Ignite Creation Date:
2025-12-25 @ 12:29 AM
Ignite Modification Date:
2025-12-31 @ 11:12 PM
Study NCT ID:
NCT04480567
Status:
ACTIVE_NOT_RECRUITING
Last Update Posted:
2024-12-12
First Post:
2020-07-08
Is NOT Gene Therapy:
True
Has Adverse Events:
False
Brief Title:
AAV Gene Therapy Study for Subjects with PKU
Sponsor:
BioMarin Pharmaceutical
Organization:
Study Overview
Official Title:
A Phase 1/2 Open-Label, Dose Escalation Study to Determine the Safety and Efficacy of BMN 307, an Adeno-Associated Virus Vector-Mediated Gene Transfer of Human Phenylalanine Hydroxylase in Subjects with Phenylketonuria
Status:
ACTIVE_NOT_RECRUITING
Status Verified Date:
2024-12
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 is a Phase 1/2, open-label, dose escalation study to evaluate the safety, efficacy and tolerability of BMN 307 in adult PKU subjects with PAH deficiency. Participants will receive a single administration of BMN 307 and will be followed for safety and efficacy.
Detailed Description:
None
Study Oversight
Has Oversight DMC:
True
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?:
False
Is an FDA AA801 Violation?: