Study Details
DNL310: A Study Drug for Children and Adolescents with Hunter Syndrome
(IRB#: IRB_00157640)
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
- Paid
Who can participate?
Gender: All genders
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
IRB#: IRB_00157640
PI: David Viskochil
Department: PEDIATRICS
Approval Date: 2023-07-26 06:00:00
Specialties: Pediatric Genetics