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Giant Axonal Disease - Healing Genes
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Giant Axonal Disease

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Intrathecal Administration of scAAV9/JeT-GAN for the Treatment of Giant Axonal Neuropathy

A Phase I Study of Intrathecal Administration of scAAV9/JeT-GAN for the Treatment of Giant Axonal Neuropathy


Phase 1

DESCRIPTION:

A trial for a an investigatory gene therapy to treat Giant Axonal Neuropathy (GAN) is recruiting patients at the National Institutes of Health Clinical Center. Patients with GAN may be eligible for this therapy utilizing a virus shell to deliver a functional copy of the GAN gene, which encodes the protein gigaxonin. The researchers hope to permanently provide normal axonal growth and function.

The treatment requires a single intrathecal (to the spine) injection of the investigatory drug and follow up will proceed during weeks 1-4; months 3, 6, 9, 18; and, years 1 to 15; closest follow up is within the first year post gene transfer. Long term safety follow up will be evaluated for up to 15 years.


PATIENT MUST:

  • Be 5 to 99 years of age
  • Have a genetic diagnosis of GAN: Identified mutation(s) on both copies of the GAN gene
  • Agree to reside within 100 miles of the study site for at least 2 months following treatment (may include housing on NIH campus)

THE STUDY INVOLVES:

  1. Screening before the treatment (including lab tests, MRIs, physical assessments, and nerve biopsies).
  2. A single intrathecal (to the spine) injection of the investigatory drug will be administered.
  3. Follow up will proceed during weeks 1-4; months 3, 6, 9, 18; and, years 1 to 15; closest follow up is within the first year post gene transfer. Long term safety follow up will be evaluated for up to 15 years.

LOCATIONS AND CONTACTS:
Trials will take place at the National Institutes of Health Clinical Center in Bethesda, MD. Map.

Contact:
Carsten G Bonnemann, M.D.  |  (301) 594-5496  |  [email protected]
 
SPONSOR INFORMATION:
National Institute of Neurological Disorders and Stroke (NINDS)
 
Or go online:
https://clinicaltrials.gov/ct2/show/NCT02362438

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