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Results for “Brain Diseases, Metabolic, Inborn”

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Showing 20 of 0 results

Large-scale testing (Phase 3)Study completedNCT00705939
What this trial is testing

Plant Cell Expressed Recombinant Human Glucocerebrosidase Extension Trial

Who this might be right for
Gaucher Disease
Pfizer 45
Not applicableLooking for participantsNCT03333200
What this trial is testing

Longitudinal Study of Neurodegenerative Disorders

Who this might be right for
MLDKrabbe DiseaseALD+24 more
University of Pittsburgh 1,500
Not applicableStudy completedNCT02785744
What this trial is testing

Genzyme Osteopenia/Osteoporosis Study

Who this might be right for
Gaucher Disease
NYU Langone Health 76
Not applicableActive Not RecruitingNCT07226622
What this trial is testing

Prescreening Study to Identify Potential Wilson Disease Participants for Gene-Editing Clinical Trial

Who this might be right for
Wilson Disease
Prime Medicine, Inc. 30
Not applicableUnknownNCT05548751
What this trial is testing

Physiotherapy Assessment Based on the ICF Model in The Lesch-Nyhan Syndrome: Case Report

Who this might be right for
Lesch-Nyhan Syndrome
Uskudar University 1
Testing effectiveness (Phase 2)Active Not RecruitingNCT04480567
What this trial is testing

AAV Gene Therapy Study for Subjects with PKU

Who this might be right for
Phenylketonuria (PKU)
BioMarin Pharmaceutical 100
Not applicableUnknownNCT06048601
What this trial is testing

18F-florbetaben PET-CT to Non-invasively Diagnose Cardiac AL Amyloidosis

Who this might be right for
AmyloidAmyloid Cardiomyopathy
Fondazione Toscana Gabriele Monasterio 150
Not applicableUnknownNCT05255328
What this trial is testing

Clinical Long Term Evaluation of Glutamine Supplement in MELAS Syndrome

Who this might be right for
MELAS Syndrome
Hospital Universitario 12 de Octubre 9
Testing effectiveness (Phase 2)Study completedNCT02909400
What this trial is testing

The KHENERGY Study

Who this might be right for
Mitochondrial DiseasesMitochondrial MyopathiesMitochondrial Encephalomyopathies+2 more
Khondrion BV 20
Not applicableUnknownNCT01914536
What this trial is testing

A Natural History Study of Adult Onset Pompe Disease Using Muscle MRI

Who this might be right for
Pompe Disease
Fundació Institut de Recerca de l'Hospital de la Santa Creu i Sant Pau 30
Early research (Phase 1)Active Not RecruitingNCT06247670
What this trial is testing

Study of CMP-CPS-001 in Healthy Volunteers and Participants With Abnormal Heterozygous OTC Genotype

Who this might be right for
Healthy VolunteersOTC Deficiency
CAMP4 Therapeutics Corporation 120
Not applicableStudy completedNCT01619722
What this trial is testing

Study of a National Cohort of Adult Patients With Phenylketonuria

Who this might be right for
PKUHyperphenylalaninemia
University Hospital, Tours 220
Not applicableStudy completedNCT01065753
What this trial is testing

Multi-faceted Evaluations Following Weight Reduction in Subjects With Metabolic Syndrome

Who this might be right for
Metabolic Syndrome
Taichung Veterans General Hospital 40
Testing effectiveness (Phase 2)Active Not RecruitingNCT05445323
What this trial is testing

Gene Therapy for Cardiomyopathy Associated With Friedreich's Ataxia

Who this might be right for
Friedreich AtaxiaCardiomyopathy, Secondary
Lexeo Therapeutics 8
Not applicableStudy completedNCT00830583
What this trial is testing

Pompe Prevalence Study in Patients With Muscle Weakness Without Diagnosis

Who this might be right for
Pompe's Disease
Centre Hospitalier Universitaire de Nice 400
Not applicableStudy completedNCT01391637
What this trial is testing

Long-Term Follow-Up Study of Human Stem Cells Transplanted in Subjects With Connatal Pelizaeus-Merzbacher Disease (PMD)

Who this might be right for
Pelizaeus-Merzbacher DiseasePMD
StemCells, Inc. 4
Not applicableStudy completedNCT05356377
What this trial is testing

Preliminary Study of Brain Effects of Palynziq-Related Changes in Phenylalanine in Individuals With PKU

Who this might be right for
Phenylketonurias
University of Missouri-Columbia 6
Not applicableActive Not RecruitingNCT07011329
What this trial is testing

Brain Alterations Based on Multiplex MR Imaging in Nasopharyngeal Carcinoma Patients After Treatment, and Their Correlation With Clinical Cognitive Scores.

Who this might be right for
Nasopharyngeal Carcinoma (NPC)
Hubei Cancer Hospital 60
Not applicableStudy completedNCT06180421
What this trial is testing

Mitochondrial Video Assessment Source Material Study

Who this might be right for
PolG, Primary Mitochondrial Myopathy
The Emmes Company, LLC 28
Not applicableWithdrawnNCT02298647
What this trial is testing

Biomarker for Gangliosidosis: BioGM1/BioGM2 (BioGM1/GM2)

Who this might be right for
Hepato-splenomegalyDysostosis MultiplexSeizures+1 more
CENTOGENE GmbH Rostock
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