NCT04227600

A Study of JR-171 in Patients With Mucopolysaccharidosis I

Phase I/II Study of JR-171 ㏌ Patients With Mucopolysaccharidosis Type I

• Status: Completed
• Phase: Phase 1 / Phase 2
• Study type: Interventional
• Enrollment: 18 (actual)
• Sponsor: JCR Pharmaceuticals Co., Ltd. · Industry
• Sex: All
• Age: 0 Years
• Healthy volunteers: Not accepted

Summary

Phase I/II, open-label, multicenter, multinational (Japan, Brazil and US),designed to evaluate the safety, pharmacokinetics and explore the efficacy for the treatment of mucopolysaccharidosis type I (MPS I).

Detailed description

In the JR-171-102 study, subjects will receive either 2.0 or 4.0 mg/kg/week of JR-171 intravenously at the same dosages received at Week 12 of the JR-171-101 study.

Conditions

• Mucopolysaccharidosis I

Interventions

Type	Name	Description
DRUG	JR-171 (lepunafusp alfa)	IV infusion

Timeline

• Start date: 2020-09-01
• Primary completion: 2022-08-02
• Completion: 2022-08-02
• First posted: 2020-01-13
• Last updated: 2025-03-19
• Results posted: 2025-03-19

Locations

6 sites across 3 countries: United States, Brazil, Japan

Regulatory

• FDA-regulated drug study