JR-171 for Hurler Syndrome
What You Need to Know Before You Apply
What is the purpose of this trial?
This trial aims to test the safety and effectiveness of a new treatment called JR-171 (lepunafusp alfa) for individuals with Mucopolysaccharidosis I (MPS I), a rare genetic disorder that impairs the body's ability to break down certain sugars. Researchers will evaluate how the body processes the drug and whether it improves symptoms. Participants will receive the treatment through an intravenous (IV) infusion, with doses adjusted to determine the optimal amount. The trial is open to those diagnosed with MPS I who have been taking another treatment, laronidase, for at least three months. As a Phase 1 trial, this research focuses on understanding how the treatment works in people, offering participants the opportunity to be among the first to receive this new treatment.
Do I need to stop my current medications to join the trial?
The trial protocol does not specify if you need to stop taking your current medications. However, it mentions that participants must have been on a stable dosage of laronidase for at least 2 weeks before starting the trial, unless they are laronidase naïve or have had a specific treatment called HSCT.
Is there any evidence suggesting that JR-171 (lepunafusp alfa) is likely to be safe for humans?
Research has shown that JR-171, also known as lepunafusp alfa, has been tested for safety in treating mucopolysaccharidosis type I (MPS I). A 52-week study found that JR-171 is generally safe for long-term use in people with this condition. The results revealed no major safety issues, suggesting that the treatment is likely well-tolerated. In these studies, patients received JR-171 through an IV drip, and researchers tested different doses to ensure safety at various levels. While more research is needed, these early results are promising for those considering joining a trial.12345
Why do researchers think this study treatment might be promising?
Most treatments for Mucopolysaccharidosis I (MPS I) focus on enzyme replacement therapy, such as laronidase, to help break down the complex sugars that build up in the body. However, JR-171 (lepunafusp alfa) offers something new by crossing the blood-brain barrier, potentially addressing neurological symptoms that other treatments cannot reach. Researchers are excited about JR-171 because it not only involves a unique delivery method—an IV infusion with dose escalation—but it also holds promise for more comprehensive symptom management by targeting the brain directly, which is a significant step forward in treating MPS I.
What evidence suggests that JR-171 might be an effective treatment for MPS I?
Research shows that JR-171, also known as lepunafusp alfa, could help treat mucopolysaccharidosis type I (MPS I). Studies suggest this treatment might improve both physical and brain-related symptoms of MPS I. JR-171 is a specially designed protein that combines an enzyme with an antibody to better target cells. Early findings indicate it may improve conditions in patients with nerve-related MPS I over a 52-week period. These initial results are promising for those considering joining this trial, which includes different treatment arms with varying doses of JR-171.12356
Are You a Good Fit for This Trial?
Inclusion Criteria
Timeline for a Trial Participant
Screening
Participants are screened for eligibility to participate in the trial
Treatment
Participants receive JR-171 intravenously with dose escalation, either 2.0 or 4.0 mg/kg/week
Follow-up
Participants are monitored for safety and effectiveness after treatment
What Are the Treatments Tested in This Trial?
Interventions
- JR-171 (lepunafusp alfa)
Find a Clinic Near You
Who Is Running the Clinical Trial?
JCR Pharmaceuticals Co., Ltd.
Lead Sponsor