Status:
TERMINATED
Study to Characterize Rate of Ureagenesis in Patients With Ornithine Transcarbamylase (OTC) Deficiency
Lead Sponsor:
Ultragenyx Pharmaceutical Inc
Conditions:
Ornithine Transcarbamylase Deficiency
Eligibility:
All Genders
18+ years
Brief 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 a...
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 as...
Eligibility Criteria
Inclusion
- Key
- Willing and able to provide written informed consent.
- For symptomatic patients:
- Confirmed clinical diagnosis of OTC deficiency and enzymatic, biochemical, or molecular testing.
- Documented history of ≥ 1 symptomatic hyperammonemic episode with ammonia level ≥ 100 μmol/L
- Patients on ongoing daily ammonia scavenger therapy must be at a stable dose(s) for ≥ 4 weeks prior to Visit 1 (Baseline)
- For asymptomatic patients: confirmed diagnosis of OTC deficiency by family history and documented by molecular testing.
- Willing and able to comply with the study procedures and requirements, including clinic visits, blood and urine collections, questionnaires, and cognitive assessments.
- Key
Exclusion
- Liver transplant, including hepatocyte cell therapy/transplant.
- History of liver disease
- Significant hepatic inflammation or cirrhosis
- Participation in another investigational medicine study within 3 months of Screening
- Participation (current or previous) in another gene transfer study
- Pregnant or nursing
- Other protocol specific criteria may apply
Key Trial Info
Start Date :
October 6 2020
Trial Type :
OBSERVATIONAL
Allocation :
ACTUAL
End Date :
December 15 2021
Estimated Enrollment :
1 Patients enrolled
Trial Details
Trial ID
NCT04717453
Start Date
October 6 2020
End Date
December 15 2021
Last Update
February 18 2022
Active Locations (1)
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1
PPD Phase 1 Clinic - Orlando
Orlando, Florida, United States, 32806