Phase 1/2 Open-Label, Multicenter Study to Assess the Safety, Tolerability and Efficacy of a Single Dose of PBGM01 Delivered Into the Cisterna Magna of Subjects With Type 1 (Early Onset) and Type 2a (Late Onset) Infantile GM1 Gangliosidosis

This study aims to assess the safety, tolerability and efficacy of a single dose of PBGM01 delivered via intracisterna magna injection in pediatric participants with GM1 Gangliosidosis. Safety and tolerability of PBFM01, by measuring changes in adverse events (AEs), neurological evaluation results, safety laboratory tests, and brain imaging. Efficacy of PBGM01 by the number of developmental milestones achieved at 2 years and over 5 years post-dose. 

Sponsor

Passage Bio, Inc. 

Status of enrollment

Accepting new patients

Ages Eligible for Study

Type 1 (Early Onset Infantile) GM1 participants must be ≥4 months and <24 months of age at enrollment for Cohorts 3 and 4, and ≥1 month and <12 months of age at enrollment for Cohort 6 with onset of signs or symptoms of GM1 before 6 months of age. Type 2a (Late Onset Infantile) GM1 participants must be ≥6 months and <36 months of age at enrollment for Cohorts 1 and 2 and ≥6 months and ≤24 months of age at enrollment for Cohort 5 with onset of signs or symptoms of GM1 between 6 and 18 months of age.

Genders Eligible for Study

Females and males

Disease indication

GM1 Gangliosidosis

Principal Investigator

Caroline Hastings, MD

Contact

Caroline Hastings, MD

[email protected]

 

Additional study eligibility details can be found at UCSF Clinical Trials.