A Study of Intrathecal SHP611 in Children With Metachromatic Leukodystrophy

The main aim of the study is to determine if SHP611 given by injection into the spinal fluid that surrounds the brain and spinal cord (intrathecal; IT) prolongs the time for children with Metachromatic Leukodystrophy (MLD) to retain the ability to move from place to place. Other aims of the study are to determine the effects of intrathecal administration of SHP611 on movement and speech functions and to learn how well SHP611 injected in the spinal fluid that surrounds the brain and spinal cord is tolerated. Study participants will receive SHP611 for about 2 years with the possibility of an extended treatment period.

NCT Number	NCT03771898
Lead Sponsor	Shire Collaborators: Takeda Development Center Americas, Inc.
Conditions	Metachromatic Leukodystrophy (MLD)
Enrollment	36 participants
Start Date	2019-05-13
Primary Completion	2023-03-08 (estimated)
Study Completion	2026-05-06 (estimated)
Updated on ClinicalTrials.gov	2026-07-10