A Study of Intrathecal SHP611 in Children With Metachromatic Leukodystrophy

Summary

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.

Timeline

Start

2019-05-13

Primary completion

2023-03-08

Completion

2026-05-06

Outcome

Missed primary endpoint

registry analysis (superiority test); Group A, SHP611 vs GLIA-MLD Matched External Control for Group A; p =0.585 NCT03771898 ↗

Drugs

Indications