Spinal fluid injections aim to preserve movement in devastating childhood brain disease
NCT ID NCT03771898
Summary
This study is testing whether an experimental drug called SHP611, injected into the spinal fluid, can help children with a severe genetic brain disease called MLD keep their ability to walk and move for longer. The trial involves about 36 children who will receive the injections for about two years. Researchers will compare their progress to data from similar children who did not receive the treatment to see if it slows the disease's progression.
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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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Attikon University General Hospital
Chaïdári, Attica, 124 62, Greece
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Birmingham Children's Hospital NHS Foundation Trust
Birmingham, B4 6NH, United Kingdom
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British Columbia Children's Hospital
Vancouver, British Columbia, V6H 3V4, Canada
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CHU Lenval
Nice, 06200, France
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Children's Hospital of Philadelphia
Philadelphia, Pennsylvania, 19104, United States
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Children's Hospital of Pittsburgh
Pittsburgh, Pennsylvania, 15224, United States
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Childrens Hospital Colorado
Aurora, Colorado, 80045, United States
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Cincinnati Children's Hospital Medical Center
Cincinnati, Ohio, 45229, United States
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Hospital Universitario Austral - PIN
Ciudad Autónoma Buenos Aires, Buenos Aires, B1629AHJ, Argentina
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Hospital Universitario Cruces
Barakaldo, Vizcaya, 48903, Spain
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Hospital Vall d'Hebrón
Barcelona, 8035, Spain
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Hospital de Clínicas de Porto Alegre
Porto Alegre, 90035-903, Brazil
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Hospital for Sick Children
Toronto, Ontario, M5G 1X8, Canada
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Hôpital Bicêtre - Paris Sud
Le Kremlin-Bicêtre, 94275, France
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IRCCS Ospedale Pediatrico Bambino Gesù - INCIPIT - PIN
Roma, 165, Italy
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Kanazawa University Hospital
Kanazawa, 920-8641, Japan
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Los Angeles Biomedical Research Institute at Harbor-UCLA
Torrance, California, 90502, United States
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Mayo Clinic - PPDS
Rochester, Minnesota, 55905, United States
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Montreal Children's Hospital
Montreal, Quebec, H3H 1P3, Canada
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New York University Langone Medical Center
New York, New York, 10016, United States
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Rare Disease Research, LLC
Atlanta, Georgia, 30303, United States
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Stollery Children's Hospital University of Alberta
Edmonton, Alberta, T6G 2R7, Canada
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Tel Aviv Sourasky Medical Center
Tel Aviv, 64239, Israel
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UZ Antwerpen
Edegem, 2650, Belgium
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University of Iowa Stead Family Children's Hospital
Iowa City, Iowa, 52242, United States
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University of Utah
Salt Lake City, Utah, 84108, United States
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Universitätsklinikum Hamburg Eppendorf
Hamburg, 20246, Germany
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Universitätsklinikum Tübingen
Tübingen, 72076, Germany
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VU Medisch Centrum
Amsterdam, 1081 HV, Netherlands
Conditions
Explore the condition pages connected to this study.