An Open-label Phase I/II Study of JR-446 in Mucopolysaccharidosis Type IIIB

JRCT ID: jRCT2071240043

Registered date:30/07/2024

Basic Information

• Recruitment status: Pending
• Health condition(s) or Problem(s) studied: Mucopolysaccharidosis IIIB
• Date of first enrollment: 01/08/2024
• Target sample size: 10
• Study type: Interventional
• Intervention(s): JR-446 will be administered intravenously at multiple doses. Once the optimal dose is determined, all subjects will receive the same dose intravenously.

Outcome(s)

Primary Outcome

1.To evaluate the tolerability of JR-446 in MPSIIIB patients
Incidence and severity of infusion-associated reactions, including anaphylaxis
[Time Frame: up to 4 years (multiple visits)]
2. To explore the potential efficacy of JR-446
Change from baseline in heparan sulfate levels in cerebrospinal fluid (CSF)
[Time Frame: up to 4 years (multiple visits)]

Secondary Outcome

1. Change from baseline in cognitive function
[Time Frame: up tp 4 years (multiple visits)]
2. Change from baseline in HS concentration in serum and urine
[Time Frame: up tp 4 years (multiple visits)]
3. Plasma drug concentration
[Time Frame: up tp 4 years (multiple visits)]

Key inclusion & exclusion criteria

• Age minimum: Not applicable
• Age maximum: < 18age old
• Gender: Both

Include criteria

-Chronological age of <18 years
-Confirmed diagnosis of MPS III

Exclude 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.

Related Information

• Primary Sponsor: Wakita Ryo
• Secondary Sponsor: MEDIPAL HOLDINGS CORPORATION
• Secondary ID(s): NCT06488924