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Results for “Leukodystrophy, Metachromatic”

51 trials

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Showing 20 of 51 results

Not applicableNo Longer AvailableNCT02084121
What this trial is testing

Allogeneic Stem Cell Transplantation for the Treatment of Multiple Sclerosis (Compassionate Use)

Who this might be right for
Metachromatic Leukodystrophy
Talaris Therapeutics Inc.
Not applicableLooking for participantsNCT01793168
What this trial is testing

Rare Disease Patient Registry & Natural History Study - Coordination of Rare Diseases at Sanford

Who this might be right for
Rare DisordersUndiagnosed DisordersDisorders of Unknown Prevalence+340 more
Sanford Health 20,000
Large-scale testing (Phase 3)Active Not RecruitingNCT04283227
What this trial is testing

OTL-200 in Patients With Late Juvenile Metachromatic Leukodystrophy (MLD)

Who this might be right for
Lysosomal Storage DiseasesMetachromatic Leukodystrophy
Orchard Therapeutics 6
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
Early research (Phase 1)Study completedNCT00418561
What this trial is testing

Metazym for the Treatment of Patients With Late Infantile Metachromatic Leukodystrophy (MLD)

Who this might be right for
Metachromatic Leukodystrophy (MLD)
Shire 13
Testing effectiveness (Phase 2)Study completedNCT01801709
What this trial is testing

Intracerebral Gene Therapy for Children With Early Onset Forms of Metachromatic Leukodystrophy

Who this might be right for
Metachromatic Leukodystrophy
Institut National de la Santé Et de la Recherche Médicale, France 5
Testing effectiveness (Phase 2)Study completedNCT01510028
What this trial is testing

Multicenter Study of HGT-1110 Administered Intrathecally in Children With Metachromatic Leukodystrophy (MLD)

Who this might be right for
Metachromatic Leukodystrophy (MLD)
Shire 24
Testing effectiveness (Phase 2)Looking for participantsNCT01962415
What this trial is testing

Reduced Intensity Conditioning for Non-Malignant Disorders Undergoing UCBT, BMT or PBSCT

Who this might be right for
Primary Immunodeficiency (PID)Congenital Bone Marrow Failure SyndromesInherited Metabolic Disorders (IMD)+4 more
Paul Szabolcs 100
Testing effectiveness (Phase 2)Active Not RecruitingNCT02171104
What this trial is testing

MT2013-31: Allo HCT for Metabolic Disorders and Severe Osteopetrosis

Who this might be right for
Mucopolysaccharidosis DisordersHurler SyndromeHunter Syndrome+26 more
Masonic Cancer Center, University of Minnesota 149
Not applicableEnded earlyNCT04008849
What this trial is testing

A Follow-up Study in Patients With Inherited Metabolic Disorders (IMD) Who Underwent Hematopoietic Stem Cell Transplantation (HSCT) With MGTA-456

Who this might be right for
Inherited Metabolic Disorders (IMD)
Magenta Therapeutics, Inc. 3
Testing effectiveness (Phase 2)Active Not RecruitingNCT03771898
What this trial is testing

Intrathecal SHP611 in Children With Metachromatic Leukodystrophy

Who this might be right for
Metachromatic Leukodystrophy (MLD)
Shire 36
Not applicableLooking for participantsNCT03725670
What this trial is testing

Direct Lentiviral Injection Gene Therapy for MLD

Who this might be right for
Metachromatic Leukodystrophy (MLD)
Shenzhen Geno-Immune Medical Institute 10
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 applicableStudy completedNCT04628364
What this trial is testing

The Natural History of Metachromatic Leukodystrophy Study (HOME Study)

Who this might be right for
Metachromatic Leukodystrophy
National Organization for Rare Disorders 21
Not applicableLooking for participantsNCT03047369
What this trial is testing

The Myelin Disorders Biorepository Project

Who this might be right for
LeukodystrophyWhite Matter DiseaseLeukoencephalopathies+63 more
Children's Hospital of Philadelphia 12,000
Testing effectiveness (Phase 2)Ended earlyNCT00668564
What this trial is testing

Hematopoietic Stem Cell Transplantation (HCT) for Inborn Errors of Metabolism

Who this might be right for
Hurler's SyndromeMaroteaux-Lamy SyndromeSly Syndrome+8 more
Masonic Cancer Center, University of Minnesota 18
Not applicableUnknownNCT04324775
What this trial is testing

Comparison of Acute Effect of Different Manual Techniques on Muscle Fascia Stiffness

Who this might be right for
Manual Lymphatic DrainageSwedish MassageMuscle Fascia Stiffness
Marmara University 30
Not applicableEnded earlyNCT01963650
What this trial is testing

Natural History Study of Children With Metachromatic Leukodystrophy

Who this might be right for
Lipid Metabolism DisordersMetachromatic Leukodystrophy (MLD)Nervous System Diseases+11 more
Shire 1
Not applicableWithdrawnNCT05755568
What this trial is testing

Learn About Metachromatic Leukodystrophy (MLD) in Children in Spain

Who this might be right for
Metachromatic Leukodystrophy (MLD)
Takeda
Not applicableStudy completedNCT05687474
What this trial is testing

Baby Detect : Genomic Newborn Screening

Who this might be right for
Congenital Adrenal HyperplasiaFamilial Hyperinsulinemic Hypoglycemia 1Phosphoglucomutase 1 Deficiency+121 more
Centre Hospitalier Universitaire de Liege 6,824
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