Race to stop ALS before it starts: drug trial targets genetic time bomb
NCT ID NCT04856982
Summary
This study is testing whether the drug tofersen can delay or prevent the onset of ALS in adults who carry a specific genetic mutation linked to the disease but do not yet have symptoms. The trial involves 158 participants who will receive either the drug or a placebo to see if treatment can stop the disease from becoming clinically active. The main goal is to see how many people develop full ALS within two years of starting the study.
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Contacts and locations
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Locations
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Austin Neuromuscular Center
Austin, Texas, 78756, United States
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Azienda Ospedaliero-Universitaria Città della Salute e della Scienza di Torino
Torino, 10124, Italy
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California Pacific Medical Center Research Institute
San Francisco, California, 94107, United States
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Centrum Medyczne NeuroProtect
Warsaw, 01-684, Poland
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Columbia University Medical center
New York, New York, 10032, United States
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Genge Partners
Montreal, Quebec, H4A 3T2, Canada
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Groupe Hospitalier Pitie-Salpetriere
Paris, Paris, 75651, France
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Hanyang University Seoul Hospital
Seoul, 04763, South Korea
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Holy Cross Hospital
Fort Lauderdale, Florida, 33308, United States
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HonorHealth Neurology
Scottsdale, Arizona, 85258, United States
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Hospital Sao Paulo
São Paulo, São Paulo, 04037-002, Brazil
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Hospital Universitari i Politecnic La Fe
Valencia, 46026, Spain
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Johns Hopkins Hospital
Baltimore, Maryland, 21287, United States
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Kagoshima University Hospital
Kagoshima, Kagoshima-ken, 890-8520, Japan
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Macquarie University Hospital
Macquarie Park, New South Wales, 2109, Australia
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Massachusetts General Hospital, MA
Charlestown, Massachusetts, 02129, United States
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Medizinische Hochschule Hannover
Hanover, Lower Saxony, 30625, Germany
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NeuroProtect Sp. z o.o.
Warsaw, Masovian Voivodeship, 01-684, Poland
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Norrlands Universitetssjukhus
Umeå, 90185, Sweden
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Northwestern Medicine
Chicago, Illinois, 60611, United States
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PSEG Centro de Pesquisa Clinica
São Paulo, 04038-002, Brazil
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Sunnybrook Health Sciences Centre
Toronto, Ontario, M4N 3M5, Canada
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The Emory Clinic
Atlanta, Georgia, 30322-4200, United States
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UZ Leuven
Leuven, 3000, Belgium
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Universitaetsklinikum Ulm
Ulm, Baden-Wurttemberg, 89081, Germany
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University Hospital of Umea
Umeå, Västerbotten County, 90185, Sweden
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University of Calgary
Calgary, Alberta, T2N4Z6, Canada
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University of California San Diego Medical Center
La Jolla, California, 92093-0949, United States
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University of Miami School of Medicine
Miami, Florida, 33136, United States
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University of Sheffield
Sheffield, South Yorkshire, S10 2RX, United Kingdom
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University of Tokyo Hospital
Bunkyō City, Tokyo-To, 113-8655, Japan
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Washington University School of Medicine
St Louis, Missouri, 63110, United States
Conditions
Explore the condition pages connected to this study.