Ignite Creation Date:
2024-05-05 @ 11:07 PM
Last Modification Date:
2024-10-26 @ 10:28 AM
Study NCT ID:
NCT01257737
Status:
COMPLETED
Last Update Posted:
2018-05-04
First Post:
2010-12-02
Brief Title:
To Evaluate the Safety of Long-term Use of HPN-100 in the Management of Urea Cycle Disorders UCDs
Sponsor:
Horizon Therapeutics LLC
Organization:
Horizon Pharma Ireland Ltd Dublin Ireland
Study Overview
Official Title:
Long Term Use of HPN-100 in Urea Cycle Disorders
Status:
COMPLETED
Status Verified Date:
2024-07
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 was an open-label long-term safety study of HPN-100 RAVICTI glycerol phenylbutyrate in participants with a urea cycle disorder UCD who completed the safety extensions of HPN-100-005 NCT00947544 HPN-100-005SE HPN-100-006 NCT00947297 HPN-100-007 or HPN-100-012 NCT01347073 HPN-100-012SE The initial studies were 1- to 2-week crossover studies and their associated safety extensions were 12-month open-label studies All participants who completed the initial studies were eligible to enroll in the associated safety extension studies and new participants were also permitted to enroll directly into the safety extension studies
Detailed Description:
The duration of treatment in this study was open-ended Participants were to return for clinic visits as prescribed by the investigator and were to be seen at a minimum of every 6 months At each clinic visit participants were queried about any adverse events AEs or hyperammonemic crises HACs that occurred since the last visit Physical and neurological examinations were performed and blood samples were collected for the analysis of ammonia amino acid panels and routine clinical laboratory safety tests Participants underwent neuropsychological testing at baseline every 12 months thereafter and at the final study visit
Study acquired from Horizon in 2024
Study acquired from Horizon in 2024
Study Oversight
Has Oversight DMC:
None
Is a FDA Regulated Drug?:
True
Is a FDA Regulated Device?:
False
Is an Unapproved Device?:
None
Is a PPSD?:
None
Is a US Export?:
None
Is an FDA AA801 Violation?:
None