An Open-label Phase I/II Study of JR-446 in Mucopolysacch... | NCT06488924

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

NCT06488924

Sponsor

JCR Pharmaceuticals Co., Ltd.

Status

Recruiting

Last Update Posted

Sep 2, 2025Actual

Enrollment

10Estimated

Phase

Phase 1Phase 2

Conditions

Mucopolysaccharidosis III-B

Interventions

JR-446

Countries

Japan

Protocol Section

Identification Module Status Module Sponsor/Collaborators Module Oversight Module Description Module Conditions Module Design Module Arms/Interventions Module Outcomes Module Eligibility Module Contacts/Locations Module References Module IPD Sharing Statement Module

Results Section

Participant Flow Module Baseline Characteristics Module Outcome Measures Module Adverse Events Module More Info Module

Annotation Section

Annotation Module

Document Section

Large Document Module

Derived Section

Miscellaneous Info Module Condition Browse Module Intervention Browse Module

Protocol Section

Identification Module

NCT ID

Results Section

No data available

No data is available for this block.

Annotation Section

No data available

No data is available for this block.

Document Section

No data available

No data is available for this block.

Derived Section

Miscellaneous Info Module

Version Holder

Status Module

Record Verification Date

Aug 2025

Overall Recruitment Status or Expanded Access Status

Recruiting

Last Known Status

Not provided

Delayed Posting

Not provided

Why Stopped

Not provided

Expanded Access Info

Start Date

Nov 5, 2024Actual

Primary Completion Date

Apr 30, 2030Estimated

Completion Date

Apr 30, 2030Estimated

First Submitted Date

Jun 28, 2024

First Submission Date that Met QC Criteria

Jun 28, 2024

First Posted Date

Jul 5, 2024Actual

Results Waived

Not provided

Results First Submitted Date

Not provided

Results First Submitted that Met QC Criteria

Not provided

Results First Posted Date

Not provided

Certification/Extension (aka Delayed Results) First Submitted Date

Not provided

Certification/Extension First Submitted that Passed QC Review

Not provided

Certification/Extension First Posted Date

Not provided

Last Update Submitted Date

Aug 25, 2025

Last Update Posted Date

Sep 2, 2025Actual

Oversight Module

Has Data Monitoring Committee (DMC)

Is FDA Regulated Drug

Is FDA Regulated Device

Is Unapproved Device

Not provided

Pediatric Postmarket Surveillance of a Device Product

Not provided

Product Exported from US

FDAAA801 Violation

Not provided

Description Module

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 provided

Design Module

Study Type

Interventional

Number of References to an Expanded Access Study

Not provided

Expanded Access Types

Not provided

Patient Registry

Not provided

Target Follow-Up Duration

Not provided

Phases

Phase 1Phase 2

Interventional Study Design

Allocation

Biospecimen

No data available

No data is available for this block.

Enrollment

10Estimated

Label	Type	Description	Intervention Names
JR-446	Experimental		Drug: JR-446

Name	Type	Description	Arm Group Labels	Other Names
JR-446	Drug	IV infusion	JR-446

Outcomes Module

Primary Outcomes

Measure	Description	Time Frame
To establish the safety and tolerability of JR-446 in MPSIIIB patients		up to 4 years (multiple visits)
To determine the pharmacodynamic effects of JR-446 in MPSIIIB patients		up to 4 years (multiple visits)

Secondary Outcomes

Measure	Description	Time Frame
To assess PK profile of JR-446 in MPSIIIB patients		up to 4 years (multiple visits)
To explore the potential efficacy of JR-446 in the treatment of patients with MPS IIIB		up to 4 years (multiple visits)

Other Outcomes

Not provided

Eligibility Module

Eligibility Criteria

Inclusion Criteria:

Chronological age of <18 years
Confirmed diagnosis of MPS IIIB

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.

Accepts Healthy Volunteers

Sex

All

Sex/Gender Based

Not provided

Sex/Gender Description

Not provided

Minimum Age

Not provided

Maximum Age

17 Years

Standard Ages

Child

Study Population

Not provided

Sampling Method

Not provided

Contacts/Locations Module

Central Contacts

Name	Role	Phone	Extension	Email
JCR Pharmaceuticals Co., Ltd.	Contact	+81-797-32-8582		clinical_development@jp.jcrpharm.com

Overall Officials

Not provided

Locations

Facility	Status	City	State	ZIP	Country	Contacts
Hiroshima University Hospital	Recruiting	Hiroshima			Japan

Condition Browse Module

MeSH Terms

ID	Term
D009084	Mucopolysaccharidosis III

Ancestor Terms

ID	Term
D009083	Mucopolysaccharidoses
D002239	Carbohydrate Metabolism, Inborn Errors
D008661	Metabolism, Inborn Errors
D030342	Genetic Diseases, Inborn

Browse Leaves

Not provided

Browse Branches

Not provided