Mucopolysaccharidosis II Clinical Trial
Official title:
A Phase I/II Study of JR-141 in Patients With Mucopolysaccharidosis Type II
Verified date | November 2022 |
Source | JCR Pharmaceuticals Co., Ltd. |
Contact | n/a |
Is FDA regulated | No |
Health authority | |
Study type | Interventional |
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
Status | Completed |
Enrollment | 14 |
Est. completion date | October 4, 2017 |
Est. primary completion date | October 4, 2017 |
Accepts healthy volunteers | No |
Gender | Male |
Age group | 6 Years and older |
Eligibility | 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. |
Country | Name | City | State |
---|---|---|---|
Japan | Gifu Clinical site | Gifu | |
Japan | Fukuoka Clinical site | Kurume | |
Japan | Tokyo clinical site1 | Minato | |
Japan | Osaka Clinical site1 | Osaka | |
Japan | Saitama Clinical site | Saitama | |
Japan | Tokyo clinical site2 | Setagaya | |
Japan | Osaka Clinical site2 | Suita | |
Japan | Tottori Clinical site | Yonago |
Lead Sponsor | Collaborator |
---|---|
JCR Pharmaceuticals Co., Ltd. |
Japan,
Type | Measure | Description | Time frame | Safety issue |
---|---|---|---|---|
Primary | Number of participants with Adverse Events | Adverse events
Laboratory tests Vital signs 12-lead electrocardiogram Antibody Infusion associated reaction |
4 weeks | |
Secondary | Plasma Pharmacokinetic parameter [Maximum Plasma Concentration [Cmax]] | Plasma concentration of JR-141 | 4 weeks | |
Secondary | Plasma Pharmacokinetic parameter [Area Under the Curve [AUC]] | Plasma concentration of JR-141 | 4 weeks | |
Secondary | Urinary and serum heparan sulfate (HS) /dermatan sulfate (DS) | 4 weeks | ||
Secondary | HS/DS in CSF | JR-141 concentration in CSF | 4 weeks | |
Secondary | Urinary total GAG | 4 weeks | ||
Secondary | Liver and spleen volumes | 4 weeks | ||
Secondary | Cardiac function | 4 weeks |
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