Figure 2.
ACTIVATE/LTE study design. Key eligibility criteria: (1) age of ≥18 years; (2) documented 2 mutant alleles in PKLR with 1 missense mutation (excluding patients homozygous for R479H mutation or who have 2 nonmissense mutations, without another missense mutation); (3) ACTIVATE: not regularly transfused (≤4 transfusion episodes in the previous year); baseline hemoglobin of ≤10 g/dL; (4) LTE study: completed the fixed-dose period of ACTIVATE and demonstrated clinical benefit from mitapivat treatment, or were assigned to the placebo arm in ACTIVATE and elected to continue to the LTE study. ∗Stratified by average of screening hemoglobin values (<8.5 g/dL vs ≥8.5 g/dL) and PKLR gene mutation category (missense/missense vs missense/nonmissense). BID, twice daily; R, randomized.

ACTIVATE/LTE study design. Key eligibility criteria: (1) age of ≥18 years; (2) documented 2 mutant alleles in PKLR with 1 missense mutation (excluding patients homozygous for R479H mutation or who have 2 nonmissense mutations, without another missense mutation); (3) ACTIVATE: not regularly transfused (≤4 transfusion episodes in the previous year); baseline hemoglobin of ≤10 g/dL; (4) LTE study: completed the fixed-dose period of ACTIVATE and demonstrated clinical benefit from mitapivat treatment, or were assigned to the placebo arm in ACTIVATE and elected to continue to the LTE study. ∗Stratified by average of screening hemoglobin values (<8.5 g/dL vs ≥8.5 g/dL) and PKLR gene mutation category (missense/missense vs missense/nonmissense). BID, twice daily; R, randomized.

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