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NCT Number: NCT06095388

Phase I/II Study 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.

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This study is active but is not currently recruiting participants.

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Key information

Conditions

Mucopolysaccharidosis III-A Carbohydrate Metabolism, Inborn Errors Congenital, Hereditary, and Neonatal Diseases and Abnormalities Connective Tissue Diseases Genetic Diseases, Inborn Lysosomal Storage Diseases Metabolic Diseases Metabolism, Inborn Errors Mucinoses Mucopolysaccharidoses Mucopolysaccharidosis III Nutritional and Metabolic Diseases Skin and Connective Tissue Diseases

Age range

1 year–18 year

Sex eligibility

All sexes

Study type

Interventional

Phase

Phase 1 / Phase 2

Primary location

Universitätsklinikum Hamburg-Eppendorf

Hamburg, Germany

Who can participate

Healthy volunteers accepted: No

Only the study team can determine whether someone qualifies for participation.

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.

Treatment and study plan

JR-441

Drug

IV infusion

Other names: posnafusp alfa

Primary outcomes

  1. 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

  2. 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

Secondary outcomes

  1. Plasma drug concentration

    Time frame: up to 5 years (multiple visits)

  2. Plasma PK parameters

    Time frame: up to 5 years (multiple visits)

  3. Change from baseline in heparan sulfate levels in cerebrospinal fluid (CSF), serum and urine

    Time frame: up to 5 years (multiple visits)

  4. 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)

Sponsors and collaborators

Lead sponsor

JCR Pharmaceuticals Co., Ltd.

Industry

Registry information

Official study title

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

Important dates

Study start
2023
Primary completion
2029
Study completion
2029
First posted
Oct 23, 2023
Registry last updated
Aug 26, 2025

OpenTrials presents study information sourced from ClinicalTrials.gov. The official registry record should be consulted for the latest information.

View the official ClinicalTrials.gov record (opens in a new tab)

This listing is for discovery and informational purposes only. It is not medical advice, does not guarantee that a study is recruiting, and does not determine eligibility. Contact the study team and a qualified healthcare professional when considering participation.

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