New hope for rare metabolic disorder: can a repurposed drug ease debilitating fatigue?
NCT ID NCT06887777
Summary
This study is testing if a drug called Ravicti can help control symptoms of a rare genetic disorder called pyruvate dehydrogenase deficiency (PDH). It will involve about 15 children and young adults (ages 2-25) with specific genetic forms of PDH. The main goal is to see if taking Ravicti for 6 months reduces severe fatigue and improves daily functioning and quality of life.
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Contacts and locations
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Study contacts
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Contact
Phone: •••-•••-•••• Email: •••••@•••••
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Contact
Phone: •••-•••-•••• Email: •••••@•••••
Locations
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Hôpital Universitaire Necker - Enfants Malades
RECRUITINGParis, France, 75015, France
Contact Phone: •••-•••-•••• Email: •••••@•••••
Conditions
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