Recruiting NCT06488924

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

Phase I / Phase II Interventional Mucopolysaccharidosis III-B

For patients and families

In plain language

An automatic summary of structured registry data. It is an orientation aid, not a substitute for the official protocol or a physician assessment.

What is being studied: The protocol lists: JR-446.
Who it may be relevant to: Registry conditions: Mucopolysaccharidosis III-B. Basic parameters: up to 17 years · All.
What needs checking: Age, condition and sex are only basic indicators. Prior treatment, laboratory values and other mandatory requirements appear in the eligibility criteria below.
Where it takes place: Japan
Next step: Save the trial, show it to the treating physician, and confirm current recruitment with the study center. Costs, documents and travel →

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Overview

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.

Interventions

Drug JR-446
IV infusion

Primary outcome measures

To establish the safety and tolerability of JR-446 in MPSIIIB patients [Time frame: up to 4 years (multiple visits)]
To determine the pharmacodynamic effects of JR-446 in MPSIIIB patients [Time frame: up to 4 years (multiple visits)]

Secondary outcome measures (2)

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

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.

Criteria are shown verbatim from the registry (in English). Final eligibility is always assessed by the study center.

Healthy volunteers: No

Study design

Allocation: N/A
Model: Single group
Masking: Open label
Primary purpose: Treatment

Study locations

Japan · 3 centers

Hiroshima University Hospital — Hiroshima
University of the Ryukyus Hospital — Okinawa
National Center for Child Health and Development — Tokyo

Identifiers

NCT: NCT06488924 · JR-446-101

Primary sources (government registries)

ClinicalTrials.gov (US NIH)

View this study on ClinicalTrials.gov ↗