Clinical Trial: A Study of JR-141 in Patients With Mucopolysaccharidosis Type II
Study Status: Recruiting
Recruit Status: Recruiting
Study Type: Interventional
Official Title: A Phase I/II Study of JR-141 in Patients With Mucopolysaccharidosis Type II
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
Detailed Summary:
Sponsor: JCR Pharmaceuticals Co., Ltd.
Current Primary Outcome: Number of participants with Adverse Events [ Time Frame: 4 weeks ]
- Adverse events
- Laboratory tests
- Vital signs
- 12-lead electrocardiogram
- Antibody
- Infusion associated reaction
Original Primary Outcome: Same as current
Current Secondary Outcome:
- Plasma Pharmacokinetic parameter [Maximum Plasma Concentration [Cmax]] [ Time Frame: 4 weeks ]Plasma concentration of JR-141
- Plasma Pharmacokinetic parameter [Area Under the Curve [AUC]] [ Time Frame: 4 weeks ]Plasma concentration of JR-141
- Urinary and serum heparan sulfate (HS) /dermatan sulfate (DS) [ Time Frame: 4 weeks ]
- HS/DS in CSF [ Time Frame: 4 weeks ]JR-141 concentration in CSF
- Urinary total GAG [ Time Frame: 4 weeks ]
- Liver and spleen volumes [ Time Frame: 4 weeks ]
- Cardiac function [ Time Frame: 4 weeks ]
Original Secondary Outcome: Same as current
Information By: JCR Pharmaceuticals Co., Ltd.
Dates:
Date Received: April 10, 2017
Date Started: March 30, 2017
Date Completion: December 2017
Last Updated: April 24, 2017
Last Verified: April 2017