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Results for “Inherited Metabolic Disorder of Nervous System”

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Showing 20 of 0 results

Large-scale testing (Phase 3)WithdrawnNCT00935753
What this trial is testing

Trial of Kuvan in Lesch-Nyhan Disease

Who this might be right for
Behavioral Manifestations of Lesch-Nyhan Disease
University of California, San Diego
Very early researchUnknownNCT06249581
What this trial is testing

Pharmacokinetics of AUX-001 40mg Once-daily in Healthy Subjects Under Fasting and Fed Conditions

Who this might be right for
Chronic Stable Angina
Auxilius Pharma sp.z.o.o. 16
Not applicableUnknownNCT04956965
What this trial is testing

High Resolution Three-dimensional Maps of the Right Chambers in Patient Diagnosed With Cardiac Amyloidosis

Who this might be right for
Cardiac AmyloidosisCardiac Disease
University Hospital Center of Martinique 24
Not applicableLooking for participantsNCT05943002
What this trial is testing

Patient-reported, Health Economic and Psychosocial Outcomes in Friedreich Ataxia

Who this might be right for
Friedreich Ataxia
German Center for Neurodegenerative Diseases (DZNE) 200
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 completedNCT04522245
What this trial is testing

Evaluation of Brain Activity Changes After a Behavior Change Weight Loss Intervention

Who this might be right for
ObesityBehavior, HealthWeight Loss
Noom Inc. 70
Post-approval studies (Phase 4)Study completedNCT00455195
What this trial is testing

Late-Onset Treatment Study Extension Protocol

Who this might be right for
Pompe Disease (Late-Onset)Glycogen Storage Disease Type II (GSD-II)Glycogenesis Type II+1 more
Genzyme, a Sanofi Company 81
Large-scale testing (Phase 3)Looking for participantsNCT06451757
What this trial is testing

KHENERFIN Study: A Trial to Evaluate the Efficacy and Safety of Sonlicromanol in Primary Mitochondrial Diseases

Who this might be right for
Mitochondrial DiseasesMaternally Inherited Diabetes and Deafness (MIDD)Mitochondrial Encephalomyopathy, Lactic Acidosis and Stroke-like Episodes (MELAS)+1 more
Khondrion BV 220
Testing effectiveness (Phase 2)Study completedNCT00250939
What this trial is testing

RhGAA 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 5
Testing effectiveness (Phase 2)Study completedNCT00004488
What this trial is testing

Phase II Randomized Study of Alendronate Sodium for Osteopenia in Patients With Gaucher's Disease

Who this might be right for
Gaucher's DiseaseOsteopenia
Children's Hospital Medical Center, Cincinnati 82
Not applicableStudy completedNCT03939052
What this trial is testing

Protein Requirements in Adults With Phenylketonuria (PKU)

Who this might be right for
Phenylketonuria
University of British Columbia 6
Early research (Phase 1)Study completedNCT02939547
What this trial is testing

Study of the Pharmacokinetics of Trappsol and Effects on Potential Biomarkers of Niemann-Pick C1 (NPC1)

Who this might be right for
Niemann-Pick Disease, Type C1
Cyclo Therapeutics, Inc. 13
Not applicableStudy completedNCT03631238
What this trial is testing

The Dual Impact of Homocysteine and Cholesterol on Cognitive Functions

Who this might be right for
HypercholesterolemiaCognitive DysfunctionHyperhomocysteinemia
Assiut University 41
Not applicableEnrolling By InvitationNCT06763549
What this trial is testing

COR-INSIGHT: Optimizing Cardiovascular and Cardiopulmonary Outcomes with AI-Driven Multiplexed Indications Using COR ECG Wearable

Who this might be right for
Cardiopulmonary FailureMyocardial Infarction (MI)Heart Decompensation+38 more
Peerbridge Health, Inc 15,000
Large-scale testing (Phase 3)Active Not RecruitingNCT05515536
What this trial is testing

Assess the Safety and Efficacy of Vatiquinone in Participants With Friedreich Ataxia

Who this might be right for
Friedreich Ataxia
PTC Therapeutics 130
Not applicableUnknownNCT05448716
What this trial is testing

Left Atrial Strain and Supraventricular Arrhythmia Burden in Cardiac Light Chain Amyloidosis Following Chemotherapy

Who this might be right for
Supraventricular ArrhythmiaLeft Atrial AbnormalityAmyloid Cardiomyopathy+1 more
Istanbul University - Cerrahpasa 30
Not applicableStudy completedNCT03847753
What this trial is testing

Exploring the Comorbidity Between Mental Disorders and General Medical Conditions

Who this might be right for
Organic, Including Symptomatic, Mental DisordersSubstance UseSchizophrenia+38 more
University of Aarhus 5,940,299
Not applicableNot Yet RecruitingNCT04484051
What this trial is testing

Growth Hormone Study in Adults With Prader-Willi Syndrome

Who this might be right for
Prader-Willi Syndrome
Erasmus Medical Center 25
Not applicableStudy completedNCT03864029
What this trial is testing

Retrospective Observational Safety Effectiveness With Kuvan in hpA

Who this might be right for
Tetrahydrobiopterin Deficiency
BioMarin Pharmaceutical 26
Testing effectiveness (Phase 2)Study completedNCT03274856
What this trial is testing

GLWL-01 in Patients With Prader-Willi Syndrome

Who this might be right for
Prader-Willi Syndrome
GLWL Research Inc. 19
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