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Results for “Adrenoleukodystrophy, Neonatal”

68 trials

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Showing 20 of 68 results

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 completedNCT03864523
What this trial is testing

Effect of Pioglitazone Administered to Patients With Adrenomyeloneuropathy

Who this might be right for
AdrenomyeloneuropathyX-linked Adrenoleukodystrophy
Pujol, Aurora, M.D. 18
Large-scale testing (Phase 3)Study completedNCT04303416
What this trial is testing

Plasma Exchange With Albumin in AMN Patients

Who this might be right for
AdrenomyeloneuropathyAdrenoleukodystrophy
Pujol, Aurora, M.D. 5
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
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
Large-scale testing (Phase 3)Study completedNCT02961803
What this trial is testing

MD1003-AMN MD1003 in Adrenomyeloneuropathy

Who this might be right for
AdrenomyeloneuropathyAdrenoleukodystrophyAMN
MedDay Pharmaceuticals SA 67
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
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
Testing effectiveness (Phase 2)WithdrawnNCT05146284
What this trial is testing

Study to Assess PXL770 in Subjects With Adrenomyeloneuropathy (AMN) Form of X-linked Adrenoleukodystrophy (X-ALD or ALD)

Who this might be right for
Adrenomyeloneuropathy
Poxel SA
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
Not applicableUnknownNCT04090268
What this trial is testing

Precision Exercise in Children With Malignant Hemopathies

Who this might be right for
Leukemia, Myeloid, AcuteLeukemia, B-cellLeukemia, T Cell+5 more
University of Milano Bicocca 380
Not applicableLooking for participantsNCT05939232
What this trial is testing

Registry of X-linked Adrenoleukodystrophy

Who this might be right for
X-linked Adrenoleukodystrophy
Beijing Tiantan Hospital 200
Testing effectiveness (Phase 2)Study completedNCT03513328
What this trial is testing

Conditioning Regimen for Allogeneic Hematopoietic Stem-Cell Transplantation

Who this might be right for
Bone Marrow Failure SyndromeThalassemiaSickle Cell Disease+13 more
University of Florida 6
Not applicableAvailableNCT01881334
What this trial is testing

Expanded Access to T-cell Depleted Haplo-Identical Stem Cells for Patients Receiving Haplo-Identical and Unrelated Cord Blood Transplants

Who this might be right for
Hematologic MalignanciesInborn Errors of Metabolism DisordersImmune Deficiencies
Joanne Kurtzberg, MD
Testing effectiveness (Phase 2)WithdrawnNCT03196765
What this trial is testing

Safety, Pharmacokinetics and Pharmacodynamics of NV1205 in Pediatric Male Subjects With Adrenoleukodystrophy

Who this might be right for
X-Linked Adrenoleukodystrophy
NeuroVia, Inc.
Testing effectiveness (Phase 2)Study completedNCT01495260
What this trial is testing

A Clinical Trial for AMN: Validation of Biomarkers of Oxidative Stress, Efficacy and Safety of a Mixture of Antioxidants

Who this might be right for
Adrenomyeloneuropathy
Pujol, Aurora, M.D. 13
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
Large-scale testing (Phase 3)Ended earlyNCT00545597
What this trial is testing

A Phase III Trial of Lorenzo's Oil in Adrenomyeloneuropathy

Who this might be right for
AdrenomyeloneuropathyAdrenoleukodystrophy
Eunice Kennedy Shriver National Institute of Child Health and Human Development (NICHD) 240
Testing effectiveness (Phase 2)Ended earlyNCT00004418
What this trial is testing

Effect of Glycerol Trierucate on Clinical Course of Adrenoleukodystrophy

Who this might be right for
Adrenoleukodystrophy
Hugo W. Moser Research Institute at Kennedy Krieger, Inc. 126
Not applicableStudy completedNCT01165060
What this trial is testing

The Effect of Bezafibrate on the Level of Very Long Chain Fatty Acids (VLCFA) in X-linked Adrenoleukodystrophy (X-ALD)

Who this might be right for
X-linked AdrenoleukodystrophyAdrenomyeloneuropathy
Academisch Medisch Centrum - Universiteit van Amsterdam (AMC-UvA) 10
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