The clinical study designed to evaluate the safety and efficacy of Agios Pharmaceutical’s AG-348 in adult patients with pyruvate kinase deficiency has opened at Boston Children’s Hospital.  This is the first site of a multicenter study that will open in North America and Europe.

AG-348 is an oral medication being studied as a possible treatment for PK deficiency. Researchers believe that this medicine may allow a patient with pyruvate kinase deficiency to maintain a normal amount of healthy red blood cells, and avoid other complications associated with this disease.

“This is a Phase 2, open label, two arm, multicenter, randomized, dose-ranging study during which adult patients with PK deficiency will receive multiple doses of AG-348 for up to 24 weeks. Up to 25 patients will be randomized to two doses of AG-348; a third arm (up to 25 patients) may be added, if required. AG-348 will be administered orally at various doses; if implemented, the dose of Arm 3 will be determined based on ongoing review. Data will be reviewed on a regular basis and study design, dose and schedule will be adapted based on these reviews. The study will evaluate the safety and tolerability of multiple doses of AG-348, pharmacokinetic (PK) and pharmacodynamic (PD) profile of AG-348 and early indicators of clinical efficacy.” ClinicalTrials.gov

For more information about this study go to ClinicalTrials.gov.