What side effects are associated with this type of intervention?

The most common treatment for Hunter Syndrome, particularly enzyme replacement therapy (ERT) like Idursulfase, is generally well-tolerated but can have side effects. These include infusion-associated reactions, which are typically mild to moderate and self-limiting. Common reactions may include fever, headache, rash, and mild to moderate infusion site reactions. Serious allergic reactions are rare but can occur. Long-term safety data is still being evaluated, but current evidence suggests that serious adverse effects are uncommon.

What prior FDA approvals exist?

Hunter Syndrome, also known as Mucopolysaccharidosis Type II (MPS II), has been treated with enzyme replacement therapies (ERTs). The FDA approved Idursulfase (brand name Elaprase) in 2006, which is a recombinant form of the human iduronate-2-sulfatase enzyme. This treatment helps to replace the deficient enzyme in patients with Hunter Syndrome, thereby reducing the accumulation of glycosaminoglycans (GAGs) in the body. Idursulfase is administered via intravenous infusion and has been shown to improve walking capacity and reduce spleen and liver size in affected individuals.

What other types of research exist?

A promising novel alternative to Idursulfase for Hunter Syndrome patients is JR-141, which is currently being evaluated in an extension study for its long-term safety and efficacy. Patients should discuss with their healthcare provider about the availability and suitability of JR-141 as a treatment option in 2024.

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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, a-2, a-3: week 157, 209, 261] [a-4: week 131, 183, 235, 287]

Awards & highlights

Pivotal Trial

No Placebo-Only Group

Summary

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.

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.

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.

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, a-2, a-3: week 157, 209, 261] [a-4: week 131, 183, 235, 287]

Screening ~ 3 weeks

Treatment ~ Varies

Follow Up ~[a-1, a-2, a-3: week 157, 209, 261] [a-4: week 131, 183, 235, 287]

This trial's timeline: 3 weeks for screening, Varies for treatment, and [a-1, a-2, a-3: week 157, 209, 261] [a-4: week 131, 183, 235, 287] for reporting.

Treatment Details

Study Objectives

Study objectives can provide a clearer picture of what you can expect from a treatment.

Primary study objectives

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

Awards & Highlights

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.

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

Do I qualify?Apply below to find out

Research Highlights

Information in this section is not a recommendation. We encourage patients to speak with their healthcare team when evaluating any treatment decision.

Mechanism Of Action

Side Effect Profile

Prior Approvals

Other Research

Enzyme Replacement Therapy (ERT) with Idursulfase is a common treatment for Hunter Syndrome (Mucopolysaccharidosis Type II). The mechanism of action involves supplementing the deficient or absent iduronate-2-sulfatase enzyme in patients, which is crucial for breaking down glycosaminoglycans (GAGs). Accumulation of GAGs in various tissues leads to the symptoms and complications of Hunter Syndrome. By providing the missing enzyme, ERT helps reduce GAG accumulation, thereby alleviating symptoms and improving the quality of life for patients. This treatment is significant as it directly addresses the underlying enzyme deficiency, offering a targeted approach to managing the disease.