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

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

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Recruiting

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Phase 1 — Early testing in a small group of people (usually 20–80) to evaluate safety, determine safe dosage, and identify side effects.

Trial locations(3 sites)

Japan

Hiroshima University Hospital, Hiroshima

University of the Ryukyus Hospital, Okinawa

National Center for Child Health and Development, Tokyo

Key details

Phase

Early testing / Safety & dosing

Sponsor

JCR Pharmaceuticals Co., Ltd.

Enrollment target

~10 participants

Started

November 2024

Primary completion

April 2030

Last updated September 2025

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