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

Mucopolysaccharidosis II Clinical Trial

Official title:

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

Clinical Trial Details — Status: Completed

Administrative data

• NCT number: NCT03128593
• Other study ID #: JR-141-101
• Status: Completed
• Phase: Phase 1/Phase 2
• Start date: March 30, 2017
• Est. completion date: October 4, 2017

Study information

• Verified date: November 2022
• Source: JCR Pharmaceuticals Co., Ltd.
• Contact: n/a
• Is FDA regulated: No
• Study type: Interventional

Clinical Trial 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

Recruitment information / eligibility

• 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.

Related Conditions & MeSH terms

• Mucopolysaccharidoses
• Mucopolysaccharidosis II

Intervention

Drug:
• JR-141
IV infusion (lyophilized powder), 0.01-2.0 mg/kg/week

Locations

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

Sponsors (1)

Lead Sponsor	Collaborator
JCR Pharmaceuticals Co., Ltd.

Country where clinical trial is conducted

Japan, 

Outcome

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

External Links

•   Iduronate-2-Sulfatase with Anti-human Transferrin Receptor Antibody for Neuropathic Mucopolysaccharidosis II: A Phase 1/2 Trial