Clinical Trial

Pyruvate Kinase Deficiency

Study Description

Extension Study of AG-348 in Adult Participants With Pyruvate Kinase Deficiency Previously Enrolled in AG-348-006 or AG348-C-007

This is a multicenter, open-label, extension study to evaluate the long-term safety, tolerability, and efficacy of treatment with AG-348 in participants who were previously enrolled in Study AG348-C-006 or Study AG348-C-007.

Location

Locations Selected Location

Methods

Pharmaceutical medication involved Pharmaceutical medication involved
Recruiting patients only Recruiting patients only

Drug - AG-348

Participants will receive 5, 20, or 50 mg twice a day for up to 192 weeks (not including dose taper) unless the dose is modified for reasons related to safety.

Additional Information

Official Study Title

An Open-Label, Multicenter, Extension Study of AG-348 in Adult Subjects With Pyruvate Kinase Deficiency Previously Enrolled in AG-348 Studies

Clinical Trial ID

NCT03853798

ParticipAid ID

aADX7e