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Results for “Brain Diseases, Metabolic, Inborn”

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Showing 20 of 0 results

Not applicableWithdrawnNCT06560346
What this trial is testing

Understanding the Natural History Early in the Course or Presentation of Friedreich Ataxia

Who this might be right for
Friedreich AtaxiaRare Diseases
Friedreich's Ataxia Research Alliance
Not applicableEnded earlyNCT05004701
What this trial is testing

Impact of the Lockdown Due to the COVID Pandemic in French Wilson's Disease Patients

Who this might be right for
Wilson's Disease and COVID-19
Fondation Ophtalmologique Adolphe de Rothschild 120
Testing effectiveness (Phase 2)Ended earlyNCT00526071
What this trial is testing

Open-label Long-term Safety Study of AT1001 (Migalastat Hydrochloride) in Participants With Fabry Disease Who Have Completed a Previous AT1001 Study

Who this might be right for
Fabry Disease
Amicus Therapeutics 23
Post-approval studies (Phase 4)Looking for participantsNCT06875245
What this trial is testing

Comparing Efficacy of 8-Week and 12-Week Faricimab Initial Follow-Up Treatment Intervals

Who this might be right for
Wet Age-related Macular Degeneration
Faculty Hospital Kralovske Vinohrady 100
Not applicableLooking for participantsNCT07521930
What this trial is testing

Interfacing With NeuroTechnology to Expand Neural Throughput (INTENT)

Who this might be right for
Tetraplegia/TetraparesisAmyotrophic Lateral Sclerosis (ALS)Muscular Disorders, Atrophic+6 more
Johns Hopkins University 5
Not applicableLooking for participantsNCT07478172
What this trial is testing

Effects of Whole-body Electrical Muscle Stimulation Exercise on Adults With Neuromuscular Disease

Who this might be right for
Neuromuscular Diseases (NMD)Amyotrophic Lateral SclerosisMyasthenia Gravis+15 more
University of Missouri-Columbia 50
Not applicableStudy completedNCT01870375
What this trial is testing

Longitudinal Studies of Brain Structure and Function in MPS Disorders

Who this might be right for
Mucopolysaccharidosis Type IMucopolysaccharidosis Type IIMucopolysaccharidosis Type VI+2 more
University of Minnesota 100
Testing effectiveness (Phase 2)Ended earlyNCT00668564
What this trial is testing

Hematopoietic Stem Cell Transplantation (HCT) for Inborn Errors of Metabolism

Who this might be right for
Hurler's SyndromeMaroteaux-Lamy SyndromeSly Syndrome+8 more
Masonic Cancer Center, University of Minnesota 18
Not applicableLooking for participantsNCT02497534
What this trial is testing

Biomarkers in Friedreich's Ataxia

Who this might be right for
Friedreich's Ataxia
University of Florida 203
Large-scale testing (Phase 3)Study completedNCT00656409
What this trial is testing

Conjugate Pneumococcal Vaccine in Ataxia Telangiectasia (AT)

Who this might be right for
Ataxia Telangiectasia (AT)
Institute of Child Health 30
Testing effectiveness (Phase 2)Enrolling By InvitationNCT04833907
What this trial is testing

RAAV-Olig001-ASPA Gene Therapy for Treatment of Children With Typical Canavan Disease

Who this might be right for
Canavan Disease
Myrtelle Inc. 24
Large-scale testing (Phase 3)Study completedNCT06193200
What this trial is testing

Evaluate the Neurological Effects of EryDex on Subjects With A-T

Who this might be right for
Ataxia Telangiectasia
Quince Therapeutics S.p.A. 105
Not applicableNot Yet RecruitingNCT05911919
What this trial is testing

Validation of a Prognostic Biomarker Using Brain Diffusion MRI in X-linked Adrenoleukodystrophy

Who this might be right for
X-linked Adrenoleukodystrophy
Assistance Publique - Hôpitaux de Paris 200
Early research (Phase 1)Study completedNCT04519567
What this trial is testing

Multiple Ascending Dose Study of CTI-1601 Versus Placebo in Subjects With Friedreich's Ataxia

Who this might be right for
Friedreich Ataxia
Larimar Therapeutics, Inc. 27
Not applicableLooking for participantsNCT04896281
What this trial is testing

Phenylalanine-free Diet for Patients With Secondary Hyperphenylalaninemia in ICU

Who this might be right for
Non-Phenylketonuric Hyperphenylalaninemia
Chang Gung Memorial Hospital 100
Testing effectiveness (Phase 2)Temporarily pausedNCT04015557
What this trial is testing

Effect of Acetaminophen and N-Acetylcysteine on Liver Metabolism on Homocystinuria

Who this might be right for
CBS Deficiency
Hospital de Clinicas de Porto Alegre 10
Not applicableUnknownNCT05904366
What this trial is testing

Magnetic Resonance Spectroscopy in Acid Sphingomyelinase Deficiency

Who this might be right for
Asmd, Visceral Type
Eline C. B. Eskes 34
Not applicableUnknownNCT06332105
What this trial is testing

Evaluation of Low Phenylalanine Formulas

Who this might be right for
Phenylketonurias
Ajinomoto Co., Inc. 19
Not applicableLooking for participantsNCT05493605
What this trial is testing

Cardiac Involvement in Wilson's Disease

Who this might be right for
Wilson's Disease
Fondation Ophtalmologique Adolphe de Rothschild 150
Not applicableUnknownNCT06179394
What this trial is testing

Evaluation of Cognitive Dysfunction and Psychiatric Comorbidities

Who this might be right for
Wilson Disease
Assiut University 40
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