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ARTICLE | Clinical News

Triheptanoin: Phase II started

March 17, 2014 7:00 AM UTC

Ultragenyx began a double-blind, placebo-controlled, international Phase II trial to evaluate oral triheptanoin given 4 times daily in up to 50 patients ages 3-17 years with glucose transporter type-1 deficiency syndrome who are not on or fully compliant with a ketogenic diet and continue to have seizures. The trial comprises a 6-week observational baseline period, an 8-week treatment period and a 38-week, open-label extension period. The company said patients who complete treatment through week 52 may continue treatment in a separate open-label extension study. ...