A Study of Intrathecal SHP611 in Children With Metachromatic Leukodystrophy (NCT03771898) | Clinical Trial Compass
Active — Not RecruitingPhase 2
A Study of Intrathecal SHP611 in Children With Metachromatic Leukodystrophy
United States36 participantsStarted 2019-05-13
Plain-language 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.
Who can participate
Age range6 Months – 72 Months
SexALL
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Inclusion criteria
✓. Low ASA activity in leukocytes (compared to laboratory normal range).
✓. Elevated sulfatides in urine.
Exclusion criteria
✕. The participant has had, or may have, an allergic reaction to the materials of construction.
✕. The participant has shown an intolerance to an implanted device.
✕. The participant's body size is too small to support the size of the SOPH-A-PORT Mini S Access Port.
✕. The participant's drug therapy requires substances known to be incompatible with the materials of construction.
✕. The participant has a known or suspected local or general infection.
✕. The participant is at risk of abnormal bleeding due to a medical condition or therapy.
✕. The participant has one or more spinal abnormalities that could complicate safe implantation or fixation.
What they're measuring
1
Percent Probability of Free of Loss of Locomotion in the Last Time Interval Up to 2 Years (Week 106) Based on GMFC-MLD for SHP611 Group A and GLIA-MLD Matched External Control