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Results for “Brain Diseases, Metabolic, Inborn”

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Showing 20 of 0 results

Testing effectiveness (Phase 2)Looking for participantsNCT06818838
What this trial is testing

A Clinical Study Evaluating LY-M001 Injection in the Treatment of Adult Patients With Type I Gaucher Disease

Who this might be right for
Gaucher Disease Type 1
Lingyi Biotech Co., Ltd. 12
Not applicableStudy completedNCT01517048
What this trial is testing

Brain-Derived Neurotrophic Factor in Obesity and Brain Function

Who this might be right for
ObesityGenetic DisorderMental Retardation+1 more
Eunice Kennedy Shriver National Institute of Child Health and Human Development (NICHD) 93
Early research (Phase 1)Ended earlyNCT04669535
What this trial is testing

A Dose-escalation and Safety & Efficacy Study of AXO-AAV-GM2 in Tay-Sachs or Sandhoff Disease

Who this might be right for
Tay-Sachs DiseaseSandhoff Disease
Terence Flotte 9
Not applicableStudy completedNCT01899950
What this trial is testing

Longitudinal Study of Cognition With Niemann-Pick Disease, Type C

Who this might be right for
Niemann-Pick Disease, Type C
Mayo Clinic 70
Testing effectiveness (Phase 2)UnknownNCT01951989
What this trial is testing

Intra-monocyte Imiglucerase Kinetics in Gaucher Disease

Who this might be right for
Gaucher Disease
University Hospital, Clermont-Ferrand 60
Not applicableStudy completedNCT00778206
What this trial is testing

PKUDOS: Phenylketonuria (PKU) Demographic, Outcomes, and Safety Registry

Who this might be right for
PhenylketonuriaHyperphenylalaninaemia
BioMarin Pharmaceutical 1,887
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 applicableEnded earlyNCT04270604
What this trial is testing

Biological Sample Collection for Research and Biobanking

Who this might be right for
Parkinson DiseaseDiabetes Mellitus, Type 1Macular Degeneration+4 more
New York Stem Cell Foundation Research Institute 1,279
Early research (Phase 1)Study completedNCT06143904
What this trial is testing

Evaluate Absolute Bioavailability, Absorption, Metabolism, and Excretion of Genz-112638 in Healthy Male Participants

Who this might be right for
Gaucher's Disease Type I
Sanofi 10
Not applicableUnknownNCT04212195
What this trial is testing

Cohort Research on Wilson's Disease

Who this might be right for
Wilson's Disease
University College, London 500
Not applicableStudy completedNCT03122925
What this trial is testing

TCA Cycle in the Dentate in Friedreich's Ataxia

Who this might be right for
Friedreich Ataxia
University of Minnesota 20
Large-scale testing (Phase 3)Looking for participantsNCT07465718
What this trial is testing

Trientine Tetrahydrochloride Administered Once a Day for the First Line Treatment of Wilson's Disease Patients.

Who this might be right for
Wilson's Disease
Orphalan 38
Not applicableStudy completedNCT03120013
What this trial is testing

Rehabilitative Trial With Cerebello-Spinal tDCS in Neurodegenerative Ataxia

Who this might be right for
Ataxia, CerebellarCerebellar AtaxiaSpinocerebellar Ataxias+8 more
Azienda Socio Sanitaria Territoriale degli Spedali Civili di Brescia 21
Large-scale testing (Phase 3)Ended earlyNCT00122980
What this trial is testing

Stroke With Transfusions Changing to Hydroxyurea

Who this might be right for
HemochromatosisCerebrovascular AccidentAnemia, Sickle Cell+1 more
St. Jude Children's Research Hospital 134
Not applicableStudy completedNCT00006142
What this trial is testing

Study of a Phenylalanine Restricted Diet During Pregnancy to Prevent Symptoms in Offspring of Patients With Phenylketonuria

Who this might be right for
Phenylketonuria
National Center for Research Resources (NCRR)
Not applicableUnknownNCT05951790
What this trial is testing

Inspiratory Muscle Training (IMT) in Adult People With Pompe Disease

Who this might be right for
Glycogen Storage Disease Type IIPompe Disease (Late-onset)
Fondazione Don Carlo Gnocchi ETS 20
Testing effectiveness (Phase 2)Study completedNCT01942590
What this trial is testing

Safety and Efficacy of Clenbuterol in Individuals With Late-onset Pompe Disease and Receiving Enzyme Replacement Therapy

Who this might be right for
Pompe Disease
Dwight Koeberl, M.D., Ph.D. 17
Early research (Phase 1)Study completedNCT01747980
What this trial is testing

Safety and Pharmacokinetics of Oral PRX-112 in Gaucher Disease Patients

Who this might be right for
Gaucher Disease
Protalix 16
Not applicableStudy completedNCT05409846
What this trial is testing

Screening of Fabry Disease in Portuguese Patients With Idiopathic Cardiomyopathies

Who this might be right for
Fabry Disease
Universidade do Porto 409
Not applicableUnknownNCT02605603
What this trial is testing

SRT in Comparison to ERT on Immune Aspects and Bone Involvement in Gaucher Disease

Who this might be right for
Gaucher Disease
Lysosomal and Rare Disorders Research and Treatment Center, Inc. 30
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