Niemann-Pick Type C1

Study of VTS-301 (2-hydroxypropyl-β-cyclodextrin) to Treat Niemann-Pick Type C1 (NPC1) Disease

An Open-Label, Fixed-Sequence, Ascending-Dose, First-in-Human Study to Assess the Safety, Tolerability, Pharmacokinetics, Pharmacodynamics, and Efficacy of Intravenous Infusions of ATB200 Co-Administered With Oral AT2221 in Adult Subjects With Pompe Disease

About: This study evaluates the efficacy and safety of 2-hydroxypropyl-β-cyclodextrin (VTS-270) in patients with neurologic manifestations of Niemann-Pick Type C1 (NPC1) Disease. Approximately two-thirds of patients will receive the study drug, 2-hydroxypropyl-β-cyclodextrin (HP-β-CD), while the remaining study participants will receive sham control.

Ages Eligible for Study: 4 Years to 21 Years (Child, Adult)

Sexes Eligible for Study: All

Status:  Active but no longer recruiting. Accepting Transfers.

For more information about this study, including eligibility criteria, please go to the studies page. If you are interested in participating in this study or have questions about research, please contact Gee Kim, Clinical Research Coordinator.