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Results for “Brain Diseases, Metabolic, Inborn”

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Phase
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Showing 20 of 0 results

Testing effectiveness (Phase 2)Ended earlyNCT04145037
What this trial is testing

Lentiviral Vector Gene Therapy - The Guard1 Trial of AVR-RD-02 for Subjects With Type 1 Gaucher Disease

Who this might be right for
Gaucher Disease
AVROBIO 8
Testing effectiveness (Phase 2)Study completedNCT00467935
What this trial is testing

Scotoma Reduction in AMD Patients Treated With Ranibizumab

Who this might be right for
Macular Degeneration
Retina Research Foundation 20
Not applicableUnknownNCT03590548
What this trial is testing

Clinical Audit of Managment of Gausher Disease in Children

Who this might be right for
Gaucher Disease
Yasmeen Abd Elbaset Hamzaa 1
Large-scale testing (Phase 3)Study completedNCT05218655
What this trial is testing

A Safety Study for Previously Treated Vatiquinone (PTC743) Participants With Inherited Mitochondrial Disease

Who this might be right for
Inherited Mitochondrial Disease
PTC Therapeutics 101
Not applicableEnded earlyNCT06308718
What this trial is testing

Long-term Follow-up Study to Evaluate Safety and Efficacy of FBX-101 in Krabbe Patients

Who this might be right for
Krabbe Disease
Forge Biologics, Inc 2
Not applicableStudy completedNCT02952482
What this trial is testing

Newborn Screening for Adrenoleukodystrophy

Who this might be right for
Adrenoleukodystrophy
National Taiwan University Hospital 45,796
Not applicableUnknownNCT05301218
What this trial is testing

CAlcified Lesion Intervention Planning Steered by OCT.

Who this might be right for
Coronary Artery CalcificationAngioplasty
Institut Mutualiste Montsouris 140
Testing effectiveness (Phase 2)Ended earlyNCT00004418
What this trial is testing

Effect of Glycerol Trierucate on Clinical Course of Adrenoleukodystrophy

Who this might be right for
Adrenoleukodystrophy
Hugo W. Moser Research Institute at Kennedy Krieger, Inc. 126
Early research (Phase 1)Ended earlyNCT00875160
What this trial is testing

A Study in Type 1 Gaucher Patients to Evaluate the Pharmacokinetics, Safety and Pharmacodynamics of AT2101

Who this might be right for
Type 1 Gaucher Disease
Amicus Therapeutics 1
Not applicableNot Yet RecruitingNCT07671859
What this trial is testing

PKU Microtablets Case Studies

Who this might be right for
Phenylketonuria (PKU)
Nutricia UK Ltd 20
Not applicableStudy completedNCT03519646
What this trial is testing

Eliglustat on Gaucher Disease Type IIIB

Who this might be right for
Gaucher Disease, Type III
National Taiwan University Hospital 4
Testing effectiveness (Phase 2)Study completedNCT01603446
What this trial is testing

L-arginine Therapy on Endothelium-dependent Vasodilation & Mitochondrial Metabolism in MELAS Syndrome

Who this might be right for
MELAS Syndrome
The Hospital for Sick Children 7
Testing effectiveness (Phase 2)Study completedNCT01685216
What this trial is testing

Efficacy and Safety Study of Velaglucerase Alfa in Children and Adolescents With Type 3 Gaucher Disease

Who this might be right for
Gaucher Disease, Type 3
Shire 7
Not applicableStudy completedNCT01924026
What this trial is testing

Neurocognitive Outcomes in Mild Hyperphenylalaninemia (MHP)MHP Study

Who this might be right for
PhenylketonuriaMild Hyperphenylalaninemia
The Hospital for Sick Children 10
Testing effectiveness (Phase 2)Study completedNCT00433147
What this trial is testing

AT2101 (Afegostat Tartrate) in Adult Patients With Type 1 Gaucher Disease Currently Receiving Enzyme Replacement Therapy

Who this might be right for
Gaucher Disease, Type 1Type 1 Gaucher DiseaseGaucher Disease
Amicus Therapeutics 30
Not applicableStudy completedNCT01325025
What this trial is testing

Imaging Study of the White Matter Lesions in Children With Metachromatic Leucodystrophy

Who this might be right for
Late Infantile Metachromatic Leukodystrophy
Assistance Publique - Hôpitaux de Paris 29
Testing effectiveness (Phase 2)Study completedNCT03917225
What this trial is testing

A Clinical Study to Evaluate the Effect of MIN-102 on the Progression of Friedreich's Ataxia in Male and Female Patients

Who this might be right for
Friedreich Ataxia
Minoryx Therapeutics, S.L. 39
Testing effectiveness (Phase 2)Active Not RecruitingNCT02843035
What this trial is testing

Venglustat in Combination With Cerezyme in Adult Patients With Gaucher Disease Type 3 With Venglustat Monotherapy Extension

Who this might be right for
Gaucher Disease Type 1Gaucher Disease Type 3
Genzyme, a Sanofi Company 12
Testing effectiveness (Phase 2)Study completedNCT04604548
What this trial is testing

The KHENEREXT Study

Who this might be right for
Mitochondrial DiseasesMitochondrial DNA tRNALeu(UUR) m.3243A<G MutationMaternally Inherited Diabetes and Deafness (MIDD)+2 more
Khondrion BV 11
Not applicableAvailableNCT07487389
What this trial is testing

Zilganersen Expanded Access Program for Individuals With Alexander Disease

Who this might be right for
Alexander Disease
Ionis Pharmaceuticals, Inc.
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