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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 part 1: baseline, 2,3,4,5 week part2: baseline, 2, 4, 6, 8, 10, 12, 13 week
Awards & highlights

Study 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).

Eligible Conditions
  • Hurler Syndrome

Timeline

Screening ~ 3 weeks
Treatment ~ Varies
Follow Up ~part 1: baseline, 2,3,4,5 week part2: baseline, 2, 4, 6, 8, 10, 12, 13 week
This trial's timeline: 3 weeks for screening, Varies for treatment, and part 1: baseline, 2,3,4,5 week part2: baseline, 2, 4, 6, 8, 10, 12, 13 week for reporting.

Treatment Details

Study Objectives

Outcome measures can provide a clearer picture of what you can expect from a treatment.
Primary outcome measures
Number of participants with Adverse Events
Secondary outcome measures
Assessment of pharmacokinetic parameter
Assessment of plasma drug concentration
Change From Baseline Drug concentration in Cerebrospinal Fluid.
+15 more

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
11 Previous Clinical Trials
292 Total Patients Enrolled

Frequently Asked Questions

These questions and answers are submitted by anonymous patients, and have not been verified by our internal team.
Recent research and studies
clinicaltrials.govStudy
A Study of JR-171 in Patients With Mucopolysaccharidosis I
2020. "A Study of JR-171 in Patients With Mucopolysaccharidosis I". ClinicalTrials.gov. https://clinicaltrials.gov/ct2/show/NCT04227600.
~4 spots leftby Apr 2025
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