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Results for “Leukodystrophy”

87 trials

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Showing 20 of 87 results

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)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
Not applicableLooking for participantsNCT03333200
What this trial is testing

Longitudinal Study of Neurodegenerative Disorders

Who this might be right for
MLDKrabbe DiseaseALD+24 more
University of Pittsburgh 1,500
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 applicableEnrolling By InvitationNCT03655223
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)Active Not RecruitingNCT07222371
What this trial is testing

An Open-label, Single Center, Single Participant Study of an Experimental Antisense Oligonucleotide Treatment for TUBB4A-related Leukodystrophy

Who this might be right for
Genetic DiseaseTUBB4A-Related Leukodystrophy
University of California, San Diego 1
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
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
Large-scale testing (Phase 3)Ended earlyNCT00654433
What this trial is testing

ALD-101 Adjuvant Therapy of Unrelated Umbilical Cord Blood Transfusion (UCBT) in Patients With Inherited Metabolic Diseases

Who this might be right for
Inherited Metabolic DiseasesLysosomal Storage DisordersPeroxisomal Storage Diseases+2 more
Aldagen 40
Large-scale testing (Phase 3)Study completedNCT04270682
What this trial is testing

Study to Evaluate Patients With Cerebrotendinous Xanthomatosis (RESTORE)

Who this might be right for
CTX
Mirum Pharmaceuticals, Inc. 19
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 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)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
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)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 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 completedNCT01938014
What this trial is testing

Lysosomal Storage Disease: Health, Development, and Functional Outcome Surveillance in Preschool Children

Who this might be right for
Mucopolysaccharidosis Type I (MPS I)Mucopolysaccharidosis Type II (MPS II)Mucopolysaccharidosis Type III (MPS III)+2 more
University of Chicago 19
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
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 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
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