Vagus nerve gene therapy (scAAV9/JeT-GAN) for giant axonal neuropathy
Official title: A Phase I/II Open-label Intraneural Administration of scAAV9/JeT-GAN Into the Vagus Nerve to Determine the Safety and Efficacy for Patients With Giant Axonal Neuropathy (GAN) Caused by a Mutation in the GAN Gene
Run by University of Texas Southwestern Medical Center · NCT07543991 · Data updated 2026-11-12
This Phase 1 test delivers a gene therapy (scAAV9/JeT-GAN) directly into the vagus nerve to see if it can help people with confirmed giant axonal neuropathy who previously received intrathecal AAV/GAN.
Quick facts
| Phase | Phase 1 |
|---|---|
| Study type | Interventional |
| Ages | 10 Years to 25 Years |
| Sex | All |
| Enrollment | 4 (estimated) |
| Sponsor | University of Texas Southwestern Medical Center Academic / other |
| Locations | 1 site (Dallas, Texas) |
| Status | Recruiting |
| Started | March 1, 2026 |
| Main results expected | December 31, 2031 |
| Study ends | June 30, 2032 |
| Registry record updated | April 22, 2026 |
| Trial ID | NCT07543991 on ClinicalTrials.gov |
Is this trial a fit for you?
✓Likely a good fit
Ideal candidates are individuals with genetically confirmed GAN who completed a prior intrathecal AAV/GAN treatment and the required five-year follow-up, are medically able to undergo the procedure, and have a parent or legal guardian available to provide consent and attend visits.
✕Probably not a fit
People without a confirmed GAN genetic diagnosis, those who have not previously received intrathecal AAV/GAN with the required follow-up, or those with medical conditions that preclude vagus nerve injection are unlikely to be eligible or benefit.
A plain-English reading of the study's rules. Only the study team can confirm whether you qualify.
What this trial studies
This Phase 1, open-label study delivers a single intraneural injection of scAAV9/JeT-GAN into the vagus nerve to target autonomic and peripheral nervous system involvement in GAN. Enrollment is limited to individuals with genetically confirmed GAN who have completed five years of follow-up after prior intrathecal AAV/GAN treatment. Participants will be closely monitored for safety, autonomic and respiratory function changes, and biological markers of gene delivery. The trial is conducted at the University of Texas Southwestern/Children's Health site in Dallas, Texas.
Why it matters
Potential benefit: If successful, this approach could improve autonomic control and reduce life-threatening respiratory and swallowing complications in people with GAN.
How similar studies have gone: A prior intrathecal scAAV9/JeT-GAN trial delivered gene therapy to the CNS/PNS and showed biological activity, but direct vagus nerve (intraneural) delivery to target autonomic function is a novel approach with limited human data.
Who can join: the full rules
Show full inclusion / exclusion criteria
Inclusion Criteria: 1. Confirmed diagnosis of GAN disease by: 1. Genomic DNA mutation analysis demonstrating homozygous or compound heterozygous, pathogenic and/or confirmed pathogenic variants in the GAN gene; 2. Clinical history or symptoms to ANS dysfunction. 2. Previously treated with IT AAV/GAN and completion of 5 year follow up prior to enrollment. 3. Parents/l LAR willing to accompany the participant to all study visits and who will provide consent for their child's participation. 4. Subject able to comply with all protocol requirements and procedures. 5. Up to date on childhood vaccinations according to Centers for Disease Control (CDC) guidelines. Annual influenza and COVID-19 vaccinations are highly recommended. 6. Female participants of child-bearing potential must have a negative urine and/or negative serum pregnancy test at screening/baseline; (a) Female participants must agree to use an effective form of birth control during study participation. Exclusion Criteria: 1. Inability to participate in study procedures (as determined by the site investigator). 2. Inability to be safely sedated in the opinion of the clinical anesthesiologist. 3. Concomitant illness or requirement for chronic drug treatment that in the opinion of the Principal Investigator (PI) creates unnecessary risks for gene transfer. 4. The presence of significant non-GAN related CNS impairment or behavioral disturbances that would confound the scientific rigor or interpretation of results of the study. 5. Have received an investigational drug within 30 days prior to screening or plan to receive an investigational drug (other than this gene therapy) during the study. 6. Currently participating in another interventional (drug/device) clinical trial. 7. Experienced an SAE (serious adverse event) related to scAAV9/JeT-GAN while participating in the first GAN IT study. 8. Contraindication to scAAV9/JeT-GAN or any of its ingredients. 9. Contraindication to any of the immune suppression medications used in this study. 10. Clinically significant abnormal laboratory values (GGT, ALT, and AST, or total bilirubin \> 3 × ULN, creatinine ≥ 1.5 mg/dL, hemoglobin \[Hgb\] \< 6 or \> 20 g/dL; white blood cell \[WBC\] \> 20,000 per cmm) prior to gene replacement therapy
Where it's running
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Children's Health
Dallas, Texas, United States Recruiting
Study contacts
- Study coordinator: Samantha Bridges, BSN
- Email: samantha.bridges@utsouthwestern.edu
- Phone: 214-456-3696
How to take part
- Review the eligibility criteria above with your treating physician.
- Check the official listing on ClinicalTrials.gov for the latest contact details and recruitment status.
- Contact the study coordinator or principal investigator to ask for pre-screening. Pre-screening is free and never obligates you to enroll.