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Results for “Leukodystrophies”

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Showing 20 of 0 results

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)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 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
Testing effectiveness (Phase 2)Not Yet RecruitingNCT07740512
What this trial is testing

Safety, Tolerability, Pharmacokinetics, and Preliminary Efficacy of PLX-200 in Pediatric Patients (Master Protocol)

Who this might be right for
Lysosomal Storage DisordersSandhoff DiseaseKrabbe Disease+2 more
Polaryx Therapeutics, Inc. 24
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 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
Early research (Phase 1)Study completedNCT02595489
What this trial is testing

A Pilot Study of Vitamin D in Boys With X-linked Adrenoleukodystrophy

Who this might be right for
X-linked Adrenoleukodystrophy
Stanford University 21
Early research (Phase 1)Study completedNCT01586455
What this trial is testing

Human Placental-Derived Stem Cell Transplantation

Who this might be right for
Mucopolysaccharidosis IMucopolysaccharidosis VIAdrenoleukodystrophy+13 more
New York Medical College 43
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
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 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
Testing effectiveness (Phase 2)Active Not RecruitingNCT06816498
What this trial is testing

Personalized Antisense Oligonucleotide Therapy for A Single Participant With LMNB1 Mutation Associated Autosomal Dominant Leukodystrophy (ADLD)

Who this might be right for
Autosomal Dominant Leukodystrophy
n-Lorem Foundation 1
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 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
Testing effectiveness (Phase 2)Study completedNCT01887938
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
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)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
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 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
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