Rare disease study: what happens when you stop the treatment?
NCT ID NCT05579327
Summary
This study tested what happens when males with a rare genetic disorder called MCT8 deficiency stop taking their medication, tiratricol. For 30 days, some participants received a placebo (dummy pill) instead of their regular drug to see if their thyroid hormone levels would rise too high, requiring them to restart treatment. The goal was to understand how necessary ongoing treatment is for controlling this condition.
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Contacts and locations
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Locations
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Addenbrooke's Hospital
Cambridge, United Kingdom
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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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Rare Disease Research, LLC
Kissimmee, Florida, 34746, United States
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Rare Disease Research, LLC
Atlanta, Georgia, 30329, United States
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Rare Disease Research, LLC
Hillsborough, North Carolina, 27278, United States
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SSM Health Cardinal Glennon Children's Hospital
St Louis, Missouri, 63104, United States
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Tranquil Clinical and Research Consulting Services
Webster, Texas, 77598, United States
Conditions
Explore the condition pages connected to this study.