Actively Recruiting
Intraneural Administration of scAAV9/JeT-GAN Into the Vagus Nerve for Patients With Giant Axonal Neuropathy (GAN)
Led by University of Texas Southwestern Medical Center · Updated on 2026-04-22
4
Participants Needed
1
Research Sites
330 weeks
Total Duration
On this page
AI-Summary
What this Trial Is About
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 first people to receive the drug intraneurally. As a result, information about the safety and effectiveness of the route of administration is incomplete and all of the possible side effects are not yet known.
CONDITIONS
Official Title
Intraneural Administration of scAAV9/JeT-GAN Into the Vagus Nerve for Patients With Giant Axonal Neuropathy (GAN)
Who Can Participate
Eligibility Criteria
You may qualify if you...
- Confirmed diagnosis of GAN by genetic testing showing pathogenic variants in the GAN gene
- Clinical history or symptoms of autonomic nervous system dysfunction
- Previously treated with intrathecal scAAV9/JeT-GAN and completed 5-year follow-up
- Parents or legal representatives willing to attend all study visits and provide consent
- Ability to comply with all study procedures
- Up to date on childhood vaccinations per CDC guidelines; annual influenza and COVID-19 vaccines highly recommended
- Female participants of child-bearing potential must have negative pregnancy test at screening and agree to use effective birth control during the study
You will not qualify if you...
- Unable to participate in study procedures as determined by investigators
- Unable to be safely sedated according to clinical anesthesiologist
- Existing illnesses or chronic drug treatments that pose risks for gene therapy
- Significant CNS impairments or behavioral issues unrelated to GAN that affect study results
- Received any investigational drug within 30 days before screening or plan to receive other investigational drugs during study
- Currently in another interventional clinical trial
- Experienced serious adverse event related to scAAV9/JeT-GAN in previous trial
- Allergic or contraindicated to scAAV9/JeT-GAN or its ingredients
- Contraindicated to immune suppression medications used in this study
- Abnormal lab values (e.g., liver enzymes, creatinine, hemoglobin, white blood cell count) outside set limits before gene therapy
AI-Screening
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Trial Site Locations
Total: 1 location
1
Children's Health
Dallas, Texas, United States, 75235
Actively Recruiting
Research Team
S
Samantha Bridges, BSN
CONTACT
K
Kristy Riddle, BSN
CONTACT
How is the study designed?
Study Type
INTERVENTIONAL
Masking
NONE
Allocation
NA
Model
SINGLE_GROUP
Primary Purpose
TREATMENT
Number of Arms
1
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