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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)Study completedNCT02991144
What this trial is testing

Safety and Dose-Finding Study of DTX301 (scAAV8OTC) in Adults With Late-Onset Ornithine Transcarbamylase (OTC) Deficiency

Who this might be right for
Ornithine Transcarbamylase (OTC) Deficiency
Ultragenyx Pharmaceutical Inc 16
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
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
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
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
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)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
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
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
Not applicableLooking for participantsNCT01793168
What this trial is testing

Rare Disease Patient Registry & Natural History Study - Coordination of Rare Diseases at Sanford

Who this might be right for
Rare DisordersUndiagnosed DisordersDisorders of Unknown Prevalence+340 more
Sanford Health 20,000
Not applicableLooking for participantsNCT02780297
What this trial is testing

Prospective Research Rare Kidney Stones (ProRKS)

Who this might be right for
HyperoxaluriaCystinuriaDent Disease+2 more
Mayo Clinic 220
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
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
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
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
Very early researchStudy completedNCT01659645
What this trial is testing

Anti-1-amino-3-[18F]Fluorocyclobutyl-1-carboxylic Acid (Anti-[18F](FACBC)Positron Emission Tomography (PET-CT) of the Breast

Who this might be right for
Breast Cancer
Emory University 12
Testing effectiveness (Phase 2)Looking for participantsNCT07667387
What this trial is testing

Study of LNP.UCD.ABE in Patients With Urea Cycle Disorders

Who this might be right for
Urea Cycle DisordersCarbamoyl-Phosphate Synthase I Deficiency
Rebecca Ahrens-Nicklas 7
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 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
Testing effectiveness (Phase 2)Not Yet RecruitingNCT07410455
What this trial is testing

An Open-label, Phase 2 Pilot Study on the Efficacy and Safety of Piclidenoson in Patients With Lowe Syndrome

Who this might be right for
Lowe Syndrome
Can-Fite BioPharma 5
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