Active — Not RecruitingPhase 1/2

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

Canada3 participantsStarted 2021-03-12

Plain-language summary

GM2 gangliosidoses are a group of autosomal recessive neurodegenerative diseases characterized by a deficiency of the Hex A enzyme to catabolize GM2, thereby causing GM2 accumulation within cellular lysosomes.Hex A is composed of 2 subunits, α- and β-, coded by the HEXA and HEXB genes, respectively. The primary purpose of the current study is to assess the safety and tolerability of TSHA101 administered via IT injection.

Who can participate

Age range15 Months

SexALL

See this in plain English?

AI-rewrites the medical criteria so a patient or caregiver can understand them. Always confirm with the trial site.

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

What they're measuring

Safety and tolerability: Treatment-emergent Adverse Events (TEAEs)

Timeframe: 1 year

Safety and Tolerability: Number of participants with abnormal Laboratory assessments

Timeframe: 1 year

Safety and Tolerability: Electrocardiogram (ECG)

Timeframe: 1 year

Trial details

NCT IDNCT04798235

SponsorDr. Anupam Sehgal

Sponsor typeOTHER

Study typeINTERVENTIONAL

Primary completion2027-03-12

View on ClinicalTrials.gov More Infantile GM2 Gangliosidosis (Disorder) trials