Ignite Creation Date:
2024-05-06 @ 12:08 PM
Last Modification Date:
2024-10-26 @ 12:55 PM
Study NCT ID:
NCT03687476
Status:
WITHDRAWN
Last Update Posted:
2020-07-22
First Post:
2018-08-06
Brief Title:
Safety and Tolerability Study of VTS-270 in Pediatric Participants With Niemann-Pick Type C NPC Disease
Sponsor:
Vtesse LLC a Mallinckrodt Pharmaceuticals Company
Organization:
Mallinckrodt
Study Overview
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
Status:
WITHDRAWN
Status Verified Date:
2020-07
Last Known Status:
None
Delayed Posting:
No
If Stopped, Why?:
Business decision to Cancel
Has Expanded Access:
No
If Expanded Access, NCT#:
N/A
Has Expanded Access, NCT# Status:
N/A
Acronym:
None
Brief 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 investigators 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 Oversight
Has Oversight DMC:
None
Is a FDA Regulated Drug?:
True
Is a FDA Regulated Device?:
False
Is an Unapproved Device?:
None
Is a PPSD?:
None
Is a US Export?:
None
Is an FDA AA801 Violation?:
None