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Results for “Metabolic Inborn Brain Diseases”

1,590 trials

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Showing 20 of 1,590 results

Testing effectiveness (Phase 2)Study completedNCT01603446
What this trial is testing

L-arginine Therapy on Endothelium-dependent Vasodilation & Mitochondrial Metabolism in MELAS Syndrome

Who this might be right for
MELAS Syndrome
The Hospital for Sick Children 7
Testing effectiveness (Phase 2)Not Yet RecruitingNCT07773246
What this trial is testing

Phase 1/2 Study of KRRO-121 in Healthy Volunteers and Patients With UCD

Who this might be right for
Urea Cycle Disorders, InbornUrea Cycle DisordersOrnithine Transcarbamylase (OTC) Deficiency+8 more
Korro Bio, Inc. 30
Not applicableStudy completedNCT00343577
What this trial is testing

Antiproteinuric Agents and Fabry Disease

Who this might be right for
Fabry DiseaseProteinuria
University of Alabama at Birmingham 12
Not applicableStudy completedNCT04997772
What this trial is testing

Digital Health Platform Customized for Patients With Gaucher Disease

Who this might be right for
Gaucher DiseasePatient Reported Outcome
Shoshana Vilk 80
Post-approval studies (Phase 4)Study completedNCT04801303
What this trial is testing

Evaluation of the Effects of Calcitriol's in the Neurological Symptoms of Friedreich's Ataxia Patients

Who this might be right for
Friedreich Ataxia
Berta Alemany 20
Testing effectiveness (Phase 2)Study completedNCT01102686
What this trial is testing

Pyrimethamine as a Treatment for Late-Onset GM2-gangliosidosis (Tay-Sachs and Sandhoff Disease)

Who this might be right for
Gangliosidoses, GM2Sandhoff DiseaseTay-Sachs Disease
The Hospital for Sick Children 20
Testing effectiveness (Phase 2)Ended earlyNCT04273269
What this trial is testing

A Safety and Efficacy Study of LYS-GM101 Gene Therapy in Patients With GM1 Gangliosidosis

Who this might be right for
GM1 Gangliosidosis
LYSOGENE 5
Not applicableStudy completedNCT00351156
What this trial is testing

Study to Evaluate Blood Cell Lines From Patients With Gaucher Disease

Who this might be right for
Gaucher Disease
Amicus Therapeutics 50
Early research (Phase 1)Study completedNCT02422654
What this trial is testing

Taste Evaluation of Different Liquid Formulations With Eliglustat

Who this might be right for
Gaucher Disease
Genzyme, a Sanofi Company 8
Not applicableStudy completedNCT04649866
What this trial is testing

Coronary Artery Disease in Patients With Friedreich's Ataxia

Who this might be right for
Friedreich Ataxia
University of Pittsburgh 7
Not applicableStudy completedNCT02468570
What this trial is testing

Substudy to Evaluate Executive Function in Adults With PKU Who Are Participating in the Phase 3 Study, 165-302

Who this might be right for
Phenylketonuria
BioMarin Pharmaceutical 9
Not applicableStudy completedNCT05330039
What this trial is testing

Characterization of Intestinal Microbiota in Children With Inborn Errors of Metabolism (IEM)

Who this might be right for
Inborn Errors of MetabolismPropionic AciduriaMethylmalonic Aciduria+1 more
Emma Marie Caroline Slack 22
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
Large-scale testing (Phase 3)Study completedNCT01994889
What this trial is testing

Safety and Efficacy of Tafamidis in Patients With Transthyretin Cardiomyopathy

Who this might be right for
Transthyretin (TTR) Amyloid Cardiomyopathy
Pfizer 441
Not applicableEnded earlyNCT01806051
What this trial is testing

A Pilot Study on Diurnal Variation

Who this might be right for
Phenylketonuria (PKU)
Dr. Linda Randolph 6
Not applicableLooking for participantsNCT05473637
What this trial is testing

Taiwan Associated Genetic and Nongenetic Small Vessel Disease

Who this might be right for
Cerebral Small Vessel DiseasesCadasilHTRA1-Related Autosomal Dominant Cerebral Angiopathy+5 more
National Taiwan University Hospital 500
Testing effectiveness (Phase 2)Study completedNCT02675465
What this trial is testing

First-In-Human Study to Evaluate Safety, Tolerability, and PK of Intravenous ATB200 Alone and When Co-Administered With Oral AT2221

Who this might be right for
Pompe Disease
Amicus Therapeutics 29
Not applicableLooking for participantsNCT06969209
What this trial is testing

Brain Aging in Phenylketonuria

Who this might be right for
Phenylketonuria (PKU)
Insel Gruppe AG, University Hospital Bern 90
Not applicableStudy completedNCT03168399
What this trial is testing

Evaluation of PKU Explore

Who this might be right for
PhenylketonuriaInborn Errors of Metabolism
Vitaflo International, Ltd 22
Large-scale testing (Phase 3)Study completedNCT00158600
What this trial is testing

A Placebo-Controlled Study of Safety and Effectiveness of Myozyme (Alglucosidase Alfa) in Patients With Late-Onset Pompe Disease

Who this might be right for
Pompe Disease (Late-onset)Glycogen Storage Disease Type II (GSD-II)Acid Maltase Deficiency Disease+1 more
Genzyme, a Sanofi Company 90
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