A Phase I/II Trial of JR-446 in Mucopolysaccharidosis Type IIIB (MPS IIIB)
Part of paid clinical trials in Oakland, California.
- Sponsor
- JCR Pharmaceuticals Co., Ltd.
- Study ID
- NCT07640984
- Phase
- PHASE1/PHASE2
- Status
- Not Yet Recruiting
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Conditions
- Mucopolysaccharidosis IIIB
Eligibility Criteria
- Sex
- ALL
- Age
- N/A - 5 Years
- Healthy Volunteers
- Not accepted
Interventions
- JR-446 — DRUGIV infusion
Study Details
This is a global, open-label, Phase I/II, interventional trial in participants younger than 6 years of age with Mucopolysaccharidosis Type IIIB (MPS IIIB), designed to assess the safety and tolerability of JR-446, determine its pharmacodynamic effects, and explore its potential to demonstrate early clinical effects on disease-relevant outcomes.
Key Dates
- Start date
- Jun 18, 2026
- Status verified
- Jun 2026
- Primary completion
- Jun 30, 2028
- Completion
- Sep 30, 2028
Study Design
- Enrollment
- 12 participants (estimated)
- Allocation
- NA
- Intervention model
- SINGLE_GROUP
- Primary purpose
- TREATMENT
Arms
- Experimental: JR-446Arm: 1
Primary Outcome Measure
To establish the safety and tolerability of JR-446 in MPS IIIB patients following repeated dose administration [ Time Frame: Up to 1 year (multiple visits) ]
Central Contacts
- JCR Pharmaceuticals Co., Ltd.+81-797-32-8582
Locations (3)
| Facility | City | State | ZIP | Site coordinators |
|---|---|---|---|---|
| Children's Hospital Oakland Research Institution, Attn: 2900 Wing | Oakland | California | 94609 | |
| UPMC Children's Hospital of Pittsburgh | Pittsburgh | Pennsylvania | 15224 | |
| Lysosomal and Rare Disorders Research and Treatment Center (LDRTC) | Fairfax | Virginia | 22030 |