Natural History Study of Children With Metachromatic Leukodystrophy

Status Terminated

Clinical Trial Summary

The purpose of this study is evaluate the natural course of disease progression related to gross motor function in children with metachromatic leukodystrophy (MLD).

Clinical Trial Description

Metachromatic leukodystrophy (MLD) is an inherited, autosomal recessive disorder of lipid metabolism characterized by deficient activity of the lysosomal enzyme, arylsulfatase A (ASA). MLD is a rare genetic disease that occurs in most parts of the world. The estimated overall incidence of the disease in the western world is approximately 1 in 100,000 live births. This study is a multicenter, observational, longitudinal study that plans to enroll up to 30 patients with onset of MLD-related signs and symptoms prior to 30 months of age and who are less than 12 years of age. Patients will participate in this study for approximately 114 weeks (Screening through Follow-up) and will be assessed at defined intervals for disease status. ;

Study Design

Related Conditions & MeSH terms

Brain Diseases
Central Nervous System Diseases
Demyelinating Diseases
Genetic Diseases, Inborn
Hereditary Central Nervous System Demyelinating Diseases
Leukodystrophy, Metachromatic
Lipid Metabolism Disorders
Lysosomal Storage Diseases
Metabolic Diseases
Metabolic Inborn Brain Diseases
Metabolism, Inborn Errors
Metachromatic Leukodystrophy (MLD)
Nervous System Diseases
Sphingolipidoses
Sulfatidosis

Clinical Trial Details

NCT number	NCT01963650
Study type	Observational
Source	Takeda
Contact
Status	Terminated
Phase
Start date	November 2, 2015
Completion date	April 8, 2016

View Details

See also
Status	Clinical Trial	Phase
Completed	`NCT05086809` - Investigation of an Updated Bone-anchored Sound Processor	N/A
Completed	`NCT05080777` - Pilot Pragmatic Clinical Trial to Embed Tele-Savvy Into Health Care Systems	N/A
Recruiting	`NCT03222375` - SQUED™ Series 28.1 Home-use and Treatment of Autowave Reverberator of Autism	N/A
Completed	`NCT03295786` - Clinical Study to Test the Safety of CDNF by Brain Infusion in Patients With Parkinson's Disease	Phase 1/Phase 2
Completed	`NCT01922258` - Safety and Tolerability Study of Flexible Dosing of Brexpiprazole in the Treatment of Subjects With Agitation Associated With Dementia of the Alzheimer's Type	Phase 3
Completed	`NCT01915368` - Determining Optimal Post-Stroke Exercise (DOSE)	N/A
Terminated	`NCT03270189` - Effect of the Visual Information Change in Functional Dystonia	N/A
Active, not recruiting	`NCT03911388` - HSV G207 in Children With Recurrent or Refractory Cerebellar Brain Tumors	Phase 1
Completed	`NCT02798406` - Combination Adenovirus + Pembrolizumab to Trigger Immune Virus Effects	Phase 2
Suspended	`NCT04912115` - Randomized, Double-Blind, Active Placebo-Controlled Study of Ketamine to Treat Levodopa-Induced Dyskinesia	Phase 2
Completed	`NCT03994822` - pRESET for Occlusive Stroke Treatment	N/A
Completed	`NCT03336645` - Open-label Study of Midazolam Hydrochloride Oromucosal Solution (MHOS/SHP615) in Children With Status Epilepticus (Convulsive) in a Healthcare Setting in Japan	Phase 3
Completed	`NCT04283253` - Predictors of Response in Chronic Stroke	N/A
Not yet recruiting	`NCT06048523` - Prospective Cohort Study of Neurogenetic Diseases	N/A
Completed	`NCT02684825` - Detection of Silent Atrial Fibrillation aFter Ischemic StrOke	N/A
Active, not recruiting	`NCT02284126` - Topical Vancomycin for Neurosurgery Wound Prophylaxis	Phase 3
Completed	`NCT05815836` - Precision Medicine in Stroke
Completed	`NCT00739518` - Refinement and Assessment of New Magnetic Resonance Imaging Technologies for Neurological Exams	N/A
Recruiting	`NCT03678194` - Treating Depression on a Day-to-day Basis: Development of a Tool for Physicians Based on a Smartphone Application	N/A
Recruiting	`NCT04698421` - Collection of Biological Samples From Patients With Rare Neurological Diseases