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Virus Therapy

Gene Therapy for Giant Axonal Neuropathy

Phase 1
Waitlist Available
Led By Carsten G Bonnemann, M.D.
Research Sponsored by Taysha Gene Therapies, Inc.
Eligibility Criteria Checklist
Specific guidelines that determine who can or cannot participate in a clinical trial
Must have
Age 3 years or older with genetic diagnosis of GAN: Identified pathogenic variant(s) on both copies of the GAN gene
Willingness to undergo a nerve biopsy at baseline and at 12 months after treatment
Must not have
Forced vital capacity <= 50% of predicted value (if patient is >/= 5 years old)
Unwilling to undergo lumbar puncture at baseline and up to 2 to 3 times during follow up during the first year after treatment
Timeline
Screening 3 weeks
Treatment Varies
Follow Up 12 months
Awards & highlights
No Placebo-Only Group

Summary

This trial is testing whether a gene transfer is safe and shows potential to help people with Giant Axonal Neuropathy by delivering a gene that will produce the protein Gigaxonin.

Who is the study for?
This trial is for people aged 3 and older diagnosed with Giant Axonal Neuropathy (GAN). Participants must be able to consent, agree to use contraception if of childbearing potential, undergo nerve biopsies, and live near the NIH for a month post-treatment. Pregnant or breastfeeding individuals, those dependent on ventilators, with significant infections or heart issues, prior gene therapy/stem cell transplants, recent immunosuppressive treatments or vaccinations are excluded.
What is being tested?
The trial tests scAAV9/JeT-GAN gene transfer via spinal tap injection into cerebrospinal fluid. It aims to deliver functional Gigaxonin genes using a modified virus carrier to help GAN patients. The study involves extensive screening and follow-up over 15 years with various health tests including MRIs and biopsies.
What are the potential side effects?
Potential side effects include reactions related to the spinal tap procedure such as headache or back pain; immune responses against the viral vector; discomfort from sedation during certain tests; risks associated with long-term monitoring like infection from intravenous lines.

Eligibility Criteria

Inclusion Criteria

You may be eligible if you check “Yes” for the criteria below
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I am 3 years or older and have a genetic diagnosis of GAN.
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I am willing to have a nerve biopsy now and in 12 months.

Exclusion Criteria

You may be eligible for the trial if you check “No” for criteria below:
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My lung function is significantly reduced.
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I am not willing to have a lumbar puncture at the start and 2-3 times in the first year after treatment.
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I haven't had chemotherapy, radiotherapy, or immunosuppressive therapy in the last 30 days.
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I do not have any serious infections needing treatment.
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I do not have any brain or spinal issues that could affect spinal fluid tests or safety checks.
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I have diabetes or a significant abnormal result on a glucose tolerance test.
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I am taking medication that could affect the pressure inside my skull.
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I have GAN but do not experience noticeable weakness or loss of function.
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I have had bacterial meningitis before.
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I have been diagnosed with cardiomyopathy.
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I have kidney issues shown by high protein levels in my urine.
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I need a machine to help me breathe during the day.

Timeline

Screening ~ 3 weeks
Treatment ~ Varies
Follow Up ~12 months
This trial's timeline: 3 weeks for screening, Varies for treatment, and 12 months for reporting.

Treatment Details

Study Objectives

Study objectives can provide a clearer picture of what you can expect from a treatment.
Primary study objectives
To assess the safety of the vector
Secondary study objectives
Assessment of motor and sensory disease symptoms compare to baseline
Assessment of vector shedding following treatment
Determine safety and tolerability of gene transfer in patients with null mutations receiving immunosuppression
+2 more

Awards & Highlights

No Placebo-Only Group
All patients enrolled in this study will receive some form of active treatment.

Trial Design

4Treatment groups
Experimental Treatment
Group I: 5XExperimental Treatment1 Intervention
3rd dose increase in escalation scheme
Group II: 3.3XExperimental Treatment1 Intervention
2nd dose increase in escalation scheme
Group III: 1XExperimental Treatment1 Intervention
Lowest dose in the escalation scheme
Group IV: 10XExperimental Treatment1 Intervention
Highest dose in the escalation scheme

Find a Location

Who is running the clinical trial?

National Institute of Neurological Disorders and Stroke (NINDS)NIH
1,377 Previous Clinical Trials
651,816 Total Patients Enrolled
Taysha Gene Therapies, Inc.Lead Sponsor
4 Previous Clinical Trials
41 Total Patients Enrolled
Carsten G Bonnemann, M.D.Principal InvestigatorNational Institute of Neurological Disorders and Stroke (NINDS)
2 Previous Clinical Trials
5,725 Total Patients Enrolled

Media Library

scAAV9/JeT-GAN (Virus Therapy) Clinical Trial Eligibility Overview. Trial Name: NCT02362438 — Phase 1
Giant Axonal Neuropathy Research Study Groups: 10X, 1X, 5X, 3.3X
Giant Axonal Neuropathy Clinical Trial 2023: scAAV9/JeT-GAN Highlights & Side Effects. Trial Name: NCT02362438 — Phase 1
scAAV9/JeT-GAN (Virus Therapy) 2023 Treatment Timeline for Medical Study. Trial Name: NCT02362438 — Phase 1
~8 spots leftby Jun 2030
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