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Virus Therapy

Gene Therapy for Spastic Paraplegia

Phase 1

Waitlist Available

Research Sponsored by The Hospital for Sick Children

Eligibility Criteria Checklist

Specific guidelines that determine who can or cannot participate in a clinical trial

Must have

Confirmed diagnosis of SPG50 disease by genomic DNA mutation analysis demonstrating homozygous or compound heterozygous, pathogenic and/or potentially pathogenic variants in the AP4M1 gene

Clinical history or examination features consistent with SPG50 and that include neurologic dysfunction

Must not have

Inability of the patient to undergo any other procedure required in this study

Have received an investigational drug within 30 days prior to screening or plan to receive an investigational drug (other than gene therapy) during the study

Timeline

Screening 3 weeks

Treatment Varies

Follow Up through study completion, an average of 5 years

Awards & highlights

Summary

This trial tests the safety and efficacy of a gene therapy for a rare genetic disorder. The patient will be administered a single intrathecal infusion and followed for 5 years.

Who is the study for?

This trial is for children under 5 with SPG50, a genetic disorder affecting movement. They must have specific mutations in the AP4M1 gene and be able to follow study procedures. Kids can't join if they've had other recent treatments, are on certain drugs, or have conditions that make the study's procedures risky.

What is being tested?

The trial tests MELPIDA, a gene therapy delivered directly into spinal fluid to treat SPG50. It's an initial safety test involving one dose and monitoring over five years with several follow-up visits after dosing.

What are the potential side effects?

Since this is a first-in-human study of MELPIDA, potential side effects aren't fully known but may include reactions at the injection site, immune responses to the treatment or its ingredients, and typical risks associated with lumbar punctures.

Eligibility Criteria

Inclusion Criteria

You may be eligible if you check “Yes” for the criteria below

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I have SPG50, confirmed by a DNA test showing specific mutations in the AP4M1 gene.

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I have symptoms or a diagnosis of SPG50 with neurological issues.

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I am under 5 years old.

Exclusion Criteria

You may be eligible for the trial if you check “No” for criteria below:

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I cannot undergo certain procedures required in this study.

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I haven't taken any experimental drugs in the last 30 days and don't plan to during the study.

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I am allergic or cannot take MELPIDA or its components.

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I am allergic or cannot take certain immune suppression drugs.

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I have a health condition that prevents me from having a lumbar puncture or using anesthetics.

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I have a condition that makes spinal taps unsafe for me.

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I do not have brain-related issues that could affect study results.

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I do not have an active infection when starting treatment.

Timeline

Screening ~ 3 weeks3 visits

Treatment ~ Varies

Follow Up ~ through study completion, an average of 5 years

Screening ~ 3 weeks

Treatment ~ Varies

Follow Up ~through study completion, an average of 5 years

This trial's timeline: 3 weeks for screening, Varies for treatment, and through study completion, an average of 5 years for reporting.

Treatment Details

Study Objectives

Outcome measures can provide a clearer picture of what you can expect from a treatment.

Primary outcome measures

Change from baseline in nerve conduction amplitude

Change from baseline in nerve conduction velocity

Determination of liver safety

+2 more

Secondary outcome measures

Stability or improvement in spasticity

Other outcome measures

Motor function, neuropsychological, and disease burden assessments

motor function, neuropsychological, and disease burden assessments

Trial Design

1Treatment groups

Experimental Treatment

Group I: MELPIDAExperimental Treatment1 Intervention

A single intrathecal infusion of 10 mL at 1E14 vg/mL for a total dose of 1E15 vg (open-label)

0 / 11Eligibility criteria met

Find a Location

Who is running the clinical trial?

The Hospital for Sick ChildrenLead Sponsor

692 Previous Clinical Trials

6,946,742 Total Patients Enrolled

~0 spots leftby Mar 2027

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