U.S., Feb. 11 -- ClinicalTrials.gov registry received information related to the study (NCT07398872) titled 'Safety and Efficacy of AAV9. hMCOLN1co For Patients With Mucolipidosis Type IV' on Feb. 03.
Brief Summary: Safety and Efficacy of AAV9.hMCOLN1co for patients with Mucolipidosis Type IV(MLIV): A Single-Center, Interventional, Open-Label, Single-Arm Clinical Study. The goal of this clinical trial is to evaluate whether a gene therapy can safely treat children with MLIV.
Study Start Date: Jan. 13
Study Type: INTERVENTIONAL
Condition:
Mucolipidosis Type IV
Intervention:
BIOLOGICAL: AAV9.hMCOLN1co
A single intrathecal infusion of 10 mL at 2E13 vg/mL for a total dose of 2E14 vg
Recruitment Status: ENROLLING_BY_INVITATION
Sponsor:...