Ignite Creation Date:
2025-12-24 @ 11:46 AM
Ignite Modification Date:
2025-12-31 @ 1:29 PM
Study NCT ID:
NCT05166161
Status:
RECRUITING
Last Update Posted:
2025-05-08
First Post:
2021-12-08
Is NOT Gene Therapy:
True
Has Adverse Events:
False
Brief Title:
A Long-Term Safety Study of PTC923 in Participants With Phenylketonuria
Sponsor:
PTC Therapeutics
Organization:
Study Overview
Official Title:
A Phase 3 Open-Label Study of PTC923 (Sepiapterin) in Phenylketonuria
Status:
RECRUITING
Status Verified Date:
2025-05
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:
The main purpose of this study is to evaluate the long-term safety of PTC923 in participants with phenylketonuria, and to evaluate the changes from baseline in dietary phenylalanine (Phe)/protein consumption.
Detailed Description:
Eligible participants are:
Feeder participants: those who have completed a Phase 3 PTC Therapeutics (PTC) sponsored feeder study.
Non-feeder controlled participants: those who have not completed a feeder study and have blood Phe levels \<360 μmol/L at study entry.
Non-feeder uncontrolled participants: those who have not completed a feeder study and have blood Phe levels ≥360 μmol/L at study entry.
Feeder participants: those who have completed a Phase 3 PTC Therapeutics (PTC) sponsored feeder study.
Non-feeder controlled participants: those who have not completed a feeder study and have blood Phe levels \<360 μmol/L at study entry.
Non-feeder uncontrolled participants: those who have not completed a feeder study and have blood Phe levels ≥360 μmol/L at study entry.
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?: