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Results for “Brain Diseases, Metabolic, Inborn”

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Showing 20 of 0 results

Not applicableStudy completedNCT02345135
What this trial is testing

Susceptibility to Infections in Ataxia Telangiectasia

Who this might be right for
Ataxia TelangiectasiaInfectionsImmunodeficiency+1 more
Johann Wolfgang Goethe University Hospital 41
Testing effectiveness (Phase 2)Study completedNCT01624311
What this trial is testing

Pilot Study For Hypothermia Treatment In Hyperammonemic Encephalopathy In Neonates And Very Young Infants

Who this might be right for
Urea Cycle DisordersOrganic Acidemias
Uta Lichter-Konecki 5
Early research (Phase 1)Study completedNCT01065558
What this trial is testing

Safety and Tolerability of the D1 Dopamine Receptor Antagonist Ecopipam in Patients With Lesch-Nyhan Disease

Who this might be right for
Lesch-Nyhan Disease
Psyadon Pharma 5
Not applicableStudy completedNCT02798458
What this trial is testing

Evaluation of the Gastrointestinal Manifestation of Fabry's Disease

Who this might be right for
Fabry's Disease
Massachusetts General Hospital 48
Large-scale testing (Phase 3)Study completedNCT01760564
What this trial is testing

Application of Miglustat in Patients With Niemann-Pick Type C

Who this might be right for
Niemann-Pick Disease Type C
National Taiwan University Hospital 5
Not applicableNot Yet RecruitingNCT06615011
What this trial is testing

Bardet Beidle Syndrome in a Syrian Adolescent : a Rare Case Report

Who this might be right for
Bardet Biedl Syndrome (BBS)
Al Baath University 100
Not applicableStudy completedNCT05487378
What this trial is testing

Evaluation of Phe Fluctuation in PKU Pts Treated With PKU GOLIKE Versus Standard Amino Acid Protein Substitute.

Who this might be right for
Phenylketonurias
APR Applied Pharma Research s.a. 16
Not applicableStudy completedNCT01096758
What this trial is testing

Follow-up of Adult Phenylketonuria (PKU) Patients

Who this might be right for
Nutritional DeficienciesPhenylketonuria
Ludwig-Maximilians - University of Munich 40
Not applicableStudy completedNCT02311283
What this trial is testing

Pilot Study: Urea Cycle Disorders Practice Patterns and Outcomes Assessment

Who this might be right for
Urea Cycle Disorders
University of South Florida 87
Large-scale testing (Phase 3)Ended earlyNCT00432822
What this trial is testing

Long-Term Tetrahydrobiopterin Treatment in PKU Patients of 0-18 Years - Study on Phenylalanine Tolerance and Safety

Who this might be right for
Phenylalanine Hydroxylase Deficiencies
Orphanetics Pharma Entwicklungs GmbH 50
Not applicableActive Not RecruitingNCT03655223
What this trial is testing

Early Check: Expanded Screening in Newborns

Who this might be right for
Spinal Muscular AtrophyFragile X SyndromeFragile X - Premutation+182 more
RTI International 30,000
Testing effectiveness (Phase 2)Study completedNCT00718627
What this trial is testing

Human Heterologous Liver Cells for Infusion in Children With Urea Cycle Disorders

Who this might be right for
Urea Cycle DisordersCarbamoylphosphate Synthetase I DeficiencyOrnithine Transcarbamylase Deficiency+1 more
Cytonet GmbH & Co. KG 12
Not applicableStudy completedNCT04353466
What this trial is testing

Assessing the Impact of Elelyso on Bone Involvement Currently Treated With Other ERTs

Who this might be right for
Gaucher Disease, Type 1
Shaare Zedek Medical Center 30
Testing effectiveness (Phase 2)Study completedNCT00325962
What this trial is testing

The Effects of 6R-BH4 on Blood Pressure in Subjects With Poorly Controlled Systemic Hypertension

Who this might be right for
Hypertension
BioMarin Pharmaceutical 84
Early research (Phase 1)Study completedNCT00634660
What this trial is testing

Safety and Tolerability Study of rAvPAL-PEG to Treat Phenylketonuria

Who this might be right for
Phenylketonuria
BioMarin Pharmaceutical 25
Not applicableStudy completedNCT02023086
What this trial is testing

Clinical Investigation on the Blood Oxygenation at the Optic Nerve Head in Fabry Patients

Who this might be right for
Fabry Disease
Université de Montréal 8
Post-approval studies (Phase 4)UnknownNCT02426905
What this trial is testing

Study to Assess Long-Term Outcomes of Trientine in Wilson Disease Patients Withdrawn From Therapy With d-Penicillamine

Who this might be right for
Wilson Disease
Univar BV 90
Testing effectiveness (Phase 2)Looking for participantsNCT07748403
What this trial is testing

The Safety and Efficacy of Prime Editing (PM577) in Participants With Wilson Disease (WD)

Who this might be right for
Wilson DiseaseWilson's DiseaseWilsons Disease
Prime Medicine, Inc. 42
Not applicableNot Yet RecruitingNCT07485660
What this trial is testing

Care Pathway for Patients With Fabry's Disease (Fabry-PATH)

Who this might be right for
Fabry Disease
University Hospital, Toulouse 200
Not applicableWithdrawnNCT01331642
What this trial is testing

Biomarker for Gaucher Disease: BioGaucher (BioGaucher)

Who this might be right for
SplenomegalyHepatomegaly
CENTOGENE GmbH Rostock
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