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Results for “Brain Diseases, Metabolic, Inborn”

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Showing 20 of 0 results

Not applicableWithdrawnNCT03284658
What this trial is testing

Biomarker for the Early Diagnosis and Monitoring in Tyrosinemia Type 1 (BioTyrosin)

Who this might be right for
TyrosinosisHepatorenal TyrosinemiaFumarylacetoacetase Deficiency+2 more
CENTOGENE GmbH Rostock
Not applicableStudy completedNCT02440932
What this trial is testing

Impact of Phenylketonuria-type Diet on Appetite, Appetite Hormones and Diet Induced Thermogenesis

Who this might be right for
Phenylketonuria (PKU)
University of Glasgow 26
Testing effectiveness (Phase 2)Study completedNCT06949358
What this trial is testing

Evaluate Safety and Tolerability of Olipudase Alfa in Pediatric and Adult Participants With Acid Sphingomyelinase Deficiency (ASMD) Who Completed the DFI12712 or the LTS13632 Study in France

Who this might be right for
Acid Sphingomyelinase Deficiency
Sanofi 3
Large-scale testing (Phase 3)Study completedNCT01074944
What this trial is testing

Eliglustat Tartrate (Genz-112638) in Patients With Gaucher Disease to Evaluate Once Daily Versus Twice Daily Dosing (EDGE)

Who this might be right for
Gaucher Disease
Genzyme, a Sanofi Company 170
Testing effectiveness (Phase 2)Ended earlyNCT03454893
What this trial is testing

Open Label, Study Of Efficacy and Safety Of AVR-RD-01 for Treatment-Naive Subjects With Classic Fabry Disease

Who this might be right for
Fabry Disease
AVROBIO 15
Not applicableUnknownNCT01075438
What this trial is testing

Immunogenicity of Pneumococcal Vaccines in Ataxia-telangiectasia Patients

Who this might be right for
Ataxia Telangiectasia
Sheba Medical Center 20
Testing effectiveness (Phase 2)Study completedNCT00084084
What this trial is testing

Replagal Enzyme Replacement Therapy for Children With Fabry Disease

Who this might be right for
Fabry Disease
Shire 17
Not applicableLooking for participantsNCT06316752
What this trial is testing

The Natural History of Sialidosis Type I

Who this might be right for
Observational Study
National Taiwan University Hospital 30
Testing effectiveness (Phase 2)Study completedNCT01599286
What this trial is testing

Short-Term Outcome of N-Carbamylglutamate in the Treatment of Acute Hyperammonemia

Who this might be right for
Propionic Acidemia, Type I and/or Type IIMethylmalonic AcidemiaCarbamoyl-Phosphate Synthase I Deficiency Disease+1 more
Mendel Tuchman 35
Not applicableUnknownNCT03446586
What this trial is testing

Hereditary Hepatorenal Tyrosinemia Natural History in Egypt and the Arab World (Multicenter Clinical Study)

Who this might be right for
Hereditary Tyrosinemia, Type I
Yassin Abdelghaffar Charity Center for Liver Disease and Research 50
Not applicableUnknownNCT03542370
What this trial is testing

Umbrella Review of the DASH Dietary Pattern and Cardiometabolic Risk

Who this might be right for
Cardiovascular Risk FactorCardiovascular DiseasesCoronary Heart Disease+6 more
University of Toronto 1
Not applicableStudy completedNCT05096988
What this trial is testing

Evaluation of PKU Sphere Liquid

Who this might be right for
Phenylketonurias
Vitaflo International, Ltd 19
Large-scale testing (Phase 3)UnknownNCT06081062
What this trial is testing

Evaluate the Safety and Efficacy of Fabagal® (Agalsidase Beta) in Patients With Fabry Disease

Who this might be right for
Fabry Disease
ISU Abxis Co., Ltd. 24
Not applicableUnknownNCT05719337
What this trial is testing

Multi-Modality Echocardiographic Techniques in Pathological Left Ventricular Hypertrophy Adults

Who this might be right for
Left Ventricular Hypertrophy
First Hospital of China Medical University 660
Not applicableLooking for participantsNCT07412782
What this trial is testing

REMS25: Study on the Use of REMS Technology in Diseases Commonly Associated With Reduced Bone Mineral Density (BMD)

Who this might be right for
Osteogenesis ImperfectaOsteoporosisHypogonadisms+6 more
Meyer Children's Hospital IRCCS 100
Testing effectiveness (Phase 2)Study completedNCT01560286
What this trial is testing

Evaluate Subcutaneously Administered rAvPAL-PEG in Patients With Phenylketonuria for 24 Weeks

Who this might be right for
Phenylketonuria
BioMarin Pharmaceutical 24
Not applicableStudy completedNCT05671770
What this trial is testing

Study Based on Electronic Health RecOrds to Identify Patients at High-risk of Fabry DiseasE (HOPE Fabry)

Who this might be right for
Fabry Disease
Sanofi 15
Large-scale testing (Phase 3)Study completedNCT00007020
What this trial is testing

Compassionate Treatment of Patients With Inborn Errors of Bile Acid Metabolism With Cholic Acid

Who this might be right for
Infantile Refsum's DiseaseZellweger SyndromeAdrenoleukodystrophy+2 more
Mirum Pharmaceuticals, Inc. 85
Testing effectiveness (Phase 2)Ended earlyNCT05154890
What this trial is testing

A Multiple Ascending Dose Study of ACN00177 (Pegtarviliase) in Subjects With CBS Deficiency

Who this might be right for
Homocystinuria Due to Cystathionine Beta-Synthase Deficiency
Aeglea Biotherapeutics 13
Large-scale testing (Phase 3)Active Not RecruitingNCT05280548
What this trial is testing

Evaluate the Effect of Venglustat Tablets on Left Ventricular Mass Index in Male and Female Adult Participants With Fabry Disease

Who this might be right for
Fabry Disease
Sanofi 104
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