safety of Lenti-D Drug Product (also known as elivaldogene autotemcel or Skysona, hereafter referred to as eli-cel) after myeloablative conditioning with busulfan and fludarabine in subjects with CALD

A study to explore the safety, tolerability, pharmacokinetic profile, and potential efficacy of Guanabenz in patients with early childhood onset Vanishing White Matter (VWM)

To evaluate safety, tolerability pharmacokinetic profile; to explore pharmacodynamics; to evaluate major effects of Guanabenz on quantitative MRI and clinical parameters in pediatric patients with VWM; explore biomarkers in blood and cerebrospinal…

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…

Old Title: International, multi-center, open label 9-month FOLLOW-UP extension study assessing the long term safety and tolerability of PXT3003 in patients with Charcot-Marie-Tooth Disease type 1A.;New title : International, multi-center, open label FOLLOW-UP extension study assessing the long term safety and tolerability of PXT3003 in patients with Charcot-Marie-Tooth Disease type 1A.

The primary objectives:Period 1:The primary objective is to assess the long-term (up to 2 years) safety and tolerability of two doses of PXT3003.Period 2:For patients continuing after V9, the main objective will be to offer patients the opportunity…

A phase 1/2a, open-label trial to investigate the safety, tolerability, pharmacokinetics and pharmacodynamics of multiple ascending doses of intrathecally administered VO659 in participants with spinocerebellar ataxia types 1, 3 and Huntington*s disease

This study has been transitioned to CTIS with ID 2024-514328-18-00 check the CTIS register for the current data. Primary Objective:To evaluate the safety and tolerability of multiple doses of intrathecal lumbar bolus administrations of VO659 in…

Longterm Follow-up of Subjects With Cerebral Adrenoleukodystrophy Who Were Treated With Lenti-D Drug Product

This study has been transitioned to CTIS with ID 2024-513904-33-00 check the CTIS register for the current data. • Monitor for long-term safety of the eli-cel administered in parent clinical studies• Monitor for long-term efficacy of eli-cel…

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.

A Multicenter, Open-Label, Extension Study to Evaluate the Long-term Safety and Efficacy of Patisiran in Patients with Familial Amyloidotic Polyneuropathy Who Have Completed a Prior Clinical Study with Patisiran

To assess the safety and efficacy of long-term dosing with patisiran in transthyretin-mediated amyloidosis (ATTR) patients with familial amyloidotic polyneuropathy (FAP)

An open-label, adaptive multiple-dose study to investigate the pharmacokinetics and pharmacodynamics of RO7234292 in csf and plasma, and safety and tolerability following intrathecal administration in patients with Huntington's disease.

To date, non-clinical and clinical data have been generated to support further investigation of RO7234292 in patients with early manifest HD. Building on the recently completed Phase I/IIa study and the ongoing OLE study, this Phase Ia study (…

Establishing biomarkers and clinical endpoints in myotonic dystrophy type 1 (END-DM1)

The overall goal of this study is to accelerate the development of new therapies for DM1 by validating new clinical assessments for measuring disease status and collecting data and biological samples to help understand disease progression and…