Page last updated: October 23, 2023
For web accessibility options: Click/tap the floating blue icon on the right.
Phase I/II Study of JR-441 in Patients With Mucopolysaccharidosis Type IIIA
A Phase I/ II, open-label, randomized, 2-arm study, designed to evaluate the safety and explore efficacy of the study drug in development for the treatment of MPS IIIA patients. Studying weekly infusions of JR-441 in patients with Mucopolysaccharidosis Type IIIA.
A uniqueness of JCR’s approach is the uses of its “J-Brain Cargo” technology, to bring the protein therapeutics across the blood-brain barrier. Typically, ERTs are unable to cross the blood-brain barrier and provide therapeutic benefit to the brain. However, JCR’s therapy fuses the enzyme to an antibody, and the antibody recognizes a receptor on the blood-brain barrier and actively cycles the fused enzyme-antibody into the brain.
Number of participants: 12 children with MPS IIIA (estimated), ages 12 months old to 18 years old
Duration: Up to 5 years
Locations: Hamburg, Germany
Qualifications to Participate:
- Chronological age of ≥1 year and ≤18 years.
- Confirmed diagnosis of MPS IIIA.
- Body weight ≥ 10 kg.
- 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.
More information: View the listing on ClinicalTrials.gov. This includes information for contacting the trial investigator about enrolling.