New hope for hunter syndrome: brain-targeting drug enters final trial
NCT ID NCT04573023
First seen Nov 01, 2025 · Last updated May 24, 2026 · Updated 27 times
Summary
This phase 3 study tests JR-141, an enzyme replacement therapy designed to cross into the brain and treat Hunter syndrome (MPS II). About 86 participants receive the drug to see if it reduces harmful sugar buildup in spinal fluid and improves thinking skills. The goal is to slow or stop brain damage, but patients will need lifelong treatment.
Disclaimer
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This is a summary of
the original study
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Summaries may miss details or leave out important information. Before applying or accepting participation, make sure you have read and understood the full study. Curemydisease.com takes no responsibility whatsoever for anything missed, misunderstood, or acted upon as a result of our summary — we know it does not capture everything.
This is a summary of the original study . Summaries may miss details or leave out important information. Before applying or accepting participation, make sure you have read and understood the full study. Curemydisease.com takes no responsibility whatsoever for anything missed, misunderstood, or acted upon as a result of our summary — we know it does not capture everything.
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Contacts and locations
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Locations
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Ann & Robert H. Lurie Children's Hospital of Chicago
Chicago, Illinois, 60611, United States
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Children's Hospital of Philadelphia
Philadelphia, Pennsylvania, 19104, United States
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Columbia University
New York, New York, 10032, United States
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Ege University Children Hospital
Izmir, Turkey (Türkiye)
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Fundación Cardio Infantil - Instituto de Cardiología
Bogotá, Colombia
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Gazi University Medicine Faculty Hospital
Ankara, Turkey (Türkiye)
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Great Ormond Street Hospital for Children NHS Trust - Metabolic Medicine
London, United Kingdom
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Ha'Emek Medical Center
Afula, Israel
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Hospital Sant Joan de Déu
Barcelona, Spain
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Hospital Universitario Austral
Buenos Aires, Argentina
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Hospital de Clínicas de Porto Alegre
Porto Alegre, Brazil
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Hôpital Armand Trousseau
Paris, France
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Hôpital Femme Mère Enfant
Bron, France
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Instituto de Genética e Erros Inatos do Metabolismo
São Paulo, Brazil
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Osp. Pediatrico Bambino Gesù, IRCCS
Rome, Italy
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Phoenix Children's Hospital
Phoenix, Arizona, 27599-7487, United States
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SphinCS GmbH
Höchheim, Germany
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UCSF Benioff Children's Hospital Oakland
Oakland, California, 94609, United States
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University of Minnesota
Minneapolis, Minnesota, 55455, United States
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University of North Carolina at Chapel Hill Medical School Wing E
Chapel Hill, North Carolina, 27599-7487, United States
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Universitätsklinikum Giessen
Giessen, Germany
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Universitätsklinikum Hamburg-Eppendorf
Hamburg, Germany
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Universitätsmedizin Mainz
Mainz, Germany
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Uniwersytecki Szpital Dziecięcy
Krakow, Poland
Conditions
Explore the condition pages connected to this study.