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

Niemann-Pick Disease, Type C Clinical Trial

Official title:
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

Clinical Trial 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.

Clinical Trial 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. ;

Study Design

Related Conditions & MeSH terms

Clinical Trial Details
NCT number NCT03687476
Study type Interventional
Source Mallinckrodt
Contact
Status Withdrawn
Phase Phase 2
Start date May 2020
Completion date October 30, 2022
View Details
See also
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