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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
Timeline
Screening 3 weeks
Treatment Varies
Follow Up 12 months
Awards & highlights
Study 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.Check my eligibility
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.See study design
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 belowSelect...
I am 3 years or older and have a genetic diagnosis of GAN.
Select...
I am willing to have a nerve biopsy now and in 12 months.
Timeline
Screening ~ 3 weeks3 visits
Treatment ~ Varies
Follow Up ~ 12 months
Screening ~ 3 weeks
Treatment ~ Varies
Follow Up ~12 months
Treatment Details
Study Objectives
Outcome measures can provide a clearer picture of what you can expect from a treatment.Primary outcome measures
To assess the safety of the vector
Secondary outcome measures
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 moreTrial 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
1,339 Previous Clinical Trials
649,285 Total Patients Enrolled
Taysha Gene Therapies, Inc.Lead Sponsor
4 Previous Clinical Trials
27 Total Patients Enrolled
Carsten G Bonnemann, M.D.Principal InvestigatorNational Institute of Neurological Disorders and Stroke (NINDS)
1 Previous Clinical Trials
5,650 Total Patients Enrolled
Media Library
Eligibility Criteria:
This trial includes the following eligibility criteria:- Your lab test results show a significant abnormality, according to the doctor.My lung function is significantly reduced.You have had bad reactions to the drugs used for anesthesia.I am not willing to have a lumbar puncture at the start and 2-3 times in the first year after treatment.You have an important heart rhythm issue according to the doctor, anesthesiologist, or heart specialist.I haven't had chemotherapy, radiotherapy, or immunosuppressive therapy in the last 30 days.You have tested positive for tuberculosis.I do not have any serious infections needing treatment.I do not have any brain or spinal issues that could affect spinal fluid tests or safety checks.I have diabetes or a significant abnormal result on a glucose tolerance test.I am 3 years or older and have a genetic diagnosis of GAN.I am willing to have a nerve biopsy now and in 12 months.I am taking medication that could affect the pressure inside my skull.I have GAN but do not experience noticeable weakness or loss of function.I agree to use contraception or have a partner who cannot become pregnant for 6 months after treatment.I agree to take pregnancy tests and use birth control or have a partner who is sterilized.I have had bacterial meningitis before.You have a device implanted in your brain to help drain fluid.I do not expect to need major surgery in the next 12-18 months.You have an important heart problem according to the study doctor, anesthesiologist, or heart specialist.I have been diagnosed with cardiomyopathy.I have kidney issues shown by high protein levels in my urine.Not growing or gaining weight as expected.I need a machine to help me breathe during the day.I am over 17 and can consent, or over 7 and can assent to treatment.
Research Study Groups:
This trial has the following groups:- Group 1: 10X
- Group 2: 1X
- Group 3: 5X
- Group 4: 3.3X
Awards:
This trial has 1 awards, including:- No Placebo-Only Group - All patients enrolled in this study will receive some form of active treatment.
Timeline:
This trial has the following timeline:- Screening: It may take up to 3 Weeks to process to see if you qualify in this trial.
- Treatment: The duration you will receive the treatment varies.
- Follow Ups: You may be asked to continue sharing information regarding the trial for 6 Months after you stop receiving the treatment.
Frequently Asked Questions
These questions and answers are submitted by anonymous patients, and have not been verified by our internal team.
Is it possible to join the experiment of this clinical trial?
"This trial is inviting up to 21 applicants with giant axonal neuropathy who are between 3 and 99 years old."
Answered by AI
Is enrollment for this trial open currently?
"Affirmative. According to the data found on clinicaltrials.gov, this medical experiment was published on April 24th 2015 and is currently recruiting participants. 21 individuals are needed from 1 study site."
Answered by AI
Can you provide assurance as to the security of scAAv9/JeT-GAN use in humans?
"Due to the preliminary nature of this clinical trial, there is limited data available regarding the safety and efficacy of scAAv9/JeT-GAN. Thus, our assessment has given it a score of 1 on a scale from 1 to 3."
Answered by AI
Is the eligibility criterion for this research limited to adults?
"The age range of participants accepted into this medical study is between 3 and 99 years."
Answered by AI
How many participants are being given treatment in this experiment?
"Affirmative. Clinicaltrials.gov attests that this clinical trial is actively sourcing participants, having been initially posted on April 24th 2015 and most recently updated on September 13th 2022. This study requires the recruitment of 21 patients from a single medical site."
Answered by AI
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