NCT04453085

An Extension Study of JR-171-101 Study in Patients With Mucopolysaccharidosis Type I (MPS I)

An Extension Study of JR-171-101 Study in Patients With Mucopolysaccharidosis Type I

• Status: Completed
• Phase: Phase 1 / Phase 2
• Study type: Interventional
• Enrollment: 14 (actual)
• Sponsor: JCR Pharmaceuticals Co., Ltd. · Industry
• Sex: All
• Age: —
• Healthy volunteers: Not accepted

Summary

Phase I/II, open label, multicenter, multinational (Japan, Brazil and the US) extension study of JR-171-101 (NCT04227600) for the treatment of MPS I

Detailed description

Patients who have completed the Part2 of JR-171-101 study (NCT04227600) and fulfill all eligibility criteria can be enrolled in this JR-171-102 study. 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 doses received at Week 12 of the JR-171-101 study.

Conditions

• Mucopolysaccharidosis I

Interventions

Type	Name	Description
DRUG	JR-171	Intravenous (IV) infusion

Timeline

• Start date: 2021-10-28
• Primary completion: 2025-05-02
• Completion: 2025-05-02
• First posted: 2020-07-01
• Last updated: 2025-08-28

Locations

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

Regulatory

• FDA-regulated drug study