Safety and Tolerability Study of VTS-270 in Pediatric Participants With Niemann-Pick Type C (NPC) Disease

Summary

This is a Phase-2, multicenter, multiple dose, open-label, 2-part evaluation study which will primarily assess the safety and tolerability of VTS-270 (2-hydroxypropyl beta-cyclodextrin \[HP-β-CD\]) in pediatric participants with age \<4 years.

Timeline

Start

2020-05

Primary completion

2022-10-30

Completion

2022-10-30

Outcome

Outcome not reported

Stopped (Business): “Business decision to Cancel”

Drugs

Indications