Evaluating JR-441 for treating Mucopolysaccharidosis Type IIIA
Phase I/II Study of Weekly Infusions of JR-441 in Patients With Mucopolysaccharidosis Type IIIA
This study is testing a new drug called JR-441 to see if it is safe and effective for treating children and young adults with Mucopolysaccharidosis Type IIIA.
Quick facts
| Phase | Phase1; Phase2 |
|---|---|
| Study type | Interventional |
| Enrollment | 12 (estimated) |
| Ages | 1 Year to 18 Years |
| Sex | All |
| Sponsor | JCR Pharmaceuticals Co., Ltd. Industry-sponsored |
| Locations | 1 site (Hamburg) |
| Trial ID | NCT06095388 on ClinicalTrials.gov |
What this trial studies
This is a Phase I/II, open-label, randomized study designed to assess the safety and explore the efficacy of the investigational drug JR-441 in patients diagnosed with Mucopolysaccharidosis Type IIIA. The study will involve participants aged between 1 and 18 years who meet specific inclusion criteria. The trial will be conducted in two arms to compare the effects of the treatment. The primary focus is to gather data on how well the drug works and its safety profile in this patient population.
Who should consider this trial
Good fit: Ideal candidates for this study are children and adolescents aged 1 to 18 years with a confirmed diagnosis of MPS IIIA.
Not a fit: Patients with a history of successful gene therapy or hematopoietic stem cell transplantation may not benefit from this study.
Why it matters
Potential benefit: If successful, this treatment could significantly improve the quality of life for patients with Mucopolysaccharidosis Type IIIA.
How similar studies have performed: While this approach is novel for MPS IIIA, similar studies in other mucopolysaccharidoses have shown promising results.
Eligibility criteria
Show full inclusion / exclusion criteria
Inclusion Criteria: * Chronological age of ≥1 year and ≤18 years. * Confirmed diagnosis of MPS IIIA. * Body weight ≥ 10 kg. Exclusion Criteria: * Prior experience to gene therapy or HSCT with successful engraftment. * Past use of another investigational drug or product in last 4 months or 5 half-lives (whichever is longer) before signing ICF. * Current participation in a clinical trial or past participation (within 30 days of enrolment into this study) in a study involving invasive procedures. * Past use of Genistein or Kineret (anakinra) within 4 months before signing ICF. * Serious drug allergy or hypersensitivity. * Contraindication for lumbar puncture or MRI. * History of bleeding disorder or current use of medications that, in the opinion of the investigator, place them at risk of bleeding following lumbar puncture. The above information is not intended to contain all considerations relevant to a patient's potential participation in this clinical trial.
Where this trial is running
Hamburg
- Universitätsklinikum Hamburg-Eppendorf — Hamburg, Germany (Recruiting)
Study contacts
- Study coordinator: JCR Pharmaceuticals Co., Ltd.
- Email: clinical_development@jp.jcrpharm.com
- Phone: +81-(0)797-32-8582
How to participate
- Review the eligibility criteria above with your treating physician.
- Visit the official trial page on ClinicalTrials.gov for the most current contact information and recruitment status.
- Contact the listed study coordinator or principal investigator to request pre-screening. Pre-screening is free and never obligates you to enroll.