Ornithine transcarbamylase deficiency
Conditions
Brief summary
The incidence of AEs and SAEs for each dosing cohort assessed by severity and relationship to study product.
Detailed description
The change from baseline (Day 0 of Study 301OTC01) in AUC0-24 for plasma ammonia over time to 208 weeks following IV administration of DTX301, The change from baseline (the baseline of each subject before DTX301 administration in Study 301OTC01) in the rate of ureagenesis (as measured by the generation of [13 C]urea over 4 hours) as determined by gas chromatography mass spectrometry over time to 416 weeks following IV administration of DTX301 The rate of ureagenesis was assessed prior to Amendment 3; ureagenesis data collected will be analyzed as a secondary efficacy endpoint.
Interventions
Sponsors
Ultragenyx Pharmaceutical Inc.
Eligibility
Sex/Gender
All
Age
18 Years to No maximum
Design outcomes
Primary
| Measure | Time frame |
|---|---|
| The incidence of AEs and SAEs for each dosing cohort assessed by severity and relationship to study product. | — |
Secondary
| Measure | Time frame |
|---|---|
| The change from baseline (Day 0 of Study 301OTC01) in AUC0-24 for plasma ammonia over time to 208 weeks following IV administration of DTX301, The change from baseline (the baseline of each subject before DTX301 administration in Study 301OTC01) in the rate of ureagenesis (as measured by the generation of [13 C]urea over 4 hours) as determined by gas chromatography mass spectrometry over time to 416 weeks following IV administration of DTX301 The rate of ureagenesis was assessed prior to Amendment 3; ureagenesis data collected will be analyzed as a secondary efficacy endpoint. | — |
Countries
France, Spain
Outcome results
None listed