NCT04713475View on ClinicalTrials.gov

Study of Safety, Tolerability and Efficacy of PBGM01 in Pediatric Participants With GM1 Gangliosidosis

PHASE1/PHASE2Active, not recruitingINTERVENTIONAL
Enrollment

26

Participants

Timeline

Start Date

March 17, 2021

Primary Completion Date

February 28, 2026

Study Completion Date

February 28, 2029

Conditions
GM1 GangliosidosisGM1 Gangliosidosis, Type IGM1 Gangliosidosis, Type 2Beta-Galactosidase-1 (GLB1) Deficiency
Interventions
BIOLOGICAL

PBGM01

AAVhu68 viral vector

Trial Locations (7)

19104

The Children's Hospital of Philadelphia, Philadelphia

55455

University of Minnesota, Minneapolis

94158

Benioff Children's Hospital, Oakland

08901

Children's Hospital at St. Peter's University Hospital, New Brunswick

Unknown

Hospital de Clínicas de Porto Alegre (HCPA), Porto Alegre

Gazi University, Ankara

Great Ormond Street Hospital, London

Sponsors
All Listed Sponsors
lead

Gemma Biotherapeutics

INDUSTRY

NCT04713475 - Study of Safety, Tolerability and Efficacy of PBGM01 in Pediatric Participants With GM1 Gangliosidosis | Biotech Hunter | Biotech Hunter