Ignite Creation Date:
2025-12-25 @ 1:19 AM
Ignite Modification Date:
2026-02-26 @ 4:18 PM
Study NCT ID:
NCT03128593
Status:
COMPLETED
Last Update Posted:
2022-11-14
First Post:
2017-04-10
Is Gene Therapy:
True
Has Adverse Events:
False
Brief Title:
A Study of JR-141 in Patients With Mucopolysaccharidosis Type II
Sponsor:
JCR Pharmaceuticals Co., Ltd.
Organization:
Study Overview
Official Title:
A Phase I/II Study of JR-141 in Patients With Mucopolysaccharidosis Type II
Status:
COMPLETED
Status Verified Date:
2022-11
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:
The purpose of this study in patients with mucopolysaccharidosis type II (MPS II) is below,
* to collect the safety information of JR-141
* to evaluate the plasma pharmacokinetics of JR-141
* to explore the efficacy of JR-141 on MPS II-related central nervous system symptoms and general symptoms
* to collect the safety information of JR-141
* to evaluate the plasma pharmacokinetics of JR-141
* to explore the efficacy of JR-141 on MPS II-related central nervous system symptoms and general symptoms
Detailed Description:
None
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?: