Phase I/II Study of JR-441 in Patients With Mucopolysaccharidosis Type IIIA

Study Overview
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Brief Summary
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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.

Official Title
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Phase I/II Study of Weekly Infusions of JR-441 in Patients With Mucopolysaccharidosis Type IIIA

Conditions
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Mucopolysaccharidosis III-A

Intervention / Treatment
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* Drug: JR-441
* Drug: JR-441


Participation Criteria
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Eligibility Criteria
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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.

Ages Eligible for Study
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Minimum Age: 1 Year
Maximum Age: 18 Years

Sexes Eligible for Study
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All

Accepts Healthy Volunteers
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No

Study Plan
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How is the study designed?
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Design Details

Primary Purpose: Treatment
Allocation: Randomized
Intervention Model: Parallel Assignment
Masking: None (Open Label)


Arms and Interventions

| Participant Group/Arm | Intervention/Treatment |
| --- | --- |
| Experimental: JR-441 low dose<br> | Drug: JR-441<br>* IV infusion<br>|
| Experimental: JR-441 high dose<br> | Drug: JR-441<br>* IV infusion<br>|

What is the study measuring?
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Primary Outcome Measures

| Outcome Measure | Measure Description | Time Frame |
| --- | --- | --- |
| To evaluate the tolerability of JR-441 in MPSIIIA patients | Adverse events will be reported and graded, laboratory tests will be conducted and vital signs will be monitored | up to 5 years (multiple visits) | 
| To assess the safety of JR-441 in MPSIII-A patients | Number and severity of infusion-associated reactions, including anaphylaxis | up to 5 years (multiple visits) | 

Secondary Outcome Measures

| Outcome Measure | Measure Description | Time Frame |
| --- | --- | --- |
| 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 | Bayley Scales of Infant and Toddler Development, Third Edition (BSID-III) and/or Kaufman Assessment Battery for Children, Second Edition (KABC-II) | up to 5 years (multiple visits) |