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
Study Type
INTERVENTIONAL
Allocation
NA
Purpose
TREATMENT
Masking
NONE
Enrollment
14
IV infusion (lyophilized powder), 0.01-2.0 mg/kg/week
Gifu Clinical site
Gifu, Japan
Fukuoka Clinical site
Kurume, Japan
Tokyo clinical site1
Minato, Japan
Osaka Clinical site1
Osaka, Japan
Number of participants with Adverse Events
* Adverse events * Laboratory tests * Vital signs * 12-lead electrocardiogram * Antibody * Infusion associated reaction
Time frame: 4 weeks
Plasma Pharmacokinetic parameter [Maximum Plasma Concentration [Cmax]]
Plasma concentration of JR-141
Time frame: 4 weeks
Plasma Pharmacokinetic parameter [Area Under the Curve [AUC]]
Plasma concentration of JR-141
Time frame: 4 weeks
Urinary and serum heparan sulfate (HS) /dermatan sulfate (DS)
Time frame: 4 weeks
HS/DS in CSF
JR-141 concentration in CSF
Time frame: 4 weeks
Urinary total GAG
Time frame: 4 weeks
Liver and spleen volumes
Time frame: 4 weeks
Cardiac function
Time frame: 4 weeks
This platform is for informational purposes only and does not constitute medical advice. Always consult a qualified healthcare professional.
Saitama Clinical site
Saitama, Japan
Tokyo clinical site2
Setagaya City, Japan
Osaka Clinical site2
Suita, Japan
Tottori Clinical site
Yonago, Japan