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Results for “Leukodystrophies”

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Active, Not Recruiting
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Enrolling by Invitation

Showing 20 of 0 results

Not applicableLooking for participantsNCT05443906
What this trial is testing

Home Exercise for Individuals With Neurodegenerative Disease

Who this might be right for
Neurodegenerative DiseasesLeukodystrophyAtaxia+5 more
Hugo W. Moser Research Institute at Kennedy Krieger, Inc. 30
Not applicableNot Yet RecruitingNCT03725670
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
Not applicableNot Yet RecruitingNCT07046338
What this trial is testing

Lentiviral Hematopoietic Stem Cell Gene Therapy for MLD

Who this might be right for
Metachromatic Leukodystrophy (MLD)
Shenzhen Geno-Immune Medical Institute 10
Testing effectiveness (Phase 2)Ended earlyNCT00681811
What this trial is testing

Open-Label Extension Study of Recombinant Human Arylsulfatase A (HGT-1111) in Late Infantile MLD

Who this might be right for
Late Infantile Metachromatic Leukodystrophy
Shire 11
Testing effectiveness (Phase 2)WithdrawnNCT02715505
What this trial is testing

Safety and Exploratory Efficacy of HSC835 in Patients With Inherited Metabolic Disorders (IMD)

Who this might be right for
Inherited Metabolic Disorders IMD
Novartis Pharmaceuticals
Testing effectiveness (Phase 2)Study completedNCT00730314
What this trial is testing

Unrelated Hematopoietic Stem Cell Transplantation(HSCT) for Genetic Diseases of Blood Cells

Who this might be right for
Sickle Cell DiseaseThalassemiaAnemia+9 more
Children's Hospital Los Angeles 25
Not applicableWithdrawnNCT00639132
What this trial is testing

The Natural History of Metachromatic Leukodystrophy

Who this might be right for
Metachromatic Leukodystrophy
University of Pittsburgh
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
Not applicableLooking for participantsNCT03639285
What this trial is testing

Natural History, Diagnosis, and Outcomes for Leukodystrophies

Who this might be right for
Leukodystrophy
University of Utah 600
Not applicableActive Not RecruitingNCT03655223
What this trial is testing

Early Check: Expanded Screening in Newborns

Who this might be right for
Spinal Muscular AtrophyFragile X SyndromeFragile X - Premutation+182 more
RTI International 30,000
Testing effectiveness (Phase 2)Study completedNCT03392987
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
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
Testing effectiveness (Phase 2)WithdrawnNCT03304717
What this trial is testing

Reverse Transcriptase Inhibitors in Aicardi Goutières Syndrome

Who this might be right for
Aicardi Goutières Syndrome
Children's Hospital of Philadelphia
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
Early research (Phase 1)Looking for participantsNCT02254863
What this trial is testing

UCB Transplant of Inherited Metabolic Diseases With Administration of Intrathecal UCB Derived Oligodendrocyte-Like Cells

Who this might be right for
AdrenoleukodystrophyBatten DiseaseMucopolysaccharidosis II+9 more
Joanne Kurtzberg, MD 40
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 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 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)Enrolling By InvitationNCT04833907
What this trial is testing

RAAV-Olig001-ASPA Gene Therapy for Treatment of Children With Typical Canavan Disease

Who this might be right for
Canavan Disease
Myrtelle Inc. 24
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
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