Ignite Creation Date:
2024-05-06 @ 1:01 PM
Last Modification Date:
2024-10-26 @ 1:07 PM
Study NCT ID:
NCT03910621
Status:
COMPLETED
Last Update Posted:
2022-05-25
First Post:
2019-03-08
Brief Title:
Safety and Efficacy of Miglustat in Chinese NPC Patients
Sponsor:
Actelion
Organization:
Actelion
Study Overview
Official Title:
A Single Arm Uncontrolled 12 Months Clinical Study to Evaluate the Safety and Efficacy of Miglustat Zavesca for the Treatment of Niemann Pick Type C Disease NPC in Chinese Subjects
Status:
COMPLETED
Status Verified Date:
2022-05
Last Known Status:
None
Delayed Posting:
No
If Stopped, Why?:
Not Stopped
Has Expanded Access:
False
If Expanded Access, NCT#:
N/A
Has Expanded Access, NCT# Status:
N/A
Acronym:
None
Brief Summary:
This is a prospective multi-center open-label non-randomized single-arm Phase IV confirmatory study
Approximately 19 subjects with Niemann Pick Type C disease NPC will be enrolled in this study The study will be conducted at 2 sites in China
Approximately 19 subjects with Niemann Pick Type C disease NPC will be enrolled in this study The study will be conducted at 2 sites in China
Detailed Description:
This is a prospective multi-center open-label non-randomized single-arm Phase IV confirmatory studyThe study is conducted in Chinese subjects aged 4 years and older with Niemann Pick Type C disease NPC Approximately 19 subjects will be enrolled in this study The study will be conducted at 2 sites in China Patients will be treated with miglustat for 12 months efficacy and safety outcomes will be measured
Study Oversight
Has Oversight DMC:
None
Is a FDA Regulated Drug?:
False
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