A Study of JR-141 in Patients With Mucopolysaccharidosis Type II

Phase 1

Completed

• Conditions: Mucopolysaccharidosis II
• Interventions: Drug: JR-141

• Registration Number: NCT03128593
• Lead Sponsor: JCR Pharmaceuticals Co., Ltd.

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 Description

Not available

Study Timeline

• Study Start: 2017-03-30
• Study First Submitted: 2017-04-10
• Study First Submitted QC: 2017-04-20
• Study First Posted: 2017-04-25
• Primary Completion: 2017-10-04
• Study Completion: 2017-10-04
• Status Verified: 2022-11
• Last Update Submitted: 2022-11-10
• Last Update Posted: 2022-11-14

Recruitment & Eligibility

• Status: COMPLETED
• Sex: Male
• Target Recruitment: 14

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.

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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.

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Study & Design

• Study Type: INTERVENTIONAL
• Study Design: SEQUENTIAL

Arm && Interventions

Group	Intervention	Description
Experimental: JR-141	JR-141	-

Primary Outcome Measures

Name	Time	Method
Number of participants with Adverse Events	4 weeks	* Adverse events; * Laboratory tests; * Vital signs; * 12-lead electrocardiogram; * Antibody; * Infusion associated reaction

Secondary Outcome Measures

Name	Time	Method
Plasma Pharmacokinetic parameter [Maximum Plasma Concentration [Cmax]]	4 weeks	Plasma concentration of JR-141
Urinary total GAG	4 weeks
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
Liver and spleen volumes	4 weeks
Cardiac function	4 weeks

Trial Locations

Locations (8)

Osaka Clinical site2; 🇯🇵 Suita, Japan

Fukuoka Clinical site; 🇯🇵 Kurume, Japan

Tottori Clinical site; 🇯🇵 Yonago, Japan

Tokyo clinical site1; 🇯🇵 Minato, Japan

Tokyo clinical site2; 🇯🇵 Setagaya, Japan

Gifu Clinical site; 🇯🇵 Gifu, Japan

Saitama Clinical site; 🇯🇵 Saitama, Japan

Osaka Clinical site1; 🇯🇵 Osaka, Japan