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A Study of AG-348 in Adult Participants With Pyruvate Kinase (PK) Deficiency

Pyruvate Kinase Deficiency Clinical Trial

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

Clinical Trial Summary

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.

Clinical Trial Description

This is a Phase 2, open label, two arm, multicenter, randomized, dose-ranging study during which adult participants with PK deficiency will receive multiple doses of AG-348 for up to 24 weeks (Core Period); eligible participants may enter an Extension Period to receive AG-348 for up to 8 additional years. Data will be reviewed on a regular basis and study design, dose and schedule will be adapted based on these reviews. The study will evaluate the safety and tolerability of multiple doses of AG-348, pharmacokinetic and pharmacodynamic (PD) profile of AG-348 and early indicators of clinical efficacy. ;

Study Design

Related Conditions & MeSH terms

Clinical Trial Details
NCT number NCT02476916
Study type Interventional
Source Agios Pharmaceuticals, Inc.
Contact
Status Active, not recruiting
Phase Phase 2
Start date June 26, 2015
Completion date March 2025
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
Completed NCT03559699 - A Study Evaluating the Efficacy and Safety of AG-348 in Regularly Transfused Adult Participants With Pyruvate Kinase Deficiency (PKD) Phase 3
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
Recruiting NCT03481738 - Pyruvate Kinase Deficiency Global Longitudinal Registry