NCT06692712

Phase 3 Efficacy Study With Concurrent Control of IT MELPIDA in SPG50.Concurrent Controls.

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: Recruiting
• Phase: Phase 3
• Study type: Interventional
• Enrollment: 24 (estimated)
• Sponsor: Elpida Therapeutics SPC · Industry
• Sex: All
• Age: 21 Months – 78 Months
• Healthy volunteers: Not accepted

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.

Conditions

• Hereditary Spastic Paraplegia Type 50

Interventions

Type	Name	Description
GENETIC	MELPIDA	Gene Therapy agent

Timeline

• Start date: 2026-02-01
• Primary completion: 2032-02-28
• Completion: 2032-06-01
• First posted: 2024-11-18
• Last updated: 2026-01-21

Locations

2 sites across 2 countries: United States, Spain

Regulatory

• FDA-regulated drug study