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Results for “Metabolic Inborn Brain Diseases”

1,542 trials

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Showing 20 of 1,542 results

Not applicableStudy completedNCT02851862
What this trial is testing

A Natural History of Late Onset Tay-Sachs Disease

Who this might be right for
GM2 Gangliosidosis
Massachusetts General Hospital 10
Testing effectiveness (Phase 2)Study completedNCT00283959
What this trial is testing

A 12-Week Safety and Pharmacodynamic Study of AT1001 (Migalastat Hydrochloride) in Participants With Fabry Disease

Who this might be right for
Fabry Disease
Amicus Therapeutics 4
Testing effectiveness (Phase 2)Study completedNCT03580122
What this trial is testing

The Effect of Arginine on Classic Galactosemia

Who this might be right for
Classic Galactosemia
Academisch Ziekenhuis Maastricht 4
Not applicableUnknownNCT04388969
What this trial is testing

World Data on Ambroxol for Patients With GD and GBA Related PD

Who this might be right for
Gaucher DiseaseParkinson DiseaseGBA Gene Mutation
Shaare Zedek Medical Center 300
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 applicableStudy completedNCT04353466
What this trial is testing

Assessing the Impact of Elelyso on Bone Involvement Currently Treated With Other ERTs

Who this might be right for
Gaucher Disease, Type 1
Shaare Zedek Medical Center 30
Not applicableWithdrawnNCT02385162
What this trial is testing

Biomarker for Glycogen Storage Diseases (BioGlycogen)

Who this might be right for
Fructose Metabolism, Inborn ErrorsGlycogen Storage DiseaseGlycogen Storage Disease Type I+7 more
CENTOGENE GmbH Rostock
Testing effectiveness (Phase 2)Study completedNCT00925054
What this trial is testing

Dose-Finding Study to Evaluate the Safety, Efficacy, & Tolerability of Multiple Doses of rAvPAL-PEG in Subjects With PKU

Who this might be right for
Phenylketonuria
BioMarin Pharmaceutical 40
Large-scale testing (Phase 3)Study completedNCT02319005
What this trial is testing

ENDEAVOUR: Phase 3 Multicenter Study of Revusiran (ALN-TTRSC) in Patients With Transthyretin (TTR) Mediated Familial Amyloidotic Cardiomyopathy (FAC)

Who this might be right for
Transthyretin (TTR) Mediated Familial Amyloidotic Cardiomyopathy (FAC)Amyloidosis, HereditaryAmyloid Neuropathies, Familial+3 more
Alnylam Pharmaceuticals 206
Testing effectiveness (Phase 2)Ended earlyNCT01195753
What this trial is testing

Human Heterologous Liver Cells for Infusion in Children With Urea Cycle Disorders

Who this might be right for
Urea Cycle Disorders
Cytonet GmbH & Co. KG 10
Large-scale testing (Phase 3)Study completedNCT00697073
What this trial is testing

Study to Assess the Safety and Tolerability of Idebenone in the Treatment of Friedreich's Ataxia Patients

Who this might be right for
Friedreich's Ataxia
Santhera Pharmaceuticals 68
Not applicableNot Yet RecruitingNCT06258577
What this trial is testing

Screening for Gaucher Disease and Acid Sphingomyelinase Deficiency

Who this might be right for
Gaucher Disease
Chung-Hsing Wang 50
Large-scale testing (Phase 3)Study completedNCT01422187
What this trial is testing

Extension Study of Taliglucerase Alfa in Adult Subjects With Gaucher Disease

Who this might be right for
Gaucher Disease
Pfizer 19
Early research (Phase 1)WithdrawnNCT01787578
What this trial is testing

Safety and Pharmacodynamic Study of Sobetirome in X-Linked Adrenoleukodystrophy (X-ALD)

Who this might be right for
X-Linked AdrenoleukodystrophyAdrenomyeloneuropathy
Thomas S. Scanlan
Not applicableNo Longer AvailableNCT02084121
What this trial is testing

Allogeneic Stem Cell Transplantation for the Treatment of Multiple Sclerosis (Compassionate Use)

Who this might be right for
Metachromatic Leukodystrophy
Talaris Therapeutics Inc.
Not applicableStudy completedNCT01931644
What this trial is testing

At-Home Research Study for Patients With Autoimmune, Inflammatory, Genetic, Hematological, Infectious, Neurological, CNS, Oncological, Respiratory, Metabolic Conditions

Who this might be right for
All Diagnosed Health ConditionsADD/ADHDAlopecia Areata+58 more
Sanguine Biosciences 17,667
Early research (Phase 1)Not Yet RecruitingNCT06735638
What this trial is testing

Outside the Cage (OTC) Robotic Esophagectomy

Who this might be right for
Esophageal Cancer
Centre hospitalier de l'Université de Montréal (CHUM) 31
Not applicableStudy completedNCT02719249
What this trial is testing

Fabry Disease Screening in ESRD Patients in West of France

Who this might be right for
ESRD
Rennes University Hospital 500
Testing effectiveness (Phase 2)Study completedNCT00924703
What this trial is testing

Long-Term Extension of Previous rAvPAL-PEG Protocols in Subjects With PKU (PAL-003)

Who this might be right for
Phenylketonuria
BioMarin Pharmaceutical 68
Early research (Phase 1)Not Yet RecruitingNCT06772870
What this trial is testing

A Single Ascending Dose Study of DT-216P2 in Normal Healthy Participants

Who this might be right for
Friedreich Ataxia
Design Therapeutics, Inc. 36
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