JR-141 for Hunter Syndrome

Recruiting at 28 trial locations

Age: Any Age

Sex: Any

Travel: May Be Covered

Time Reimbursement: Varies

Trial Phase: Phase 3

Recruiting

Sponsor: JCR Pharmaceuticals Co., Ltd.

Must be taking: Enzyme replacement

Disqualifiers: Gene therapy, HSCT, Brain surgery, others

No Placebo Group

Pivotal Trial (Near Approval)

Prior Safety Data

Approved in 1 Jurisdiction

Trial Summary

What is the purpose of this trial?

This trial is testing a new medicine for patients with MPS II to see if it is safe and effective. Previous research in animals helped design this trial to evaluate the new medicine for MPS II patients.

Will I have to stop taking my current medications?

The trial does not specify if you need to stop taking your current medications. However, if you are on stable enzyme replacement therapy with idursulfase, you can continue it during the study.

Research Team

Eligibility Criteria

This trial is for patients with Hunter Syndrome (MPS II). Eligible participants include those diagnosed with MPS II, either treatment-naïve or on stable enzyme therapy. They must agree to use effective contraception and sign consent forms. There are specific age-related cognitive criteria for two separate cohorts: Cohort A includes children aged 36-71 months with certain developmental scores, while Cohort B includes individuals aged 6 years or older with an IQ of 70 or higher.

Inclusion Criteria

Patients or patients whose partners are of child-bearing potential agree to use a medically accepted, highly effective method of contraception being use of condoms from the time of informed consent

For children 36-42 months old, their measured development score must be 85 or less. For children 43-71 months old, either their development quotient should be between 20 to 85, or their composite standard score on a specific test should be 85 or less. Children 30-35 months old must be assessed as having a severe condition by an Expert Board.

I am 6 or older with an IQ of 70 or more and fit the specific test criteria for Cohort B.

Exclusion Criteria

I have had a successful stem cell transplant.

Judged by the principal investigator or subinvestigator to be ineligible to participate in the study due to a history of serious drug allergy or sensitivity including anesthesia or hypersensitivity to any component of JR-141

I have a genetic mutation linked to developmental delays or seizures.

Trial 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 or standard of care idursulfase (ELAPRASE®) for the treatment of MPS II

105 weeks

Weekly visits for treatment administration

Follow-up

Participants are monitored for safety and effectiveness after treatment, including assessments of antibodies and cognitive testing

4 weeks

Visits at Week 26, 53, 78, and 105

Treatment Details

Interventions

• Idursulfase (Enzyme Replacement Therapy)
• JR-141 (Enzyme Replacement Therapy)

Trial Overview The STARLIGHT Phase III trial is testing the safety and effectiveness of a drug called JR-141 compared to Idursulfase in treating MPS II. Participants will be randomly assigned to receive either JR-141 alone, JR-141 combined with Idursulfase, or only Idursulfase in a blinded manner where assessors do not know which treatment each participant receives.

Participant Groups

Group I: administered as the standard of care: idursulfase (ELAPRASE®) Experimental Treatment 1 Intervention

standard of care-controlled study

Group II: Rescue arm Experimental Treatment 1 Intervention

Group III: 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+