New Brain-Targeting drug enters final testing for rare genetic disease
NCT ID NCT04573023
Summary
This study is testing an investigational drug called JR-141 for Hunter syndrome (MPS II), a rare genetic disorder. It aims to see if JR-141 can better control the disease, particularly its effects on brain development and thinking skills, compared to the current standard treatment. The trial involves 86 participants, including young children and older patients, and is in its final phase of testing.
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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.