Ignite Creation Date:
2024-05-06 @ 2:58 PM
Last Modification Date:
2024-10-26 @ 1:40 PM
Study NCT ID:
NCT04480567
Status:
ACTIVE_NOT_RECRUITING
Last Update Posted:
2021-10-07
First Post:
2020-07-08
Brief Title:
AAV Gene Therapy Study for Subjects With PKU
Sponsor:
BioMarin Pharmaceutical
Organization:
BioMarin Pharmaceutical
Study Overview
Official Title:
A Phase 12 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:
2021-10
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 12 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:
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