Ignite Creation Date:
2024-05-05 @ 11:30 PM
Last Modification Date:
2024-10-26 @ 10:35 AM
Study NCT ID:
NCT01347073
Status:
COMPLETED
Last Update Posted:
2024-07-11
First Post:
2011-04-29
Brief Title:
Study of the Safety Pharmacokinetics and Efficacy of HPN-100 in Pediatric Subjects With Urea Cycle Disorders UCDs
Sponsor:
Amgen
Organization:
Amgen
Study Overview
Official Title:
A Switch-Over Open-Label Study of the Safety Pharmacokinetics and Efficacy of HPN-100 Followed by Long-Term Treatment With HPN-100 in Pediatric Subjects Under 6 Years of Age With Urea Cycle Disorders UCDs
Status:
COMPLETED
Status Verified Date:
2024-06
Last Known Status:
None
Delayed Posting:
No
If Stopped, Why?:
Not Stopped
Has Expanded Access:
False
If Expanded Access, NCT#:
N/A
Has Expanded Access, NCT# Status:
N/A
Acronym:
None
Brief Summary:
This non-randomized open-label study was approximately one year in duration and consisted of a short term NaPBA to HPN-100 switchover part involving two overnight stays followed by a 12-month long term treatment period involving monthly visits
Detailed Description:
This was an open-label study consisting of a 10-day switch-over period during which subjects were switched from their prescribed dose of sodium phenylbutyrate BUPHENYLTM or NaPBA to a dose of HPN-100 that delivered the same amount of the active ingredient PBA followed by long-term treatment with HPN-100 for up to 12 months The study was designed to capture information important for evaluating safety Pharmacokinetics and efficacy while recognizing sampling limitations in young children and current standard of care Patients eligible for this study included pediatric patients from 29 days to 6 years of age with either a diagnosed or clinically suspected Urea Cycle Disorders UCD who are receiving a stable dose of the powder formulation of NaPBA Subjects were clinically stable and had been receiving a stable dose NaPBA powder for at least 5 days at the time of enrollment
During the switch-over part of the study subjects switched from NaPBA to HPN-100 in one step and had two overnight stays with 24 hour blood sampling the first of which was on Day 1 while still taking NaPBA and the second of which was on approximately Day 10 while taking HPN-100 Subjects then continued in the long-term treatment phase which was 12 months in duration
Study acquired from Horizon in 2024
During the switch-over part of the study subjects switched from NaPBA to HPN-100 in one step and had two overnight stays with 24 hour blood sampling the first of which was on Day 1 while still taking NaPBA and the second of which was on approximately Day 10 while taking HPN-100 Subjects then continued in the long-term treatment phase which was 12 months in duration
Study acquired from Horizon in 2024
Study Oversight
Has Oversight DMC:
None
Is a FDA Regulated Drug?:
None
Is a FDA Regulated Device?:
None
Is an Unapproved Device?:
None
Is a PPSD?:
None
Is a US Export?:
None
Is an FDA AA801 Violation?:
None