First-in-Human Study of TSHA-101 Gene Therapy for Treatment of Infantile Onset GM2 Gangliosidosis

Plain-language summary

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.

Trial details

Phase

PHASE1/PHASE2

Status

Active, not recruiting

Sponsor

Dr. Anupam Sehgal

Intervention

TSHA-101

Start date

3/12/2021

Estimated completion

3/12/2027

Countries

Canada

Related genes

Locations

• Kingston, Canada

Eligibility criteria

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