Experimental drug aims to tame rare, devastating immune flares
NCT ID NCT04641442
Summary
This study is testing an investigational drug called MAS825 to see if it can prevent painful disease flares in people with rare, genetic immune conditions like NLRC4-GOF, XIAP deficiency, or CDC42 mutations. About 17 patients will receive the drug, and some will later be randomly switched to a placebo to compare results. The main goal is to see if MAS825 is safe and can effectively control the inflammation and symptoms of these lifelong diseases.
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Contacts and locations
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Locations
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Bambino Gesu Hospital
Roma, RM, 00165, Italy
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Centrum detske revmatologie a autoinflamatornich onemocneni
Prague, CZ, 121 00, Czechia
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Childrens Hospital of Philadelphia
Philadelphia, Pennsylvania, 19104-4399, United States
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Children´s Hospital of Philadelphia
Philadelphia, Pennsylvania, 19104, United States
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Cincinnati Children's Hospital
Cincinnati, Ohio, 45229, United States
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Cincinnati Childrens Hospital
Cincinnati, Ohio, 45229, United States
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Great Ormond Street Hospital
London, WC1N 3JH, United Kingdom
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Hospital Clinic Barcelona
Barcelona, Catalonia, 08036, Spain
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Novartis Investigative Site
Prague, 128 08, Czechia
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Novartis Investigative Site
Prague, 150 06, Czechia
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Novartis Investigative Site
Paris, 75970, France
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Novartis Investigative Site
Roma, RM, 00165, Italy
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Novartis Investigative Site
Chiba, 266-0007, Japan
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Novartis Investigative Site
Madrid, 28046, Spain
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Novartis Investigative Site
London, NW3 2QG, United Kingdom
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Novartis Investigative Site
London, WC1N 3JH, United Kingdom
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Seattle Childrens Hospital
Seattle, Washington, 98105, United States
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Seattle Children´s Hospital
Seattle, Washington, 98105, United States
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Texas Childrens Hospital
Houston, Texas, 77030, United States
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Texas Children´s Hospital
Houston, Texas, 77030, United States
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Ustav Imunologie 2 LF UK a FN Motol
Prague, 150 06, Czechia
Conditions
Explore the condition pages connected to this study.