Ornithine Transcarbamylase Deficiency
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CLINICAL_TRIALS trial_result · 2026-06-02Clinical Trial Update: Clinical Study of DTX301 AAV-Mediated Gene Transfer for Ornithine Transcarbamylase (OTC) Deficiency [Phase 3]
Phase 3 clinical study update for DTX301 AAV-mediated gene transfer in OTC deficiency is active not recruiting.
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CLINICAL_TRIALS research · 2026-04-22Clinical Trial Update: Study of CMP-CPS-001 in Healthy Volunteers and Participants With Abnormal Heterozygous OTC Genotype [Phase 1]
Phase 1 clinical trial update for CMP-CPS-001 in healthy volunteers and individuals with abnormal heterozygous OTC genotype; the study is ac…
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CLINICAL_TRIALS research · 2026-04-15Clinical Trial Update: An Open-label Study to Investigate ECUR-506 in Male Babies Less Than 9 Months of Age With Neonatal Onset OTC Deficiency [Phase 1, Phase 2]
This clinical trial update describes an open-label Phase 1/2 study evaluating ECUR-506 in male infants under 9 months with neonatal-onset Or…
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CLINICAL_TRIALS trial_result · 2026-02-19Clinical Trial Update: Long Term Follow Up to Evaluate DTX301 in Adults With Late-Onset OTC Deficiency
Clinical trial update providing long-term follow-up data for DTX301 in adults with late-onset Ornithine Transcarbamylase deficiency. The stu…
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CLINICAL_TRIALS research · 2026-02-17Clinical Trial Update: Clinical Study of DTX301 AAV-Mediated Gene Transfer for Ornithine Transcarbamylase (OTC) Deficiency [Phase 3]
Clinical trial update indicating that Ultragenyx's DTX301 AAV-mediated gene transfer for ornithine transcarbamylase deficiency is a Phase 3 …
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CLINICAL_TRIALS trial_result · 2025-09-26Clinical Trial Update: Study for Adolescents and Adults With Ornithine Transcarbamylase Deficiency to Evaluate Safety and Tolerability of ARCT-810 [Phase 2]
A Phase 2 study evaluating ARCT-810 for Ornithine transcarbamylase deficiency has been terminated, representing a status update on the trial…
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CLINICAL_TRIALS trial_result · 2025-07-20Clinical Trial Update: Hepatic Histopathology in Urea Cycle Disorders
This clinical trial update indicates the study on hepatic histopathology in urea cycle disorders is actively recruiting, including Ornithine…
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CLINICAL_TRIALS research · 2025-04-17Clinical Trial Update: Liver Disease in Urea Cycle Disorders
A clinical trial update indicating ongoing recruitment for a study on liver disease in patients with urea cycle disorders, including OTC def…
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CLINICAL_TRIALS research · 2025-03-17Clinical Trial Update: A Study to Evaluate the Pharmacodynamics and Safety of ARCT-810 in Participants With OTCD [Phase 2]
A Phase 2 clinical trial update describing the pharmacodynamics and safety evaluation of ARCT-810 in participants with ornithine transcarbam…
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CLINICAL_TRIALS trial_result · 2025-02-03Clinical Trial Update: Long-term Follow-up (LTFU) Study of Participants in Any iECURE Protocol Using an Investigational Product (IP)
Long-term follow-up study update for participants in any iECURE protocol using an investigational product; the trial remains in RECRUITING s…
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CLINICAL_TRIALS trial_result · 2024-08-02Clinical Trial Update: Phase 1b Study to Assess Safety, Tolerability, and Pharmacokinetics of ARCT-810 in Stable Adult Subjects With Ornithine Transcarbamylase Deficiency [Phase 1]
This update reports completion of a Phase 1b study evaluating safety, tolerability, and pharmacokinetics of ARCT-810 in adults with ornithin…
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CLINICAL_TRIALS trial_result · 2022-12-23Clinical Trial Update: Safety and Dose-Finding Study of DTX301 (scAAV8OTC) in Adults With Late-Onset Ornithine Transcarbamylase (OTC) Deficiency [Phase 1, Phase 2]
Phase 1/2 safety and dose-finding study of DTX301 (scAAV8OTC) for adults with late-onset Ornithine Transcarbamylase deficiency has completed…
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CLINICAL_TRIALS trial_result · 2017-04-14Clinical Trial Update: Investigation of Brain Nitrogen in Partial Ornithine Transcarbamylase Deficiency (OTCD) Using 1 H MRS, DTI, and fMRI
A completed clinical trial investigating brain nitrogen handling in partial ornithine transcarbamylase deficiency using MR spectroscopy, dif…
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CLINICAL_TRIALS trial_result · 2016-02-08Clinical Trial Update: Human Heterologous Liver Cells for Infusion in Children With Urea Cycle Disorders [Phase 2]
Phase 2 trial evaluating infusion of human heterologous liver cells in children with urea cycle disorders has been completed.
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CLINICAL_TRIALS trial_result · 2015-05-29Clinical Trial Update: Neurologic Injuries in Adults With Urea Cycle Disorders
A completed clinical trial update reporting results on neurologic injuries in adults with urea cycle disorders, including ornithine transcar…
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CLINICAL_TRIALS trial_result · 2015-03-25Clinical Trial Update: Phase I Study of Adenoviral Vector Mediated Gene Transfer for Ornithine Transcarbamylase in Adults With Partial Ornithine Transcarbamylase Deficiency [Phase 1]
A Phase I study evaluating an adenoviral vector mediated gene transfer for adults with partial ornithine transcarbamylase deficiency has bee…
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FDA label_change · 2013-08-05FDA Approved: BUPHENYL — Supplemental Application (Horizon Therapeutics USA, Inc.)
FDA approved a supplemental application for BUPHENYL (sodium phenylbutyrate) from Horizon Therapeutics USA, Inc.
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FDA approval · 2009-03-31FDA Approved: BUPHENYL — Supplemental Application (Horizon Therapeutics USA, Inc.)
The FDA approved a supplemental application for BUPHENYL (sodium phenylbutyrate) submitted by Horizon Therapeutics USA, Inc.