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Results for “Brain Diseases, Metabolic, Inborn”

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Showing 20 of 0 results

Not applicableNot Yet RecruitingNCT05017402
What this trial is testing

Higher Dose of Alglucosidase Alpha for Pompe Disease

Who this might be right for
Glycogen Storage Disease Type II
Taipei Veterans General Hospital, Taiwan 36
Not applicableLooking for participantsNCT00567073
What this trial is testing

Pompe Pregnancy Sub-Registry

Who this might be right for
Glycogen Storage Disease Type II (GSD-II)Pompe Disease (Late-onset)Glycogenesis 2 Acid Maltase Deficiency
Genzyme, a Sanofi Company 20
Testing effectiveness (Phase 2)Study completedNCT05758922
What this trial is testing

Phase 2 Study Evaluating the Safety, Tolerability, Pharmacokinetics and Pharmacodynamics of Oral AZ-3102 in Patients With GM2 Gangliosidosis or Niemann-Pick Type C Disease

Who this might be right for
GM2 GangliosidosisNiemann-Pick Disease, Type C
Azafaros B.V. 13
Large-scale testing (Phase 3)Study completedNCT01347073
What this trial is testing

Study of the Safety, Pharmacokinetics and Efficacy of HPN-100, in Pediatric Subjects With Urea Cycle Disorders (UCDs)

Who this might be right for
Urea Cycle Disorders
Amgen 23
Not applicableUnknownNCT02527239
What this trial is testing

Prevalence of Pompe's Disease in Respiratory Clinics

Who this might be right for
Pompe's Disease
Royal Brompton & Harefield NHS Foundation Trust 400
Large-scale testing (Phase 3)Active Not RecruitingNCT04849741
What this trial is testing

Evaluate the Safety and Efficacy of Zilganersen (ION373) in Patients With Alexander Disease (AxD)

Who this might be right for
Alexander Disease
Ionis Pharmaceuticals, Inc. 54
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 applicableStudy completedNCT00309400
What this trial is testing

The Early History of Universal Screening for Metabolic Disorders

Who this might be right for
PhenylketonuriaGalactosemiaInborn Errors of Metabolism
University of Miami 10
Testing effectiveness (Phase 2)Study completedNCT00053573
What this trial is testing

RhGAA in Patients With Infantile-onset Glycogen Storage Disease-II (Pompe Disease)

Who this might be right for
Glycogen Storage Disease Type IIPompe DiseaseAcid Maltase Deficiency Disease+1 more
Genzyme, a Sanofi Company 20
Large-scale testing (Phase 3)Active Not RecruitingNCT03911505
What this trial is testing

ZIP Study-OL Study of Safety, PK, Efficacy, PD, Immunogenicity of ATB200/AT2221 in Pediatrics Aged 0 to < 18 y.o. w/LOPD

Who this might be right for
Pompe Disease (Late-onset)
Amicus Therapeutics 21
Not applicableActive Not RecruitingNCT05596760
What this trial is testing

Promoting Goals-of-Care Discussions for Patients With Memory Problems and Their Caregivers

Who this might be right for
DementiaDementia, VascularMixed Dementias+14 more
University of Washington 1,802
Not applicableApproved For MarketingNCT00954460
What this trial is testing

Treatment Protocol of Velaglucerase Alfa for Patients With Type 1 Gaucher Disease

Who this might be right for
Gaucher Disease, Type 1
Shire
Testing effectiveness (Phase 2)Looking for participantsNCT06488313
What this trial is testing

Evaluate the Pharmacodynamics and Safety of ARCT-810 in Participants With OTCD

Who this might be right for
OTC DeficiencyOrnithine Transcarbamylase DeficiencyOTCD
Arcturus Therapeutics, Inc. 9
Not applicableLooking for participantsNCT06178120
What this trial is testing

Disease Progression in Women With X-linked Adrenoleukodystrophy

Who this might be right for
X-linked Adrenoleukodystrophy
Minoryx Therapeutics, S.L. 40
Large-scale testing (Phase 3)Study completedNCT00947297
What this trial is testing

Study of the Safety of HPN (Hyperion)-100 for the Long-Term Treatment of Urea Cycle Disorders (Treat UCD)

Who this might be right for
Urea Cycle Disorders
Amgen 60
Not applicableEnded earlyNCT05229549
What this trial is testing

Study to Evaluate the Effect on Nitrogen Retention of Two Different Posology Schemes of PKU GOLIKE PLUS 3-16 and Free AAs in Patients With Phenylketonuria

Who this might be right for
Phenylketonurias
APR Applied Pharma Research s.a. 5
Not applicableUnknownNCT04847713
What this trial is testing

Early Detection and Follow-Up of Patients With Fabry's Disease

Who this might be right for
Fabry's Disease
Hospital Clinico Universitario de Santiago 100
Not applicableLooking for participantsNCT04463316
What this trial is testing

GROWing Up With Rare GENEtic Syndromes

Who this might be right for
Prader-Willi SyndromePWS-like SyndromeSilver Russel Syndrome+30 more
dr. Laura C. G. de Graaff-Herder 600
Testing effectiveness (Phase 2)Not Yet RecruitingNCT07641140
What this trial is testing

Phase I/II Clinical Study to Evaluate the Safety, Tolerability and Efficacy of LY-M003 Injection in Adult Patients With Wilson's Disease

Who this might be right for
Wilson's Disease
Lingyi Biotech Co., Ltd. 18
Not applicableActive Not RecruitingNCT04406818
What this trial is testing

Metabolic and Hemodynamic Reserve in Pediatric SCA

Who this might be right for
Child, OnlyBrain DiseasesSickle Cell Disease+1 more
Washington University School of Medicine 120
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