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Results for “Brain Diseases, Metabolic, Inborn”

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Showing 20 of 0 results

Not applicableEnded earlyNCT05827536
What this trial is testing

Open-label, Randomized, 2-way Crossover, Monocentric, Controlled Study to Evaluate the Effect on Daily PHE Fluctuation of PKU GOLIKE Versus SoC in Patients With PKU.

Who this might be right for
Phenylketonurias
APR Applied Pharma Research s.a. 21
Not applicableStudy completedNCT01626092
What this trial is testing

Reduced-Intensity Hematopoietic Stem Cell Transplant for High Risk Lysosomal and Peroxisomal Disorders

Who this might be right for
Lysosomal Storage DiseasePeroxisomal Disorder
Masonic Cancer Center, University of Minnesota 3
Not applicableStudy completedNCT05109793
What this trial is testing

GM1 and GM2 Gangliosidosis PROspective Neurological Disease TrajectOry Study (PRONTO)

Who this might be right for
GM1 GangliosidosisSandhoff DiseaseTay-Sachs Disease
Azafaros B.V. 31
Not applicableLooking for participantsNCT02399748
What this trial is testing

A Long-term Study for the Outcome of Pompe Disease

Who this might be right for
Pompe Disease
National Taiwan University Hospital 50
Not applicableUnknownNCT05816993
What this trial is testing

Assessment of Neurological Manifestations in Gaucher Disease Patients

Who this might be right for
Gaucher Disease
Assiut University 45
Not applicableWithdrawnNCT02309632
What this trial is testing

Pancreatic Cancer Screening of High-Risk Individuals in Arkansas

Who this might be right for
Pancreatic NeoplasmsPeutz-Jegher's SyndromeBRCA1 Gene Mutation+5 more
University of Arkansas
Not applicableLooking for participantsNCT05579548
What this trial is testing

A Global, Multicenter Study to Assess Maternal, Fetal and Infant Outcomes of Exposure to Palynziq® (Pegvaliase) During Pregnancy and Breastfeeding

Who this might be right for
Phenylketonuria, Maternal
BioMarin Pharmaceutical 50
Testing effectiveness (Phase 2)Study completedNCT00730314
What this trial is testing

Unrelated Hematopoietic Stem Cell Transplantation(HSCT) for Genetic Diseases of Blood Cells

Who this might be right for
Sickle Cell DiseaseThalassemiaAnemia+9 more
Children's Hospital Los Angeles 25
Not applicableUnknownNCT00497484
What this trial is testing

Evaluation of rhGH Replacement Therapy in Patients With Pseudohypoparathyroidism Type Ia (PHP Ia)

Who this might be right for
PseudohypoparathyroidismGrowth Hormone Deficiency, Dwarfism
University of Milan
Not applicableStudy completedNCT04898829
What this trial is testing

Evaluation of PKU Explore France

Who this might be right for
Phenylketonurias
Vitaflo International, Ltd 7
Not applicableStudy completedNCT06801288
What this trial is testing

Diagnostic Role of Renal Biopsy in Patients With Fabry Disease

Who this might be right for
Fabry Disease
IRCCS Azienda Ospedaliero-Universitaria di Bologna 80
Not applicableStudy completedNCT02212288
What this trial is testing

Antioxidant Signature in Adult Patients With Phenylketonuria

Who this might be right for
PKUHyperphenylalaninemia
University Hospital, Tours 20
Large-scale testing (Phase 3)Looking for participantsNCT06904261
What this trial is testing

Migalastat in Pediatric Subjects (2 to <12 Yrs) With Fabry Disease and Amenable GLA Variants

Who this might be right for
Fabry Disease
Amicus Therapeutics 8
Testing effectiveness (Phase 2)Study completedNCT02107846
What this trial is testing

An Open-Label, Dose Escalation Study to Evaluate the Safety and the Pharmacokinetics of Oral PRX-112

Who this might be right for
Gaucher Disease
Protalix 10
Testing effectiveness (Phase 2)Study completedNCT01016366
What this trial is testing

Safety Study of Carbamylated Erythropoietin to Treat Patients With the Neurodegenerative Disorder Friedreich's Ataxia

Who this might be right for
Friedreich's Ataxia
H. Lundbeck A/S 36
Not applicableStudy completedNCT04845958
What this trial is testing

A Non-Interventional National Study in Pediatric Patients With Unexplained Enlarged Spleen

Who this might be right for
Gaucher Disease, SplenomegalyAcid SphingoMyelinase Deficiency
Sanofi 60
Not applicableActive Not RecruitingNCT04798378
What this trial is testing

NuroSleeve Powered Brace & Stimulation System to Restore Arm Function

Who this might be right for
Neurologic DiseasesHemiparesisQuadriplegia+11 more
Thomas Jefferson University 20
Not applicableStudy completedNCT02594917
What this trial is testing

Genetic and Environmental Determinants That Control Metabolism in Pulmonary Hypertension

Who this might be right for
Iron-sulfur Cluster DeficiencyPulmonary HypertensionFriedreich Ataxia
University of Pittsburgh 13
Testing effectiveness (Phase 2)Study completedNCT01560182
What this trial is testing

Gene Therapy for Metachromatic Leukodystrophy (MLD)

Who this might be right for
Lysosomal Storage DiseaseMetachromatic Leukodystrophy
Orchard Therapeutics 20
Not applicableLooking for participantsNCT05473637
What this trial is testing

Taiwan Associated Genetic and Nongenetic Small Vessel Disease

Who this might be right for
Cerebral Small Vessel DiseasesCadasilHTRA1-Related Autosomal Dominant Cerebral Angiopathy+5 more
National Taiwan University Hospital 500
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