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Results for “Inherited Metabolic Disorder of Nervous System”

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Showing 20 of 0 results

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
Testing effectiveness (Phase 2)Study completedNCT01818921
What this trial is testing

An Efficacy, Safety, and Pharmacokinetics Study of Beloranib in Obese Subjects With Prader-Willi Syndrome

Who this might be right for
ObesityOver-weightPrader-Willi Syndrome
Zafgen, Inc. 17
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
Large-scale testing (Phase 3)Active Not RecruitingNCT07236606
What this trial is testing

RGX-121-3102 Gene Therapy in Participants With MPS II (Hunter Syndrome)

Who this might be right for
MPS IIHunter Syndrome (MPS II)
REGENXBIO Inc. 2
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
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 completedNCT02380638
What this trial is testing

Effects of Whole-body Vibration Training on Body Composition in Adolescents With and Without Down Syndrome

Who this might be right for
Down Syndrome
Universidad de Zaragoza 58
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
Not applicableStudy completedNCT02789059
What this trial is testing

Muscle Oxygenation in Effort in Neuromuscular Diseases

Who this might be right for
Neuromuscular Diseases
University Hospital, Lille 17
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 applicableLooking for participantsNCT07308704
What this trial is testing

Deep Learning For Echo Analysis, Tracking, And Evaluation

Who this might be right for
CardiomyopathyIschemic Heart DiseaseCardiac Amyloidosis
Mayo Clinic 10,040,000
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
Large-scale testing (Phase 3)UnknownNCT05032326
What this trial is testing

Long-term Interventional Follow-up Study of Children With Prader-Willi Syndrome Included in the OTBB3 Clinical Trial

Who this might be right for
Prader-Willi Syndrome
University Hospital, Toulouse 80
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
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