A Study of Intrathecal SHP611 in Participants With Late Infantile Metachromatic Leukodystrophy
Status:
Active, not recruiting
Trial end date:
2022-08-15
Target enrollment:
Participant gender:
Summary
The purpose of this study is to evaluate the safety and efficacy of intrathecal (IT)
administration of SHP611 on gross motor function, using the Gross Motor Function
Classification in Metachromatic Leukodystrophy (GMFC-MLD) compared with matched historical
control data in children with metachromatic leukodystrophy (MLD).