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Virus Therapy

Gene Therapy for Spastic Paraplegia

Phase 1 & 2

Recruiting

Led By Susan T. Iannaccone, MD, FAAN

Research Sponsored by University of Texas Southwestern Medical Center

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, confirmed pathogenic variants in the AP4M1 gene

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

Timeline

Screening 3 weeks

Treatment Varies

Follow Up 60 months

Awards & highlights

Study Summary

This trial will test a new drug to treat SPG50 by injecting it into the spine, measuring safety and seeing if it has an effect on the disease.

Who is the study for?

Children aged 1-10 with SPG50, a genetic disorder causing paralysis and intellectual disability. They must be able to take steps independently or with help, stand for over 5 seconds, and have a confirmed AP4M1 gene mutation. Excluded are those on certain medications, unable to undergo MRI or lumbar puncture, recently in other trials, or with conditions that interfere with the study.Check my eligibility

What is being tested?

The trial tests MELPIDA's safety and tolerability through intrathecal injection aimed at delivering functional human AP4M1 cDNA to neurons affected by SPG50. It will monitor adverse events related to treatment and assess any improvements in disease symptoms.See study design

What are the potential side effects?

Potential side effects include reactions specific to gene therapy such as immune responses against the vector or transgene product, issues from intrathecal administration like headache or back pain, and general risks associated with sedation required for procedures.

Eligibility Criteria

Inclusion Criteria

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

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My SPG50 disease diagnosis was confirmed through genetic testing 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 can stand for more than 5 seconds.

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My ankle stiffness is mild or moderate.

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I am between 1 and 10 years old.

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I can walk 5 steps on my own or with a walker.

Timeline

Screening ~ 3 weeks3 visits

Treatment ~ Varies

Follow Up ~ 60 months

Screening ~ 3 weeks

Treatment ~ Varies

Follow Up ~60 months

This trial's timeline: 3 weeks for screening, Varies for treatment, and 60 months for reporting.

Treatment Details

Study Objectives

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

Primary outcome measures

Incidence of unanticipated treatment-related toxicities, Grade 3 or higher in participants with SPG50

Secondary outcome measures

Stability or improvement in spasticity in participants with SPG50 as measured by the Modified Ashworth scale (MAS)

Stability or improvement in spasticity in participants with SPG50 as measured by the Tardieu scale

Trial Design

1Treatment groups

Experimental Treatment

Group I: Treatment ArmExperimental Treatment1 Intervention

MELPIDA, a gene therapy product

0 / 6Eligibility criteria met

Find a Location

Who is running the clinical trial?

University of Texas Southwestern Medical CenterLead Sponsor

1,048 Previous Clinical Trials

1,053,733 Total Patients Enrolled

Cure SPG50UNKNOWN

Susan T. Iannaccone, MD, FAANPrincipal InvestigatorUT Southwestern Medical Center

Media Library

MELPIDA (Virus Therapy) Clinical Trial Eligibility Overview. Trial Name: NCT05518188 — Phase 1 & 2

Spastic Paraplegia Research Study Groups: Treatment Arm

Spastic Paraplegia Clinical Trial 2023: MELPIDA Highlights & Side Effects. Trial Name: NCT05518188 — Phase 1 & 2

MELPIDA (Virus Therapy) 2023 Treatment Timeline for Medical Study. Trial Name: NCT05518188 — Phase 1 & 2

Frequently Asked Questions

These questions and answers are submitted by anonymous patients, and have not been verified by our internal team.

Does this research project encompass people who have not yet reached retirement age?

"Participants that meet the qualifying criteria of this study must be between 1 and 10 years old."

Answered by AI

Are there open slots for participants in this clinical research?

"Per the information on clinicaltrials.gov, recruitment for this trial is ongoing and was initially posted on February 15th 2023. The data has been most recently updated as of January 31st 2023."

Answered by AI

What is the total participant count for this clinical experiment?

"Affirmative. Records hosted on clinicaltrials.gov indicate that this study is actively searching for test subjects, having first been posted in February 15th 2023 and last updated January 31st 2023. The trial needs to acquire 2 participant from a single medical centre."

Answered by AI

What eligibility criteria must be met to participate in this experiment?

"Two minors, within the age bracket of 1 and 10 years old who have microcephaly are now eligible to join this medical trial. In addition to these criteria, participants must possess a homozygous or compound heterozygous variant in AP4M1 gene; display signs of SPG50-related neurologic dysfunction; receive parental/guardian approval prior to taking part in the study; be able comply with all protocol procedures as well as demonstrate an ability to stand for over 5 seconds OR take five steps independently or with aid of a walker OR exhibit Modified Ashworth Scale score 2 or lower (Ankles)."

Answered by AI

Recent research and studies

clinicaltrials.govStudy

Melpida: Recombinant Adeno-associated Virus (Serotype 9) Encoding a Codon Optimized Human AP4M1 Transgene (hAP4M1opt)

Susan T Iannaccone 2023. "Melpida: Recombinant Adeno-associated Virus (Serotype 9) Encoding a Codon Optimized Human AP4M1 Transgene (hAP4M1opt)". ClinicalTrials.gov. https://clinicaltrials.gov/ct2/show/NCT05518188.