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Results for “Metabolic Inborn Brain Diseases”

1,522 trials

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Showing 20 of 1,522 results

Not applicableLooking for participantsNCT06610019
What this trial is testing

Cardiovascular Multimodality Imaging Study

Who this might be right for
Non-ischemic CardiomyopathyCardiomyopathiesHypertrophic Cardiomyopathy+7 more
Montefiore Medical Center 5,000
Not applicableLooking for participantsNCT03190837
What this trial is testing

A Long-term Follow-up Study of Gaucher Disease

Who this might be right for
Gaucher Disease
Duke University 200
Not applicableStudy completedNCT05388357
What this trial is testing

AI Quantitative Coronary Angiography Versus Optical Coherent Tomography Guidance for Coronary Stent Implantation

Who this might be right for
Coronary VesselsProcedure PCI
Asan Medical Center 400
Early research (Phase 1)Ended earlyNCT00875160
What this trial is testing

A Study in Type 1 Gaucher Patients to Evaluate the Pharmacokinetics, Safety and Pharmacodynamics of AT2101

Who this might be right for
Type 1 Gaucher Disease
Amicus Therapeutics 1
Early research (Phase 1)Study completedNCT01730482
What this trial is testing

Assess the Absorption, Metabolism and Excretion of Migalastat Hydrochloride (AT1001-014)

Who this might be right for
Fabry Disease
Amicus Therapeutics 6
Not applicableStudy completedNCT00484549
What this trial is testing

Fabry : National Initiative of Screening

Who this might be right for
Ischemic Stroke Hospitalized
University Hospital, Clermont-Ferrand 889
Not applicableEnded earlyNCT04768348
What this trial is testing

Natural History Clinical Study in Adult PKU

Who this might be right for
PhenylketonuriasPKU
Homology Medicines, Inc 7
Not applicableStudy completedNCT01776918
What this trial is testing

Energy Requirements in Mitochondrial Disease

Who this might be right for
Mitochondrial DiseaseChronic Metabolic Disorder
University of British Columbia 23
Very early researchLooking for participantsNCT06207552
What this trial is testing

Evaluation of the Safety, Tolerability and Efficacy of a Gene Therapy Drug for the Treatment of Pediatric Fabry Disease

Who this might be right for
Fabry Disease
Children's Hospital of Fudan University 6
Not applicableStudy completedNCT01879995
What this trial is testing

Phenylketonuria and Hyperphenylalaninemia Nutrition Study

Who this might be right for
Phenylketonuria (PKU) and Hyperphenylalaninemia
University of Zurich 30
Large-scale testing (Phase 3)Ended earlyNCT04378075
What this trial is testing

Evaluate Efficacy and Safety of Vatiquinone for Treating Mitochondrial Disease in Participants With Refractory Epilepsy

Who this might be right for
Mitochondrial DiseasesDrug Resistant EpilepsyLeigh Disease+5 more
PTC Therapeutics 68
Not applicableStudy completedNCT00055016
What this trial is testing

Registry of Fabry Disease - A Multicenter Observational Study

Who this might be right for
Fabry Disease
National Institute of Neurological Disorders and Stroke (NINDS) 100
Not applicableNot Yet RecruitingNCT06663878
What this trial is testing

An Exploratory Study to Evaluate the Tolerability and Safety of MWAV201 in Subjects With Wilson Disease

Who this might be right for
Wilson Disease
Xinhua Hospital, Shanghai Jiao Tong University School of Medicine 9
Early research (Phase 1)Looking for participantsNCT06150716
What this trial is testing

Orbit Study: A Study to Evaluate the Safety, Pharmacokinetics, and Pharmacodynamics of Intrathecally Administered ION356 in Participants With Pelizaeus Merzbacher Disease (PMD)

Who this might be right for
Pelizaeus-Merzbacher Disease
Ionis Pharmaceuticals, Inc. 24
Not applicableUnknownNCT02120235
What this trial is testing

Investigating Lysosomal Storage Diseases in Minority Groups

Who this might be right for
Lysosomal Storage DisordersGaucher DiseaseFabry Disease+2 more
O & O Alpan LLC 20,000
Large-scale testing (Phase 3)Study completedNCT02334748
What this trial is testing

Canakinumab in Patients With Systemic Juvenile Idiopathic Arthritis or Hereditary Periodic Fevers Who Participated in the CACZ885G2301E1, CACZ885G2306 or CACZ885N2301 Studies

Who this might be right for
Systemic Juvenile Idiopathic ArthritisHereditary Periodic Fevers
Novartis Pharmaceuticals 31
Not applicableEnded earlyNCT00078091
What this trial is testing

Genetics and Clinical Characteristics of Bardet-Biedl Syndrome

Who this might be right for
Bardet-Biedl Syndrome
National Human Genome Research Institute (NHGRI) 194
Testing effectiveness (Phase 2)Study completedNCT00283933
What this trial is testing

A 24-Week Safety and Pharmacodynamic Study of AT1001 (Migalastat Hydrochloride) in Participants With Fabry Disease

Who this might be right for
Fabry Disease
Amicus Therapeutics 5
Not applicableLooking for participantsNCT06796920
What this trial is testing

Epidural Spinal Cord Stimulation for Lower-limb Impairment in Adrenomyeloneuropathy

Who this might be right for
Adrenomyeloneuropathy Without Cerebral Involvement
Third Military Medical University 10
Not applicableUnknownNCT06169358
What this trial is testing

Screening Patients With Fabry Disease in Patients With Hypertrophic Cardiomyopathy or Left Ventricular Hypertrophy

Who this might be right for
Hypertrophic CardiomyopathyLeft Ventricular HypertrophyFabry Disease
Qilu Hospital of Shandong University 627
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