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

Phase 1

Recruiting

• Conditions: Mucopolysaccharidosis III-A
• Interventions: Drug: JR-441

• Registration Number: NCT06095388
• Lead Sponsor: JCR Pharmaceuticals Co., Ltd.

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.

Detailed Description

Not available

Study Timeline

• Study First Submitted: 2023-09-13
• Status Verified: 2023-10
• Study Start: 2023-10-04
• Study First Submitted QC: 2023-10-18
• Last Update Submitted: 2023-10-18
• Study First Posted: 2023-10-23
• Last Update Posted: 2023-10-23
• Primary Completion: 2029-10-31
• Study Completion: 2029-10-31

Recruitment & Eligibility

• Status: RECRUITING
• Sex: All
• Target Recruitment: 12

Inclusion Criteria

• Chronological age of ≥1 year and ≤18 years.
• Confirmed diagnosis of MPS IIIA.
• Body weight ≥ 10 kg.

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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.

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Study & Design

• Study Type: INTERVENTIONAL
• Study Design: PARALLEL

Arm && Interventions

Group	Intervention	Description
JR-441 low dose	JR-441	-
JR-441 high dose	JR-441	-

Primary Outcome Measures

Name	Time	Method
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 Outcome Measures

Name	Time	Method
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)

Trial Locations

Locations (1)

Universitätsklinikum Hamburg-Eppendorf; 🇩🇪 Hamburg, Germany