Defunct Company
Total Trials
6
As Lead Sponsor
As Collaborator
0
Total Enrollment
81
NCT01474343
Intracerebral Gene Therapy for Sanfilippo Type A Syndrome
Phase: Phase 1/2
Role: Lead Sponsor
Start: Aug 31, 2011
Completion: May 31, 2013
NCT02053064
Long-term Follow-up of Sanfilippo Type A Patients Treated by Intracerebral SAF-301 Gene Therapy
Start: May 31, 2013
Completion: Jun 30, 2017
NCT02746341
Natural History Study of Patients With MPS IIIA
Phase: N/A
Start: Apr 30, 2016
Completion: May 31, 2019
NCT03612869
Study of AAVrh10-h.SGSH Gene Therapy in Patients With Mucopolysaccharidosis Type IIIA (MPS IIIA)
Phase: Phase 2/3
Start: Dec 17, 2018
Completion: Mar 31, 2022
NCT04310163
Interviews and Video Capture in Patients With GM1 Gangliosidosis
Start: Apr 20, 2020
Completion: May 5, 2023
NCT04273269
A Safety and Efficacy Study of LYS-GM101 Gene Therapy in Patients With GM1 Gangliosidosis
Start: May 11, 2021
Completion: May 22, 2023