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New treatment option for Giant Axonal Neuropathy (GAN)
Official title Intraneural Administration of scAAV9/JeT-GAN Into the Vagus Nerve for Patients With Giant Axonal Neuropathy (GAN)
ClinicalTrials.gov ID: NCT07543991
What this study is testing
What is scAAV9/JeT-GAN?
scAAV9/JeT-GAN is an investigational medicine, given as an injectable medicine, being studied as a potential treatment for giant axonal neuropathy (gan).
Plain-language explanation of the investigational treatment - it is being studied and is not an approved or proven therapy. The study team can confirm the details.
- What it's testing
- Giant axonal neuropathy (GAN) is a rare pediatric disorder caused by autosomal recessive mutations in the GAN gene. GAN is a multisystem, neurodegenerative disorder affecting the peripheral nervous system (PNS), central nervous system (CNS) and autonomic nervous system (ANS).
- Phase 1: an early, usually small safety study
A plain-language read of the study's public ClinicalTrials.gov listing. The study team confirms the details.
Who can take part
Ages 10 to 25
You may be able to join if
- Confirmed diagnosis of GAN disease by:
- Genomic DNA mutation analysis demonstrating homozygous or compound heterozygous, pathogenic and/or confirmed pathogenic variants in the GAN gene;
- Clinical history or symptoms to ANS dysfunction.
- Previously treated with IT AAV/GAN and completion of 5 year follow up prior to enrollment.
- Parents/l LAR willing to accompany the participant to all study visits and who will provide consent for their child's participation.
You likely can't join if
- Inability to participate in study procedures (as determined by the site investigator).
- Inability to be safely sedated in the opinion of the clinical anesthesiologist.
- Concomitant illness or requirement for chronic drug treatment that in the opinion of the Principal Investigator (PI) creates unnecessary risks for...
- The presence of significant non-GAN related CNS impairment or behavioral disturbances that would confound the scientific rigor or interpretation of...
- Have received an investigational drug within 30 days prior to screening or plan to receive an investigational drug (other than this gene therapy)...
- Currently participating in another treatment (drug/device) clinical trial.
See the full eligibility criteria
- Confirmed diagnosis of GAN disease by:
- Genomic DNA mutation analysis demonstrating homozygous or compound heterozygous, pathogenic and/or confirmed pathogenic variants in the GAN gene;
- Clinical history or symptoms to ANS dysfunction.
- Previously treated with IT AAV/GAN and completion of 5 year follow up prior to enrollment.
- Parents/l LAR willing to accompany the participant to all study visits and who will provide consent for their child's participation.
- Subject able to comply with all protocol requirements and procedures.
- Up to date on childhood vaccinations according to Centers for Disease Control (CDC) guidelines. Annual influenza and COVID-19 vaccinations are highly recommended.
- 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...
- Inability to participate in study procedures (as determined by the site investigator).
- Inability to be safely sedated in the opinion of the clinical anesthesiologist.
- Concomitant illness or requirement for chronic drug treatment that in the opinion of the Principal Investigator (PI) creates unnecessary risks for gene transfer.
- 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.
- 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.
- Currently participating in another treatment (drug/device) clinical trial.
- Experienced an SAE (serious adverse event) related to scAAV9/JeT-GAN while participating in the first GAN IT study.
- Contraindication to scAAV9/JeT-GAN or any of its ingredients.
- Contraindication to any of the immune suppression medications used in this study.
- Clinically significant abnormal laboratory values (GGT, ALT, and AST, or total bilirubin \> 3 × ULN, creatinine ≥ 1.5 mg/dL, hemoglobin [Hgb] \ 20 g/dL; white blood cell [WBC] \> 20,000 per cmm) prior to gene...
The study team makes the final eligibility decision.
Where it's taking place
- Dallas, Texas, United States
Questions & answers
Do participants get paid in this trial?
This listing doesn't specify compensation. Many trials still reimburse travel or offer a stipend, so it's worth asking the study team when you connect.
Is it free to join, and do I need insurance?
Searching and applying through BridgeMD is free. In clinical trials the study-related treatment and visits are generally provided at no cost to you. You usually don't need insurance to take part - confirm specifics with the study team.
How long does this study last?
The listing doesn't state an exact length. The study team walks you through the schedule and number of visits before you decide to enroll.
Who can join this trial?
This study is enrolling all sexes, 10 years to 25 years. The study team makes the final eligibility decision.
Where is this trial taking place?
Study sites include Dallas, Texas, United States. Enter your location above to see the nearest site and check your eligibility.
Explore other conditions
BridgeMD is an information and trial-matching tool - not medical advice, and not the study sponsor. Details come from ClinicalTrials.gov; the study team decides eligibility.