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Results for “Peroxisomal Biogenesis Disorder”

85 trials

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Showing 20 of 85 results

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
Testing effectiveness (Phase 2)Study completedNCT03856866
What this trial is testing

Hydroxychloroquine Administration for Reduction of Pexophagy

Who this might be right for
Zellweger SyndromePeroxisome Biogenesis Disorders
The Hospital for Sick Children 3
Early research (Phase 1)UnknownNCT05969977
What this trial is testing

A First-in-Human Phase 1 Study of Plasmalogen Precursor PPI-1011 in Healthy Adult Volunteers to Assess Safety, Tolerability, and Pharmacokinetics

Who this might be right for
Rhizomelic Chondrodysplasia Punctata
MED-LIFE DISCOVERIES LP 56
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
Not applicableUnknownNCT04031287
What this trial is testing

RCDP Natural History Study

Who this might be right for
Rhizomelic Chondrodysplasia Punctata
MED-LIFE DISCOVERIES LP 75
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 applicableLooking for participantsNCT01668186
What this trial is testing

Longitudinal Natural History Study of Patients With Peroxisome Biogenesis Disorders (PBD)

Who this might be right for
Peroxisome Biogenesis DisorderZellweger Spectrum DisorderRCDP - Rhizomelic Chondrodysplasia Punctata+8 more
McGill University Health Centre/Research Institute of the McGill University Health Centre 244
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
Not applicableLooking for participantsNCT02435940
What this trial is testing

Inherited Retinal Degenerative Disease Registry

Who this might be right for
Eye Diseases HereditaryRetinal DiseaseAchromatopsia+25 more
Foundation Fighting Blindness 20,000
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 applicableStudy completedNCT03163771
What this trial is testing

Evaluation of a New Strategy for the Diagnosis of Peroxisomal Diseases

Who this might be right for
Peroxisomal DisordersDiagnoses Disease
University Hospital, Lille 8
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.
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