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Results for “Leukodystrophy, Metachromatic”

51 trials

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Showing 20 of 51 results

Not applicableStudy completedNCT01626092
What this trial is testing

Reduced-Intensity Hematopoietic Stem Cell Transplant for High Risk Lysosomal and Peroxisomal Disorders

Who this might be right for
Lysosomal Storage DiseasePeroxisomal Disorder
Masonic Cancer Center, University of Minnesota 3
Not applicableUnknownNCT00005900
What this trial is testing

Study of Pulmonary Complications in Pediatric Patients With Storage Disorders Undergoing Allogeneic Hematopoietic Stem Cell Transplantation

Who this might be right for
I Cell DiseaseFucosidosisGloboid Cell Leukodystrophy+9 more
Fairview University Medical Center 10
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
Not applicableWithdrawnNCT01536327
What this trial is testing

Biomarker for Metachromatic Leukodystrophy (BioMeta) Disease

Who this might be right for
Peripheral NeuropathyMuscle Weakness
CENTOGENE GmbH Rostock
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
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)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
Testing effectiveness (Phase 2)Ended earlyNCT01372228
What this trial is testing

Phase I/II Pilot Study of Mixed Chimerism to Treat Inherited Metabolic Disorders

Who this might be right for
Hurler Syndrome (MPS I)Hurler-Scheie SyndromeHunter Syndrome (MPS II)+9 more
Talaris Therapeutics Inc. 3
Not applicableStudy completedNCT02699190
What this trial is testing

LeukoSEQ: Whole Genome Sequencing as a First-Line Diagnostic Tool for Leukodystrophies

Who this might be right for
LeukodystrophyWhite Matter Disease4H Syndrome+61 more
Children's Hospital of Philadelphia 236
Testing effectiveness (Phase 2)Active Not RecruitingNCT03392987
What this trial is testing

A Safety and Efficacy Study of Cryopreserved OTL-200 for Treatment of Metachromatic Leukodystrophy (MLD)

Who this might be right for
Lysosomal Storage DiseaseMetachromatic Leukodystrophy
Orchard Therapeutics 10
Not applicableStudy completedNCT00004378
What this trial is testing

Stem Cell Transplantation (SCT) for Genetic Diseases

Who this might be right for
ThrombocytopeniaMetachromatic LeukodystrophyFanconi's Anemia+3 more
National Center for Research Resources (NCRR)
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
Large-scale testing (Phase 3)Study completedNCT00176904
What this trial is testing

Stem Cell Transplant for Inborn Errors of Metabolism

Who this might be right for
AdrenoleukodystrophyMetachromatic LeukodystrophyGloboid Cell Leukodystrophy+8 more
Masonic Cancer Center, University of Minnesota 135
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 completedNCT01303146
What this trial is testing

Efficacy METAZYM for the Treatment Metachromatic Leukodystrophy Treated With Hematopoietic Stem Cell Transplantation

Who this might be right for
Metachromatic Leukodystrophy
Assistance Publique - Hôpitaux de Paris 1
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
Testing effectiveness (Phase 2)Study completedNCT01043640
What this trial is testing

Allogeneic Bone Marrow Transplant for Inherited Metabolic Disorders

Who this might be right for
MucopolysaccharidosisHurler SyndromeHunter Syndrome+10 more
Masonic Cancer Center, University of Minnesota 46
Not applicableStudy completedNCT01325025
What this trial is testing

Imaging Study of the White Matter Lesions in Children With Metachromatic Leucodystrophy

Who this might be right for
Late Infantile Metachromatic Leukodystrophy
Assistance Publique - Hôpitaux de Paris 29
Testing effectiveness (Phase 2)Study completedNCT01560182
What this trial is testing

Gene Therapy for Metachromatic Leukodystrophy (MLD)

Who this might be right for
Lysosomal Storage DiseaseMetachromatic Leukodystrophy
Orchard Therapeutics 20
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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