Study Details
Testing a new drug called Trappsol® Cyclo in patients with Niemann-Pick Disease Type C1
(IRB#: IRB_00161309)
Niemann-Pick Disease Type C1 (NPC1) is a disease that may cause problems in the brain and the body. This study will test a drug called Trappsol® Cyclo in children and adults with NPC1 to see if it helps treat their disease. Participants in this study will be randomized to be treated with Trappsol® Cyclo or a placebo in an IV infusion. The IV infusion will happen in the clinic every 2 weeks. Being in this study requires up to 100 study visits at the clinic. Later in the study, participants on the placebo can be switched to Trappsol® Cyclo.
- 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
Will I be paid for my time?
Yes
IRB#: IRB_00161309
PI: Lorenzo Botto
Department: PEDIATRIC GENETICS
Approval Date: 2023-08-02 06:00:00
Specialties: Pediatric Genetics