Phase 1 Trial Tests scAAV9/JeT-GAN Gene Therapy for Giant Axonal Neuropathy
Summary
The NIH has published a Phase 1 clinical trial (NCT07543991) evaluating the safety and tolerability of intraneural administration of scAAV9/JeT-GAN gene therapy directly into the left vagus nerve for patients with Giant Axonal Neuropathy (GAN). The study enrolls participants who have previously received scAAV9/JeT-GAN via intrathecal administration and now require treatment specifically targeting autonomic nervous system symptoms. GAN is a rare, fatal, pediatric neurodegenerative disorder with no approved therapies.
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What changed
The NIH registered a new Phase 1 clinical trial on ClinicalTrials.gov for scAAV9/JeT-GAN gene therapy administered intraneurally into the left vagus nerve for Giant Axonal Neuropathy (GAN). This trial follows a prior intrathecal study and specifically addresses autonomic nervous system involvement. The study is classified as investigational, meaning the drug is not yet approved by any regulatory authority. Affected parties include pharmaceutical companies sponsoring gene therapy trials for rare pediatric neurodegenerative diseases, clinical investigators conducting gene therapy studies, and patients with GAN seeking trial enrollment. Sponsors of similar intrathecal-to-intraneural delivery pathway programs should monitor this trial's progress as a potential regulatory precedent.
Archived snapshot
Apr 22, 2026GovPing captured this document from the original source. If the source has since changed or been removed, this is the text as it existed at that time.
Intraneural Administration of scAAV9/JeT-GAN Into the Vagus Nerve for Patients With Giant Axonal Neuropathy (GAN)
Phase 1 NCT07543991 Kind: PHASE1 Apr 22, 2026
Abstract
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...
Conditions: Giant Axonal Neuropathy (GAN)
Interventions: scAAV9/JeT-GAN
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