An Open-label Phase I/II Study of JR-446 in Mucopolysaccharidosis Type IIIB
NCT ID: NCT06488924
Last Updated: 2025-09-02
Study Results
The study team has not published outcome measurements, participant flow, or safety data for this trial yet. Check back later for updates.
Basic Information
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RECRUITING
PHASE1/PHASE2
10 participants
INTERVENTIONAL
2024-11-05
2030-04-30
Brief Summary
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Detailed Description
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Conditions
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Study Design
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NA
SINGLE_GROUP
TREATMENT
NONE
Study Groups
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JR-446
JR-446
IV infusion
Interventions
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JR-446
IV infusion
Eligibility Criteria
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Inclusion Criteria
* Confirmed diagnosis of MPS IIIB
Exclusion Criteria
* 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.
17 Years
ALL
No
Sponsors
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JCR Pharmaceuticals Co., Ltd.
INDUSTRY
Responsible Party
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Locations
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Hiroshima University Hospital
Hiroshima, , Japan
University of the Ryukyus Hospital
Okinawa, , Japan
National Center for Child Health and Development
Tokyo, , Japan
Countries
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Central Contacts
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Facility Contacts
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Satoshi Okada
Role: primary
Koichi Nakanishi
Role: primary
Yuichi Abe
Role: primary
Other Identifiers
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JR-446-101
Identifier Type: -
Identifier Source: org_study_id
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