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Results for “Brain Diseases, Metabolic, Inborn”

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Showing 20 of 0 results

Not applicableLooking for participantsNCT01581424
What this trial is testing

Natural History and Structural Functional Relationships in Fabry Renal Disease Treatment Outcomes(Changes)in Fabry Renal Disease Study

Who this might be right for
Fabry Disease
University of Minnesota 50
Large-scale testing (Phase 3)Study completedNCT03921541
What this trial is testing

Efficacy and Safety of Pegzilarginase in Patients With Arginase 1 Deficiency

Who this might be right for
Arginase I DeficiencyHyperargininemia
Immedica Pharma AB 32
Not applicableLooking for participantsNCT06094205
What this trial is testing

Feasibility of the BrainGate2 Neural Interface System in Persons With Tetraplegia (BG-Speech-02)

Who this might be right for
AnarthriaDysarthriaTetraplegia+5 more
Leigh R. Hochberg, MD, PhD. 2
Not applicableEnded earlyNCT00260299
What this trial is testing

Dietary Cholesterol and Defects in Cholesterol Synthesis in Mevalonate Kinase Deficiency

Who this might be right for
Mevalonic AciduriaMevalonate Kinase DeficiencyImmune System Diseases+2 more
Oregon Health and Science University 1
Not applicableStudy completedNCT00515398
What this trial is testing

Evaluate the Effects of Pharmacological Chaperones in Cells From Patients With Pompe Disease

Who this might be right for
Pompe DiseaseGlycogen Storage Disease Type II
Amicus Therapeutics 30
Not applicableUnknownNCT01716741
What this trial is testing

Identification of Undiagnosed Gaucher Disease

Who this might be right for
Gaucher Disease
Brigham and Women's Hospital 100
Not applicableStudy completedNCT01632111
What this trial is testing

Pulmonary Involvement in Patients With Fabry Disease

Who this might be right for
Fabry Disease
University of Zurich 110
Testing effectiveness (Phase 2)UnknownNCT06130228
What this trial is testing

Nutritional Therapy in Late-onset Pompe Disease

Who this might be right for
Pompe DiseaseMuscle LossObesity+5 more
McMaster University 28
Testing effectiveness (Phase 2)Study completedNCT04165239
What this trial is testing

The KHENERGYZE Study

Who this might be right for
Mitochondrial DiseasesMitochondrial MyopathiesMitochondrial Encephalomyopathies+2 more
Khondrion BV 27
Not applicableUnknownNCT06116071
What this trial is testing

Biomarkers Related to Bone in Pediatric Gaucher Disease

Who this might be right for
Gaucher DiseaseBone Diseases
Lysosomal and Rare Disorders Research and Treatment Center, Inc. 20
Not applicableEnded earlyNCT00827762
What this trial is testing

Behavioral Effects of Kuvan in Children With Mild Phenylketonuria

Who this might be right for
Phenylketonuria
Washington University School of Medicine 2
Not applicableEnded earlyNCT04433728
What this trial is testing

Life With Phenylketonuria. Adult Neurological Outcome of PCU Screened Patients From 1971 to 2002.

Who this might be right for
Phenylketonurias
University Hospital, Lille 42
Not applicableStudy completedNCT03440905
What this trial is testing

Proxy-Reported Symptoms and Quality of Life Survey in Zellweger Spectrum Disorders

Who this might be right for
Zellweger Spectrum
University of South Florida 92
Testing effectiveness (Phase 2)Study completedNCT00345605
What this trial is testing

Arginine and Buphenyl in Patients With Argininosuccinic Aciduria (ASA), a Urea Cycle Disorder

Who this might be right for
Argininosuccinic AciduriaAmino Acid Metabolism, Inborn ErrorsUrea Cycle Disorders
Brendan Lee 12
Testing effectiveness (Phase 2)Study completedNCT03214588
What this trial is testing

Efficacy, Tolerability, and Pharmacokinetics of Multiple Doses of Oral TAK-831 in Adults With Friedreich Ataxia

Who this might be right for
Friedreich Ataxia
Neurocrine Biosciences 67
Large-scale testing (Phase 3)Ended earlyNCT03403205
What this trial is testing

Efficacy and Safety of ALXN1840 Administered for 48 Weeks Versus Standard of Care in Participants With Wilson Disease

Who this might be right for
Wilson Disease
Alexion Pharmaceuticals, Inc. 214
Large-scale testing (Phase 3)Study completedNCT03746522
What this trial is testing

Setmelanotide (RM-493), Melanocortin-4 Receptor (MC4R) Agonist, in Bardet-Biedl Syndrome (BBS) and Alström Syndrome (AS) Participants With Moderate to Severe Obesity

Who this might be right for
Bardet Biedl Syndrome (BBS)Alström Syndrome (AS)
Rhythm Pharmaceuticals, Inc. 52
Not applicableActive Not RecruitingNCT02327845
What this trial is testing

Phenotype, Genotype & Biomarkers in ALS and Related Disorders

Who this might be right for
Amyotrophic Lateral SclerosisFrontotemporal DementiaPrimary Lateral Sclerosis+3 more
University of Miami 708
Not applicableStudy completedNCT01378182
What this trial is testing

Efficacy of Invitro Expanded Bone Marrow Derived Allogeneic Mesenchymal Stem Cell Transplantation Via Portal Vein or Hepatic Artery or Peripheral Vein in Patients With Wilson Cirrhosis

Who this might be right for
Wilson's Disease
Murat Kantarcioglu 10
Not applicableUnknownNCT05174559
What this trial is testing

Additional Dietary Large Neutral Amino Acids (LNAA) for Improved Symptoms in Adult Classical Phenylktonuria (PKU)

Who this might be right for
Phenylketonurias
University of Southern California 10
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