Ignite Creation Date:
2024-05-06 @ 1:11 PM
Last Modification Date:
2024-10-26 @ 1:10 PM
Study NCT ID:
NCT03952156
Status:
TERMINATED
Last Update Posted:
2023-08-29
First Post:
2019-05-09
Brief Title:
Gene Therapy Clinical Study in Adult PKU
Sponsor:
Homology Medicines Inc
Organization:
Homology Medicines Inc
Study Overview
Official Title:
A Phase 12 Open-Label Randomized Concurrently-Controlled Dose Escalation Study to Evaluate the Safety and Efficacy of HMI-102 in Adult PKU Subjects With PAH Deficiency
Status:
TERMINATED
Status Verified Date:
2023-08
Last Known Status:
None
Delayed Posting:
No
If Stopped, Why?:
Homology Medicines 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:
pheNIX
Brief Summary:
This is a Phase 12 open-label randomized concurrently-controlled dose escalation study to evaluate the safety and efficacy of HMI-102 in adult PKU subjects with PAH deficiency Participants will receive a single administration of HMI-102 and will be followed for safety and efficacy for 1 year
Detailed Description:
Part 1 of this study will evaluate the safety and efficacy of HMI-102 gene therapy in adult subjects with PKU due to PAH deficiency Subjects will receive a single dose of HMI-102 administered intravenously Up to 3 dose levels of HMI-102 may be investigated in this study At a given dose level a minimum of 2 subjects will be enrolled and dosed Dosing of the first two subjects will be staggered Following evaluation of data from the first 2 subjects in a cohort a decision can be made to either escalate to the next dose level or expand the cohort at the selected dose level Additional doses may be added by HMI to investigate intermediate or higher doses
In Part 2 dose expansion evaluation of up to 2 dose levels is planned Subjects will be randomized to receive HMI-102 or a concurrent delayed treatment control arm Subjects in the delayed treatment control will be eligible to receive HMI-102 after 28 weeks
In Part 2 dose expansion evaluation of up to 2 dose levels is planned Subjects will be randomized to receive HMI-102 or a concurrent delayed treatment control arm Subjects in the delayed treatment control will be eligible to receive HMI-102 after 28 weeks
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