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Results for “Brain Diseases, Metabolic, Inborn”

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Phase
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Status
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Active, Not Recruiting
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Enrolling by Invitation

Showing 20 of 0 results

Not applicableStudy completedNCT04408300
What this trial is testing

Study of Retinal Vascular Parameters in Patients With Wilson's Disease

Who this might be right for
Wilson Disease
Fondation Ophtalmologique Adolphe de Rothschild 124
Not applicableStudy completedNCT01869972
What this trial is testing

Biological Variation of Phenylalanine in Patients With Hyperphenylalaninemia

Who this might be right for
PhenylketonuriaHyperphenylalaninemia
Hamilton Health Sciences Corporation 32
Not applicableEnrolling By InvitationNCT06880250
What this trial is testing

Efficacy and Safety of Enzyme Replacement Therapy in Patients With Fabry Disease

Who this might be right for
Fabry Disease
NPO Petrovax 100
Not applicableStudy completedNCT04084301
What this trial is testing

Impact of Cardiopulmonary Bypass Flow on Renal Oxygenation, Blood Flow and Tubular Injury

Who this might be right for
Extracorporeal Circulation; ComplicationsRenal FailureCirculation Disorder+4 more
Sahlgrenska University Hospital 100
Early research (Phase 1)Study completedNCT01489995
What this trial is testing

Migalastat Food Effect Study

Who this might be right for
Fabry Disease
Amicus Therapeutics 20
Testing effectiveness (Phase 2)Study completedNCT03759639
What this trial is testing

N-Acetyl-L-Leucine for Niemann-Pick Disease, Type C (NPC)

Who this might be right for
Niemann-Pick Disease, Type C
IntraBio Inc 33
Testing effectiveness (Phase 2)WithdrawnNCT04094948
What this trial is testing

Phase II Clinical Trial of Clenbuterol in Adult Patients With Pompe Disease

Who this might be right for
Pompe Disease (Late-onset)
Duke University
Not applicableUnknownNCT04639999
What this trial is testing

Impact of MIgalastat TheRApy on CaRdiac Function in patiEnts With Fabry's Cardiomyopathy (MIRACRE-Fabry Trial)

Who this might be right for
Fabry Disease
Yonsei University 20
Testing effectiveness (Phase 2)Study completedNCT00947544
What this trial is testing

Study of the Safety and Tolerability of HPN-100 Compared to Sodium Phenylbutyrate in Children With Urea Cycle Disorders

Who this might be right for
Urea Cycle Disorders
Amgen 17
Not applicableStudy completedNCT00006057
What this trial is testing

Diagnostic and Screening Study of Genetic Disorders

Who this might be right for
Tay-Sachs DiseasePorphyria, ErythropoieticLeukodystrophy, Globoid Cell+1 more
National Center for Research Resources (NCRR) 50
Large-scale testing (Phase 3)Study completedNCT00811681
What this trial is testing

Effect of Pioglitazone Administered to Patients With Friedreich's Ataxia: Proof of Concept

Who this might be right for
Friedreich's Ataxia
Assistance Publique - Hôpitaux de Paris 40
Testing effectiveness (Phase 2)Study completedNCT00048906
What this trial is testing

Alpha-Galactosidase A Replacement Therapy for Fabry Disease

Who this might be right for
Fabry Disease
National Institute of Neurological Disorders and Stroke (NINDS) 3
Early research (Phase 1)Study completedNCT03952234
What this trial is testing

L-Citrulline Dose Finding Safety Study in MELAS

Who this might be right for
MELAS Syndrome
Baylor College of Medicine 10
Testing effectiveness (Phase 2)Looking for participantsNCT03406611
What this trial is testing

Pegtibatinase as a Treatment for Patients With Classical Homocystinuria (HCU) (Also Known as the COMPOSE Study)

Who this might be right for
Homocystinuria
Travere Therapeutics, Inc. 39
Not applicableNot Yet RecruitingNCT07406009
What this trial is testing

The Psychosocial Functioning of Adults With Phenylketonuria.

Who this might be right for
Phenylketonuria (PKU)
Central Hospital, Nancy, France 30
Testing effectiveness (Phase 2)UnknownNCT05710367
What this trial is testing

Effects Of Sodium Glucose Cotranspoter 2 Inhibitors On Heart And Kidneys In Fabry Disease Patients

Who this might be right for
Fabry Disease
Albina Nowak, MD 46
Large-scale testing (Phase 3)Study completedNCT01732471
What this trial is testing

Phase 3 Open-label Study to Evaluate the Response and Safety of Kuvan® in Subjects With Phenylketonuria

Who this might be right for
Phenylketonuria
Merck KGaA, Darmstadt, Germany 90
Large-scale testing (Phase 3)Study completedNCT03614234
What this trial is testing

Open Label Extension of 2 mg/kg Pegunigalsidase Alfa (PRX-102) Every 4 Weeks in Adult Fabry Disease Patients

Who this might be right for
Fabry Disease
Chiesi Farmaceutici S.p.A. 29
Not applicableStudy completedNCT04907162
What this trial is testing

Musculoskeletal Nociceptive Pain in Participants With Neuromuscular Disorders

Who this might be right for
Pompe Disease (Late-onset)Myotonic Dystrophy Type 1 (DM1)Myotonic Dystrophy Type 2+4 more
LMU Klinikum 82
Not applicableLooking for participantsNCT01532791
What this trial is testing

Natural History Study - Mitochondrial Disease

Who this might be right for
MELAS or m.3243 A>G Mitochondrial DNA Mutation Carrier
Columbia University 300
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