Hope for rare brain disorder: experimental drug aims to aid development in toddlers
NCT ID NCT02396459
Summary
This study is testing whether a drug called tiratricol (Triac) can help young boys with a rare genetic disorder called Allan-Herndon-Dudley Syndrome (MCT8 deficiency). The condition causes severe developmental problems in the brain and harmful overactivity in other body parts. Researchers will give the drug to boys under 30 months old for nearly two years to see if it improves their motor skills and development while reducing other harmful symptoms.
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Contacts and locations
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Locations
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Charité - Universitätsmedizin Berlin Institut fur experimental paediatrische endokrinologie
Berlin, 13353, Germany
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Charles University and Motol University Hospital; The department of peadiatrics of the 2nd faculty of medicine
Prague, 15006, Czechia
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Children's Hospital of Philadelphia
Philadelphia, Pennsylvania, 19104, United States
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Erasmus MC
Rotterdam, 3015 GD, Netherlands
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Oregon Health & Science University (OHSU) Doernbecher Childrens Hospital
Portland, Oregon, 97239, United States
Conditions
Explore the condition pages connected to this study.