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Results for “Brain Diseases, Metabolic, Inborn”

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Showing 20 of 0 results

Early research (Phase 1)Study completedNCT04176991
What this trial is testing

Single Ascending Dose Study of CTI-1601 Versus Placebo in Subjects With Friedreich's Ataxia

Who this might be right for
Friedreich Ataxia
Larimar Therapeutics, Inc. 28
Testing effectiveness (Phase 2)Not Yet RecruitingNCT07750990
What this trial is testing

An Extension Study of S-606001 in Participants With Late-onset Pompe Disease (LOPD)

Who this might be right for
Pompe Disease
Shionogi 60
Not applicableUnknownNCT05026489
What this trial is testing

G6PD Deficiency in Infarction Patients in Shaanxi Province

Who this might be right for
Cerebral InfarctionG6PD Deficiency
First Affiliated Hospital Xi'an Jiaotong University 1,000
Early research (Phase 1)Study completedNCT00001410
What this trial is testing

PEG-Glucocerebrosidase for the Treatment of Gaucher Disease

Who this might be right for
Gaucher's Disease
National Institute of Mental Health (NIMH) 18
Large-scale testing (Phase 3)Study completedNCT01371825
What this trial is testing

Safety, Tolerability, Efficacy, Pharmacokinetics, and Pharmacodynamics of Sebelipase Alfa in Children With Growth Failure Due to Lysosomal Acid Lipase Deficiency

Who this might be right for
Lysosomal Acid Lipase DeficiencyWolman Disease
Alexion Pharmaceuticals, Inc. 9
Testing effectiveness (Phase 2)Ended earlyNCT05394064
What this trial is testing

Evaluate Administration of SBT101 Gene Therapy in Adult Patients With Adrenomyeloneuropathy (AMN)

Who this might be right for
AMNAMN Gene MutationX-ALD
SwanBio Therapeutics, Inc. 9
Not applicableNot Yet RecruitingNCT07731971
What this trial is testing

Digital Outcome Assessment Using AI Active Gaming and Motion Capture in Friedreich Ataxia

Who this might be right for
Friedreich Ataxia
University of Exeter 40
Early research (Phase 1)Study completedNCT00078481
What this trial is testing

Phase 1 Trial of Idebenone to Treat Patients With Friedreich's Ataxia

Who this might be right for
Friedreich Ataxia
National Institute of Neurological Disorders and Stroke (NINDS) 16
Large-scale testing (Phase 3)Looking for participantsNCT07778836
What this trial is testing

Evaluate the Efficacy and Safety of Subcutaneous Nomlabofusp in Subjects With Friedreich's Ataxia

Who this might be right for
Friedreich AtaxiaFriedreich's Ataxia
Larimar Therapeutics, Inc. 150
Not applicableStudy completedNCT02702765
What this trial is testing

SCD163 and sMR in Wilsons Disease - Associations With Disease Severity and Fibrosis

Who this might be right for
Wilsons Disease
University of Aarhus 33
Early research (Phase 1)Looking for participantsNCT07672756
What this trial is testing

A Clinical Study on the Safety and Tolerability of PL54 Injection in Adult Patients With Phenylketonuria (PKU)

Who this might be right for
Phenylketonuria (PKU)
Chongqing Peg-Bio Biopharm Co., Ltd. 48
Testing effectiveness (Phase 2)Study completedNCT03378531
What this trial is testing

AEB1102 (Pegzilarginase) in Patients With Arginase 1 Deficiency

Who this might be right for
Arginase I DeficiencyHyperargininemia
Immedica Pharma AB 14
Large-scale testing (Phase 3)Study completedNCT02612129
What this trial is testing

Arimoclomol Prospective Study in Participants Diagnosed With Niemann-Pick Disease Type C

Who this might be right for
Niemann-Pick Disease, Type C
ZevraDenmark 50
Not applicableUnknownNCT02935283
What this trial is testing

Neuroimaging and Neuropsychological Outcomes in Urea Cycle Disorders

Who this might be right for
Urea Cycle Disorders
Children's National Research Institute 56
Post-approval studies (Phase 4)Not Yet RecruitingNCT06575829
What this trial is testing

Treatment Frequency Reduction in Pompe Disease

Who this might be right for
Pompe Disease (Late-onset)GAA DeficiencyGlycogen Storage Disease Type II+1 more
Erasmus Medical Center 10
Post-approval studies (Phase 4)UnknownNCT04088513
What this trial is testing

Safety and Efficacy of Aspirin in Stroke Patients With Glucose-6-phosphate Dehydrogenase Deficiency (SAST)

Who this might be right for
G6PD DeficiencyStroke
First Affiliated Hospital, Sun Yat-Sen University 440
Not applicableWithdrawnNCT00795197
What this trial is testing

A Screening Study Evaluating Disease Status of Gaucher Type I Patients

Who this might be right for
Gaucher Disease, Type 1
Genzyme, a Sanofi Company
Not applicableEnded earlyNCT01963650
What this trial is testing

Natural History Study of Children With Metachromatic Leukodystrophy

Who this might be right for
Lipid Metabolism DisordersMetachromatic Leukodystrophy (MLD)Nervous System Diseases+11 more
Shire 1
Not applicableActive Not RecruitingNCT06095713
What this trial is testing

German Observational Multicenter Study of Patients With Fabry Disease Under Enzyme Replacement Therapy With Pegunigalsidase-alfa

Who this might be right for
Fabry Disease
Universität Münster 60
Testing effectiveness (Phase 2)Study completedNCT04273165
What this trial is testing

Safety and Efficacy of Etravirine in Friedreich Ataxia Patients

Who this might be right for
Friedreich Ataxia
IRCCS Eugenio Medea 30
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