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

NCT06069687 · Status: ACTIVE_NOT_RECRUITING · Phase: PHASE1 · Type: INTERVENTIONAL · Enrollment: 1

Last updated 2025-05-14

No results posted yet for this study

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

BIOLOGICAL

MELPIDA

A single intrathecal infusion of 10 mL at 1E14 vg/mL for a total dose of 1E15 vg

Sponsors & Collaborators

  • The Hospital for Sick Children

    lead OTHER

Study Design

Allocation
NA
Purpose
OTHER
Masking
NONE
Model
SINGLE_GROUP

Eligibility

Min Age
0 Years
Max Age
4 Years
Sex
MALE
Healthy Volunteers
No

Timeline & Regulatory

Start
2022-03-11
Primary Completion
2027-03-31
Completion
2027-03-31

Countries

  • Canada

Study Locations

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Read the full study record

This page highlights key information. For complete eligibility criteria, study locations, investigator contacts, and the full protocol, visit the original record on ClinicalTrials.gov.

View NCT06069687 on ClinicalTrials.gov