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Results for “Amino-Acid Transport Disorder”

27 trials

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Showing 20 of 27 results

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
Not applicableLooking for participantsNCT04602325
What this trial is testing

Systemic Biomarkers of Brain Injury From Hyperammonemia

Who this might be right for
Urea Cycle DisorderOrganic AcidemiaMaple Syrup Urine Disease+3 more
Children's National Research Institute 24
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 applicableStudy completedNCT05687474
What this trial is testing

Baby Detect : Genomic Newborn Screening

Who this might be right for
Congenital Adrenal HyperplasiaFamilial Hyperinsulinemic Hypoglycemia 1Phosphoglucomutase 1 Deficiency+121 more
Centre Hospitalier Universitaire de Liege 6,824
Testing effectiveness (Phase 2)Ended earlyNCT02418598
What this trial is testing

AADC Gene Therapy for Parkinson's Disease

Who this might be right for
Parkinson Disease
Jichi Medical University 2
Not applicableLooking for participantsNCT06065852
What this trial is testing

National Registry of Rare Kidney Diseases

Who this might be right for
Adenine Phosphoribosyltransferase DeficiencyAH AmyloidosisAHL Amyloidosis+81 more
UK Kidney Association 35,000
Testing effectiveness (Phase 2)Study completedNCT02830880
What this trial is testing

FACBC Prostate Therapy Response

Who this might be right for
Prostate Cancer
Emory University 7
Early research (Phase 1)WithdrawnNCT04014712
What this trial is testing

O2 Transport and Utilization in Health and Lung Disease

Who this might be right for
COPDTetrahydrobiopterin DeficiencyOxidative Stress
University of Massachusetts, Amherst
Early research (Phase 1)Study completedNCT00001328
What this trial is testing

Gene Therapy for the Treatment of Brain Tumors

Who this might be right for
Brain NeoplasmNeoplasm Metastasis
National Institute of Neurological Disorders and Stroke (NINDS) 15
Early research (Phase 1)Study completedNCT03065192
What this trial is testing

Safety and Efficacy Study of VY-AADC01 for Advanced Parkinson's Disease

Who this might be right for
Idiopathic Parkinson's DiseaseParkinson's DiseaseBasal Ganglia Disease+6 more
Neurocrine Biosciences 16
Not applicableStudy completedNCT01574287
What this trial is testing

Anti-3-[18F]FACBC Imaging of Parathyroid Adenomas

Who this might be right for
Parathyroid Disease
David M. Schuster, MD 4
Not applicableStudy completedNCT00359515
What this trial is testing

Genetic Analysis of Oculocerebrorenal Syndrome of Lowe

Who this might be right for
Lowe Syndrome
National Human Genome Research Institute (NHGRI) 120
Not applicableActive Not RecruitingNCT03636438
What this trial is testing

Long Term Follow Up to Evaluate DTX301 in Adults With Late-Onset OTC Deficiency

Who this might be right for
Ornithine Transcarbamylase (OTC) Deficiency
Ultragenyx Pharmaceutical Inc 11
Not applicableLooking for participantsNCT07379463
What this trial is testing

Amino Acid Transporter System PET/CT Imaging in AATS-Related Diseases

Who this might be right for
Amino-Acid Transport DisorderPrimary HyperparathyroidismGlioma
Tianjin Medical University 500
Early research (Phase 1)Study completedNCT01973543
What this trial is testing

Safety Study of AADC Gene Therapy (VY-AADC01) for Parkinson's Disease

Who this might be right for
Parkinson's Disease
Neurocrine Biosciences 15
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
Early research (Phase 1)Ended earlyNCT00004498
What this trial is testing

Phase I Study of Adenoviral Vector Mediated Gene Transfer for Ornithine Transcarbamylase in Adults With Partial Ornithine Transcarbamylase Deficiency

Who this might be right for
Ornithine Transcarbamylase Deficiency Disease
University of Pennsylvania 21
Not applicableStudy completedNCT01314560
What this trial is testing

Study of the Pathophysiological Mechanisms Involved in Bleeding Events

Who this might be right for
Oculocerebrorenal Syndrome
Assistance Publique - Hôpitaux de Paris 30
Not applicableUnknownNCT02633436
What this trial is testing

Research on the Role of SLC1A5 in the Development of Esophageal Cancer

Who this might be right for
Esophageal CancerAmino Acid Transport Disorder
Fourth Affiliated Hospital of Guangxi Medical University 100
Large-scale testing (Phase 3)Active Not RecruitingNCT05345171
What this trial is testing

Clinical Study of DTX301 AAV-Mediated Gene Transfer for Ornithine Transcarbamylase (OTC) Deficiency

Who this might be right for
OTC Deficiency
Ultragenyx Pharmaceutical Inc 37
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