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Results for “OTCD”

61 trials

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Showing 20 of 61 results

Testing effectiveness (Phase 2)WithdrawnNCT01791192
What this trial is testing

Study of FTY720 in Patients With Uveitis

Who this might be right for
Acute Noninfectious Posterior, Intermediate, or Pan Uveitis
Novartis Pharmaceuticals
Testing effectiveness (Phase 2)WithdrawnNCT03767270
What this trial is testing

Safety, Tolerability and PK/PD Evaluation of Intravenous Administration of MRT5201 in Patients With OTC Deficiency

Who this might be right for
Ornithine Transcarbamylase Deficiency
Translate Bio, Inc.
Not applicableEnrolling By InvitationNCT03655223
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
Large-scale testing (Phase 3)UnknownNCT01824030
What this trial is testing

FFR or OCT Guidance to RevasculariZe Intermediate Coronary Stenosis Using Angioplasty

Who this might be right for
Ischemic Heart Disease
Catholic University of the Sacred Heart 400
Not applicableStudy completedNCT04248062
What this trial is testing

Patient and Observer Reported Outcome Measurements in Inborn Errors of Metabolism

Who this might be right for
Inborn Errors of MetabolismUrea Cycle DisorderMaple Syrup Urine Disease+5 more
University Children's Hospital, Zurich 69
Testing effectiveness (Phase 2)Looking for participantsNCT06255782
What this trial is testing

An Open-label Study to Investigate ECUR-506 in Male Babies Less Than 9 Months of Age With Neonatal Onset OTC Deficiency

Who this might be right for
Ornithine Transcarbamylase DeficiencyOrnithine Transcarbamylase Deficiency DiseaseOrnithine Carbamoyltransferase Deficiency (Disorder)+1 more
iECURE, Inc. 20
Early research (Phase 1)Ended earlyNCT00004386
What this trial is testing

Phase I Pilot Study of Liver-Directed Gene Therapy for Partial Ornithine Transcarbamylase Deficiency

Who this might be right for
Ornithine Transcarbamylase Deficiency Disease
Eunice Kennedy Shriver National Institute of Child Health and Human Development (NICHD)
Early research (Phase 1)UnknownNCT00004307
What this trial is testing

Study of Treatment and Metabolism in Patients With Urea Cycle Disorders

Who this might be right for
Amino Acid Metabolism, Inborn Errors
National Center for Research Resources (NCRR) 66
Early research (Phase 1)Study completedNCT04442347
What this trial is testing

Phase 1b Study to Assess Safety, Tolerability, and Pharmacokinetics of ARCT-810 in Stable Adult Subjects With Ornithine Transcarbamylase Deficiency

Who this might be right for
Ornithine Transcarbamylase Deficiency
Arcturus Therapeutics, Inc. 16
Not applicableUnknownNCT05910151
What this trial is testing

Selective Screening of Children for Hereditary Metabolic Diseases by Tandem Mass Spectrometry in Kazakhstan

Who this might be right for
Propionic/Methylmalonic AcidemiasMaple Syrup Urine DiseaseCitrullinemia+26 more
West Kazakhstan Medical University 2,250
Not applicableLooking for participantsNCT04908319
What this trial is testing

Hepatic Histopathology in Urea Cycle Disorders

Who this might be right for
Urea Cycle DisorderOrnithine Transcarbamylase DeficiencyCitrullinemia 1+7 more
Baylor College of Medicine 70
Not applicableActive Not RecruitingNCT07485413
What this trial is testing

Looking for VUS to Confirm Dominant Wolfram-like Syndrome Instead of Recessive Wolfram Syndrome

Who this might be right for
Optical Coherence Tomography (OCT) of the RetinaMutation
Hôpital Necker-Enfants Malades 45
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 applicableStudy completedNCT03269461
What this trial is testing

Serial OCT Evaluation of Tissue Coverage in Patients Submitted to Inspiron Drug Eluting Stent Implantation

Who this might be right for
Coronary Artery Disease
Scitech Produtos Medicos SA 60
Not applicableLooking for participantsNCT06805695
What this trial is testing

Long-term Follow-up (LTFU) Study of Participants in Any iECURE Protocol Using an Investigational Product (IP)

Who this might be right for
Ornithine Transcarbamylase DeficiencyOrnithine Transcarbamylase Deficiency DiseaseUrea Cycle Disorders, Inborn
iECURE, Inc. 13
Testing effectiveness (Phase 2)Study completedNCT01599286
What this trial is testing

Short-Term Outcome of N-Carbamylglutamate in the Treatment of Acute Hyperammonemia

Who this might be right for
Propionic Acidemia, Type I and/or Type IIMethylmalonic AcidemiaCarbamoyl-Phosphate Synthase I Deficiency Disease+1 more
Mendel Tuchman 35
Not applicableStudy completedNCT01080859
What this trial is testing

Vessel Wall Response of the Bio-Active-stent and Everolimus-Eluting Stent Assessed By Optical Coherence Tomography

Who this might be right for
Acute Coronary Syndrome
The Hospital District of Satakunta 40
Testing effectiveness (Phase 2)Looking for participantsNCT06488313
What this trial is testing

Evaluate the Pharmacodynamics and Safety of ARCT-810 in Participants With OTCD

Who this might be right for
OTC DeficiencyOrnithine Transcarbamylase DeficiencyOTCD
Arcturus Therapeutics, Inc. 9
Testing effectiveness (Phase 2)Ended earlyNCT03181828
What this trial is testing

Manipulating the Gut Microbiome Study

Who this might be right for
Urea Cycle Disorder
Nicholas Ah Mew 4
Not applicableUnknownNCT02820064
What this trial is testing

Effects of Hypoxia and Inflammation on Citrulline Synthesis by Ornithine Transcarbamylase in Human Enterocytes

Who this might be right for
HypoxiaInflammationChronic Obstructive Pulmonary Disease
University Hospital, Grenoble 30
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