Phase I/II Study of JR-441 in Patients With Mucopolysaccharidosis Type IIIA

August 19, 2025 updated by: JCR Pharmaceuticals Co., Ltd.

Phase I/II Study of Weekly Infusions of JR-441 in Patients With Mucopolysaccharidosis Type IIIA

A Phase I/ II, open-label, randomized, 2-arm study, designed to evaluate the safety and explore efficacy of the study drug in development for the treatment of MPS IIIA patients.

Study Overview

Status

Active, not recruiting

Conditions

Intervention / Treatment

Study Type

Interventional

Enrollment (Actual)

15

Phase

  • Phase 2
  • Phase 1

Contacts and Locations

This section provides the contact details for those conducting the study, and information on where this study is being conducted.

Study Locations

  • Germany
      • Hamburg, Germany
        • Universitatsklinikum Hamburg-Eppendorf

Participation Criteria

Researchers look for people who fit a certain description, called eligibility criteria. Some examples of these criteria are a person's general health condition or prior treatments.

Eligibility Criteria

Ages Eligible for Study

  • Child
  • Adult

Accepts Healthy Volunteers

No

Description

Inclusion Criteria:

  • Chronological age of ≥1 year and ≤18 years.
  • Confirmed diagnosis of MPS IIIA.
  • Body weight ≥ 10 kg.

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.

Study Plan

This section provides details of the study plan, including how the study is designed and what the study is measuring.

How is the study designed?

Design Details

  • Primary Purpose: Treatment
  • Allocation: Randomized
  • Interventional Model: Parallel Assignment
  • Masking: None (Open Label)

Arms and Interventions

Participant Group / Arm
Intervention / Treatment
Experimental: JR-441 low dose
Drug: JR-441
IV infusion
Other Names:
  • posnafusp alfa
Experimental: JR-441 high dose
Drug: JR-441
IV infusion
Other Names:
  • posnafusp alfa
Experimental: JR-441 medium dose
Drug: JR-441
IV infusion
Other Names:
  • posnafusp alfa

What is the study measuring?

Primary Outcome Measures

Outcome Measure
Measure Description
Time Frame
To evaluate the tolerability of JR-441 in MPSIIIA patients
Time Frame: up to 5 years (multiple visits)
Adverse events will be reported and graded, laboratory tests will be conducted and vital signs will be monitored
up to 5 years (multiple visits)
To assess the safety of JR-441 in MPSIII-A patients
Time Frame: up to 5 years (multiple visits)
Number and severity of infusion-associated reactions, including anaphylaxis
up to 5 years (multiple visits)

Secondary Outcome Measures

Outcome Measure
Measure Description
Time Frame
Plasma drug concentration
Time Frame: up to 5 years (multiple visits)
up to 5 years (multiple visits)
Plasma PK parameters
Time Frame: up to 5 years (multiple visits)
up to 5 years (multiple visits)
Change from baseline in heparan sulfate levels in cerebrospinal fluid (CSF), serum and urine
Time Frame: up to 5 years (multiple visits)
up to 5 years (multiple visits)
Change from baseline in cognitive function
Time Frame: up to 5 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)
up to 5 years (multiple visits)

Collaborators and Investigators

This is where you will find people and organizations involved with this study.

Sponsor

JCR Pharmaceuticals Co., Ltd.

Study record dates

These dates track the progress of study record and summary results submissions to ClinicalTrials.gov. Study records and reported results are reviewed by the National Library of Medicine (NLM) to make sure they meet specific quality control standards before being posted on the public website.

Study Major Dates

Study Start (Actual)

October 4, 2023

Primary Completion (Estimated)

October 31, 2029

Study Completion (Estimated)

October 31, 2029

Study Registration Dates

First Submitted

September 13, 2023

First Submitted That Met QC Criteria

October 18, 2023

First Posted (Actual)

October 23, 2023

Study Record Updates

Last Update Posted (Estimated)

August 26, 2025

Last Update Submitted That Met QC Criteria

August 19, 2025

Last Verified

August 1, 2025

More Information

Terms related to this study

Additional Relevant MeSH Terms

Other Study ID Numbers

  • JR-441-101
  • 2022-002314-17 (EudraCT Number)

Drug and device information, study documents

Studies a U.S. FDA-regulated drug product

No

Studies a U.S. FDA-regulated device product

No

This information was retrieved directly from the website clinicaltrials.gov without any changes. If you have any requests to change, remove or update your study details, please contact register@clinicaltrials.gov. As soon as a change is implemented on clinicaltrials.gov, this will be updated automatically on our website as well.

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