Evaluating JR-446 for treating Mucopolysaccharidosis Type IIIB
An Open-label Phase I/II Study of JR-446 in Mucopolysaccharidosis Type IIIB
This study is testing a new drug called JR-446 to see if it can help children with Mucopolysaccharidosis Type IIIB feel better and improve their health.
Quick facts
| Phase | Phase1; Phase2 |
|---|---|
| Study type | Interventional |
| Enrollment | 10 (estimated) |
| Ages | N/A to 17 Years |
| Sex | All |
| Sponsor | JCR Pharmaceuticals Co., Ltd. Industry-sponsored |
| Locations | 3 sites (Hiroshima and 2 other locations) |
| Trial ID | NCT06488924 on ClinicalTrials.gov |
What this trial studies
This is an open-label Phase I/II clinical trial designed to assess the safety and preliminary efficacy of JR-446 in patients diagnosed with Mucopolysaccharidosis Type IIIB. The study will involve children under 18 years of age who have a confirmed diagnosis of this rare genetic disorder. Participants will receive the investigational drug JR-446, and the outcomes will be monitored to determine its potential benefits and risks. The trial aims to gather important data that could inform future treatment options for this condition.
Who should consider this trial
Good fit: Ideal candidates for this study are children under 18 years old with a confirmed diagnosis of MPS IIIB.
Not a fit: Patients who have previously undergone gene therapy or hematopoietic stem cell transplantation with successful engraftment may not benefit from this study.
Why it matters
Potential benefit: If successful, this treatment could provide a new therapeutic option for children suffering from Mucopolysaccharidosis Type IIIB.
How similar studies have performed: While there have been studies on gene therapies for similar conditions, the specific approach of JR-446 in MPS IIIB is novel and has not been extensively tested.
Eligibility criteria
Show full inclusion / exclusion criteria
Inclusion Criteria: * Chronological age of \<18 years * Confirmed diagnosis of MPS IIIB 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
Hiroshima and 2 other locations
- Hiroshima University Hospital — Hiroshima, Japan (Recruiting)
- University of the Ryukyus Hospital — Okinawa, Japan (Recruiting)
- National Center for Child Health and Development — Tokyo, Japan (Recruiting)
Study contacts
- Study coordinator: JCR Pharmaceuticals Co., Ltd.
- Email: clinical_development@jp.jcrpharm.com
- Phone: +81-797-32-8582
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.