JR-141 for Hunter Syndrome

Recruiting at 5 trial locations

Overseen By JCR Pharmaceuticals Co., Ltd.

Age: Any Age

Sex: Any

Trial Phase: Phase 3

Sponsor: JCR Pharmaceuticals Co., Ltd.

Must not be taking: Gene therapy, NSAIDs

Disqualifiers: Gene therapy, Drug allergy, others

No Placebo Group All trial participants will receive the active study treatment (no placebo)

Pivotal Trial (Near Approval) This treatment is in the last trial phase before FDA approval

Prior Safety Data This treatment has passed at least one previous human trial

Approved in 1 Jurisdiction This treatment is already approved in other countries

Compensation: Varies

Number of Visits: Unknown

Duration: 2 years

80 Participants Needed

Trial Summary

What is the purpose of this trial?

This trial is testing a medication to see if it is safe and effective for patients with MPS II, a rare genetic disorder. The drug works by replacing or fixing a missing or malfunctioning enzyme to help reduce symptoms. Idursulfase has been used to treat MPS II by replacing the deficient enzyme.

Do I need to stop my current medications for the trial?

The trial information does not specify if you need to stop taking your current medications. However, if you are using drugs that affect bleeding or platelet function, you should stop them 14 days before a lumbar catheter insertion.

Eligibility Criteria

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.

Inclusion Criteria

I agree to use effective birth control during and up to 90 days after the study for men, 30 days for women.

Subject who participated in the Parent Study (JR-141-GS31) and completed the assessments at Week 105 in Cohort A or Week 53 in Cohort B, and in the opinion of the principal investigator has no safety concerns to enter this study

I (or my legal guardian) have signed the consent form to participate in the study.

Exclusion Criteria

I switched from JR-141 to idursulfase in a previous study.

I have received gene therapy treatment before.

Refusal to sign the ICF

Timeline

Screening

Participants are screened for eligibility to participate in the trial

2-4 weeks

Treatment

Participants receive JR-141 2.0 mg/kg/week for the treatment of MPS II

2 years

Follow-up

Participants are monitored for safety and effectiveness after treatment

4 weeks

Open-label extension

An extension study to evaluate the long-term safety and efficacy of JR-141

Long-term

Treatment Details

Interventions

• JR-141

Trial Overview 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.

Participant Groups

Group I: JR-141 2.0 mg/kg/week Experimental Treatment 1 Intervention

JR-141 is already approved in Japan for the following indications:

Approved in Japan as IZCARGO for:

• Mucopolysaccharidosis type II (MPS II)
• Hunter syndrome

Who Is Running the Clinical Trial?

JCR Pharmaceuticals Co., Ltd.

Lead Sponsor

Trials

13

Recruited

320+