Mucopolysaccharidosis III-A
Conditions
Brief summary
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.
Interventions
DRUGJR-441
IV infusion
Sponsors
JCR Pharmaceuticals Co., Ltd.
Study design
Allocation
RANDOMIZED
Intervention model
PARALLEL
Primary purpose
TREATMENT
Masking
NONE
Eligibility
Sex/Gender
ALL
Age
1 Years to 18 Years
Healthy volunteers
No
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. 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 | Description |
|---|---|---|
| To evaluate the tolerability of JR-441 in MPSIIIA patients | up to 5 years (multiple visits) | Adverse events will be reported and graded, laboratory tests will be conducted and vital signs will be monitored |
| To assess the safety of JR-441 in MPSIII-A patients | up to 5 years (multiple visits) | Number and severity of infusion-associated reactions, including anaphylaxis |
Secondary
| Measure | Time frame | Description |
|---|---|---|
| Plasma drug concentration | up to 5 years (multiple visits) | — |
| Plasma PK parameters | up to 5 years (multiple visits) | — |
| Change from baseline in heparan sulfate levels in cerebrospinal fluid (CSF), serum and urine | up to 5 years (multiple visits) | — |
| Change from baseline in cognitive function | up to 5 years (multiple visits) | Bayley Scales of Infant and Toddler Development, Third Edition (BSID-III) and/or Kaufman Assessment Battery for Children, Second Edition (KABC-II) |
Countries
Germany
Outcome results
None listed