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The purpose of this study is to assess the safety and efficacy of the intrathecally-delivered drug SHP611 on gross motor function in babies and young children with late-infantile metachromatic leukodystrophy (MLD). MLD is a rare, genetic, neurodegenerative disease that manifests between 6 months and 2 years of age. In MLD, the body can not properly make an enzyme called arylsulfatase A (ASA). This leads to a loss of myelin, which protects the body's nerves, causing nerve damage that can lead to problems with mobility, speech, and more.
Subject to certain inclusion criteria, this study is enrolling participants ages 6 months through 6 years of age who have been diagnosed with LIMLD.
Boys and Girls: Ages 6 months through 6 years of age
Participants will receive SHP611 intrathecally (into the spinal fluid via catheter) once a week for about 4 years. At predetermined intervals during weekly visits for infusions, participants will also receive medical examinations and motor function testing. Imaging (such as an MRI) and biological sample collection may also occur.
Duration: 210 weeks (about 4 years)
Shire Human Genetic Therapies
Study Description at National Institutes of Health
Neurodevelopment in Rare Disorders Research
Deepa Soundara Rajan, MD
For more information about the study or enrollment, please contact:
Dawn Kolar, Clinical Research Coordinator
Children's Hospital's main campus is located in the Lawrenceville neighborhood. Our main hospital address is:
UPMC Children’s Hospital of Pittsburgh
One Children’s Hospital Way
4401 Penn Ave.
Pittsburgh, PA 15224
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