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Gene Therapy for Tay-Sachs and Sandhoff Diseases
Study Summary
This trial is designed to study the safety and efficacy of a gene therapy treatment for two rare, fatal, pediatric neurodegenerative disorders caused by defects in the HEXA or HEXB genes.
Eligibility Criteria
Inclusion Criteria
You may be eligible if you check “Yes” for the criteria belowTimeline
Treatment Details
Study Objectives
Outcome measures can provide a clearer picture of what you can expect from a treatment.Trial Design
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- You have a heart condition that makes it unsafe for you to have gene transfer surgery, as determined by the doctor.I am cleared for gene therapy by a neurosurgeon after an exam and MRI.I am not allergic or unable to take sirolimus or trimethoprim/sulfamethoxazole.I do not have any serious infections, including HIV or Hepatitis.I was born full-term and have a genetic diagnosis of TSD or SD.I require a ventilator to help me breathe.My child is between 6-20 months old and has been diagnosed with infantile-onset TSD or SD.I am between 2 and 12 years old and have juvenile-onset condition.Young participants must show at least 2 specific age-appropriate signs or abilities, as confirmed by the site examiner during screening and before starting treatment.I have had a swallowing test in the last 6 months.If you are a baby and want to join the study, you need to be able to do at least 3 of the following things, as confirmed by the doctor at the screening and before starting the treatment: [list the skills here].My child has been able to sit without support for at least 5 seconds.I have had seizures that medication couldn't control.I am willing to stop taking Zavesca or Tanganil 30 days before screening starts.My child was diagnosed with Tay-Sachs disease or Sandhoff disease early in life.I have a genetic diagnosis of TSD or SD due to HEXA or HEXB gene mutations.I have lost a significant amount of weight in the last 3 months.I do not have spinal conditions that prevent lumbar puncture procedures.I can travel to the study site for all required visits.I have swallowing problems that can't be fixed with thicker drinks or a special bottle.I can sit without support for at least 5 seconds.You have metal devices inside your body that would prevent you from having certain types of imaging tests.I am willing to stop taking Zavesca or Tanganil 30 days before screening starts.I have been diagnosed with juvenile-onset Tay-Sachs or Sandhoff disease.My genetic test shows I have a G269S or W474C mutation in HEXA.You have important abnormal test results from lab tests.I can travel to the study site for all required visits.My parent or guardian does not understand or agree to the study requirements.A neurosurgeon has approved me for gene transfer surgery.I haven't had chemotherapy, radiotherapy, or immunosuppressive therapy in the last 30 days.I have had several lung infections from inhaling food or liquid in the past year.I am between 2 and 12 years old with a diagnosis of juvenile-onset TSD or SD.My child is between 6 to 20 months old.
- Group 1: AXO-AAV-GM2
- No Placebo-Only Group - All patients enrolled in this study will receive some form of active treatment.
- 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
What is the current size of this trial's participant pool?
"Correct. Clinicaltrials.gov data confirms this trial, which was launched on the 15th of January 2021, is recruiting candidates at present. Two medical centres are enrolling 18 patients in total."
How likely are adverse effects to present in patients taking AXO-AAV-GM2 at the recommended starting dose?
"Our team at Power identified AXO-AAV-GM2 Starting Dose as a Level 1 treatment, due to the limited clinical data defining its safety and efficacy."
Is this experiment open to participants of legal age?
"According to the criteria outlined by this medical trial, eligible participants must lie between 6 months and 12 years of age. Moreover, 7 trials are available for those under 18 whereas 3 specifically caters to seniors aged 65 or older."
Is my participation permissible in this exploration?
"This trial necessitates the recruitment of 18 children with Tay-Sachs disease within the ages 6 Months to 12 years. Patients must have a genetically diagnosed TSD or SD mutation in either HEXA gene or HEXB gene, an ability to travel regularly for assessment visits, be over 2 and below 12 if they suffer from juvenile onset, between 6 and 20 months at time of transfer if suffering from infantile onset; furthermore they should possess current or past capability to sit without support for 5 seconds as well as agreement on ceasing any off-label Zavesca® (miglustat) and/or Tanganil"
Are there any unfilled positions for participants in this research?
"Yes, the evidence on clinicaltrials.gov indicates that this experiment is presently looking for participants. This research first began recruiting in mid-January 2021 and was last modified at the end of January 2021. The investigators are hoping to enroll 18 individuals across two sites."
Who else is applying?
What state do they live in?
What site did they apply to?
What portion of applicants met pre-screening criteria?
How many prior treatments have patients received?
What questions have other patients asked about this trial?
Why did patients apply to this trial?
How responsive is this trial?
Typically responds via
Most responsive sites:
- University of Massachusetts Medical Health Center: < 48 hours
Average response time
- < 2 Days
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