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

NCT06095388 | Active Not Recruiting Phase 1

• 1 other identifier: JR-441-101
• Study Type: interventional
• Target: 15
• Locations: 1 country
• Sites: 1

Brief Summary

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.

Conditions

Mucopolysaccharidosis III-A

Outcome Measures

Primary Outcomes (2)

• To evaluate the tolerability of JR-441 in MPSIIIA patients

  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

  Number and severity of infusion-associated reactions, including anaphylaxis

  up to 5 years (multiple visits)

Secondary Outcomes (4)

• Plasma drug concentration

  up to 5 years (multiple visits)

• Plasma PK parameters

  up to 5 years (multiple visits)

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

  up to 5 years (multiple visits)

• Change from baseline in cognitive function

  up to 5 years (multiple visits)

Study Arms (3)

JR-441 low dose

EXPERIMENTAL

Drug: JR-441

JR-441 medium dose

EXPERIMENTAL

Drug: JR-441

JR-441 high dose

EXPERIMENTAL

Drug: JR-441

Interventions

JR-441 DRUG

IV infusion

Also known as: posnafusp alfa

JR-441 low dose

Eligibility Criteria

• Age: 1 Year - 18 Years
• Sex: all
• Healthy Volunteers: No
• Age Groups: Child (0-17), Adult (18-64)

You may qualify if:

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

You may not qualify if:

• 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.

Sponsors & Collaborators

• JCR Pharmaceuticals Co., Ltd.: lead

Study Sites (1)

Universitätsklinikum Hamburg-Eppendorf

Hamburg, Germany

Location

MeSH Terms

Conditions

Mucopolysaccharidosis III

Condition Hierarchy (Ancestors)

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

Study Design

• Study Type: interventional
• Phase: phase 1
• Allocation: RANDOMIZED
• Masking: NONE
• Purpose: TREATMENT
• Intervention Model: PARALLEL
• Sponsor Type: INDUSTRY
• Responsible Party: SPONSOR

Study Record Dates

• First Submitted: September 13, 2023
• First Posted: October 23, 2023
• Study Start: October 4, 2023
• Primary Completion (Estimated): October 31, 2029
• Study Completion (Estimated): October 31, 2029
• Last Updated: August 26, 2025

Record last verified: 2025-08

Locations

DE (1)