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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 belowSelect...
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:Select...
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
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 moreSecondary 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)
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Who is running the clinical trial?
The Hospital for Sick ChildrenLead Sponsor
692 Previous Clinical Trials
6,946,742 Total Patients Enrolled
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