Clinical trials: Ornithine Transcarbamylase Deficiency
Healthy Volunteers, OTC Deficiency
Study of CMP-CPS-001 in Healthy Volunteers and Participants With Abnormal Heterozygous OTC Genotype
The objective of this clinical study is to evaluate the safety, tolerability, pharmacokinetics, and pharmacodynamics of single and multiple ascending doses of CMP-CPS-001 administered as a subcutaneous injection in adult healthy…
- Trial name
- NCT06247670
- Testing
- CMP-CPS-001
- Where
- 2 sites in Australia, Netherlands
- Volunteers
- 120 people, healthy volunteers welcome
- Sponsor
- CAMP4 Therapeutics Corporation
Urea Cycle Disorder
UNLOCKED: A Phase 2, Open-label Trial With KB195 in Subjects With a Urea Cycle Disorder
UNLOCKED: A Phase 2 Trial to Evaluate the Efficacy and Safety of KB195 in Subjects with a Urea Cycle Disorder with Inadequate Control on Standard of Care
- Trial name
- UNLOCKED (NCT03933410)
- Testing
- KB195
- Where
- 19 sites in United States, Belgium and 5 more
- Volunteers
- 13 people
- Sponsor
- Kaleido Biosciences
OTC Deficiency
Clinical Study of DTX301 AAV-Mediated Gene Transfer for Ornithine Transcarbamylase (OTC) Deficiency
The primary objective is to evaluate the efficacy of DTX301 on the improvement of ornithine transcarbamylase (OTC) function by maintaining safe plasma ammonia levels.
- Trial name
- NCT05345171
- Testing
- DTX301, Oral Corticosteroids, Sodium Acetate
- Where
- 16 sites in United States, Argentina and 8 more
- Volunteers
- 37 people
- Sponsor
- Ultragenyx Pharmaceutical Inc
Analgesia
A Study Investigating the Pharmacokinetic Profiles of Four Extended Release Paracetamol Formulations
A study investigating the pharmacokinetic profiles of four extended release paracetamol formulations
- Trial name
- NCT01568749
- Testing
- Paracetamol, Paracetamol formulation 1, Paracetamol Formulation 2
- Where
- United States
- Volunteers
- 14 people, healthy volunteers welcome
- Sponsor
- GlaxoSmithKline
OTC Deficiency, Ornithine Transcarbamylase Deficiency
A Study to Evaluate the Pharmacodynamics and Safety of ARCT-810 in Participants With OTCD
Evaluate the safety and pharmacodynamics of multiple doses of ARCT-810 in adolescent and adult participants with OTC deficiency.
- Conditions
- OTC Deficiency, Ornithine Transcarbamylase Deficiency, OTCD
- Trial name
- NCT06488313
- Testing
- ARCT-810
- Where
- United States
- Volunteers
- 9 people
- Sponsor
- Arcturus Therapeutics, Inc.
Ornithine Transcarbamylase Deficiency
Safety, Tolerability and Pharmacokinetics of ARCT-810 in Healthy Adult Subjects
Determine the safety, tolerability and pharmacokinetics of single doses of ARCT-810 in healthy adult subjects.
- Trial name
- NCT04416126
- Testing
- ARCT-810
- Where
- New Zealand
- Volunteers
- 30 people, healthy volunteers welcome
- Sponsor
- Arcturus Therapeutics, Inc.
Coronary Artery Disease
Observational Study of OCT in a Patients Undergoing FFR
The purpose of this study is to define and evaluate optical coherence tomography (OCT) stent guidance parameters through prospective data collection in percutaneous coronary intervention (PCI) procedures of de novo lesions.
- Trial name
- ILUMIEN I (NCT01663896)
- Testing
- OCT stent guidance
- Where
- 36 sites in United States, Australia and 12 more
- Volunteers
- 418 people
- Sponsor
- Abbott Medical Devices
Urea Cycle Disorder
Observational Study That Will Collect Information on Patients With Urea Cycle Disorders (UCDs)
THRIVE is an observational study that will collect information on patients with UCDs. THRIVE will follow enrolled participants for up to 10 years.
- Trial name
- THRIVE (NCT01948427)
- Where
- United States
- Volunteers
- 203 people
- Sponsor
- Amgen
Ornithine Transcarbamylase Deficiency
A Study to Assess Plasma Ammonia Time-Normalized Area Under the Curve and Rate of Ureagenesis in Healthy Adult Subjects
The objective of the study is to characterize 24-hour plasma ammonia levels, characterize urea production rates in healthy normal subjects.
- Trial name
- NCT04269122
- Testing
- No Intervention
- Where
- United States
- Volunteers
- 120 people, healthy volunteers welcome
- Sponsor
- Ultragenyx Pharmaceutical Inc
Ornithine Transcarbamylase (OTC) Deficiency
Long Term Follow Up to Evaluate DTX301 in Adults With Late-Onset OTC Deficiency
Determine the long-term safety of DTX301 following a single intravenous (IV) dose in adults with late-onset ornithine transcarbamylase (OTC) deficiency.
- Trial name
- CAPtivate (NCT03636438)
- Testing
- No Intervention
- Where
- 9 sites in United States, Canada and 3 more
- Volunteers
- 11 people
- Sponsor
- Ultragenyx Pharmaceutical Inc
Ornithine Transcarbamylase Deficiency
Phase 1b Study to Assess Safety, Tolerability, and Pharmacokinetics of ARCT-810 in Stable Adult Subjects With Ornithine Transcarbamylase Deficiency
Determine the safety, tolerability and pharmacokinetics of single doses of ARCT-810 in clinically stable patients (stable on standard of care treatment, e.g.
- Trial name
- NCT04442347
- Testing
- ARCT-810
- Where
- 8 sites in United States
- Volunteers
- 16 people
- Sponsor
- Arcturus Therapeutics, Inc.
Ornithine Transcarbamylase Deficiency, Wilson Disease
Adeno-Associated Virus (AAV) Antibody Study in Subjects OTC Deficiency, GSDIa, and Wilson Disease
The objective of this observational study is to evaluate the seroprevalence of anti-AAV antibodies in subjects with Ornithine Transcarbamylase (OTC) deficiency, Glycogen Storage Disease Type Ia (GSDIa), and Wilson Disease
- Conditions
- Ornithine Transcarbamylase Deficiency, Wilson Disease, Glycogen Storage Disease Type IA
- Trial name
- NCT04909346
- Where
- 5 sites in United States, Brazil and 3 more
- Volunteers
- 51 people
- Sponsor
- Ultragenyx Pharmaceutical Inc
Ornithine Transcarbamylase Deficiency
Study to Characterize Rate of Ureagenesis in Patients With Ornithine Transcarbamylase (OTC) Deficiency
The objectives of the study are to characterize urea production rates in patients with OTC, characterize the association of rate of ureagenesis and disease severity in OTC patients, characterize the association of rate of…
- Trial name
- NCT04717453
- Testing
- No Intervention
- Where
- United States
- Volunteers
- 1 people
- Sponsor
- Ultragenyx Pharmaceutical Inc
Urea Cycle Disorders, Inborn, Urea Cycle Disorders
Phase 1/2 Study of KRRO-121 in Healthy Volunteers and Patients With UCD
Phase 1/2 Study of KRRO-121 is a randomized, placebo-controlled study in Healthy Volunteers (Phase 1 SAD and MAD) and Study Participants with Urea Cycle Disorders (Phase 2).
- Conditions
- Urea Cycle Disorders, Inborn, Urea Cycle Disorders, Ornithine Transcarbamylase (OTC) Deficiency, Arginase 1 Deficiency
- Trial name
- ANCHOR (NCT07773246)
- Testing
- KRRO-121
- Where
- Australia
- Volunteers
- 30 people, healthy volunteers welcome
- Sponsor
- Korro Bio, Inc.
Ornithine Transcarbamylase Deficiency
Safety, Tolerability and PK/PD Evaluation of Intravenous Administration of MRT5201 in Patients With OTC Deficiency
This Phase 1/2, first-in-human study will evaluate the safety and tolerability of single escalating doses of MRT5201 administered intravenously to subjects with OTC Deficiency (OTCD).
- Trial name
- STEP-OTC (NCT03767270)
- Testing
- MRT5201
- Sponsor
- Translate Bio, Inc.
Ornithine Transcarbamylase Deficiency, OTC Deficiency
Study for Adolescents and Adults With Ornithine Transcarbamylase Deficiency to Evaluate Safety and Tolerability of ARCT-810
The primary objective is to evaluate the safety and tolerability of repeated doses of intravenously administered ARCT-810.
- Conditions
- Ornithine Transcarbamylase Deficiency, OTC Deficiency, OTCD
- Trial name
- NCT05526066
- Testing
- ARCT-810
- Where
- 14 sites in Belgium, France and 4 more
- Volunteers
- 8 people
- Sponsor
- Arcturus Therapeutics, Inc.
Ornithine Transcarbamylase Deficiency, Ornithine Transcarbamylase Deficiency Disease
Long-term Follow-up (LTFU) Study of Participants in Any iECURE Protocol Using an Investigational Product (IP)
This LTFU is being conducted to assess long-term safety and durability of response in participants dosed with IP in a parent protocol, and to collect longitudinal natural history in enrolled but not dosed participants who also…
- Conditions
- Ornithine Transcarbamylase Deficiency, Ornithine Transcarbamylase Deficiency Disease, Urea Cycle Disorders, Inborn
- Trial name
- NCT06805695
- Testing
- No Intervention
- Where
- 3 sites in United States, United Kingdom
- Volunteers
- 13 people
- Sponsor
- iECURE, Inc.
Acute Myocardial Infarction, Restenoses, Coronary
Abbott Vascular Medical Device Registry
The AV-MDR is a prospective, non-randomized, open-label, multi-center registry. The purpose of the AV-MDR study is to proactively collect and evaluate clinical data on the usage of the devices in scope within their intended use…
- Conditions
- Acute Myocardial Infarction, Restenoses, Coronary, Coronary Artery Lesions, Venous Embolism
- Trial name
- AV-MDR (NCT04573660)
- Testing
- Coronary and peripheral stents, Pacing catheters, Vascular plugs
- Where
- 37 sites in United States, Australia and 12 more
- Volunteers
- 3,784 people
- Sponsor
- Abbott Medical Devices
Coronary Artery Disease
Serial OCT Evaluation of Tissue Coverage in Patients Submitted to Inspiron Drug Eluting Stent Implantation
Prospective, multicentric, single-arm study to evaluate fast and effective tissue repair in patients undergone percutaneous coronary intervention with drug eluting stent Inspiron.
- Trial name
- REPAIR (NCT03269461)
- Testing
- Angiography and Optical Coherence Tomography evaluations
- Where
- 5 sites in Brazil
- Volunteers
- 60 people
- Sponsor
- Scitech Produtos Medicos SA
Ornithine Transcarbamylase Deficiency, Ornithine Transcarbamylase Deficiency Disease
An Open-label Study to Investigate ECUR-506 in Male Babies Less Than 9 Months of Age With Neonatal Onset OTC Deficiency
Ornithine Transcarbamylase (OTC) deficiency, the most common urea cycle disorder, is an inherited metabolic disorder caused by a genetic defect in a liver enzyme responsible for detoxifying of ammonia.
- Conditions
- Ornithine Transcarbamylase Deficiency, Ornithine Transcarbamylase Deficiency Disease, Ornithine Carbamoyltransferase Deficiency (Disorder), Urea Cycle Disorders, Inborn
- Trial name
- NCT06255782
- Testing
- ECUR-506
- Where
- 12 sites in United States, Australia and 2 more
- Volunteers
- 20 people
- Sponsor
- iECURE, Inc.