Intrathecal Administration of scAAV9/JeT-GAN for the Treatment of Giant Axonal Neuropathy
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ClinicalTrials.gov Identifier: NCT02362438 |
Recruitment Status :
Active, not recruiting
First Posted : February 13, 2015
Last Update Posted : November 21, 2023
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Tracking Information | |||||||||||
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First Submitted Date ICMJE | February 12, 2015 | ||||||||||
First Posted Date ICMJE | February 13, 2015 | ||||||||||
Last Update Posted Date | November 21, 2023 | ||||||||||
Actual Study Start Date ICMJE | April 24, 2015 | ||||||||||
Estimated Primary Completion Date | June 30, 2030 (Final data collection date for primary outcome measure) | ||||||||||
Current Primary Outcome Measures ICMJE |
To assess the safety of the vector [ Time Frame: 12 months ] Adverse event reports will be used to assess safety
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Original Primary Outcome Measures ICMJE |
To determine the relative safety of intrathecal administration of scAAV9/JeT-GAN in the treatment of Giant Axonal Neuropathy [ Time Frame: 8 weeks ] | ||||||||||
Change History | |||||||||||
Current Secondary Outcome Measures ICMJE |
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Original Secondary Outcome Measures ICMJE |
To determine the efficacy of scAAV9-JeT-GAN, measured by improvement of pathologic, histologic, physiologic, function, and clinical markers of Giant Axonal Neuropathy [ Time Frame: 3,6,9, 12 mo + yearly ] | ||||||||||
Current Other Pre-specified Outcome Measures | Not Provided | ||||||||||
Original Other Pre-specified Outcome Measures | Not Provided | ||||||||||
Descriptive Information | |||||||||||
Brief Title ICMJE | Intrathecal Administration of scAAV9/JeT-GAN for the Treatment of Giant Axonal Neuropathy | ||||||||||
Official Title ICMJE | A Phase I Study of Intrathecal Administration of scAAV9/JeT-GAN for the Treatment of Giant Axonal Neuropathy | ||||||||||
Brief Summary | Title: Intrathecal Administration of scAAV9/JeT-GAN for the Treatment of Giant Axonal Neuropathy Background: - The Gigaxonin gene lets the body make a protein chemical called Gigaxonin. Nerves need Gigaxonin to work properly. Giant Axonal Neuropathy (GAN) causes a shortage of functional Gigaxonin. Nerves stop working normally in people with GAN. This causes problems with walking and sometimes with eating, breathing, and many other activities. GAN has no cure. Over time, GAN can shorten a person s life. Researchers want to see if a gene transfer treatment may help people with GAN. Objectives: - To see if a gene transfer is safe and shows potential to help people with GAN. Eligibility: - People age 3 and older with GAN. Design:
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Detailed Description | This is an open-label and non-randomized first-in-human (Phase 1) clinical trial which incorporates an escalating single dose paradigm to assess safety of the gene transfer vector scAAV9/JeT-GAN administered intrathecally to target the brain and spinal cord of individuals with genetically confirmed Giant Axonal Neuropathy. GAN is a chronic neurodegenerative autosomal recessive disease pathologically characterized by enlarged axons with disordered intermediate filaments and microtubules. The disease pathology is due to loss-of-function variants in the GAN gene, which encodes the protein gigaxonin. Gigaxonin plays a major role in the maintenance of orderly and functional intermediate filament (IF) architecture, which is critical for axonal function. Onset of symptoms, usually at three to four years of age, generally manifests with a clumsy and unsteady gait (sensory ataxia). In the peripheral nervous system, the disease progressively affects predominantly sensory and motor nerves. By the end of the second decade of life, patients typically are wheelchair dependent with limited use of the arms and little to no use of their legs. During the second decade a tracheostomy or other means of ventilation, as well as a feeding tube, are often necessary. Death normally occurs in the second or third decade of life. We recently identified a sub-cohort of patients with a milder and later onset, yet progressive form of GAN characterized by a prolonged preservation of ambulation and less extensive white matter changes on brain MRI restricted to the infratentorial regions. These individuals are good trial candidates given the increased prospect of benefit at milder disease stages. There are no statistics on the incidence of GAN, but it is considered extremely rare and does not have an approved treatment aside from supportive care. Intrathecal delivery of a gene transfer vector carrying a normal copy of the GAN to the spinal cord and brain offers a potentially effective treatment for GAN. |
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Study Type ICMJE | Interventional | ||||||||||
Study Phase ICMJE | Phase 1 | ||||||||||
Study Design ICMJE | Allocation: Non-Randomized Intervention Model: Sequential Assignment Masking: None (Open Label) Primary Purpose: Treatment |
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Condition ICMJE |
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Intervention ICMJE | Genetic: scAAv9/JeT-GAN
scAAV9/JeT-GAN is a biological gene transfer reagent
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Study Arms ICMJE |
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Publications * |
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* Includes publications given by the data provider as well as publications identified by ClinicalTrials.gov Identifier (NCT Number) in Medline. |
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Recruitment Information | |||||||||||
Recruitment Status ICMJE | Active, not recruiting | ||||||||||
Estimated Enrollment ICMJE |
21 | ||||||||||
Original Estimated Enrollment ICMJE |
20 | ||||||||||
Estimated Study Completion Date ICMJE | April 1, 2035 | ||||||||||
Estimated Primary Completion Date | June 30, 2030 (Final data collection date for primary outcome measure) | ||||||||||
Eligibility Criteria ICMJE |
To participate in this study, subjects must meet the following criteria:
EXCLUSION/DEFERRAL CRITERIA: To participate in this study, a patient MUST NOT have the following characteristics:
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Sex/Gender ICMJE |
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Ages ICMJE | 3 Years to 99 Years (Child, Adult, Older Adult) | ||||||||||
Accepts Healthy Volunteers ICMJE | No | ||||||||||
Contacts ICMJE | Contact information is only displayed when the study is recruiting subjects | ||||||||||
Listed Location Countries ICMJE | United States | ||||||||||
Removed Location Countries | |||||||||||
Administrative Information | |||||||||||
NCT Number ICMJE | NCT02362438 | ||||||||||
Other Study ID Numbers ICMJE | 150073 15-N-0073 |
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Has Data Monitoring Committee | Not Provided | ||||||||||
U.S. FDA-regulated Product |
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IPD Sharing Statement ICMJE |
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Current Responsible Party | Taysha Gene Therapies, Inc. | ||||||||||
Original Responsible Party | National Institute of Neurological Disorders and Stroke (NINDS) | ||||||||||
Current Study Sponsor ICMJE | Taysha Gene Therapies, Inc. | ||||||||||
Original Study Sponsor ICMJE | National Institute of Neurological Disorders and Stroke (NINDS) | ||||||||||
Collaborators ICMJE | National Institute of Neurological Disorders and Stroke (NINDS) | ||||||||||
Investigators ICMJE |
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PRS Account | Taysha Gene Therapies, Inc. | ||||||||||
Verification Date | November 2023 | ||||||||||
ICMJE Data element required by the International Committee of Medical Journal Editors and the World Health Organization ICTRP |