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

Phase 1

Not yet recruiting

• Conditions: Mucopolysaccharidosis III-B
• Interventions: Drug: JR-446

• Registration Number: NCT06488924
• Lead Sponsor: JCR Pharmaceuticals Co., Ltd.

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.

Detailed Description

Not available

Study Timeline

• Status Verified: 2024-06
• Study First Submitted: 2024-06-28
• Study First Submitted QC: 2024-06-28
• Last Update Submitted: 2024-06-28
• Study First Posted: 2024-07-05
• Last Update Posted: 2024-07-05
• Study Start: 2024-08-31
• Primary Completion: 2027-03-31
• Study Completion: 2029-09-30

Recruitment & Eligibility

• Status: NOT_YET_RECRUITING
• Sex: All
• Target Recruitment: 10

Inclusion Criteria

• Chronological age of <18 years
• Confirmed diagnosis of MPS IIIB

Read More

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.

Read More

Study & Design

• Study Type: INTERVENTIONAL
• Study Design: SINGLE_GROUP

Arm && Interventions

Group	Intervention	Description
JR-446	JR-446	-

Primary Outcome Measures

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

Secondary Outcome Measures

Name	Time	Method
Change from baseline in cognitive function	up to 4 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)
Change from baseline in HS concentration in serum and urine	up to 4 years (multiple visits)
Plasma drug concentration	up to 4 years (multiple visits)

Trial Locations

Locations (3)

National Center for Child Health and Development; 🇯🇵 Tokyo, Japan

Hiroshima University Hospital; 🇯🇵 Hiroshima, Japan

University of the Ryukyus Hospital; 🇯🇵 Okinawa, Japan