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Results for “Leukodystrophy”

87 trials

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Showing 20 of 87 results

Not applicableNo Longer AvailableNCT03639844
What this trial is testing

BPX-501 T Cells Infused Post Stem Cell Transplant in Pediatrics With Non-Malignant Disorders Ineligible for BPU004 Study

Who this might be right for
Hurler SyndromeInherited Metabolic DisorderLysosomal Storage Disorder+2 more
Bellicum Pharmaceuticals
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
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 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 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
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 completedNCT00006057
What this trial is testing

Diagnostic and Screening Study of Genetic Disorders

Who this might be right for
Tay-Sachs DiseasePorphyria, ErythropoieticLeukodystrophy, Globoid Cell+1 more
National Center for Research Resources (NCRR) 50
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)Active Not RecruitingNCT01887938
What this trial is testing

An Efficacy and Safety Study of HGT-1110 in Participants With Metachromatic Leukodystrophy

Who this might be right for
Metachromatic Leukodystrophy (MLD)
Shire 24
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
Early research (Phase 1)WithdrawnNCT01003912
What this trial is testing

Fetal Umbilical Cord Blood (UCB) Transplant for Lysosomal Storage Diseases

Who this might be right for
Lysosomal Storage DiseasesInborn Errors of Metabolism
Joanne Kurtzberg, MD
Not applicableStudy completedNCT04126005
What this trial is testing

Natural History Study of Patients With Canavan Disease (CANinform Study)

Who this might be right for
Canavan Disease
Aspa Therapeutics 67
Not applicableEnded earlyNCT06308718
What this trial is testing

Long-term Follow-up Study to Evaluate Safety and Efficacy of FBX-101 in Krabbe Patients

Who this might be right for
Krabbe Disease
Forge Biologics, Inc 2
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)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
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 applicableStudy completedNCT00683189
What this trial is testing

Effect of Warfarin in the Treatment of Metachromatic Leukodystrophy

Who this might be right for
Metachromatic Leukodystrophy
The Cooper Health System 10
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
Testing effectiveness (Phase 2)Study completedNCT03406962
What this trial is testing

MGTA-456 in Patients With Inherited Metabolic Disorders Undergoing Hematopoietic Stem Cell Transplantation (HSCT)

Who this might be right for
Inherited Metabolic Disorders (IMD)
Magenta Therapeutics, Inc. 8
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
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