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Results for “Zellweger Syndrome”

11 trials

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Showing 11 of 11 results

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 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
Large-scale testing (Phase 3)Study completedNCT01838941
What this trial is testing

Betaine and Peroxisome Biogenesis Disorders

Who this might be right for
Peroxisome Biogenesis Disorders
McGill University Health Centre/Research Institute of the McGill University Health Centre 12
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
Not applicableActive Not RecruitingNCT03115086
What this trial is testing

The REPLACE Registry for Cholbam® (Cholic Acid)

Who this might be right for
Bile Acid Synthesis Disorders
Mirum Pharmaceuticals, Inc. 55
Testing effectiveness (Phase 2)Active Not RecruitingNCT02171104
What this trial is testing

MT2013-31: Allo HCT for Metabolic Disorders and Severe Osteopetrosis

Who this might be right for
Mucopolysaccharidosis DisordersHurler SyndromeHunter Syndrome+26 more
Masonic Cancer Center, University of Minnesota 149
Not applicableLooking for participantsNCT03047369
What this trial is testing

The Myelin Disorders Biorepository Project

Who this might be right for
LeukodystrophyWhite Matter DiseaseLeukoencephalopathies+63 more
Children's Hospital of Philadelphia 12,000
Not applicableStudy completedNCT03440905
What this trial is testing

Proxy-Reported Symptoms and Quality of Life Survey in Zellweger Spectrum Disorders

Who this might be right for
Zellweger Spectrum
University of South Florida 92
Not applicableEnded earlyNCT00004442
What this trial is testing

Study of Bile Acids in Patients With Peroxisomal Disorders

Who this might be right for
Infantile Refsum's DiseaseZellweger SyndromeBifunctional Enzyme Deficiency+1 more
University of Cincinnati 25
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
Not applicableLooking for participantsNCT06190626
What this trial is testing

Longitudinal Prospective Natural History Study of Retinopathy in Zellweger Spectrum Disorder

Who this might be right for
Zellweger Spectrum Disorder
McGill University Health Centre/Research Institute of the McGill University Health Centre 30