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

JCR Pharmaceuticals

Status and phase

Completed

Phase 2

Phase 1

Conditions

Mucopolysaccharidosis II

Treatments

Drug: JR-141

Study type

Interventional

Funder types

Industry

Identifiers

JR-141-101

Details and patient eligibility

About

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

Enrollment

14 patients

Sex

Male

Ages

6+ years old

Volunteers

No Healthy Volunteers

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.