JR-441 Enzyme Replacement | Type IIIA | Phase I-II | JCR

October 23, 2023

Page last updated: October 23, 2023

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Trial Information

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:
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.

More information: View the listing on ClinicalTrials.gov. This includes information for contacting the trial investigator about enrolling.

Trial Updates

October 2023

In a press release on October 27, 2023, JCR Pharmaceuticals announces the first patient dosed in a global phase I/II clinical trial of JR-441 for the treatment of MPS IIIA. Read the press release.

Clinical trial is first listed on ClinicalTrials.gov and marked as “Recruiting.”

August 2023

Mathias Schmidt, PhD, Vice President of JCR Pharmaceuticals, presented an update on JCR Pharmaceutical’s Sanfilippo types A & B programs during ADVANCE 2023, the virtual Sanfilippo community conference. His presentation is available for on-demand viewing.

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