NCT04771416 - Study of Safety, Tolerability and Efficacy of PBKR03 in Pediatric Subjects With Early Infantile Krabbe Disease | Biotech Hunter

Enrollment

Participants

Timeline

Start Date

February 24, 2022

Primary Completion Date

January 31, 2027

Study Completion Date

January 31, 2030

Conditions

Leukodystrophy, Globoid Cell

Interventions

BIOLOGICAL

PBKR03

Single dose of PBKR03, via intra cisterna magna

Trial Locations (9)

New York-Presbyterian, New York

The Children's Hospital of Philadelphia, Philadelphia

Ann & Robert Lurie, Chicago

University of Utah School of Medicine, Salt Lake City

Hospital De Clinicas De Porto Alegre, Porto Alegre

Montreal Children's Hospital, Montreal

Shaare Zadek Medical Center, Jerusalem

Amsterdam UMC, Amsterdam

Manchester University, Manchester