Brain-saving drug for rare hunter syndrome enters final testing
NCT ID NCT04573023
First seen Nov 01, 2025 · Last updated May 14, 2026 · Updated 25 times
Summary
This phase 3 study tests a drug called JR-141 in 86 people with Hunter syndrome (MPS II), a rare genetic disease that damages the body and brain. The goal is to see if the drug can reduce harmful substances in the spinal fluid and improve thinking and learning skills. Participants receive the drug or an active control, and researchers monitor safety and effectiveness over time.
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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
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