At a glance
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A Global, Multicenter, Single-arm, Matched External Control Study of Intrathecal SHP611 in Subjects With Late Infantile Metachromatic Leukodystrophy
In Brief
A Phase 2 clinical trial evaluating SHP611 for Metachromatic Leukodystrophy (MLD). Active but no longer recruiting, targeting 36 participants across 30 sites in 14 countries.
Detailed 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.
Study Details
Timeline
Interventions
Participants will receive 150 mg of SHP611 IT via IDDD or LP once weekly for 106 weeks.