Gene Therapy
Total Trials
5
As Lead Sponsor
2
As Collaborator
3
Total Enrollment
112,094
NCT03047369
The Myelin Disorders Biorepository Project
Phase: N/A
Role: Collaborator
Start: Dec 8, 2016
Completion: Dec 8, 2030
NCT04041102
Natural History Study of Infantile and Juvenile GM1 Gangliosidosis (GM1) Patients
Start: Jun 12, 2020
Completion: Dec 31, 2026
NCT05368038
ScreenPlus: A Comprehensive, Flexible, Multi-disorder Newborn Screening Program
Start: May 10, 2021
Completion: Aug 31, 2029
NCT04747431
A Study of PBFT02 in Participants With FTD and Mutations in the Granulin Precursor (GRN) or C9ORF72 Genes
Phase: Phase 1/2
Role: Lead Sponsor
Start: Sep 14, 2021
Completion: Aug 31, 2031
NCT04771416
Study of Safety, Tolerability and Efficacy of PBKR03 in Pediatric Subjects With Early Infantile Krabbe Disease
Start: Feb 24, 2022
Completion: Jan 31, 2030
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