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

Mucopolysaccharidosis III-A Clinical Trial

Official title:

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

Clinical Trial Details — Status: Recruiting

Administrative data

• NCT number: NCT06095388
• Other study ID #: JR-441-101
• Secondary ID: 2022-002314-17
• Status: Recruiting
• Phase: Phase 1/Phase 2
• Start date: October 4, 2023
• Est. completion date: October 31, 2029

Study information

• Verified date: October 2023
• Source: JCR Pharmaceuticals Co., Ltd.
• Contact: JCR Pharmaceuticals Co., Ltd.
• Phone: +81-(0)797-32-8582
• Email: clinical_development[@]jp.jcrpharm.com
• Is FDA regulated: No
• Study type: Interventional

Clinical Trial 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.

Recruitment information / eligibility

• Status: Recruiting
• Enrollment: 12
• Est. completion date: October 31, 2029
• Est. primary completion date: October 31, 2029
• Accepts healthy volunteers: No
• Gender: All
• Age group: 1 Year to 18 Years

Eligibility

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.

Related Conditions & MeSH terms

• Mucopolysaccharidoses
• Mucopolysaccharidosis III
• Mucopolysaccharidosis III-A

Intervention

Drug:
• JR-441
IV infusion
• JR-441
IV infusion

Locations

Country	Name	City	State
Germany	Universitätsklinikum Hamburg-Eppendorf	Hamburg

Sponsors (1)

Lead Sponsor	Collaborator
JCR Pharmaceuticals Co., Ltd.

Country where clinical trial is conducted

Germany, 

Outcome

Type	Measure	Description	Time frame	Safety issue
Primary	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)
Primary	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	Plasma drug concentration		up to 5 years (multiple visits)
Secondary	Plasma PK parameters		up to 5 years (multiple visits)
Secondary	Change from baseline in heparan sulfate levels in cerebrospinal fluid (CSF), serum and urine		up to 5 years (multiple visits)
Secondary	Change from baseline in cognitive function	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)