NCT06069687

Administration of MELPIDA to Determine the Safety and Efficacy for Patients With Spastic Paraplegia Type 50

A Phase 1 Open-label Intrathecal Administration of MELPIDA to Determine the Safety and Efficacy for Patients With Spastic Paraplegia Type 50 (SPG50) Caused by a Mutation in the AP4M1 Gene

• Status: Active Not Recruiting
• Phase: Phase 1
• Study type: Interventional
• Enrollment: 1 (actual)
• Sponsor: The Hospital for Sick Children · Academic / Other
• Sex: Male
• Age: 0 Years – 4 Years
• Healthy volunteers: Not accepted

Summary

This will be a first-in-human Phase I, open-label, single dose clinical study of MELPIDA administered intrathecally (IT) through a lumbar puncture (LP) to a single subject with confirmed pathogenic mutations in the Adaptor Related Protein Complex 4 Subunit Mu 1 (AP4M1) gene. The primary outcome will be the determination of the safety and tolerability of MELPIDA in patients with SPG50, based on development of toxicity. The secondary outcome will be a preliminary exploration of efficacy of the treatment. MELPIDA, is a recombinant serotype 9 adeno-associated virus (AAV) encoding a codon-optimized human AP4M1 transgene and will be administer to the patient via a single intrathecal infusion of 10 mL at 1E14 vg/mL for a total dose of 1E15 vg. The total study duration is 5 years post dosing and the participant will be tested at screening/baseline (-28 to -7 days), return for dosing, and then follow-up visits post-dosing on Days 7 (+/-2), 30 (+/-2), 60 (+/-2), 90 (+/-14), 180 (+/-14), 270 (+/-14), 360 (+/-14), 540 (+/-14), and 720 (+/-14) days, then annually for the last 3 years.

Conditions

• Spastic Paraplegia Type 50

Interventions

Type	Name	Description
BIOLOGICAL	MELPIDA	A single intrathecal infusion of 10 mL at 1E14 vg/mL for a total dose of 1E15 vg

Timeline

• Start date: 2022-03-11
• Primary completion: 2027-03-01
• Completion: 2027-03-01
• First posted: 2023-10-06
• Last updated: 2025-05-14

Locations

1 site across 1 country: Canada