Mucopolysaccharidosis type IIIA (MPS IIIA)
Conditions
Brief summary
1. Occurrence of adverse events (AEs), 2. Changes in safety laboratory tests (hematology, coagulation, blood biochemistry, iron-related tests, and urinalysis), 3. Changes in vital signs (pulse rate, body temperature, blood pressure, respiratory rate, and percutaneous oxygen saturation), 4. Changes in 12-lead electrocardiogram (ECG), 5. Number and severity of infusion-associated reactions (IARs), occurrence of anaphylaxis
Detailed description
1. Plasma drug concentration, 2. Plasma PK parameters, 3. Changes in the following parameters from baseline to each evaluation time point: (1) Heparan sulfate (HS concentration in CSF and serum, and HS concentration relative to creatinine concentration in urine. (2) Cognitive function assessed by Cognitive scales and/or Nonverbal Index (NVI); (3) Adaptive behavior assessment
Interventions
DRUGJR-441
Sponsors
Jcr Pharmaceuticals Co. Ltd.
Eligibility
Sex/Gender
All
Age
0 Years to 17 Years
Design outcomes
Primary
| Measure | Time frame |
|---|---|
| 1. Occurrence of adverse events (AEs), 2. Changes in safety laboratory tests (hematology, coagulation, blood biochemistry, iron-related tests, and urinalysis), 3. Changes in vital signs (pulse rate, body temperature, blood pressure, respiratory rate, and percutaneous oxygen saturation), 4. Changes in 12-lead electrocardiogram (ECG), 5. Number and severity of infusion-associated reactions (IARs), occurrence of anaphylaxis | — |
Secondary
| Measure | Time frame |
|---|---|
| 1. Plasma drug concentration, 2. Plasma PK parameters, 3. Changes in the following parameters from baseline to each evaluation time point: (1) Heparan sulfate (HS concentration in CSF and serum, and HS concentration relative to creatinine concentration in urine. (2) Cognitive function assessed by Cognitive scales and/or Nonverbal Index (NVI); (3) Adaptive behavior assessment | — |
Countries
Germany
Outcome results
None listed