Ignite Creation Date:
2024-05-06 @ 5:11 PM
Last Modification Date:
2024-10-26 @ 2:23 PM
Study NCT ID:
NCT05222178
Status:
TERMINATED
Last Update Posted:
2023-10-10
First Post:
2021-12-21
Brief Title:
Safety and Efficacy of HMI-103 in Participants With Classical PKU Due to PAH Deficiency
Sponsor:
Homology Medicines Inc
Organization:
Homology Medicines Inc
Study Overview
Official Title:
A Phase 1 Open-Label Dose-Escalation Study to Evaluate the Safety and Efficacy of HMI-103 Administered Intravenously in Adult Participants With Classical PKU Due to PAH Deficiency
Status:
TERMINATED
Status Verified Date:
2023-10
Last Known Status:
None
Delayed Posting:
No
If Stopped, Why?:
Homology Medicines Inc has discontinued the development of this program
Has Expanded Access:
False
If Expanded Access, NCT#:
N/A
Has Expanded Access, NCT# Status:
N/A
Acronym:
None
Brief Summary:
This is an open-label sequential ascending dose-escalation Phase 1 study to evaluate the safety and efficacy of a single intravenous IV administration of HMI-103 a gene editing development candidate in adult participants aged 18 to 55 years inclusive with classical PKU due to PAH deficiency who have uncontrolled disease despite Phe restricted dietary management
Detailed Description:
This is an open-label sequential ascending dose-escalation Phase 1 study to evaluate the safety and efficacy of a single intravenous administration of HMI-103 a gene editing development candidate in adult participants aged 18 to 55 years inclusive with classical PKU due to PAH deficiency who have uncontrolled disease despite Phe-restricted dietary management Up to 3 dose levels of HMI-103 may be investigated At a given dose level 3 participants are planned to be enrolled and dosed Participant dosing will be staggered
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