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A Study of JR-171 in Patients With Mucopolysaccharidosis I

NCT04227600 · Status: COMPLETED · Phase: PHASE1/PHASE2 · Type: INTERVENTIONAL · Enrollment: 18

Last updated 2025-03-19

Study results available
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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).

Conditions

  • Mucopolysaccharidosis I

Interventions

DRUG

JR-171 (lepunafusp alfa)

IV infusion

Sponsors & Collaborators

  • JCR Pharmaceuticals Co., Ltd.

    lead INDUSTRY

Study Design

Allocation
NON_RANDOMIZED
Purpose
TREATMENT
Masking
NONE
Model
PARALLEL

Eligibility

Min Age
0 Years
Sex
ALL
Healthy Volunteers
No

Timeline & Regulatory

Start
2020-09-01
Primary Completion
2022-08-02
Completion
2022-08-02
FDA Drug
Yes

Countries

  • United States
  • Brazil
  • Japan

Study Locations

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Read the full study record

This page highlights key information. For complete eligibility criteria, study locations, investigator contacts, and the full protocol, visit the original record on ClinicalTrials.gov.

View NCT04227600 on ClinicalTrials.gov