This first-in-human study evaluates a gene therapy called TSHA-101 in infantile-onset GM2 gangliosidosis. It is open to infants aged 15 months or younger who have a genetically and enzymatically confirmed diagnosis of GM2 gangliosidosis. It is in Phase 1/Phase 2 and is currently active but not recruiting new participants.
The summary above is a plain-language rendering of the official record. The original English title is shown for reference.
Criteria are reproduced from the ClinicalTrials.gov record in the original English. Only the trial team can determine eligibility.
Key Inclusion Criteria: * male or female with age less than or equal to 15 months * diagnosis of GM2 gangliosidosis with genetic and enzymatic documentation of infantile disease Key Exclusion Criteria: * a second neurodevelopmental disorder independent of the HEXA or HEXB * inability to tolerate sedation or intrathecal administration * invasive ventilatory support * concomitant illness, allergies or known hypersensitivity to the required immunosuppression regimen