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NCT07543991 · ClinicalTrials.gov registry record · Phase 1
Intraneural Administration of scAAV9/JeT-GAN Into the Vagus Nerve for Patients With Giant Axonal Neuropathy (GAN)
A Phase 1 study, sponsored by University of Texas Southwestern Medical Center.
- Recruiting
- Registry status
- Phase 1
- Development phase
- 4
- Enrollment target
NCT07543991 is a Phase 1 study that is actively recruiting participants, run by University of Texas Southwestern Medical Center. The registered enrollment target is 4 participants.
The verdict
NCT07543991, a Phase 1 study, is actively recruiting participants, sponsored by University of Texas Southwestern Medical Center.
- RECRUITING
- Registry status
- Phase 1
- Development phase
- 4 participants
- Enrollment target
Study Summary
Giant axonal neuropathy (GAN) is a rare pediatric disorder caused by autosomal recessive mutations in the GAN gene. GAN is a multisystem, neurodegenerative disorder affecting the peripheral nervous system (PNS), central nervous system (CNS) and autonomic nervous system (ANS). GAN is a fatal disease with many patients not surviving past early adulthood due to aspiration pneumonia and pulmonary complications. Currently, there are no approved drugs or other therapies for the treatment of GAN; and only supportive care therapies exist, leaving an unmet medical need to treat this rare, progressive, and ultimately fatal neurodegenerative disease. The drug used in this study (scAAV9/JeT-GAN) has been studied in a previous gene therapy clinical trial by which the drug was administered as a single injection into the spinal canal (intrathecal \[IT\] administration) to treat the symptoms associated with the CNS and PNS neurodegeneration; however, this administration method did not address the symptoms associated with neurodegeneration of the ANS. To treat the symptoms associated with ANS, this study has been designed to evaluate the safety and tolerability of a single dose of scAAV9/JeT-GAN administered directly into the left vagus nerve (intraneurally) in participants who have previously received scAAV9/JeT-GAN administered intrathecally. This study involves the use of an investigational drug called scAAV9/JeT-GAN "Investigational" means that the drug has not been approved by the U.S. Food \& Drug Administration (FDA) for the treatment of GAN and the progression of neurodegeneration to the CNS, PNS and ANS. This is the first study in humans to administer the drug directly into the left vagus nerve. We want to find out what effects, good and/or bad, scAAV9/JeT-GAN has when administered directly into the vagus nerve. The safety of intrathecal (IT) administration of scAAV9/JeT-GAN has been established in a prior research study; however, the people in this study will be the
Interventions
- GENETIC scAAV9/JeT-GAN
Trial Details
| Field | Value |
|---|---|
| Enrollment Target | 4 participants |
| Start Date | 2026-03-01 |
| Est. Completion | 2032-06-30 |
| Phase | Phase 1 |
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Full Details on ClinicalTrials.gov ↗What the Registry Record Tells You About NCT07543991
The ClinicalTrials.gov registry entry for NCT07543991 describes a study currently listed as recruiting, categorized as Phase 1. The registered enrollment target is 4 participants, a figure that helps gauge the scale of data the investigators plan to collect. The listed sponsor is University of Texas Southwestern Medical Center, which has 837 total studies on file at ClinicalTrials.gov.
The record links to 0 conditions, and to 1 intervention - of which scAAV9/JeT-GAN is the first listed.
NCT07543991 reports 0 study locations.
Frequently Asked Questions
What is clinical trial NCT07543991 about?
NCT07543991 is a clinical study titled "Intraneural Administration of scAAV9/JeT-GAN Into the Vagus Nerve for Patients With Giant Axonal Neuropathy (GAN)". Giant axonal neuropathy (GAN) is a rare pediatric disorder caused by autosomal recessive mutations in the GAN gene. GAN is a multisystem, neurodegenerative disorder affecting the peripheral nervous system (PNS), central nervous system (CNS) and autonomic nervous system (ANS). GAN is a fatal disease...
What is the current status of trial NCT07543991?
This trial is currently recruiting. It is a Phase 1 study. The enrollment target is 4 participants. The study started on 2026-03-01. Estimated completion is 2032-06-30.
What interventions are being tested in trial NCT07543991?
The interventions under investigation include: scAAV9/JeT-GAN (GENETIC).
Who is sponsoring clinical trial NCT07543991?
This trial is sponsored by University of Texas Southwestern Medical Center, which has 837 total clinical trials registered on ClinicalTrials.gov.
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