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A Study Evaluating the Efficacy and Safety of AG-348 in Regularly Transfused Adult Participants With Pyruvate Kinase Deficiency (PKD)

Pyruvate Kinase Deficiency Clinical Trial

Official title:
An Open-Label Study To Evaluate the Efficacy and Safety of AG-348 in Regularly Transfused Adult Subjects With Pyruvate Kinase (PK) Deficiency

Clinical Trial Summary

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 blood transfusions. The study was composed of two parts. During Part 1, Dose Optimization Period, participants started on a dose of 5 mg AG-348 administered twice daily. Over the course of Part 1 each participant's dose of AG-348 was sequentially increased to 20 mg twice a day, followed by 50 mg twice a day depending on their tolerance. During Part 2, Fixed-Dose Period, participants received AG-348 at their optimized dose from Part 1.

Clinical Trial Description

n/a

Study Design

Related Conditions & MeSH terms

Clinical Trial Details
NCT number NCT03559699
Study type Interventional
Source Agios Pharmaceuticals, Inc.
Contact
Status Completed
Phase Phase 3
Start date June 26, 2018
Completion date November 12, 2020
View Details
See also
  Status Clinical Trial Phase
Active, not recruiting NCT03853798 - Extension Study of AG-348 in Adult Participants With Pyruvate Kinase Deficiency Previously Enrolled in AG-348-006 or AG348-C-007 Phase 3
Active, not recruiting NCT02476916 - A Study of AG-348 in Adult Participants With Pyruvate Kinase (PK) Deficiency Phase 2
Active, not recruiting NCT04105166 - Gene Therapy for Pyruvate Kinase Deficiency (PKD) Phase 1
Enrolling by invitation NCT05777993 - A Study to Provide Continued Access to Mitapivat for Participants Who Previously Completed an Agios-Sponsored Mitapivat Study Phase 4
Completed NCT04995315 - Pyruvate Kinase Deficiency Global Longitudinal Registry Substudy of Protocol AG348-C-008
Terminated NCT04964323 - Pyruvate Kinase (PK) Deficiency Global Longitudinal Registry: Patient-Reported Outcomes (PRO)
Completed NCT03866590 - Pyruvate Kinase Deficiency Epidemiological Study (PIECE)
Recruiting NCT04902833 - Acquired Pyruvate Kinase Deficiency In Clonal Myeloid Neoplasms
Completed NCT02053480 - Pyruvate Kinase Deficiency Natural History Study
Completed NCT03548220 - A Study to Evaluate Efficacy and Safety of AG-348 in Not Regularly Transfused Adult Participants With Pyruvate Kinase Deficiency (PKD) Phase 3
Not yet recruiting NCT06422351 - Clinical Trial to Evaluate the Efficacy of Gene Therapy for Pyruvate Kinase Deficiency Phase 2
Recruiting NCT03481738 - Pyruvate Kinase Deficiency Global Longitudinal Registry