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Results for “Brain Diseases, Metabolic, Inborn”

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Showing 20 of 0 results

Not applicableLooking for participantsNCT04806620
What this trial is testing

Unhide® Project: A Digital Health Platform to Collect Lifestyle Data for Brain Inflammation Research

Who this might be right for
Post-Acute COVID-19 SyndromeME/CFSRheumatic Arthritis+41 more
Brain Inflammation Collaborative 10,000
Not applicableUnknownNCT03786497
What this trial is testing

Protecting Brains and Saving Futures - the PBSF Protocol

Who this might be right for
Brain InjuriesCongenital Heart DiseaseNewborn Morbidity+14 more
Protecting Brains Saving Futures 2,268
Not applicableLooking for participantsNCT06523582
What this trial is testing

Genetic Bases of Neuroendocrine Neoplasms in Mexican Patients

Who this might be right for
Neuroendocrine NeoplasmNeuroendocrine Neoplasm of Gastrointestinal TractNeuroendocrine Neoplasm of Lung+23 more
Universidad Nacional Autonoma de Mexico 750
Testing effectiveness (Phase 2)WithdrawnNCT03767270
What this trial is testing

Safety, Tolerability and PK/PD Evaluation of Intravenous Administration of MRT5201 in Patients With OTC Deficiency

Who this might be right for
Ornithine Transcarbamylase Deficiency
Translate Bio, Inc.
Post-approval studies (Phase 4)Study completedNCT04718779
What this trial is testing

Enzyme Replacement Therapy (VPRIV) in People With Type 1 Gaucher Disease Who Were Previously Treated With Substrate Reduction Therapy

Who this might be right for
Gaucher Disease
Takeda 4
Large-scale testing (Phase 3)Study completedNCT01298141
What this trial is testing

Open-Label Treatment Protocol to Observe the Safety of Replagal (Agalsidase Alfa) Enzyme Replacement Therapy in Canadian Patients With Fabry Disease

Who this might be right for
Fabry Disease
Shire 171
Not applicableLooking for participantsNCT00912041
What this trial is testing

BrainGate2: Feasibility Study of an Intracortical Neural Interface System for Persons With Tetraplegia

Who this might be right for
TetraplegiaSpinal Cord InjuriesAmyotrophic Lateral Sclerosis+3 more
Leigh R. Hochberg, MD, PhD. 27
Post-approval studies (Phase 4)Study completedNCT00081497
What this trial is testing

The Safety and Efficacy of Fabrazyme in Patients With Fabry Disease

Who this might be right for
Fabry Disease
Genzyme, a Sanofi Company 67
Testing effectiveness (Phase 2)Study completedNCT00446550
What this trial is testing

Oral AT2101 (Afegostat Tartrate) in Treatment-naive Patients With Gaucher Disease

Who this might be right for
Gaucher DiseaseType 1 Gaucher DiseaseGaucher Disease, Type 1
Amicus Therapeutics 19
Not applicableStudy completedNCT04141891
What this trial is testing

Advancing Understanding of Transportation Options

Who this might be right for
Diabetic RetinopathyMacular DegenerationGlaucoma+29 more
University of Colorado, Denver 529
Not applicableLooking for participantsNCT01780168
What this trial is testing

The NIH MINI Study: Metabolism, Infection, and Immunity in Inborn Errors of Metabolism

Who this might be right for
Oxidative Phosphorylation DeficienciesElectron Transport Chain Disorders, MitochondrialMitochondrial Disorders+1 more
National Human Genome Research Institute (NHGRI) 500
Not applicableNot Yet RecruitingNCT07173010
What this trial is testing

Pediatric Arthropathy Beyond Inflammation: Clinical Spectrum and Diagnostic Approach at Assiut University Children Hospital

Who this might be right for
MucopolysaccharidosesProgressive Pseudorheumatoid DysplasiaFarber Disease+3 more
Assiut University 35
Testing effectiveness (Phase 2)Study completedNCT01855360
What this trial is testing

Doxycycline and TUDCA in Patients With Transthyretin Amyloid Cardiomyopathy

Who this might be right for
Amyloidosis; Heart (Manifestation)Senile Cardiac Amyloidosis
Brigham and Women's Hospital 40
Not applicableStudy completedNCT00001289
What this trial is testing

Effects of Enzyme Replacement in Gaucher's Disease

Who this might be right for
Gaucher's Disease
National Institute of Neurological Disorders and Stroke (NINDS) 70
Large-scale testing (Phase 3)Study completedNCT01614574
What this trial is testing

Study of Velaglucerase Alfa Enzyme Replacement Therapy in Japanese Patients With Gaucher Disease

Who this might be right for
Gaucher Disease
Shire 6
Not applicableStudy completedNCT04470713
What this trial is testing

Natural History Study for Pediatric Patients With Early Onset of Either GM1 Gangliosidosis, GM2 Gangliosidoses, or Gaucher Disease Type 2

Who this might be right for
GM1 GangliosidosisGM2 GangliosidosisGaucher Disease, Type 2+3 more
Idorsia Pharmaceuticals Ltd. 226
Large-scale testing (Phase 3)Looking for participantsNCT06628128
What this trial is testing

A Long-Term Study of JNT-517 in Participants With Phenylketonuria

Who this might be right for
Phenylketonuria (PKU)
Otsuka Pharmaceutical Development & Commercialization, Inc. 240
Not applicableActive Not RecruitingNCT05526664
What this trial is testing

Omics Gaucher Study: Multiomic Approach

Who this might be right for
Gaucher Disease
CENTOGENE GmbH Rostock 25
Post-approval studies (Phase 4)Study completedNCT04656600
What this trial is testing

Study to Evaluate Efficacy and Safety of Imiglucerase Treatment in Chinese Patients With Gaucher Disease Type Ⅲ

Who this might be right for
Gaucher's Disease
Sanofi 12
Post-approval studies (Phase 4)Study completedNCT06019728
What this trial is testing

A Prospective Study to Investigate Safety and Tolerability of Shorter Infusion of Fabrazyme

Who this might be right for
Fabry's Disease
Sanofi 8
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