Ignite Creation Date:
2025-12-24 @ 4:13 PM
Ignite Modification Date:
2026-01-02 @ 10:38 AM
Study NCT ID:
NCT07125066
Status:
ENROLLING_BY_INVITATION
Last Update Posted:
2025-08-15
First Post:
2025-07-30
Is Gene Therapy:
True
Has Adverse Events:
False
Brief Title:
An Individual Patient, Open Label Study to Use ACER-001 to Treat Combined D,L-2 Hydroxyglutaric Aciduria (C-2HGA)
Sponsor:
Organization:
Study Design
Study Type:
INTERVENTIONAL
Expanded Access Type Individual:
None
Expanded Access Type Intermediate:
None
Expanded Access Type Treatment:
None
Patient Registry:
None
Target Duration:
None
Design Allocation:
Design Intervention Model:
Design Intervention Model Description:
Design Masking Description:
Bio Spec Retention:
None
Bio Spec Description:
None
Enrollment Count:
Enrollment Type:
Design Primary Purpose:
Design Masking:
Phases:
Phases
| Phase Brief | Phase Text | View |
|---|---|---|
| None | PHASE1 | View |
Observational Models:
Time Perspective List:
Who Masked List: