NIPH Clinical Trials Search

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

Basic Information

Recruitment status	Recruiting
Health condition(s) or Problem(s) studied	Mucopolysaccharidosis IIIB
Date of first enrollment	01/08/2024
Target sample size	10
Countries of recruitment
Study type	Interventional
Intervention(s)	JR-446 will be administered intravenously at multiple doses. Once the optimal dose is determined, all subjects will receive the same dose intravenously.

Outcome(s)

Primary Outcome	1.To establish the safety and tolerability of JR-446 in MPSIIIB patients [Time Frame: up to 4 years (multiple visits)] 2. To determine the pharmacodynamic effects of JR-446 in MPSIIIB patients [Time Frame: up to 4 years (multiple visits)]
Secondary Outcome	1. To assess PK profile of JR-446 in MPSIIIB patients [Time Frame: up tp 4 years (multiple visits)] 2. To explore the potential efficacy of JR-446 in the treatment of patients with MPS IIIB [Time Frame: up tp 4 years (multiple visits)]

Key inclusion & exclusion criteria

Age minimum	Not applicable
Age maximum	< 18age old
Gender	Both
Include criteria	-Chronological age of <18 years -Confirmed diagnosis of MPS III
Exclude 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.