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JAPANESE
国立保健医療科学院
JRCT ID: jRCT2071240043

Registered date:30/07/2024

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

Basic Information

Recruitment status Pending
Health condition(s) or Problem(s) studiedMucopolysaccharidosis IIIB
Date of first enrollment01/08/2024
Target sample size10
Countries of recruitment
Study typeInterventional
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 Outcome1.To evaluate the tolerability of JR-446 in MPSIIIB patients Incidence and severity of infusion-associated reactions, including anaphylaxis [Time Frame: up to 4 years (multiple visits)] 2. To explore the potential efficacy of JR-446 Change from baseline in heparan sulfate levels in cerebrospinal fluid (CSF) [Time Frame: up to 4 years (multiple visits)]
Secondary Outcome1. Change from baseline in cognitive function [Time Frame: up tp 4 years (multiple visits)] 2. Change from baseline in HS concentration in serum and urine [Time Frame: up tp 4 years (multiple visits)] 3. Plasma drug concentration [Time Frame: up tp 4 years (multiple visits)]

Key inclusion & exclusion criteria

Age minimumNot applicable
Age maximum< 18age old
GenderBoth
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.

Related Information

Contact

Public contact
Name Ryo Wakita
Address 11-18, Kusunoki-cho, Ashiya-shi, 659-0015, Hyogo Hyogo Japan 659-0015
Telephone +81-797-32-8582
E-mail clinical_development@jp.jcrpharm.com
Affiliation JCR Pharmaceuticals Co., Ltd.
Scientific contact
Name Ryo Wakita
Address 11-18, Kusunoki-cho, Ashiya-shi, 659-0015, Hyogo Hyogo Japan 659-0015
Telephone +81-797-32-8582
E-mail clinical_development@jp.jcrpharm.com
Affiliation JCR Pharmaceuticals Co., Ltd.