An Open-label Phase I/II Study of JR-446 in Mucopolysaccharidosis Type IIIB

JCR Pharmaceuticals

Status and phase: Enrolling; Phase 2; Phase 1

Conditions: Mucopolysaccharidosis III-B

Treatments: Drug: JR-446

Study type: Interventional

Funder types: Industry

Identifiers

NCT06488924

JR-446-101

Details and patient eligibility

About

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

Enrollment: 10 estimated patients

Sex: All

Ages: Under 17 years old

Volunteers: No Healthy Volunteers

Inclusion criteria

• Chronological age of <18 years
• Confirmed diagnosis of MPS IIIB

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.

Trial design

Primary purpose: Treatment

Allocation: N/A

Interventional model: Single Group Assignment

Masking: None (Open label)

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10 participants in 1 patient group

JR-446

Experimental group

Treatment:

Drug: JR-446