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Results for “Brain Diseases, Metabolic, Inborn”

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Showing 20 of 0 results

Not applicableUnknownNCT05413876
What this trial is testing

Fabry Exercise Intolerance Study

Who this might be right for
Fabry DiseaseFabry Disease, Cardiac Variant
Academisch Medisch Centrum - Universiteit van Amsterdam (AMC-UvA) 30
Not applicableUnknownNCT04620876
What this trial is testing

Bimodal and Coaxial High Resolution Ophtalmic Imaging

Who this might be right for
Retinitis PigmentosaMaculopathy, Age RelatedMacular Dystrophy+8 more
Centre Hospitalier National d'Ophtalmologie des Quinze-Vingts 1,200
Early research (Phase 1)Study completedNCT04442347
What this trial is testing

Phase 1b Study to Assess Safety, Tolerability, and Pharmacokinetics of ARCT-810 in Stable Adult Subjects With Ornithine Transcarbamylase Deficiency

Who this might be right for
Ornithine Transcarbamylase Deficiency
Arcturus Therapeutics, Inc. 16
Testing effectiveness (Phase 2)Not Yet RecruitingNCT07603050
What this trial is testing

A Phase I/II Clinical Study to Evaluate the Safety and Efficacy of VGN-R08b in Patients With Type III Gaucher's Disease

Who this might be right for
Gaucher Disease Type 3
Shanghai Vitalgen BioPharma Co., Ltd. 12
Not applicableStudy completedNCT03334968
What this trial is testing

Prolidase Enzyme Activity in Stroke Patients

Who this might be right for
Stroke, IschemicProlidase Deficiency
Bezmialem Vakif University 74
Not applicableActive Not RecruitingNCT06488261
What this trial is testing

Long Term Follow-Up for Safety of AVR-RD-02

Who this might be right for
Gaucher Disease
John Bernat 2
Testing effectiveness (Phase 2)Study completedNCT04534842
What this trial is testing

Efficacy and Safety of SYNB1618 and SYNB1934 in Adult Patients With Phenylketonuria

Who this might be right for
Phenylketonuria
Synlogic 20
Early research (Phase 1)Study completedNCT00015808
What this trial is testing

Safety Study of Idebenone to Treat Friedreich's Ataxia

Who this might be right for
Friedreich Ataxia
National Institute of Neurological Disorders and Stroke (NINDS) 100
Testing effectiveness (Phase 2)Study completedNCT00176917
What this trial is testing

Stem Cell Transplantation for Hurler

Who this might be right for
Mucopolysaccharidosis IMucopolysaccharidosis VIMannosidosis+1 more
Masonic Cancer Center, University of Minnesota 41
Testing effectiveness (Phase 2)Study completedNCT02273596
What this trial is testing

Efficacy and Safety Study of WTX101 (ALXN1840) in Adult Wilson Disease Patients

Who this might be right for
Wilson Disease
Alexion Pharmaceuticals, Inc. 29
Early research (Phase 1)Study completedNCT01859624
What this trial is testing

Albuterol in Individuals With Late Onset Pompe Disease (LOPD)

Who this might be right for
Pompe Disease
Duke University 8
Not applicableStudy completedNCT01942850
What this trial is testing

International Ataxia Rating Scale in Younger Patients

Who this might be right for
Ataxia Telangiectasia
Quince Therapeutics S.p.A. 63
Post-approval studies (Phase 4)Looking for participantsNCT07477691
What this trial is testing

Immune Modulation During Palynziq® Treatment in Adults (IMPALA)

Who this might be right for
Phenylketonuria
BioMarin Pharmaceutical 12
Not applicableStudy completedNCT02998710
What this trial is testing

Natural History Study of Homocystinuria Caused by Cystathionine Beta-Synthase Deficiency (ACAPPELLA)

Who this might be right for
Homocystinuria Due to CBS Deficiency
Travere Therapeutics, Inc. 110
Large-scale testing (Phase 3)Ended earlyNCT03822013
What this trial is testing

Effects of Miglustat Therapy on Infantile Type of Sandhoff and Taysachs Diseases (EMTISTD)

Who this might be right for
GM2 GangliosidosisSupportive Care
Tehran University of Medical Sciences 30
Not applicableLooking for participantsNCT01694940
What this trial is testing

North American Mitochondrial Disease Consortium Patient Registry and Biorepository (NAMDC)

Who this might be right for
Mitochondrial DisordersMitochondrial Genetic DisordersMitochondrial Diseases+2 more
Columbia University 1,000
Not applicableEnded earlyNCT01541397
What this trial is testing

Bone Mineral Density in Adults With Hyperphenylalaninemia on Kuvan Therapy

Who this might be right for
HyperphenylalaninemiaPhenylketonuria
The University of Texas Health Science Center, Houston 6
Not applicableStudy completedNCT05083806
What this trial is testing

MSOT in Pompe Disease

Who this might be right for
Pompe DiseasePompe Disease (Late-onset)Pompe's Disease Juvenile Onset+1 more
University of Erlangen-Nürnberg Medical School 20
Large-scale testing (Phase 3)Ended earlyNCT00712426
What this trial is testing

Creatine Safety, Tolerability, & Efficacy in Huntington's Disease (CREST-E)

Who this might be right for
Huntington's Disease
Massachusetts General Hospital 553
Early research (Phase 1)Study completedNCT00258778
What this trial is testing

Phase I Single Dose-Escalation Safety Study of Human Glucocerebrosidase (prGCD)

Who this might be right for
Gaucher Disease
Protalix 6
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