Clinical trials: Pyruvate Kinase Deficiency
Pyruvate Kinase Deficiency
A Study of AG-348 in Adult Participants With Pyruvate Kinase (PK) Deficiency
Study AG348-C-003 is a multicenter study designed to evaluate the safety and efficacy of different dose levels of AG-348 (mitapivat) in participants with PK deficiency.
- Trial name
- NCT02476916
- Testing
- AG-348
- Where
- 13 sites in United States, Canada and 4 more
- Volunteers
- 52 people
- Sponsor
- Agios Pharmaceuticals, Inc.
Pyruvate Kinase Deficiency, Anemia, Hemolytic
A Study to Evaluate Efficacy and Safety of AG-348 in Not Regularly Transfused Adult Participants With Pyruvate Kinase Deficiency (PKD)
Study AG348-C-006 evaluated the efficacy and safety of orally administered AG-348 as compared with placebo in participants with pyruvate kinase (PK) deficiency, who were not regularly receiving blood transfusions.
- Trial name
- NCT03548220
- Testing
- AG-348
- Where
- 46 sites in United States, Brazil and 14 more
- Volunteers
- 80 people
- Sponsor
- Agios Pharmaceuticals, Inc.
Pyruvate Kinase Deficiency, Anemia, Hemolytic
A Study Evaluating the Efficacy and Safety of AG-348 in Regularly Transfused Adult Participants With Pyruvate Kinase Deficiency (PKD)
Study AG348-C-007 was a multicenter study designed to evaluate the efficacy and safety of treatment with AG-348 in a minimum of 20, with up to 40, participants with pyruvate kinase (PK) deficiency, who were regularly receiving…
- Trial name
- NCT03559699
- Testing
- AG-348
- Where
- 19 sites in United States, Canada and 7 more
- Volunteers
- 27 people
- Sponsor
- Agios Pharmaceuticals, Inc.
Pyruvate Kinase Deficiency
Extension Study of AG-348 in Adult Participants With Pyruvate Kinase Deficiency Previously Enrolled in AG-348-006 or AG348-C-007
This is an open-label, multicenter, extension study to evaluate the long-term safety, tolerability, and efficacy of treatment with mitapivat in participants who were previously enrolled in Study AG348-C-006 or Study AG348-C-007.
- Trial name
- NCT03853798
- Testing
- Mitapivat
- Where
- 42 sites in United States, Brazil and 14 more
- Volunteers
- 90 people
- Sponsor
- Agios Pharmaceuticals, Inc.