Intraneural Administration of scAAV9/JeT-GAN Into the Vagus Nerve for Patients With Giant Axonal Neuropathy (GAN)

Conditions

• Giant Axonal Neuropathy (GAN)

Eligibility Criteria

Sex

ALL

Age

10 Years - 25 Years

Healthy Volunteers

Not accepted

Interventions

• scAAV9/JeT-GAN — GENETIC
  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. This is the first study in humans to administer the drug directly into the left vagus nerve.

Study Details

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.

Key Dates

Start date

Mar 1, 2026

Status verified

Apr 2026

Primary completion

Dec 31, 2031

Completion

Jun 30, 2032

Study Design

Enrollment

4 participants (estimated)

Allocation

NA

Intervention model

SINGLE_GROUP

Primary purpose

TREATMENT

Arms

• Experimental: Open Label administration of scAAV9/JeT-GAN
  Patients will receive a single injection of scAAV9/JeT-GAN, given directly into the left vagus nerve. scAAV9/JeT-GAN is an injectable drug that contains optimized human gigaxonin (the protein that is mutated in GAN) DNA. scAAV9/JeT-GAN is delivered to cells through a modified viral vector. The vector is the vehicle that transports the gigaxonin DNA to cells. The vector used to transport gigaxonin DNA, is virus called adeno-associated virus serotype 9 (AAV9); however, this virus has been modified so as not cause an illness or infection.

Primary Outcome Measure

Safety of scAAV9/JeT-GAN when delivered to the Vagus Nerve - Based on the Number and Severity of Adverse Events Attributable to Toxicity [ Time Frame: 3 years post treatment ]

Central Contacts

• Samantha Bridges, BSN
  214-456-3696
  [email protected]
• Kristy Riddle, BSN
  214-456-9501
  [email protected]

Locations (1)

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