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

Study Results

Results pending

The study team has not published outcome measurements, participant flow, or safety data for this trial yet. Check back later for updates.

Basic Information

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Recruitment Status

ACTIVE_NOT_RECRUITING

Clinical Phase

PHASE1/PHASE2

Total Enrollment

3 participants

Study Classification

INTERVENTIONAL

Study Start Date

2021-03-12

Study Completion Date

2027-03-12

Brief Summary

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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.

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Detailed Description

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Conditions

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Infantile GM2 Gangliosidosis (Disorder)

Study Design

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Allocation Method

NA

Intervention Model

SINGLE_GROUP

Primary Study Purpose

TREATMENT

Blinding Strategy

NONE

Study Groups

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TSHA-101

Subjects who will receive one-time intrathecal TSHA-101, brain volume based sliding scale for dosage

Group Type EXPERIMENTAL

TSHA-101

Intervention Type BIOLOGICAL

AAV9 viral vector containing HEXA and HEXB genes to be administered via Intrathecal injection

Interventions

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TSHA-101

AAV9 viral vector containing HEXA and HEXB genes to be administered via Intrathecal injection

Intervention Type BIOLOGICAL

Eligibility Criteria

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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

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

Maximum Eligible Age

15 Months

Eligible Sex

ALL

Accepts Healthy Volunteers

No