JR-171 for Hurler Syndrome

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

14 Participants Needed

Trial Summary

What is the purpose of this trial?

This trial continues to test JR-171, an IV medication, in patients with MPS I. The goal is to find the best dose to help manage their symptoms by aiding in the breakdown of certain substances in their bodies.

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.

What data supports the effectiveness of the drug JR-171 for Hurler Syndrome?

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 related condition, MPS I. This suggests it might help improve symptoms in patients with neuronopathic MPS I, which includes Hurler Syndrome, without serious safety concerns. ^{1 2 3 4 5}

Eligibility Criteria

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.

A patient from whom written informed consent can be obtained. If the patient is aged under 18 years (20 years in case of Japan) at the time of assent or willingness to participate in the study cannot be confirmed due to MPS I-related intellectual disability, informed permission from the patient's legally acceptable representative (e.g., his/her parents or guardians) need to be obtained instead of his/her consent. Even in this case, written informed consent should be obtained from the patient, wherever possible

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

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

Treatment Details

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.

Participant Groups

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+

Findings from Research

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]

References

1. England pubmed.ncbi.nlm.nih.gov

Neurocognitive and somatic stabilization in pediatric patients with severe Mucopolysaccharidosis Type I after 52 weeks of intravenous brain-penetrating insulin receptor antibody-iduronidase fusion protein (valanafusp alpha): an open label phase 1-2 trial. [2019]

2. United States pubmed.ncbi.nlm.nih.gov

Enzyme replacement with transferrin receptor-targeted α-L-iduronidase rescues brain pathology in mucopolysaccharidosis I mice. [2023]

3. United States pubmed.ncbi.nlm.nih.gov

Long-term safety and clinical outcomes of intrathecal heparan-N-sulfatase in patients with Sanfilippo syndrome type A. [2022]

4. England pubmed.ncbi.nlm.nih.gov

Post-transplant laronidase augmentation for children with Hurler syndrome: biochemical outcomes. [2021]

5. England pubmed.ncbi.nlm.nih.gov

Transplantation of allogeneic CD34-selected stem cells after fludarabine-based conditioning regimen for children with mucopolysaccharidosis 1H (M. Hurler). [2013]