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Results for “Leukodystrophies”

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Showing 20 of 0 results

Testing effectiveness (Phase 2)Study completedNCT00383448
What this trial is testing

HSCT for High Risk Inherited Inborn Errors

Who this might be right for
AdrenoleukodystrophyMetachromatic LeukodystrophyGloboid Cell Leukodystrophy+6 more
Masonic Cancer Center, University of Minnesota 38
Not applicableEnrolling By InvitationNCT05368038
What this trial is testing

ScreenPlus: A Comprehensive, Flexible, Multi-disorder Newborn Screening Program

Who this might be right for
Acid Sphingomyelinase DeficiencyCeroid Lipofuscinosis, Neuronal, 2Cerebrotendinous Xanthomatosis+11 more
Albert Einstein College of Medicine 100,000
Testing effectiveness (Phase 2)Study completedNCT03771898
What this trial is testing

Intrathecal SHP611 in Children With Metachromatic Leukodystrophy

Who this might be right for
Metachromatic Leukodystrophy (MLD)
Shire 36
Not applicableStudy completedNCT00001671
What this trial is testing

The Classification and Cause of Leukodystrophies of Unknown Cause

Who this might be right for
Lysosomal Storage Disease
National Institute of Neurological Disorders and Stroke (NINDS) 400
Not applicableLooking for participantsNCT02993796
What this trial is testing

Krabbe Disease Global Patient Registry

Who this might be right for
Krabbe Disease
State University of New York at Buffalo 60
Not applicableLooking for participantsNCT04925349
What this trial is testing

Modeling Macrophages Activation Pattern in X-linked Adrenoleukodystrophy, Metachromatic Leukodystrophy and Adult Onset Leukoencephalopathy With Axonal Spheroids and Pigmented Glia

Who this might be right for
AdrenoleukodystrophyAdrenomyeloneuropathyMetachromatic Leukodystrophy+1 more
Assistance Publique - Hôpitaux de Paris 100
Testing effectiveness (Phase 2)Temporarily pausedNCT04771416
What this trial is testing

Study of Safety, Tolerability and Efficacy of PBKR03 in Pediatric Subjects With Early Infantile Krabbe Disease

Who this might be right for
Leukodystrophy, Globoid Cell
Gemma Biotherapeutics 24
Not applicableEnded earlyNCT05020743
What this trial is testing

Natural History Study in Adult-Onset Leukoencephalopathy With Axonal Spheroids and Pigmented Glia

Who this might be right for
ALSP
Vigil Neuroscience, Inc. 56
Not applicableActive Not RecruitingNCT00787865
What this trial is testing

Diffusion Tensor Imaging (DTI) in Infants With Krabbe Disease

Who this might be right for
Krabbe Disease
University of Pittsburgh 100
Not applicableWithdrawnNCT01093105
What this trial is testing

An Observational Study of Pediatric Subjects With Globoid Cell Leukodystrophy (GLD)

Who this might be right for
Leukodystrophy, Globoid Cell
Shire
Not applicableNo Longer AvailableNCT02021266
What this trial is testing

Single Patient Expanded Access Protocol: Metabolic Boost

Who this might be right for
Metachromatic Leukodystrophy
Talaris Therapeutics Inc.
Testing effectiveness (Phase 2)WithdrawnNCT00657748
What this trial is testing

Lithium and Acetate for Canavan Disease

Who this might be right for
Canavan DiseaseInfantileDeficiency Disease+2 more
Assistance Publique - Hôpitaux de Paris
Not applicableLooking for participantsNCT04880356
What this trial is testing

Longitudinal Study of Ultra-rare Inherited Metabolic and Degenerative Neurological Diseases.

Who this might be right for
Inherited DiseaseRare DiseasesMetabolic Disease+3 more
Fondazione I.R.C.C.S. Istituto Neurologico Carlo Besta 100
Not applicableStudy completedNCT00001780
What this trial is testing

Magnetic Stimulation of the Human Nervous System

Who this might be right for
Demyelinating DiseaseHealthyLysosomal Storage Disease+2 more
National Institute of Neurological Disorders and Stroke (NINDS) 450
Not applicableWithdrawnNCT01425489
What this trial is testing

Biomarker for Krabbe Disease (BioKrabbe)

Who this might be right for
Krabbe Disease
CENTOGENE GmbH Rostock
Testing effectiveness (Phase 2)Study completedNCT04611880
What this trial is testing

Crizanlizumab for Treatment of Retinal Vasculopathy With Cerebral Leukoencephalopathy (RVCL)

Who this might be right for
RVCL - Retinal Vasculopathy Cerebral Leukoencephalopathy
Washington University School of Medicine 18
Not applicableStudy completedNCT00983879
What this trial is testing

The Natural History of Infantile Globoid Cell Leukodystrophy

Who this might be right for
Infantile Globoid Cell Leukodystrophy
Zymenex A/S 6
Not applicableStudy completedNCT01594853
What this trial is testing

Exercise Study of Function and Pathology for Women With X-linked Adrenoleukodystrophy

Who this might be right for
X-linked Adrenoleukodystrophy
Hugo W. Moser Research Institute at Kennedy Krieger, Inc. 31
Testing effectiveness (Phase 2)UnknownNCT02559830
What this trial is testing

Autologous Hematopoietic Stem Cell Gene Therapy for Metachromatic Leukodystrophy and Adrenoleukodystrophy

Who this might be right for
Metachromatic LeukodystrophyAdrenoleukodystrophy
Shenzhen Second People's Hospital 50
Testing effectiveness (Phase 2)Study completedNCT00633139
What this trial is testing

Long-term Metazym Treatment of Patients With Late Infantile Metachromatic Leukodystrophy (MLD)

Who this might be right for
Late Infantile Metachromatic Leukodystrophy
Shire 13
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