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Results for “X-Linked Adrenoleukodystrophy (X-ALD)”

66 trials

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Showing 20 of 66 results

Not applicableLooking for participantsNCT03789721
What this trial is testing

Adrenoleukodystrophy National Registry Study

Who this might be right for
ALD (Adrenoleukodystrophy)AdrenoleukodystrophyCerebral Adrenoleukodystrophy
Masonic Cancer Center, University of Minnesota 1,000
Large-scale testing (Phase 3)Study completedNCT03231878
What this trial is testing

A Clinical Study to Evaluate the Efficacy and Safety of MIN-102 (IMP) in Male AMN Patients.

Who this might be right for
Adrenoleukodystrophy
Minoryx Therapeutics, S.L. 105
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
Testing effectiveness (Phase 2)WithdrawnNCT05200104
What this trial is testing

Study to Assess PXL065 in Subjects With Adrenomyeloneuropathy (AMN) Form of X-linked Adrenoleukodystrophy (X-ALD or ALD)

Who this might be right for
ALD (Adrenoleukodystrophy)
Poxel SA
Not applicableNot Yet RecruitingNCT03727555
What this trial is testing

IT and IV Lentiviral Gene Therapy for X-ALD

Who this might be right for
X-linked Adrenoleukodystrophy
Shenzhen Geno-Immune Medical Institute 30
Large-scale testing (Phase 3)Study completedNCT00007020
What this trial is testing

Compassionate Treatment of Patients With Inborn Errors of Bile Acid Metabolism With Cholic Acid

Who this might be right for
Infantile Refsum's DiseaseZellweger SyndromeAdrenoleukodystrophy+2 more
Mirum Pharmaceuticals, Inc. 85
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
Testing effectiveness (Phase 2)Ended earlyNCT05394064
What this trial is testing

Evaluate Administration of SBT101 Gene Therapy in Adult Patients With Adrenomyeloneuropathy (AMN)

Who this might be right for
AMNAMN Gene MutationX-ALD
SwanBio Therapeutics, Inc. 9
Early research (Phase 1)Study completedNCT03500627
What this trial is testing

Evaluate the Safety, Tolerability, and Pharmacokinetics of OP-101 After Intravenous Administration in Healthy Volunteers

Who this might be right for
Healthy
Orpheris, Inc. 8
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 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
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 applicableLooking for participantsNCT06178120
What this trial is testing

Disease Progression in Women With X-linked Adrenoleukodystrophy

Who this might be right for
X-linked Adrenoleukodystrophy
Minoryx Therapeutics, S.L. 40
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
Testing effectiveness (Phase 2)Looking for participantsNCT01962415
What this trial is testing

Reduced Intensity Conditioning for Non-Malignant Disorders Undergoing UCBT, BMT or PBSCT

Who this might be right for
Primary Immunodeficiency (PID)Congenital Bone Marrow Failure SyndromesInherited Metabolic Disorders (IMD)+4 more
Paul Szabolcs 100
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
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
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
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
Not applicableLooking for participantsNCT04925349
What this trial is testing

Modeling Macrophages Activation Pattern in X-linked Adrenoleukodystrophy, Metachromatic Leukodystrophy and Adult Onset Leukoencephalopathy With Axonal Spheroids and Pigmented Glia

Who this might be right for
AdrenoleukodystrophyAdrenomyeloneuropathyMetachromatic Leukodystrophy+1 more
Assistance Publique - Hôpitaux de Paris 100
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