Ignite Creation Date:
2025-12-26 @ 11:13 PM
Ignite Modification Date:
2025-12-26 @ 11:13 PM
Study NCT ID:
NCT06692712
Status:
NOT_YET_RECRUITING
Last Update Posted:
2024-11-18
First Post:
2024-11-14
Is NOT Gene Therapy:
False
Has Adverse Events:
False
Brief Title:
Phase 3, Open-label Study to Assess the Efficacy and Safety of a Single Lumbar Intrathecal Administration of MELPIDA in Individuals with Hereditary Spastic Paraplegia Type 50 (SPG50) Versus Matched Prospective Concurrent Controls.
Sponsor:
Elpida Therapeutics SPC
Organization:
Study Overview
Official Title:
Intrathecal Administration of MELPIDA (AAV9/AP4M1) for Hereditary Spastic Paraplegia Type 50 (SPG50): a Phase 3, Open-Label Trial with Matched Prospective Concurrent Control Arm
Status:
NOT_YET_RECRUITING
Status Verified Date:
2024-11
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:
SPG50
Brief Summary:
Phase 3, open-label study to assess the efficacy and safety of a single lumbar intrathecal administration of MELPIDA in individuals with Hereditary Spastic Paraplegia Type 50 (SPG50).
Detailed Description:
MELPIDA is an AAV9-based gene therapy vector that expresses the fully functional form of AP4M1 under the control of a synthetic promoter. MELPIDA will be delivered intrathecally and is designed to achieve stable, potentially life-long expression of AP4M1 in non-dividing cells. This clinical study is a pivotal open-label phase 3 study designed to assess safety and efficacy of MELPIDA in individuals with SPG50.
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?: