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Enzyme Replacement Therapy

A Study of JR-171 in Patients With Mucopolysaccharidosis I

Phase 1 & 2
Waitlist Available
Research Sponsored by JCR Pharmaceuticals Co., Ltd.
Eligibility Criteria Checklist
Specific guidelines that determine who can or cannot participate in a clinical trial
Must have
Timeline
Screening 3 weeks
Treatment Varies
Follow Up part1: 1,2,3,4 week, part2: 1, 4, 12 week
Awards & highlights
No Placebo-Only Group

Summary

This trial is testing a new treatment for patients with mucopolysaccharidosis type I (MPS I). The goal is to see if the treatment is safe and how it behaves in the body. The treatment likely works by replacing or supplementing a missing enzyme. Laronidase, an enzyme replacement therapy, has been used to treat MPS I by supplementing the deficient alpha-L-iduronidase enzyme.

Eligible Conditions
  • Hurler Syndrome

Timeline

Screening ~ 3 weeks
Treatment ~ Varies
Follow Up ~part1: 1,2,3,4 week, part2: 1, 4, 12 week
This trial's timeline: 3 weeks for screening, Varies for treatment, and part1: 1,2,3,4 week, part2: 1, 4, 12 week for reporting.

Treatment Details

Study Objectives

Study objectives can provide a clearer picture of what you can expect from a treatment.
Primary study objectives
Number of participants with Adverse Events
Secondary study objectives
Assessment of pharmacokinetic parameter
Change From Baseline in Echocardiography.

Awards & Highlights

No Placebo-Only Group
All patients enrolled in this study will receive some form of active treatment.

Trial Design

2Treatment groups
Experimental Treatment
Group I: Part2 JR-171Experimental Treatment1 Intervention
Drug: JR-171 IV infusion, dose escalation, low dose, high dose
Group II: Part1 JR-171Experimental Treatment1 Intervention
Drug: JR-171 IV infusion, dose escalation
Treatment
First Studied
Drug Approval Stage
How many patients have taken this drug
JR-171 (lepunafusp alfa)
2020
Completed Phase 2
~20

Find a Location

Who is running the clinical trial?

JCR Pharmaceuticals Co., Ltd.Lead Sponsor
12 Previous Clinical Trials
302 Total Patients Enrolled
~4 spots leftby Dec 2025
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