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Intrathecal Administration of scAAV9/JeT-GAN for the Treatment of Giant Axonal Neuropathy
NCT02362438 · View on ClinicalTrials.gov ↗
Study Summary
Title: Intrathecal Administration of scAAV9/JeT-GAN for the Treatment of Giant Axonal Neuropathy Background: \- The Gigaxonin gene lets the body make a protein chemical called Gigaxonin. Nerves need Gigaxonin to work properly. Giant Axonal Neuropathy (GAN) causes a shortage of functional Gigaxonin. Nerves stop working normally in people with GAN. This causes problems with walking and sometimes with eating, breathing, and many other activities. GAN has no cure. Over time, GAN can shorten a person s life. Researchers want to see if a gene transfer treatment may help people with GAN. Objectives: \- To see if a gene transfer is safe and shows potential to help people with GAN. Eligibility: \- People age 3 and older with GAN. Design: * For 1 month following gene transfer participants must live full-time within 100 miles of the NIH. * Participants will be screened by phone and in person. They will take many tests. Some are listed below. Their medical records will be reviewed. Their caregivers may be contacted. * Participants will have a total of about 27 visits, weekly, monthly, and then yearly over 15 years. They will include many of the tests below. * Physical and nervous system exams. * Blood, urine, and stool samples. * Nerve, lung, heart, and eye tests. * Questionnaires. * MRI scans, nerve biopsies, and spinal taps. Participants will be sedated for some tests. * Speech, memory, muscle, and mobility tests. * Skin biopsy (small sample removed). * Participants will take many medicines. Some require intravenous lines. * Participants will get the gene transfer through an injection by spinal tap into their cerebrospinal fluid, which flows around the brain and spinal cord. The genes are packed in a modified virus that carries the genes to cells in their body. Participants safety is not guaranteed.
Conditions Studied
Interventions
- GENETIC scAAv9/JeT-GAN
Study Locations (1)
Maryland
- National Institutes of Health Clinical Center — Bethesda
Trial Details
| Field | Value |
|---|---|
| Enrollment Target | 14 participants |
| Start Date | 2015-04-24 |
| Est. Completion | 2035-04-01 |
| Phase | Phase 1 |
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Full Details on ClinicalTrials.gov ↗What the Registry Record Tells You About NCT02362438
The ClinicalTrials.gov registry entry for NCT02362438 describes a study currently listed as active not recruiting. It is categorized as Phase 1, which is the standard way researchers label where a study sits along the investigational pathway from early safety work through later efficacy and post-marketing evaluation. The registered enrollment target is 14 participants, a figure that helps gauge the scale of data the investigators plan to collect. The listed sponsor is National Institute of Neurological Disorders and Stroke (NINDS), which has 339 total studies on file at ClinicalTrials.gov, and sponsors are the parties responsible for study design, oversight, and regulatory filings.
The record links to 2 conditions, with Giant Axonal Neuropathy appearing as the primary indexed condition, and to 1 intervention — of which scAAv9/JeT-GAN is the first listed. Interventions can include drugs, devices, procedures, behavioral programs, or observational arms, and each is tracked as a separate registry field so that downstream queries can filter accurately. When a trial lists multiple interventions, it usually reflects a multi-arm design or a comparison protocol rather than a single treatment being tested in isolation. The brief summary published in the registry is the clearest source of protocol intent and should be read before drawing conclusions from any sidebar tags.
Geographic footprint matters for practical reasons: NCT02362438 reports 1 study location spanning 1 distinct geographic area — top geographies include Maryland. A larger site network tends to correlate with broader recruitment capacity, but it does not imply anything about study quality, and site-level enrollment status can diverge from the overall registry status shown above. Every data point on this page comes from the public ClinicalTrials.gov dataset and is reproduced here for reference only; it is not a medical recommendation, an endorsement of the sponsor, or an invitation to enroll. Verify current status, eligibility criteria, and contact details directly at ClinicalTrials.gov, and discuss any participation decision with your own healthcare provider.
Frequently Asked Questions
What is clinical trial NCT02362438 about?
NCT02362438 is a clinical study titled "Intrathecal Administration of scAAV9/JeT-GAN for the Treatment of Giant Axonal Neuropathy". Title: Intrathecal Administration of scAAV9/JeT-GAN for the Treatment of Giant Axonal Neuropathy Background: \- The Gigaxonin gene lets the body make a protein chemical called Gigaxonin. Nerves need Gigaxonin to work properly. Giant Axonal Neuropathy (GAN) causes a shortage of functional Gigaxonin...
What is the current status of trial NCT02362438?
This trial is currently active not recruiting. It is a Phase 1 study. The enrollment target is 14 participants. The study started on 2015-04-24. Estimated completion is 2035-04-01.
What conditions does trial NCT02362438 study?
This clinical trial studies the following conditions: Giant Axonal Neuropathy, Gene Transfer. These conditions were identified from the trial registry and reflect the primary focus areas of the research.
What interventions are being tested in trial NCT02362438?
The interventions under investigation include: scAAv9/JeT-GAN (GENETIC). Each intervention is being evaluated for safety and efficacy as part of this clinical study.
Who is sponsoring clinical trial NCT02362438?
This trial is sponsored by National Institute of Neurological Disorders and Stroke (NINDS), which has 339 total clinical trials registered on ClinicalTrials.gov. The sponsor is responsible for the study's design, funding, and regulatory compliance.
Where is trial NCT02362438 being conducted?
This trial has 1 study location across Maryland. Contact the study sites directly through ClinicalTrials.gov for enrollment availability.
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