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Enzyme Replacement Therapy
JR-141 for Hunter Syndrome
Phase 3
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 [a-1: week 157, 209, 261] [a-2: week 131, 157, 183, 209, 261] [a-3: week 131, 157, 209, 261 ] [a-4: week 131, 157, 183, 235, 287][b-1: week 78, 105, 157, 209, 261] [b-2: week 78, 105, 131, 157, 209, 261]
Awards & highlights
Study Summary
This trial will test a drug's safety and effectiveness to treat a rare genetic disorder.
Who is the study for?
This trial is for individuals with Hunter Syndrome who previously participated in the JR-141-GS31 study without safety concerns. They must be able to give consent or have a legal representative do so, agree to use effective contraception, and be capable of following the study protocol.Check my eligibility
What is being tested?
The trial is an extension study testing the long-term safety and effectiveness of JR-141 in treating Mucopolysaccharidosis Type II (Hunter Syndrome). It's open-label and multicenter, meaning everyone knows what treatment they're getting and it's conducted at multiple sites.See study design
What are the potential side effects?
While specific side effects are not listed here, participants will be monitored for any long-term safety issues related to the use of JR-141. This could include allergic reactions or other potential risks associated with ongoing treatment.
Timeline
Screening ~ 3 weeks3 visits
Treatment ~ Varies
Follow Up ~ [a-1: week 157, 209, 261] [a-2: week 131, 157, 183, 209, 261] [a-3: week 131, 157, 209, 261 ] [a-4: week 131, 157, 183, 235, 287][b-1: week 78, 105, 157, 209, 261] [b-2: week 78, 105, 131, 157, 209, 261]
Screening ~ 3 weeks
Treatment ~ Varies
Follow Up ~[a-1: week 157, 209, 261] [a-2: week 131, 157, 183, 209, 261] [a-3: week 131, 157, 209, 261 ] [a-4: week 131, 157, 183, 235, 287][b-1: week 78, 105, 157, 209, 261] [b-2: week 78, 105, 131, 157, 209, 261]
Treatment Details
Study Objectives
Outcome measures can provide a clearer picture of what you can expect from a treatment.Primary outcome measures
Change in heparan sulfate concentrations in cerebrospinal fluid from each subject's first administration of JR-141 throughout the Parent Study (JR-141-GS31) or this study to each time point
Change in neurocognitive testing, calculated by the BSID-III or KABC-II, from each subject's first administration of JR-141 throughout the Parent Study (JR-141-GS31) or this study
Change in neurocognitive testing, calculated by the VABS-II from each subject's first administration of JR-141 throughout the Parent Study (JR-141-GS31) or this study
+1 moreSecondary outcome measures
Change in 6-minute walk distance from each subject's first administration of JR-141 throughout the Parent Study (JR-141-GS31) or this study
Change in liver and spleen volume from each subject's first administration of JR-141 throughout the Parent Study (JR-141-GS31) or this study
Trial Design
1Treatment groups
Experimental Treatment
Group I: JR-141 2.0 mg/kg/weekExperimental Treatment1 Intervention
Treatment
First Studied
Drug Approval Stage
How many patients have taken this drug
JR-141
2018
Completed Phase 3
~60
Find a Location
Who is running the clinical trial?
JCR Pharmaceuticals Co., Ltd.Lead Sponsor
11 Previous Clinical Trials
231 Total Patients Enrolled
Media Library
Eligibility Criteria:
This trial includes the following eligibility criteria:- I switched from JR-141 to idursulfase in a previous study.I have received gene therapy treatment before.I agree to use effective birth control during and up to 90 days after the study for men, 30 days for women.I do not have chronic infections or a risk of abnormal bleeding that would make me ineligible.I (or my legal guardian) have signed the consent form to participate in the study.
Research Study Groups:
This trial has the following groups:- Group 1: JR-141 2.0 mg/kg/week
Awards:
This trial has 2 awards, including:- Pivotal Trial - The final step before approval, pivotal trials feature drugs that have already shown basic safety & efficacy.
- 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 patients able to join this research project at this time?
"Unfortunately, based on the information available on clinicaltrials.gov, this study is no longer searching for patients. Although this particular trial is completed, there are 30 other studies that are still looking for volunteers."
Answered by AI
Has the FDA greenlit JR-141 2.0 mg/kg/week for patient use?
"JR-141 2.0 mg/kg/week is estimated to be a safe drug by our team at Power, who gave it a score of 3. This is due in part because this medication has completed Phase 3 trials, meaning that there is some data supporting its efficacy as well as multiple rounds of data affirming its safety."
Answered by AI
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