NCT03687476

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

An Open-label, Multicenter Safety and Tolerability Study of VTS-270 (2-hydroxypropyl-β-cyclodextrin) in Pediatric Subjects Aged < 4 Years With Neurologic Manifestations of Niemann-Pick Type C (NPC) Disease

• Status: Withdrawn
• Phase: Phase 2
• Study type: Interventional
• Enrollment: 0 (actual)
• Sponsor: Vtesse, LLC, a Mallinckrodt Pharmaceuticals Company · Industry
• Sex: All
• Age: 4 Years
• Healthy volunteers: Not accepted

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.

Detailed description

In Part A of the study, participants after confirmation of the diagnosis of NPC will receive VTS-270 for 20 weeks during the active treatment period. Further, based on investigator's discretion participants would either end treatment with a follow-up visit 28 days (+/- 7) days after last treatment, or will enter to Part-B to benefit from open-label treatment.

Conditions

• Niemann-Pick Disease, Type C

Interventions

Type	Name	Description
DRUG	VTS-270	VTS-270 200 mg/mL administered as described in the arm group description.

Timeline

• Start date: 2020-05-01
• Primary completion: 2022-10-30
• Completion: 2022-10-30
• First posted: 2018-09-27
• Last updated: 2020-07-22

Regulatory

• FDA-regulated drug study