Trial results for the Phase 3 extension study (NCT03853798) investigating mitapivat in adult participants with pyruvate kinase deficiency were posted on ClinicalTrials.gov on 2025-11-18. The study evaluated the long-term safety, tolerability, and efficacy of mitapivat, with key measurements focusing on treatment-emergent adverse events.
Background
Mitapivat is an investigational drug for pyruvate kinase deficiency, a rare, inherited hemolytic anemia. This extension study aimed to gather long-term data on its use in patients previously treated in earlier trials.
Trial design
The extension study (NCT03853798) was an open-label, multicenter, Phase 3 trial that enrolled 90 adult participants with pyruvate kinase deficiency. Participants had previously been enrolled in Study AG348-C-006 or Study AG348-C-007. The study's primary objective was to evaluate the long-term safety, tolerability, and efficacy of treatment with mitapivat.
Key results
The trial results focused on the long-term safety profile of mitapivat, reporting the number of participants experiencing treatment-emergent adverse events (TEAEs) across three cohorts. The total enrollment for the study was 90 participants. Key safety findings included:
- For all TEAEs: 37 participants in Cohort 1, 33 participants in Cohort 2, and 15 participants in Cohort 3 experienced at least one TEAE.
- For serious TEAEs: 11 participants in Cohort 1, 8 participants in Cohort 2, and 4 participants in Cohort 3 experienced at least one serious TEAE.
- For related TEAEs: 22 participants in Cohort 1, 14 participants in Cohort 2, and 4 participants in Cohort 3 experienced at least one related TEAE.
- For TEAEs with severity greater than or equal to Grade 3: 16 participants in Cohort 1, 10 participants in Cohort 2, and 5 participants in Cohort 3 experienced such events.
What this means
The posted results provide long-term safety data for mitapivat in adult participants with pyruvate kinase deficiency. As an open-label extension study, these findings contribute to the overall understanding of the drug's safety and tolerability profile over an extended treatment period, which is crucial for rare chronic conditions like pyruvate kinase deficiency. The data characterizes the occurrence of various types of adverse events observed in participants continuing treatment.
Source
The information regarding these trial results was obtained from ClinicalTrials.gov, a public database of clinical studies. The results for study NCT03853798, titled "Extension Study of AG-348 in Adult Participants With Pyruvate Kinase Deficiency Previously Enrolled in AG-348-006 or AG348-C-007," were posted on 2025-11-18 on clinicaltrials.gov.