This trial will evaluate the efficacy and safety of a drug called mitapivat in children with a condition called pyruvate kinase deficiency. Children will be given either the drug or a placebo, and researchers will track how well they do.
1 Primary · 28 Secondary · Reporting Duration: Week 8: ≤30 minutes predose; Week 12: ≤30 minutes predose
Experimental Treatment
Non-Treatment Group
30 Total Participants · 3 Treatment Groups
Primary Treatment: Mitapivat · Has Placebo Group · Phase 3
Age 1 - 17 · All Participants · 7 Total Inclusion Criteria
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