2 results
Approved WMOCompleted
CORE PERIODPrimary:* Evaluate the safety and tolerability of up to 24 weeks of AG-348 administration in patients with pyruvate kinase deficiency (PK deficiency).Secondary:* Evaluate the pharmacokinetics (PK) of AG-348 and the metabolite AGI-8702.*…
Approved WMOCompleted
The primary objective of the proposed feasibility study is to determine the willingness of patients to be randomized to CLI or DBS. Furthermore it will serve as a pilot trial for a future larger randomized controlled trial. For this larger trial,…