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Results for “Peroxisomal Disorder”

87 trials

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Showing 20 of 87 results

Large-scale testing (Phase 3)Study completedNCT02334748
What this trial is testing

Canakinumab in Patients With Systemic Juvenile Idiopathic Arthritis or Hereditary Periodic Fevers Who Participated in the CACZ885G2301E1, CACZ885G2306 or CACZ885N2301 Studies

Who this might be right for
Systemic Juvenile Idiopathic ArthritisHereditary Periodic Fevers
Novartis Pharmaceuticals 31
Not applicableLooking for participantsNCT06796920
What this trial is testing

Epidural Spinal Cord Stimulation for Lower-limb Impairment in Adrenomyeloneuropathy

Who this might be right for
Adrenomyeloneuropathy Without Cerebral Involvement
Third Military Medical University 10
Not applicableLooking for participantsNCT06497829
What this trial is testing

Geranylgeraniol Supplementation in Patients With Mevalonate Kinase Deficiency

Who this might be right for
Mevalonate Kinase Deficiency
University Hospital, Motol 6
Not applicableEnded earlyNCT02204904
What this trial is testing

Observational Study to Evaluate Allogeneic HSCT Outcomes for Cerebral Adrenoleukodystrophy (CALD)

Who this might be right for
X-Linked Adrenoleukodystrophy (X-ALD)Cerebral Adrenoleukodystrophy (CALD)Adrenoleukodystrophy (ALD)
Genetix Biotherapeutics Inc. 59
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)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 applicableStudy completedNCT01626092
What this trial is testing

Reduced-Intensity Hematopoietic Stem Cell Transplant for High Risk Lysosomal and Peroxisomal Disorders

Who this might be right for
Lysosomal Storage DiseasePeroxisomal Disorder
Masonic Cancer Center, University of Minnesota 3
Not applicableLooking for participantsNCT04569162
What this trial is testing

Rhizomelic Chondrodysplasia Punctata Registry

Who this might be right for
RCDP - Rhizomelic Chondrodysplasia PunctataRCDP1RCDP2+3 more
Nemours Children's Clinic 100
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 applicableStudy completedNCT02699190
What this trial is testing

LeukoSEQ: Whole Genome Sequencing as a First-Line Diagnostic Tool for Leukodystrophies

Who this might be right for
LeukodystrophyWhite Matter Disease4H Syndrome+61 more
Children's Hospital of Philadelphia 236
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 participantsNCT04528355
What this trial is testing

Data Collection Study of Patients With Non-Malignant Disorders Undergoing UCBT, BMT or PBSCT With RIC

Who this might be right for
Primary Immunodeficiency (PID)Congenital Bone Marrow Failure SyndromesInherited Metabolic Disorders (IMD)+2 more
Paul Szabolcs 50
Not applicableWithdrawnNCT03649919
What this trial is testing

Multi-center Clinical Study on the Diagnosis and Treatment Management of Rare Neurological Disease in Children

Who this might be right for
XlsmaDMDX-ALD+1 more
Children's Hospital of Fudan University
Not applicableStudy completedNCT00004348
What this trial is testing

Study of Protein Translocation in Patients With Beta-Oxidation Disorders

Who this might be right for
Beta-Oxidation DisorderPeroxisomal Disorders
National Institute of Diabetes and Digestive and Kidney Diseases (NIDDK) 20
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 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
Not applicableLooking for participantsNCT04675749
What this trial is testing

Quality of Life in Women with X-linked Adrenoleukodystrophy

Who this might be right for
X-linked Adrenoleukodystrophy
Leipzig University Medical Center 200
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
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 applicableUnknownNCT03278899
What this trial is testing

Prospectively Assess Disease Progression in Male Children With X-ALD

Who this might be right for
X-Linked Adrenoleukodystrophy
NeuroVia, Inc. 100
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