← Back to Search

Enzyme Replacement Therapy

Part1 JR-171 for Hurler Syndrome

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

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.

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 Sep 2025
Unbiased ResultsWe believe in providing patients with all the options.
Your Data Stays Your DataWe only share your information with the clinical trials you're trying to access.
Verified Trials OnlyAll of our trials are run by licensed doctors, researchers, and healthcare companies.
Back to top