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.
- All genders
- All Ages
- Volunteers with special conditions
- In Person
Who can participate?
Gender: All genders
Age: All Ages
Volunteers: Volunteers with special conditions
Location: In Person
- 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
- 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
Will I be paid for my time?