NCT04717453

Study to Characterize Rate of Ureagenesis in Patients With Ornithine Transcarbamylase (OTC) Deficiency

A Study to Characterize Rate of Ureagenesis Utilizing Oral [1-13C] Sodium Acetate in the Spectrum of Severity of Patients With Ornithine Transcarbamylase (OTC) Deficiency

• Status: Terminated
• Phase: —
• Study type: Observational
• Enrollment: 1 (actual)
• Sponsor: Ultragenyx Pharmaceutical Inc · Industry
• Sex: All
• Age: 18 Years
• Healthy volunteers: Not accepted

Summary

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 ureagenesis and executive and verbal function and characterize the association of rate of ureagenesis and patient-reported functional status.

Detailed description

Study DTX301-CL102 is a noninterventional, observational study to characterize the rate of ureagenesis and to assess neurocognition and functional status in the spectrum of OTC deficiency and their association with biochemical characteristics. \[1-13C\]Sodium acetate will be administered orally as a tracer to measure the rate of ureagenesis.

Conditions

• Ornithine Transcarbamylase Deficiency

Interventions

Type	Name	Description
OTHER	No Intervention	No Intervention

Timeline

• Start date: 2020-10-06
• Primary completion: 2021-12-15
• Completion: 2021-12-15
• First posted: 2021-01-22
• Last updated: 2022-02-18

Locations

1 site across 1 country: United States

Regulatory

• FDA-regulated drug study