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New hope for hunter syndrome: Brain-Targeting drug in final testing

NCT ID NCT04573023

Ongoing Disease control Sponsor: JCR Pharmaceuticals Co., Ltd. Source: ClinicalTrials.gov ↗

First seen Jun 26, 2026 · Last updated Jun 27, 2026 · Updated 1 time

Summary

This Phase 3 study tests a new drug called JR-141 against the current standard treatment (idursulfase) in 86 people with Hunter syndrome (MPS II). The goal is to see if JR-141 can better reduce harmful substances in the brain and improve thinking skills. Participants can switch treatments if their condition worsens in certain ways. The trial is active but no longer recruiting.

What this could mean

Our plain-language read of the trial. This is informational only — not medical advice or a prediction.

Active substance
JR-141 (a drug given by IV infusion weekly)
What this could lead to
If it works, JR-141 could offer better control of both brain and body symptoms of Hunter syndrome than current treatment.
What could go wrong
This is a Phase 3 trial, but results are not yet reported. The drug may not prove superior to existing therapy, and switching rules add complexity. Risks include infusion reactions and unknown long-term effects.

This is an AI summary of the original study and may miss details. Read our disclaimer.

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Conditions

The condition(s) this trial relates to.

mucopolysaccharidosis type 2

As listed by the trial registrant

The condition terms exactly as the trial's registrant entered them.

MUCOPOLYSACCHARIDOSIS II

Contacts and locations

Locations

  • Ann & Robert H. Lurie Children's Hospital of Chicago

    Chicago, Illinois, 60611, United States

  • Children's Hospital of Philadelphia

    Philadelphia, Pennsylvania, 19104, United States

  • Chu De Montpellier Hopital Gui De Chauliac

    Montpellier, France

  • Columbia University

    New York, New York, 10032, United States

  • Ege University Children Hospital

    Izmir, Turkey (Türkiye)

  • Fundación Cardio Infantil - Instituto de Cardiología

    Bogotá, Colombia

  • Gazi University Medicine Faculty Hospital

    Ankara, Turkey (Türkiye)

  • Great Ormond Street Hospital for Children NHS Trust - Metabolic Medicine

    London, United Kingdom

  • Ha'Emek Medical Center

    Afula, Israel

  • Hospital Sant Joan de Déu

    Barcelona, Spain

  • Hospital Universitario Austral

    Buenos Aires, Argentina

  • Hospital de Clínicas de Porto Alegre

    Porto Alegre, Brazil

  • Hôpital Armand Trousseau

    Paris, France

  • Hôpital Femme Mère Enfant

    Lyon, France

  • Instituto de Genética e Erros Inatos do Metabolismo

    São Paulo, Brazil

  • Osp. Pediatrico Bambino Gesù, IRCCS

    Rome, Italy

  • Phoenix Children's Hospital

    Phoenix, Arizona, 27599-7487, United States

  • SphinCS GmbH

    Höchheim, Germany

  • UCSF Benioff Children's Hospital Oakland

    Oakland, California, 94609, United States

  • University of Minnesota

    Minneapolis, Minnesota, 55455, United States

  • University of North Carolina at Chapel Hill Medical School Wing E

    Chapel Hill, North Carolina, 27599-7487, United States

  • Universitätsklinikum Giessen

    Giessen, Germany

  • Universitätsklinikum Hamburg-Eppendorf

    Hamburg, Germany

  • Universitätsmedizin Mainz

    Mainz, Germany

  • Uniwersytecki Szpital Dziecięcy

    Krakow, Poland

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