Clinical Study
DNL310: A Study Drug for Children and Adolescents with Hunter Syndrome
Hunter syndrome is a disease that may cause delays in cognitive (part of the brain) development and disruptive and impaired behaviors in children and adolescents. DNL310 is a study drug for children and adolescents with Hunter Syndrome. Research is needed to learn the safety and effectiveness of DNL310. The information we gain may aid future patients.
For more information contact:
Carrie Bailey
carrie.bailey@hsc.utah.edu
8015873605
IRB#: IRB_00157640
| PI: David Viskochil
| Department: PEDIATRICS
| Approval Date: 2023-07-26 06:00:00
Specialties: Pediatric Genetics
Who can participate?
Gender: All
Age: All Ages
Volunteers: Volunteers with special conditions
Location: In Person
Inclusion Criteria:
- Ages 2 to 5 years old who have Neuronopathic Hunter syndrome OR
- Ages 6 to 16 years old who have Non-neuronopathic Hunter syndrome
- Be on maintenance of enzyme replacement therapy (ERT) and taking 4 months of Elaprase medication for Hunter syndrome
- Childbearing-age participants must use specific birth control
- Attend in person at the University of Utah
Exclusion Criteria:
- Unstable medical condition
- History of cancer
- Significant diagnosis of the central nervous system (brain and spinal cord) trauma or disorder
- Serious or life-threatening reactions to any part of the study drug that cannot be controlled
- Pregnant
Will I be paid for my time?
Yes