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Results for “Brain Diseases, Metabolic, Inborn”

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Showing 20 of 0 results

Not applicableNot Yet RecruitingNCT07802366
What this trial is testing

Acceptability and Tolerance Market Research of a Slow Release, Phenylalanine-free Protein Substitute for the Dietary Management of Phenylketonuria

Who this might be right for
Phenylketonuria (PKU) and HyperphenylalaninemiaBrain Diseases, Metabolic, InbornBrain Disorder, Metabolic+10 more
metaX Institut fuer Diatetik GmbH 10
Testing effectiveness (Phase 2)WithdrawnNCT02715505
What this trial is testing

Safety and Exploratory Efficacy of HSC835 in Patients With Inherited Metabolic Disorders (IMD)

Who this might be right for
Inherited Metabolic Disorders IMD
Novartis Pharmaceuticals
Testing effectiveness (Phase 2)Study completedNCT00813865
What this trial is testing

A Long-Term Extension Study of AT2101 (Afegostat Tartrate) in Type 1 Gaucher Patients

Who this might be right for
Gaucher DiseaseType 1 Gaucher DiseaseGaucher Disease, Type 1
Amicus Therapeutics 8
Large-scale testing (Phase 3)Study completedNCT02593773
What this trial is testing

Safety, Tolerability and Efficacy of ACTIMMUNE® Dose Escalation in Friedreich's Ataxia Study

Who this might be right for
Friedreich's Ataxia
Amgen 86
Large-scale testing (Phase 3)Study completedNCT02534844
What this trial is testing

VTS-270 to Treat Niemann-Pick Type C1 (NPC1) Disease

Who this might be right for
Niemann-Pick Disease, Type C
Mandos LLC 56
Testing effectiveness (Phase 2)Ended earlyNCT03746587
What this trial is testing

Study of Arimoclomol in Patients Diagnosed With Gaucher Disease Type 1 or 3

Who this might be right for
Gaucher Disease, Type 1Gaucher Disease, Type 3
ZevraDenmark 39
Testing effectiveness (Phase 2)Study completedNCT00001416
What this trial is testing

Bone Response to Enzyme Replacement in Gaucher's Disease

Who this might be right for
Gaucher's Disease
National Institute of Neurological Disorders and Stroke (NINDS) 100
Testing effectiveness (Phase 2)Study completedNCT03888664
What this trial is testing

Open Trail of γIFN for Friedreich Ataxia

Who this might be right for
Friedreich Ataxia
IRCCS Eugenio Medea 12
Not applicableLooking for participantsNCT06969209
What this trial is testing

Brain Aging in Phenylketonuria

Who this might be right for
Phenylketonuria (PKU)
Insel Gruppe AG, University Hospital Bern 90
Early research (Phase 1)Study completedNCT01874028
What this trial is testing

Assess the Effects in the Body of a Single Dose of Trientine Dihydrochloride in Wilson's Disease Patients

Who this might be right for
Wilson's Disease
Univar BV 20
Early research (Phase 1)Study completedNCT02800070
What this trial is testing

Autologous Stem Cell Transplantation of Cells Engineered to Express Alpha-Galactosidase A in Patients With Fabry Disease

Who this might be right for
Fabry Disease
University Health Network, Toronto 5
Not applicableEnded earlyNCT02582294
What this trial is testing

Fabry's Disease and Pregnancy (PREFAB)

Who this might be right for
Fabry's DiseasePregnancy
Groupe Hospitalier Diaconesses Croix Saint-Simon 12
Post-approval studies (Phase 4)Study completedNCT00140621
What this trial is testing

A Safety and Efficacy Study of Fabrazyme® Replacement Therapy in Patients With Cardiac Fabry Disease

Who this might be right for
Fabry Disease
Genzyme, a Sanofi Company 6
Not applicableStudy completedNCT07526909
What this trial is testing

Effect of Different Meal Types Given Before Exercise on Plasma Amino Acid Levels and Metabolic Control Parameters in Classical Phenylketonuria Patients Undergoing Aerobic and Resistance Exercises

Who this might be right for
Phenylketonuria
Hacettepe University 10
Not applicableStudy completedNCT03627416
What this trial is testing

Repetitive Transcranial Magnetic Stimulation as Therapy in Hereditary Spastic Paraplegia and Adrenomyeloneuropathy

Who this might be right for
Hereditary Spastic ParaplegiaAdrenomyeloneuropathy
Jakub Antczak 15
Not applicableLooking for participantsNCT03047369
What this trial is testing

The Myelin Disorders Biorepository Project

Who this might be right for
LeukodystrophyWhite Matter DiseaseLeukoencephalopathies+63 more
Children's Hospital of Philadelphia 12,000
Early research (Phase 1)Study completedNCT01730469
What this trial is testing

Safety and Pharmacokinetics of AT1001 (Migalastat HCl) in Healthy Subjects and Subjects With Impaired Renal Function

Who this might be right for
Fabry Disease
Amicus Therapeutics 32
Not applicableStudy completedNCT01136304
What this trial is testing

Validating a New Severity Score System for Adults With Type 1 Gaucher Disease (GD1)

Who this might be right for
Gaucher Disease
University Research Foundation for Lysosomal Storage Diseases, Inc. 173
Not applicableStudy completedNCT01980433
What this trial is testing

Inhibitory rTMS in Dystonic Wilson Patients

Who this might be right for
Wilson DiseaseMovement DisordersRepetitive Transcranial Magnetic Stimulation
Assistance Publique - Hôpitaux de Paris 14
Not applicableLooking for participantsNCT06065852
What this trial is testing

National Registry of Rare Kidney Diseases

Who this might be right for
Adenine Phosphoribosyltransferase DeficiencyAH AmyloidosisAHL Amyloidosis+81 more
UK Kidney Association 35,000
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