Table 1.

Clinical trials of pyruvate kinase activators in thalassemia

CategoryPhaseDrugNDurationIdentifierStatus
NTDT 2 (single-arm, open-label) Mitapivat 20 24 weeks NCT03692052 Completed 
Etavopivat 20 48 weeks NCT04987489
(cohort C) 
Ongoing 
3 (double-blind RCT) Mitapivat: placebo (2:1) 171 24 weeks NCT04770753 Ongoing 
TDT 2 (single-arm, open-label) Etavopivat 20 48 weeks NCT04987489
(cohort B) 
Ongoing 
3 (double-blind RCT) Mitapivat: placebo
(2:1) 
240 48 weeks NCT04770779 Ongoing 
CategoryPhaseDrugNDurationIdentifierStatus
NTDT 2 (single-arm, open-label) Mitapivat 20 24 weeks NCT03692052 Completed 
Etavopivat 20 48 weeks NCT04987489
(cohort C) 
Ongoing 
3 (double-blind RCT) Mitapivat: placebo (2:1) 171 24 weeks NCT04770753 Ongoing 
TDT 2 (single-arm, open-label) Etavopivat 20 48 weeks NCT04987489
(cohort B) 
Ongoing 
3 (double-blind RCT) Mitapivat: placebo
(2:1) 
240 48 weeks NCT04770779 Ongoing 

RCT, randomized controlled trial.

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