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Results for “Brain Diseases, Metabolic, Inborn”

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Showing 20 of 0 results

Not applicableStudy completedNCT01917344
What this trial is testing

The Brain, Neurological Features and Neuropsychological Functioning in Adults With Phenylketonuria: A Pilot Study

Who this might be right for
Phenylketonuria
Boston Children's Hospital 10
Not applicableLooking for participantsNCT04528355
What this trial is testing

Data Collection Study of Patients With Non-Malignant Disorders Undergoing UCBT, BMT or PBSCT With RIC

Who this might be right for
Primary Immunodeficiency (PID)Congenital Bone Marrow Failure SyndromesInherited Metabolic Disorders (IMD)+2 more
Paul Szabolcs 50
Testing effectiveness (Phase 2)Active Not RecruitingNCT04798235
What this trial is testing

First-in-Human Study of TSHA-101 Gene Therapy for Treatment of Infantile Onset GM2 Gangliosidosis

Who this might be right for
Infantile GM2 Gangliosidosis (Disorder)
Dr. Anupam Sehgal 3
Early research (Phase 1)Study completedNCT04416126
What this trial is testing

Safety, Tolerability and Pharmacokinetics of ARCT-810 in Healthy Adult Subjects

Who this might be right for
Ornithine Transcarbamylase Deficiency
Arcturus Therapeutics, Inc. 30
Testing effectiveness (Phase 2)Study completedNCT00051935
What this trial is testing

The Safety and Pharmacokinetics of rhGAA in Siblings With Glycogen Storage Disease Type II

Who this might be right for
Glycogen Storage Disease Type IIPompe DiseaseAcid Maltase Deficiency Disease+1 more
Genzyme, a Sanofi Company 2
Large-scale testing (Phase 3)Study completedNCT04974749
What this trial is testing

REPLAGAL® in Treatment-naive Chinese Participants With Fabry Disease

Who this might be right for
Fabry Disease
Takeda 20
Not applicableStudy completedNCT03777826
What this trial is testing

Effects of Synergy on Nutrient Intake and Acceptability in Phenylketonuria (PKU)

Who this might be right for
Phenylketonurias
Nutricia Research 13
Testing effectiveness (Phase 2)Looking for participantsNCT01962415
What this trial is testing

Reduced Intensity Conditioning for Non-Malignant Disorders Undergoing UCBT, BMT or PBSCT

Who this might be right for
Primary Immunodeficiency (PID)Congenital Bone Marrow Failure SyndromesInherited Metabolic Disorders (IMD)+4 more
Paul Szabolcs 100
Early research (Phase 1)Looking for participantsNCT07180355
What this trial is testing

SGT-212 Gene Therapy in Friedreich's Ataxia

Who this might be right for
Friedreich's Ataxia (FA)
Solid Biosciences Inc. 10
Not applicableLooking for participantsNCT06758648
What this trial is testing

Characterizing the Retinal Microvasculature in Patients with Fabry Disease: a Prospective Observational Study

Who this might be right for
Fabry DiseaseEndothelial DysfunctionMicrovasculature
Technical University of Munich 63
Early research (Phase 1)Study completedNCT05324943
What this trial is testing

A Gene Therapy Study in Patients With Gaucher Disease Type 1

Who this might be right for
Gaucher Disease, Type 1
Spur Therapeutics 10
Testing effectiveness (Phase 2)Study completedNCT01043640
What this trial is testing

Allogeneic Bone Marrow Transplant for Inherited Metabolic Disorders

Who this might be right for
MucopolysaccharidosisHurler SyndromeHunter Syndrome+10 more
Masonic Cancer Center, University of Minnesota 46
Not applicableStudy completedNCT03883750
What this trial is testing

Induced Pluripotent Stem Cells for Niemann Pick Disease

Who this might be right for
Niemann-Pick Diseases
CENTOGENE GmbH Rostock 40
Testing effectiveness (Phase 2)Active Not RecruitingNCT05972629
What this trial is testing

A Phase 1/Phase 2 Open-label Study to Evaluate the Safety, Tolerability, and Efficacy of a Single Intravenous Administration of SAR444836 in Adult Participants With Phenylketonuria

Who this might be right for
Phenylketonuria
Sanofi 32
Large-scale testing (Phase 3)Study completedNCT00697073
What this trial is testing

Study to Assess the Safety and Tolerability of Idebenone in the Treatment of Friedreich's Ataxia Patients

Who this might be right for
Friedreich's Ataxia
Santhera Pharmaceuticals 68
Large-scale testing (Phase 3)Study completedNCT03729362
What this trial is testing

A Study Comparing ATB200/AT2221 With Alglucosidase Alfa/Placebo in Adult Subjects With Late-onset Pompe Disease

Who this might be right for
Pompe Disease (Late-onset)
Amicus Therapeutics 125
Not applicableStudy completedNCT04804566
What this trial is testing

Understanding Fabry Disease Therapy Choices Through the Eyes of the Patients

Who this might be right for
Fabry Disease
Amicus Therapeutics 44
Not applicableUnknownNCT05894122
What this trial is testing

Evaluation of Capillary Blood Collection Devices (Mitra® and HemaPEN®)

Who this might be right for
PhenylketonuriaMetabolic Diseases
University Hospital, Grenoble 40
Not applicableStudy completedNCT02363153
What this trial is testing

Diet and Exercise in Pompe Disease

Who this might be right for
Pompe DiseaseGlycogen Storage Disease Type IILysosomal Storage Diseases
University of Florida 2
Testing effectiveness (Phase 2)Study completedNCT00924703
What this trial is testing

Long-Term Extension of Previous rAvPAL-PEG Protocols in Subjects With PKU (PAL-003)

Who this might be right for
Phenylketonuria
BioMarin Pharmaceutical 68
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