This is a global, open-label, Phase I/II, interventional trial in participants younger than 6 years of age with Mucopolysaccharidosis Type IIIB (MPS IIIB), designed to assess the safety and tolerability of JR-446, determine its pharmacodynamic effects, and explore its potential to demonstrate early clinical effects on disease-relevant outcomes.
Patients with confirmed MPS IIIB with a phenotype of MPS IIIB that is not classified as slowly progressive (i.e., not attenuated).
A chronological age <6 years.
Ability to complete and achieve a Standard score ≥55, using either Bailey Scale of Infant Development-4 (BSID-4) Cognitive domain or Kaufman Assessment Battery for Children-II (KABC-II) Non-Verbal Index, whichever tool is most appropriate for the participant's chronological age.
Contraindication for lumbar puncture or MRI.
A participant who has a medical condition or extenuating circumstance that, in the opinion of the principal investigator or sub-investigator, might compromise the participant's ability to comply with protocol requirements, the participant's well-being or safety, or the interpretability of the participant's clinical data.
A participant who has received any other investigational drug product (including but not limited to, tralesinidase alfa enzyme replacement therapy [TA-ERT], Genistein, KINERET [anakinra], ambroxol, miglustat) within 4 months (or 5 half-lives, whichever is longer) before the time of providing informed consent.
A participant who has received gene therapy treatment or hematopoietic stem cell transplantation (HSCT) with successful engraftment.
Single group
JR-446
DrugIV infusion
To establish the safety and tolerability of JR-446 in MPS IIIB patients following repeated dose administration
Incidence and severity of treatment-emergent adverse events; Incidence and severity of infusion-associated reactions; Incidence of clinically significant changes/abnormalities in safety laboratory tests, vital signs, and electrocardiogram.
Change From Baseline in Cerebrospinal Fluid Heparan Sulfate Concentration
Absolute Change From Baseline in Serum Heparan Sulfate Concentration
Percent Change From Baseline in Serum Heparan Sulfate Concentration
Absolute Change From Baseline in Urine Heparan Sulfate Concentration
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JCR Pharmaceuticals Co., Ltd.
Lead sponsor
MEDIPAL HOLDINGS CORPORATION
Collaborator
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