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Results for “Brain Diseases, Metabolic, Inborn”

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Enrolling by Invitation

Showing 20 of 0 results

Testing effectiveness (Phase 2)Looking for participantsNCT07721025
What this trial is testing

Study of LX2006 Gene Therapy in Friedreich Ataxia Cardiomyopathy

Who this might be right for
Friedreich AtaxiaCardiomyopathy, Secondary
Lexeo Therapeutics 26
Large-scale testing (Phase 3)Active Not RecruitingNCT02616484
What this trial is testing

Trial of Dichloroacetate in Pyruvate Dehydrogenase Complex Deficiency:

Who this might be right for
Pyruvate Dehydrogenase Complex Deficiency
Saol Therapeutics Inc 34
Not applicableStudy completedNCT02746718
What this trial is testing

Frequency of Pompe's Disease and Neuromuscular Etiologies in Patients With Restrictive Respiratory Failure Associated With Signs of Muscle Weakness

Who this might be right for
Pompe Disease
Centre Hospitalier Universitaire de Nice 50
Not applicableStudy completedNCT06683820
What this trial is testing

Triple Recipient Strategy Using Split, Domino, and Auxiliary Techniques

Who this might be right for
Hepatocellular Carcinoma (HCC)
Second Affiliated Hospital, Zhejiang University, School of Medicine 3
Early research (Phase 1)Study completedNCT02544217
What this trial is testing

A Dose-escalating Clinical Trial With KH176

Who this might be right for
MELASLHONLeigh Syndrome+2 more
Khondrion BV 32
Not applicableLooking for participantsNCT06833489
What this trial is testing

Transcriptomic Analysis to Put an End to Misdiagnosis in Patients With Rare Muscle Diseases

Who this might be right for
Rare Genetic Muscle DiseasesMuscular Dystrophy, DuchenneMuscular Dystrophy, Becker+2 more
Assistance Publique Hopitaux De Marseille 50
Post-approval studies (Phase 4)Study completedNCT05054387
What this trial is testing

China Post-marketing Surveillance (PMS) Study of Fabrazyme®

Who this might be right for
Fabry Disease
Genzyme, a Sanofi Company 22
Large-scale testing (Phase 3)Study completedNCT00943111
What this trial is testing

Eliglustat Tartrate (Genz-112638) in Patients With Gaucher Disease Who Have Reached Therapeutic Goals With Enzyme Replacement Therapy (ENCORE)

Who this might be right for
Gaucher Disease, Type 1
Genzyme, a Sanofi Company 160
Testing effectiveness (Phase 2)UnknownNCT04887311
What this trial is testing

MBM-01 (Tempol) for the Treatment of Ataxia Telangiectasia

Who this might be right for
Ataxia Telangiectasia Louis-BarAtaxia Telangiectasia in ChildrenAtaxia Telangiectasia
Matrix Biomed, Inc. 20
Not applicableLooking for participantsNCT06945081
What this trial is testing

Wilson's Disease Treated With D-Penicillamine: Characterization of Skin Damage Secondary to Treatment by Measuring Skin Elasticity

Who this might be right for
Wilson DiseaseD-PenicillamineEffect of D-penicilline on Cutaneous Elastity of Wilson's Patient
Centre Hospitalier Universitaire de Saint Etienne 120
Early research (Phase 1)Study completedNCT00001234
What this trial is testing

Gene Therapy for Gaucher's and Fabry Disease Using Viruses and Blood-Forming Cells

Who this might be right for
Gaucher's Disease
National Institute of Neurological Disorders and Stroke (NINDS) 120
Testing effectiveness (Phase 2)Study completedNCT00071877
What this trial is testing

An Open-Label Clinical Trial of Replagal Enzyme Therapy in Children Ages 7-17 Years With Fabry Disease

Who this might be right for
Fabry Disease
National Institute of Neurological Disorders and Stroke (NINDS) 25
Not applicableStudy completedNCT00731081
What this trial is testing

Study About the Evolution of Severe Late Onset Pompe Disease Patient With Pulmonary Dysfunction and Receiving Myozyme®

Who this might be right for
Pompe Disease (Late-Onset)Glycogen Storage Disease Type II (GSD II)Glycogenesis 2 Acid Maltase Deficiency
Genzyme, a Sanofi Company 8
Testing effectiveness (Phase 2)Study completedNCT00260000
What this trial is testing

Study of BH4, a New and Simple Treatment of Mild PKU

Who this might be right for
Phenylketonuria
The Kennedy Institute-National Eye Clinic 15
Large-scale testing (Phase 3)Study completedNCT00925301
What this trial is testing

Study of the Effects of Oral AT1001 (Migalastat Hydrochloride) in Patients With Fabry Disease

Who this might be right for
Fabry Disease
Amicus Therapeutics 67
Large-scale testing (Phase 3)Study completedNCT00004339
What this trial is testing

Study of Tetrathiomolybdate in Patients With Wilson Disease

Who this might be right for
Wilson Disease
National Center for Research Resources (NCRR) 90
Not applicableStudy completedNCT01470729
What this trial is testing

Biomarkers in Autosomal Dominant Cerebellar Ataxia

Who this might be right for
Spinocerebellar Ataxia Type 1Spinocerebellar Ataxia Type 2Spinocerebellar Ataxia, Autosomal Recessive 3+1 more
Assistance Publique - Hôpitaux de Paris 102
Not applicableLooking for participantsNCT07232459
What this trial is testing

[18F]FT8 PET Imaging in Immunoglobulin Light Chain Amyloidosis

Who this might be right for
AmyloidCardiac AmyloidosisAmyloidosis+1 more
Tianjin Medical University 25
Large-scale testing (Phase 3)Active Not RecruitingNCT05206773
What this trial is testing

Evaluate the Effect of Venglustat Tablets on Neuropathic and Abdominal Pain in Male and Female Participants ≥16 Years of Age With Fabry Disease

Who this might be right for
Fabry Disease
Sanofi 122
Not applicableEnrolling By InvitationNCT07560956
What this trial is testing

Quality of Life in Adults With Untreated Fabry Disease in Sweden (QoLUF)

Who this might be right for
Fabry Disease
Vastra Gotaland Region 35
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