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Results for “Brain Diseases, Metabolic, Inborn”

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Showing 20 of 0 results

Large-scale testing (Phase 3)Study completedNCT04138277
What this trial is testing

Assess the Long-term Safety and Efficacy of ATB200/AT2221 in Adult Subjects With Late-Onset Pompe Disease (LOPD)

Who this might be right for
Pompe Disease (Late-onset)
Amicus Therapeutics 119
Not applicableActive Not RecruitingNCT00787865
What this trial is testing

Diffusion Tensor Imaging (DTI) in Infants With Krabbe Disease

Who this might be right for
Krabbe Disease
University of Pittsburgh 100
Testing effectiveness (Phase 2)Study completedNCT02404337
What this trial is testing

Betaine METABOLISM OF PATIENTS With Homocystinuria

Who this might be right for
Homocystinuria
Assistance Publique - Hôpitaux de Paris 12
Not applicableStudy completedNCT04879277
What this trial is testing

Study of Low-grade Systemic Inflammation in Adult Patients With Phenylketonuria

Who this might be right for
Phenylketonuria
University Hospital, Tours 40
Not applicableLooking for participantsNCT07013292
What this trial is testing

Efficacy of Omaveloxolone Treatment for Dysphagia in French Patients With Friedreich's Ataxia

Who this might be right for
Friedreich Ataxia
Centre Hospitalier Universitaire de Nice 40
Testing effectiveness (Phase 2)Study completedNCT00229632
What this trial is testing

Idebenone to Treat Friedreich's Ataxia

Who this might be right for
Friedreich Ataxia
National Institute of Neurological Disorders and Stroke (NINDS) 51
Not applicableActive Not RecruitingNCT04875416
What this trial is testing

Phenotype, Genotype and Biomarkers 2

Who this might be right for
Amyotrophic Lateral SclerosisHereditary Spastic ParaplegiaPrimary Lateral Sclerosis+2 more
University of Miami 217
Large-scale testing (Phase 3)Study completedNCT00712348
What this trial is testing

Switchover Trial From Imiglucerase to Plant Cell Expressed Recombinant Human Glucocerebrosidase

Who this might be right for
Gaucher Disease
Pfizer 31
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
Early research (Phase 1)Looking for participantsNCT07354724
What this trial is testing

Evaluate the Safety, Pharmacokinetics, and Pharmacodynamics of DNL952 in Adult Participants With Late-Onset Pompe Disease

Who this might be right for
Late-onset Pompe Disease
Denali Therapeutics Inc. 32
Not applicableStudy completedNCT03911089
What this trial is testing

A Collection of Case Studies in Infants With UCD to Evaluate Infant Growth and the Safety of a New Medical Food for UCD

Who this might be right for
Urea Cycle DisorderInborn Errors of Metabolism
Nutricia Research 3
Not applicableLooking for participantsNCT06573723
What this trial is testing

Institutional Registry of Rare Diseases

Who this might be right for
Rare DiseasesAmyloidosisSarcoidosis+23 more
Hospital Italiano de Buenos Aires 380
Not applicableStudy completedNCT02354651
What this trial is testing

Response to Diaphragmatic Pacing in Subjects With Pompe Disease

Who this might be right for
Pompe Disease
University of Florida 9
Not applicableActive Not RecruitingNCT03090789
What this trial is testing

FA Clinical Outcome Measures

Who this might be right for
Friedreich AtaxiaNeuro-Degenerative Disease
Friedreich's Ataxia Research Alliance 2,000
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 completedNCT05099640
What this trial is testing

PTC923 in Participants With Phenylketonuria

Who this might be right for
Phenylketonuria
PTC Therapeutics 157
Testing effectiveness (Phase 2)Looking for participantsNCT07371663
What this trial is testing

An Phase Ib/II Clinical Trial of TCC1727 Combination Therapy in Advanced Solid Tumors

Who this might be right for
Solid CancersNSCLC (Advanced Non-small Cell Lung Cancer)Gastric (Stomach) Cancer+2 more
Beijing Tide Pharmaceutical Co., Ltd 266
Not applicableActive Not RecruitingNCT04349514
What this trial is testing

A Natural History Study to TRACK Brain and Spinal Cord Changes in Individuals with Friedreich Ataxia (TRACK-FA)

Who this might be right for
Friedreich Ataxia
Monash University 300
Not applicableUnknownNCT04548921
What this trial is testing

Biomarker for Friedreich's Ataxia (BioFridA)

Who this might be right for
FXN GeneFRDAHereditary Diseases
CENTOGENE GmbH Rostock 1,000
Very early researchStudy completedNCT02705547
What this trial is testing

Rosuvastatin (Crestor) in Friedreich Ataxia

Who this might be right for
Friedreich Ataxia
Children's Hospital of Philadelphia 12
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