Experimental drug aims to tame rare 'Fire' in Children's immune systems
NCT ID NCT04641442
Summary
This study is testing an investigational drug called MAS825 to see if it can safely prevent disease flares in people with rare, genetic autoinflammatory diseases like NLRC4-GOF and XIAP deficiency. The trial involves about 17 patients, including infants and adults, who have one of these specific genetic mutations. Researchers will compare patients receiving MAS825 to those on a placebo to measure if the drug reduces flare-ups and improves symptoms over several years.
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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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.