Urea Cycle Disorders
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Sign up freeTop drugs studied for this disease
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CLINICAL_TRIALS trial_result · 2024-06-20Clinical Trial Update: A Study of Glyceryl Tri-(4-phenylbutyrate) Administered Orally as a Single Dose, and Twice Daily for Seven Consecutive Days to Subjects With Hepatic Impairment With Cirrhosis and to a Control Group [Phase 1]
A Phase 1 study evaluated the safety and pharmacokinetics of glyceryl tri-(4-phenylbutyrate) given as a single dose and twice-daily dosing f…
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CLINICAL_TRIALS trial_result · 2021-12-15Clinical Trial Update: Manipulating the Gut Microbiome Study [Phase 1, Phase 2]
A Phase 1/2 clinical trial investigating manipulation of the gut microbiome for urea cycle disorder; the study was terminated with results p…
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CLINICAL_TRIALS trial_result · 2021-01-26Clinical Trial Update: PCORI Urea Cycle Disorder Study
Clinical trial update indicating completion of a PCORI-funded study on urea cycle disorders, with results posted in 2021.
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CLINICAL_TRIALS trial_result · 2020-10-19Clinical Trial Update: Study to Evaluate the Efficacy of HepaStem in Urea Cycle Disorders Paediatric Patients (HEP002) [Phase 2]
Phase 2 clinical trial evaluating the efficacy of HepaStem in paediatric patients with urea cycle disorders has completed.
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CLINICAL_TRIALS trial_result · 2020-10-19Clinical Trial Update: Safety Study of HepaStem for the Treatment of Urea Cycle Disorders (UCD) and Crigler-Najjar Syndrome (CN) [Phase 1, Phase 2]
Phase 1/2 safety study of HepaStem for urea cycle disorders and Crigler-Najjar syndrome has completed.
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CLINICAL_TRIALS trial_result · 2018-05-04Clinical Trial Update: To Evaluate the Safety of Long-term Use of HPN-100 in the Management of Urea Cycle Disorders (UCDs) [Phase 4]
Phase 4 clinical trial evaluating the long-term safety of HPN-100 for managing urea cycle disorders has completed, with results posted in 20…
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CLINICAL_TRIALS trial_result · 2018-03-07Clinical Trial Update: Pilot Study: Urea Cycle Disorders Practice Patterns and Outcomes Assessment
A completed pilot study assessing practice patterns and patient outcomes in urea cycle disorders. The study aims to inform broader managemen…
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CLINICAL_TRIALS trial_result · 2017-12-08Clinical Trial Update: Comparative Efficacy of Phenylbutyrate (PBA) vs. Benzoate in Urea Cycle Disorders [NA]
A completed clinical trial comparing phenylbutyrate to benzoate for urea cycle disorders with results posted in 2017.
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CLINICAL_TRIALS trial_result · 2017-03-09Clinical Trial Update: Protein Sorbent Properties of Montmorillonite in Vitro and in Vivo Models [NA]
A completed study evaluating montmorillonite's protein-sorbent properties in vitro and in vivo models relevant to urea cycle disorders and o…
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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 · 2016-02-08Clinical Trial Update: Human Heterologous Liver Cells for Infusion in Children With Urea Cycle Disorders [Phase 2]
Phase 2 clinical trial evaluating allogeneic hepatocyte infusions for children with urea cycle disorders was terminated.
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CLINICAL_TRIALS trial_result · 2015-10-02Clinical Trial Update: Oxidative Stress, Inflammation and Acute Decompensation in Urea Cycle Disorders
The clinical trial investigating oxidative stress, inflammation, and acute decompensation in urea cycle disorders has been terminated. No re…
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CLINICAL_TRIALS trial_result · 2015-06-01Clinical Trial Update: Pilot Study For Hypothermia Treatment In Hyperammonemic Encephalopathy In Neonates And Very Young Infants [Phase 2]
This update reports the completion of a Phase 2 pilot trial evaluating hypothermia treatment for hyperammonemic encephalopathy in neonates a…
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CLINICAL_TRIALS trial_result · 2015-05-28Clinical Trial Update: Study of the Safety, Pharmacokinetics and Efficacy of HPN-100, in Pediatric Subjects With Urea Cycle Disorders (UCDs) [Phase 3]
Phase 3 study of HPN-100 in pediatric patients with urea cycle disorders has completed and results were posted in 2015.
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CLINICAL_TRIALS trial_result · 2015-05-12Clinical Trial Update: Dose-Escalation Safety Study of HPN-100 to Treat Urea Cycle Disorders [Phase 2]
This clinical trial update reports a Phase 2 dose-escalation safety study of HPN-100 for urea cycle disorders, completed with results posted…
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CLINICAL_TRIALS trial_result · 2013-12-25Clinical Trial Update: Study of the Safety and Tolerability of HPN-100 Compared to Sodium Phenylbutyrate in Children With Urea Cycle Disorders [Phase 2]
Phase 2 trial comparing HPN-100 to sodium phenylbutyrate in children with urea cycle disorders to assess safety and tolerability. The study …
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CLINICAL_TRIALS trial_result · 2013-08-15Clinical Trial Update: Study of the Safety of HPN (Hyperion)-100 for the Long-Term Treatment of Urea Cycle Disorders (Treat UCD) [Phase 3]
Phase 3 trial assessing the safety of HPN (Hyperion)-100 for long-term treatment of urea cycle disorders has completed, with results posted …
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CLINICAL_TRIALS trial_result · 2013-08-12Clinical Trial Update: Efficacy and Safety of HPN-100 for the Treatment of Adults With Urea Cycle Disorders [Phase 3]
Amgen completed a Phase 3 trial evaluating the efficacy and safety of HPN-100 in adults with urea cycle disorders, with results published in…
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CLINICAL_TRIALS trial_result · 2013-06-26Clinical Trial Update: Study in Healthy Subjects, Patients With Urea Cycle Disorders (UCD) and Carriers of UCD Mutations to Evaluate Urea Cycle Function [NA]
A completed clinical trial by Cytonet assessing urea cycle function in healthy subjects, patients with urea cycle disorders, and carriers of…
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CLINICAL_TRIALS trial_result · 2010-01-15Clinical Trial Update: Study to Evaluate 13 C Isotope Ratio Measurement for Urea Cycle Capacity Assessment [NA]
This update notes the completion of a study evaluating 13C isotope ratio measurement for urea cycle capacity assessment, sponsored by Cytone…