Mucopolysaccharidosis III-B
Conditions
Brief summary
A Phase I/ II, open-label study, designed to evaluate the safety and explore efficacy of the study drug in development for the treatment of MPS IIIB patients.
Interventions
DRUGJR-446
IV infusion
Sponsors
JCR Pharmaceuticals Co., Ltd.
Study design
Allocation
NA
Intervention model
SINGLE_GROUP
Primary purpose
TREATMENT
Masking
NONE
Eligibility
Sex/Gender
ALL
Age
No minimum to 17 Years
Healthy volunteers
No
Inclusion criteria
* Chronological age of \<18 years * Confirmed diagnosis of MPS IIIB
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 |
|---|---|
| To establish the safety and tolerability of JR-446 in MPSIIIB patients | up to 4 years (multiple visits) |
| To determine the pharmacodynamic effects of JR-446 in MPSIIIB patients | up to 4 years (multiple visits) |
Secondary
| Measure | Time frame |
|---|---|
| To assess PK profile of JR-446 in MPSIIIB patients | up to 4 years (multiple visits) |
| To explore the potential efficacy of JR-446 in the treatment of patients with MPS IIIB | up to 4 years (multiple visits) |
Countries
Japan
Contacts
Primary ContactJCR Pharmaceuticals Co., Ltd.
Outcome results
None listed