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NCT02362438 · ClinicalTrials.gov registry record · Phase 1
Intrathecal Administration of scAAV9/JeT-GAN for the Treatment of Giant Axonal Neuropathy
A Phase 1 study of Giant Axonal Neuropathy and Gene Transfer, sponsored by National Institute of Neurological Disorders and Stroke (NINDS).
- Active
- Registry status
- Phase 1
- Development phase
- 14
- Enrollment target
- 1
- Study location
NCT02362438: Active Phase 1 study of Giant Axonal Neuropathy and Gene Transfer, sponsored by National Institute of Neurological Disorders and Stroke (NINDS).
NCT02362438 is a Phase 1 study of Giant Axonal Neuropathy and Gene Transfer that is active but no longer recruiting, run by National Institute of Neurological Disorders and Stroke (NINDS). The registered enrollment target is 14 participants, below the 60-participant average among 29,502 other Phase 1 trials with a reported enrollment target (77% lower). The trial reports 1 study location across 1 state. According to ClinicalTrials.gov, the official US trial registry.
The verdict
NCT02362438, a Phase 1 study of Giant Axonal Neuropathy and Gene Transfer, is active but no longer recruiting, sponsored by National Institute of Neurological Disorders and Stroke (NINDS).
- ACTIVE NOT RECRUITING
- Registry status
- Phase 1
- Development phase
- 14 participants
- Enrollment target
- 1
- Study location
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.
Primary Outcome
Adverse event reports will be used to assess safety
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 |
What the registry record for NCT02362438 still lists
NCT02362438 is an interventional study that assigns participants to a tested intervention. The registry caps enrollment at 14 participants, a relatively small participant target, below the 60-participant average among 29,502 other Phase 1 trials with a reported enrollment target (77% lower).
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.
NCT02362438 reports a single indexed study location in Maryland.
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.
What interventions are being tested in trial NCT02362438?
The interventions under investigation include: scAAv9/JeT-GAN (GENETIC).
Who is sponsoring clinical trial NCT02362438?
This trial is sponsored by National Institute of Neurological Disorders and Stroke (NINDS), which has 567 total clinical trials registered on ClinicalTrials.gov.
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.
Learn More About Clinical Trials
How this trial's enrollment target compares
Where NCT02362438's enrollment target sits among peer trials
14 1844th of 2000 higher than 154 of 2,000 other Phase 1 trials
participants (enrollment target), bucketed by value
Each bar is a band; taller bars hold more other Phase 1 trials. The dashed line + filled bar mark this entry. Hover or tap any bar for its full count and share, and where it sits relative to this entry.
Source ClinicalTrials.gov registry export · 2026-08-08
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