A Global Multicenter Open-label Matched Historical Control Study of Intrathecal SHP611 in Subjects with Late Infantile Metachromatic Leukodystrophy (MLD)

Brief description of study

The purpose of this study is to evaluate the safety of the study drug and the effect of the study drug on motor function (the abilities required to control the large muscles of the body for walking, running, sitting, crawling, and other activities) in subjects with late infantile MLD.MLD is a rare, inherited condition in which an enzyme is missing or not working properly. This enzyme breaks down certain fats. When those fats aren’t broken down, they interfere with the body’s ability to send information through nerves, and to properly control muscle activity. Subjects with late infantile MLD are usually diagnosed at an early age, and the disease worsens over time, with a low rate of survival in the following years.


Clinical Study Identifier: s18-01046
ClinicalTrials.gov Identifier: NCT03771898
Principal Investigator: Heather Ann Lau.


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