A Phase 2, Open Label, Randomized, Dose Ranging, Safety, Efficacy, Pharmacokinetic and Pharmacodynamic Study of AG-348 in Adult Patients with Pyruvate Kinase Deficiency

Update Il y a 4 ans
Reference: EUCTR2015-000484-13

Woman and Man

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Extract

• Evaluate the safety and tolerability of up to 24 weeks of AG 348 administration in patients with PK deficiency.


Inclusion criteria

  • Pyruvate Kinase Deficiency