Trial Profile

A Phase 1/2 Study of Intravenous Gene Transfer With an AAV9 Vector Expressing Human Beta-galactosidase in Type I and Type II GM1 Gangliosidosis

Status: Recruiting

Phase of Trial: Phase I/II

Latest Information Update: 08 May 2025

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At a glance

Drugs Bomtabegagene bavoparvovec (Primary) ; Methylprednisolone; Prednisone; Rituximab; Sirolimus
Indications Gangliosidoses
Focus Adverse reactions; First in man; Registrational

Most Recent Events

30 Apr 2025 Planned number of patients changed from 45 to 54.
19 Jan 2024 Status changed from active, no longer recruiting to recruiting.
02 Mar 2023 Planned End Date changed from 30 Apr 2026 to 1 Jan 2028.

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