Phase I study of weekly infusion of JR-441 in patients with mucopolysaccharidosis type IIIA

JRCT ID: jRCT2071240053

Registered date:26/08/2024

Basic Information

• Recruitment status: Pending
• Health condition(s) or Problem(s) studied: Mucopolysaccharidosis IIIA
• Date of first enrollment: 01/10/2024
• Target sample size: 3
• Study type: Interventional
• Intervention(s): JR-441 is administered intravenously at low or high doses once a week.

Outcome(s)

Primary Outcome

- 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 Outcome

- 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]

Key inclusion & exclusion criteria

• Age minimum: >= 1age old
• Age maximum: < 18age old
• Gender: Both

Include criteria

- Chronological age of 1 year or older and under 18 years
- Confirmed diagnosis of MPS IIIA
- Body weight of 10 kg or more at screening test

Exclude 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.

Related Information

• Primary Sponsor: Ibaraki Ryo