Mucopolysaccharidosis II
Conditions
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
Interventions
DRUGJR-141
IV infusion (lyophilized powder), 0.01-2.0 mg/kg/week
Sponsors
JCR Pharmaceuticals Co., Ltd.
Study design
Allocation
NA
Intervention model
SEQUENTIAL
Primary purpose
TREATMENT
Masking
NONE
Eligibility
Sex/Gender
MALE
Age
6 Years to No maximum
Healthy volunteers
No
Inclusion criteria
* Patients aged 6 years or older at the time of informed consent. * Patients diagnosed with MPS II. * Patients who have received idursulfase (0.5 mg/kg/week) continuously for at least 12 weeks until the initial dose of JR-141.
Exclusion criteria
* Patients with a history of hematopoietic stem cell transplantation, excluding those who need enzyme replacement therapy even after hematopoietic stem cell transplantation. * Patients in whom lumbar puncture cannot be performed. * Patients who have developed serious drug allergy or hypersensitivity that is inappropriate for participation in the study. * Patients who have received other investigational products within 4 months before enrollment in the study.
Design outcomes
Primary
| Measure | Time frame | Description |
|---|---|---|
| Number of participants with Adverse Events | 4 weeks | * Adverse events * Laboratory tests * Vital signs * 12-lead electrocardiogram * Antibody * Infusion associated reaction |
Secondary
| Measure | Time frame | Description |
|---|---|---|
| Plasma Pharmacokinetic parameter [Area Under the Curve [AUC]] | 4 weeks | Plasma concentration of JR-141 |
| Urinary and serum heparan sulfate (HS) /dermatan sulfate (DS) | 4 weeks | — |
| HS/DS in CSF | 4 weeks | JR-141 concentration in CSF |
| Plasma Pharmacokinetic parameter [Maximum Plasma Concentration [Cmax]] | 4 weeks | Plasma concentration of JR-141 |
| Liver and spleen volumes | 4 weeks | — |
| Cardiac function | 4 weeks | — |
| Urinary total GAG | 4 weeks | — |
Countries
Japan
Outcome results
None listed