JR-171 for Hurler Syndrome

No longer recruiting at 6 trial locations

Overseen By JCR Pharmaceuticals Co., Ltd.

Age: Any Age

Sex: Any

Trial Phase: Phase 1 & 2

Sponsor: JCR Pharmaceuticals Co., Ltd.

Disqualifiers: Serious drug allergy, others

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

Compensation: Varies

Number of Visits: Unknown

Duration: 156 weeks

What You Need to Know Before You Apply

What is the purpose of this trial?

This trial tests a treatment called JR-171, also known as lepunafusp alfa, for Hurler Syndrome, a severe condition affecting growth and development. Researchers are exploring two different doses to evaluate their effectiveness and monitor any side effects. This trial is open to individuals who have participated in an earlier related study and have Hurler Syndrome. Participants must either consent to the trial themselves or have a guardian provide permission. As a Phase 1/Phase 2 trial, this research aims to understand how the treatment works in people and measure its effectiveness in an initial, smaller group.

Will I have to stop taking my current medications?

The trial information does not specify whether you need to stop taking your current medications. It's best to discuss this with the trial team or your doctor.

Is there any evidence suggesting that JR-171 (lepunafusp alfa) is likely to be safe for humans?

A previous study administered JR-171 to patients with MPS I for a year. The study found that patients generally tolerated JR-171 well. Some side effects occurred, but they were mostly mild or moderate, not severe.

Research in animals suggests that JR-171 can reach the brain and help reduce harmful substances. Although animal studies do not always predict human outcomes, this finding is promising for safety.

The current trial is in an early stage, focusing primarily on the safety of JR-171 in humans. Early-stage trials often emphasize safety, so participants might experience some side effects. However, early data and research suggest that JR-171 could be safe for people with Hurler syndrome. ^{1 2 3 4 5}

Why do researchers think this study treatment might be promising?

Unlike the standard enzyme replacement therapies for Hurler Syndrome, which are often limited by their inability to effectively cross the blood-brain barrier, JR-171 uses a unique mechanism to address this challenge. JR-171, or lepunafusp alfa, combines the enzyme iduronidase with a receptor-mediated transport system, allowing it to reach the brain and potentially improve neurological outcomes. Researchers are excited about JR-171 because it offers the promise of more comprehensive treatment, targeting both the physical and cognitive symptoms of Hurler Syndrome more effectively than current options.

What evidence suggests that JR-171 might be an effective treatment for Hurler Syndrome?

Research has shown that JR-171 (lepunafusp alfa) may help treat Hurler Syndrome, also known as MPS I. In earlier studies, patients using JR-171 experienced improvements, particularly in brain-related symptoms. This trial will examine different dosages of JR-171, with participants receiving either 2.0 mg/kg/week or 4.0 mg/kg/week. The treatment combines an enzyme with a special protein that helps it reach the brain, reducing harmful substances. Interim results from a 52-week study were positive, suggesting that JR-171 effectively manages symptoms of MPS I. Although researchers are still studying this treatment, early findings are promising and support its potential effectiveness. ^{1 2 3 4 6}

Are You a Good Fit for This Trial?

This trial is for patients with Hurler Syndrome or Mucopolysaccharidosis (MPS I) who completed the JR-171-101 study. It's open to adults and minors with consent from a legal guardian. Participants must agree to use effective contraception post-treatment.

Inclusion Criteria

I agree to use effective birth control during and for one month after the trial.

You have already finished the Part 2 of the JR-171-101 study.

Exclusion Criteria

Judged by the principal investigator or subinvestigator as being unable to undergo lumbar puncture, including those who have difficulties in taking a position for lumbar puncture due to joint contracture or those who are likely to experience difficulty breathing during the lumbar puncture process

Otherwise judged by the principal investigator or subinvestigator to be ineligible to participate in the study in consideration of patient's safety

I can't do most study tests but can walk for 6 minutes and do some brain tests.

Timeline for a Trial Participant

Screening

Participants are screened for eligibility to participate in the trial

2-4 weeks

Treatment

Participants receive either 2.0 or 4.0 mg/kg/week of JR-171 intravenously

156 weeks

Follow-up

Participants are monitored for safety and effectiveness after treatment

4 weeks

Open-label extension

Participants continue receiving the study drug as part of the extension study

Long-term

What Are the Treatments Tested in This Trial?

Interventions

• JR-171 (lepunafusp alfa)

Trial Overview The trial is testing JR-171 (lepunafusp alfa), an extension of a previous study, across multiple countries. It's in Phase I/II, meaning it's early in clinical testing but expanding to assess safety and effectiveness further.

How Is the Trial Designed?

Group I: Dose level 2 Experimental Treatment 1 Intervention

4.0 mg/kg/week

Group II: Dose level 1 Experimental Treatment 1 Intervention

2.0 mg/kg/week

Who Is Running the Clinical Trial?

JCR Pharmaceuticals Co., Ltd.

Lead Sponsor

Trials

13

Recruited

320+

Published Research Related to This Trial

A fludarabine-based conditioning regimen for hematopoietic cell transplantation in five children with Hurler syndrome showed low toxicity, with no severe regimen-related toxicity observed, indicating a safer approach for these patients.

All five patients survived at a median follow-up of 32 months, demonstrating sustained donor engraftment, with three achieving full donor chimerism, suggesting that this treatment can effectively stabilize the condition and improve long-term outcomes.

NCBI (Pubmed)

pubmed.ncbi.nlm.nih.gov

Transplantation of allogeneic CD34-selected stem cells after fludarabine-based conditioning regimen for children with mucopolysaccharidosis 1H (M. Hurler). Grigull, L., Beilken, A., Schrappe, M., et al.[2013]

Citations

1. biospace.com biospace.com/jcr-pharmaceuticals-announces-52-week-interim-data-from-its-global-phase-i-ii-study-of-jr-171-in-individuals-with-mucopolysaccharidosis-type-i-mps-i

JCR Pharmaceuticals Announces 52-Week Interim Data ... JCR Pharmaceuticals Co., Ltd. announced key results from the 52-week interim data of its global phase I/II study with JR-171 in individuals ...

2. pmc.ncbi.nlm.nih.gov pmc.ncbi.nlm.nih.gov/articles/PMC10220318/

Enzyme replacement with transferrin receptor-targeted α-L ... This study provides nonclinical evidence that JR-171 might potentially prevent and even improve disease conditions in patients with neuronopathic MPS I without ...

3. delveinsight.com delveinsight.com/blog/mps-i-treatment-pipeline

4 Emerging MPS I Therapies on the Horizon ISP-001 is an autologous B cell therapy engineered to produce human alpha-L-iduronidase for treating MPS I (Hurler syndrome). The drug is ...

4. pmc.ncbi.nlm.nih.gov pmc.ncbi.nlm.nih.gov/articles/PMC10928130/

α-L-iduronidase fused with humanized anti-human transferrin ... We have developed a novel genetically modified protein consisting of IDUA fused with humanized anti-human transferrin receptor antibody (lepunafusp alfa; JR-171) ...

5. withpower.com withpower.com/trial/phase-2-mucopolysaccharidoses-9-2021-a28a4

JR-171 for Hurler Syndrome · Info for Participants Research on JR-171, a drug similar to lepunafusp alfa, shows it can reach the brain and reduce harmful substances in the body and brain in animal models of a ...

6. prnewswire.com prnewswire.com/news-releases/mucopolysaccharidosis-type-i-market-to-witness-upsurge-in-growth-at-a-cagr-of-8-during-the-forecast-period-20252034--delveinsight-302448307.html

Mucopolysaccharidosis Type I Market to Witness Upsurge ... The condition presents as a spectrum, with the most severe form being Hurler syndrome and the mildest form known as Scheie syndrome. Diagnosis ...