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Results for “Brain Diseases, Metabolic, Inborn”

1,543 trials

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Showing 20 of 1,543 results

Testing effectiveness (Phase 2)Study completedNCT02140151
What this trial is testing

Prophylactic Ranibizumab for Exudative Age-related Macular Degeneration

Who this might be right for
Age-related Macular Degeneration
Southern California Desert Retina Consultants, MC 108
Not applicableNo Longer AvailableNCT02233257
What this trial is testing

Expanded Access for Lorenzo's Oil (GTO/GTE) in Adrenoleukodystrophy

Who this might be right for
X-linked Adrenoleukodystrophy
University of Minnesota
Not applicableLooking for participantsNCT07461519
What this trial is testing

Gonadic Function and Pubertal Development in Female Patients With Classic Galactosemia

Who this might be right for
Galactosemia
IRCCS Azienda Ospedaliero-Universitaria di Bologna 60
Large-scale testing (Phase 3)Study completedNCT00158600
What this trial is testing

A Placebo-Controlled Study of Safety and Effectiveness of Myozyme (Alglucosidase Alfa) in Patients With Late-Onset Pompe Disease

Who this might be right for
Pompe Disease (Late-onset)Glycogen Storage Disease Type II (GSD-II)Acid Maltase Deficiency Disease+1 more
Genzyme, a Sanofi Company 90
Testing effectiveness (Phase 2)Study completedNCT00250939
What this trial is testing

RhGAA in Patients With Late-Onset Pompe Disease

Who this might be right for
Pompe Disease (Late-onset)Glycogen Storage Disease Type II (GSD-II)Acid Maltase Deficiency Disease+1 more
Genzyme, a Sanofi Company 5
Large-scale testing (Phase 3)Study completedNCT03485677
What this trial is testing

Safety and Efficacy of Eliglustat With or Without Imiglucerase in Pediatric Patients With Gaucher Disease (GD) Type 1 and Type 3

Who this might be right for
Gaucher's Disease Type IGaucher's Disease Type III
Sanofi 57
Large-scale testing (Phase 3)WithdrawnNCT00935753
What this trial is testing

Trial of Kuvan in Lesch-Nyhan Disease

Who this might be right for
Behavioral Manifestations of Lesch-Nyhan Disease
University of California, San Diego
Not applicableEnded earlyNCT03896607
What this trial is testing

Liver and the Spleen Stifness in Adult Patients With Gaucher's Disease Using Ultrasound Shear Wave Elastography

Who this might be right for
Gaucher DiseaseLiver FibrosisSpleen; Fibrosis
HaEmek Medical Center, Israel 18
Not applicableLooking for participantsNCT05848271
What this trial is testing

Natural History Study of Patients with HPDL Mutations

Who this might be right for
Mitochondrial EncephalomyopathiesHereditary Spastic ParaplegiaSpastic Paraplegia+4 more
University of California, San Diego 50
Large-scale testing (Phase 3)Active Not RecruitingNCT03614234
What this trial is testing

Open Label Extension of 2 mg/kg Pegunigalsidase Alfa (PRX-102) Every 4 Weeks in Adult Fabry Disease Patients

Who this might be right for
Fabry Disease
Chiesi Farmaceutici S.p.A. 29
Not applicableStudy completedNCT01776164
What this trial is testing

Early and Longitudinal Assessment of Neurodegeneration in the Brain and Spinal Cord in Friedreich's Ataxia

Who this might be right for
Friedreich Ataxia
University of Minnesota 85
Not applicableUnknownNCT05092230
What this trial is testing

Frequency of Pompe Disease in Patients With Myalgia With or Without Hyper Ckemia - Data From the Reference Center (CERCA)

Who this might be right for
Pompe Disease
University Hospital Center of Martinique 100
Testing effectiveness (Phase 2)Study completedNCT01685216
What this trial is testing

Efficacy and Safety Study of Velaglucerase Alfa in Children and Adolescents With Type 3 Gaucher Disease

Who this might be right for
Gaucher Disease, Type 3
Shire 7
Large-scale testing (Phase 3)Active Not RecruitingNCT05222906
What this trial is testing

Study to Evaluate the Efficacy and Safety of Venglustat in Adult and Pediatric Patients With Gaucher Disease Type 3

Who this might be right for
Gaucher's Disease Type III
Sanofi 43
Testing effectiveness (Phase 2)Study completedNCT00813865
What this trial is testing

A Long-Term Extension Study of AT2101 (Afegostat Tartrate) in Type 1 Gaucher Patients

Who this might be right for
Gaucher DiseaseType 1 Gaucher DiseaseGaucher Disease, Type 1
Amicus Therapeutics 8
Large-scale testing (Phase 3)Active Not RecruitingNCT02616484
What this trial is testing

Trial of Dichloroacetate in Pyruvate Dehydrogenase Complex Deficiency:

Who this might be right for
Pyruvate Dehydrogenase Complex Deficiency
Saol Therapeutics Inc 34
Not applicableStudy completedNCT02403687
What this trial is testing

Prospective Analgesic Compound Efficacy (PACE) Study

Who this might be right for
ArthritisTendonitisGout+5 more
Express Specialty Pharmacy 300
Testing effectiveness (Phase 2)Study completedNCT00084084
What this trial is testing

Replagal Enzyme Replacement Therapy for Children With Fabry Disease

Who this might be right for
Fabry Disease
Shire 17
Not applicableNot Yet RecruitingNCT07241832
What this trial is testing

Multifaceted Assessment of Patients With Wilson's Disease in a Low-Resource Setting in Upper Egypt: Service Integration, Psychosocial Burden, Dietary Practices, and the Geo-Spatial Disease Map

Who this might be right for
Wilson's Disease
Assiut University 66
Not applicableUnknownNCT04302194
What this trial is testing

Early Dietary Treated Patients With Phenylketonuria Can Achieve Normal Growth and Mental Development.

Who this might be right for
Phenylketonurias
Assiut University 50
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