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Enzyme Replacement Therapy
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 weeks 13, 26, 39, 52, 65, 78, 91, 104, 130, 156
Awards & highlights
Study Summary
This trial is testing an experimental drug for MPS I, a disease that causes progressive damage to the body. The trial is open to people in Japan, Brazil, and the US.
Who is the study for?
This trial is for patients with Hurler Syndrome or Mucopolysaccharidosis (MPS I) who completed the JR-171-101 study. It's open to adults and minors with consent from a legal guardian. Participants must agree to use effective contraception post-treatment.Check my eligibility
What is being tested?
The trial is testing JR-171 (lepunafusp alfa), an extension of a previous study, across multiple countries. It's in Phase I/II, meaning it's early in clinical testing but expanding to assess safety and effectiveness further.See study design
What are the potential side effects?
While specific side effects are not listed here, typical concerns may include reactions at the injection site, potential allergic responses, and other drug-related adverse effects which will be monitored throughout the study.
Timeline
Screening ~ 3 weeks3 visits
Treatment ~ Varies
Follow Up ~ weeks 13, 26, 39, 52, 65, 78, 91, 104, 130, 156
Screening ~ 3 weeks
Treatment ~ Varies
Follow Up ~weeks 13, 26, 39, 52, 65, 78, 91, 104, 130, 156
Treatment Details
Study Objectives
Outcome measures can provide a clearer picture of what you can expect from a treatment.Primary outcome measures
Incidence of abnormal EKG readings
Incidence of abnormal laboratory test results
Incidence of abnormal vital signs
+1 moreSecondary outcome measures
CSF opening pressure
Change From Baseline in 6-minute Walk Test Distance.
Cerebrospinal fluid shunts procedure
+14 moreTrial Design
1Treatment groups
Experimental Treatment
Group I: JR-171Experimental Treatment1 Intervention
Until the dose determination, subjects will intravenously receive either the low dose or high dose of JR-171 (the same dose as at Week 12 of the JR-171-101 study). Thereafter, all subjects will receive the optimal dose of JR-171 determined based on the results of JR-171-101 study.
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
297 Total Patients Enrolled
Media Library
Eligibility Criteria:
This trial includes the following eligibility criteria:- I agree to use effective birth control during and for one month after the trial.I can't do most study tests but can walk for 6 minutes and do some brain tests.You have already finished the Part 2 of the JR-171-101 study.
Research Study Groups:
This trial has the following groups:- Group 1: JR-171
Awards:
This trial has 1 awards, including:- No Placebo-Only Group - All patients enrolled in this study will receive some form of active treatment.
Timeline:
This trial has the following timeline:- Screening: It may take up to 3 Weeks to process to see if you qualify in this trial.
- Treatment: The duration you will receive the treatment varies.
- Follow Ups: You may be asked to continue sharing information regarding the trial for 6 Months after you stop receiving the treatment.
Frequently Asked Questions
These questions and answers are submitted by anonymous patients, and have not been verified by our internal team.
Are there any opportunities to participate in this experiment currently being offered?
"The data found on clinicaltrials.gov indicates that recruitment for this study has ceased, as the last time it was updated was August 16th 2022. Despite this inactivity, there are 28 other medical trials currently recruiting participants at present."
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