Phase I/II Study of JR-441 in Patients With Mucopolysaccharidosis Type IIIA
NCT ID: NCT06095388
Last Updated: 2025-08-26
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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ACTIVE_NOT_RECRUITING
PHASE1/PHASE2
15 participants
INTERVENTIONAL
2023-10-04
2029-10-31
Brief Summary
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Detailed Description
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Conditions
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Study Design
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RANDOMIZED
PARALLEL
TREATMENT
NONE
Study Groups
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JR-441 low dose
JR-441
IV infusion
JR-441 medium dose
JR-441
IV infusion
JR-441 high dose
JR-441
IV infusion
Interventions
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JR-441
IV infusion
JR-441
IV infusion
JR-441
IV infusion
Other Intervention Names
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Eligibility Criteria
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Inclusion Criteria
* Confirmed diagnosis of MPS IIIA.
* Body weight ≥ 10 kg.
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.
1 Year
18 Years
ALL
No
Sponsors
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JCR Pharmaceuticals Co., Ltd.
INDUSTRY
Responsible Party
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Locations
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Universitätsklinikum Hamburg-Eppendorf
Hamburg, , Germany
Countries
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Other Identifiers
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JR-441-101
Identifier Type: -
Identifier Source: org_study_id
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