Mucopolysaccharidosis IIIA
Conditions
Interventions
JR-441 is administered intravenously at low, medium, or high doses once a week.
Sponsors
Konaka Tadashi
Eligibility
Sex/Gender
All
Inclusion criteria
Inclusion criteria: - Chronological age of 1 year or older and under 18 years at the time of signing ICF - A subject with a diagnosis of MPS IIIA at the time of ICF - A subject with a minimal body weight of 10 kg at the time of screening
Exclusion criteria
Exclusion criteria: - Prior experience to gene therapy or Hematopoietic stem cell transplantation 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 The above information is not intended to contain all considerations relevant to a patient's potential participation in this clinical trial.
Design outcomes
Primary
| Measure | Time frame |
|---|---|
| - Incidence and severity of treatment-emergent adverse events - Change in safety laboratory tests - Change in vital signs - Abnormality in 12-lead electrocardiogram [Timeframe: Over the study period] | — |
Secondary
| Measure | Time frame |
|---|---|
| - Immunogenicity of JR-441 in plasma, as measured by the expression of anti-drug antibodies against JR-441 - Changes from baseline in oncentration of heparan sulfate in serum and urine [Timeframe: Over the study period] - Plasma drug concentrations and pharmacokinetics parameters [Timeframe: Multiple time points, including the point of administration of the maximum dose] - Changes from baseline in concentrations of heparan sulfate, ganglioside-monosialic acid 2, and ganglioside-monosialic acid 3 in cerebrospinal fluid [Timeframe: Baseline to Week 53] | — |
Contacts
Public ContactTadashi Konaka
JCR Pharmaceuticals Co., Ltd.
Outcome results
None listed