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 |
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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 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)] |
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Secondary Outcome | 1. 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 minimum | Not applicable |
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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. |
Related Information
Primary Sponsor | Wakita Ryo |
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Secondary Sponsor | MEDIPAL HOLDINGS CORPORATION |
Source(s) of Monetary Support | |
Secondary ID(s) | NCT06488924 |
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 |
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 |
clinical_development@jp.jcrpharm.com | |
Affiliation | JCR Pharmaceuticals Co., Ltd. |