Clinical Trial

A Study Evaluating the Efficacy and Safety of Mitapivat (AG-348) in Participants With Sickle Cell Disease (RISE UP)

Active, Not Recruiting Phase 2/3
View on ClinicalTrials.gov →
Summary
This clinical trial is a Phase 2/3 study that will determine the recommended dose of mitapivat and evaluate the efficacy and safety of mitapivat in sickle cell disease by testing how well mitapivat works compared to placebo to increase the amount of hemoglobin in the blood and to reduce or prevent the occurrence of sickle cell pain crises. In addition, the long-term effect of mitapivat on efficacy and safety will be explored in an open-label extension portion.
Protocol Amendment History 55 amendments
This ClinicalTrials.gov record has been amended 55 times since 2021-08-30; most recent amendment 2026-06-19.
Status change: Recruiting → Active, Not Recruiting 2024-11-18
Status change: Active, Not Recruiting → Recruiting 2023-11-29
Status change: Recruiting → Active, Not Recruiting 2022-12-22
Status change: Not Yet Recruiting → Recruiting 2022-01-05
Trial Details
NCT Number NCT05031780
Lead Sponsor Agios Pharmaceuticals, Inc.
Conditions Sickle Cell Disease
Enrollment 286 participants
Start Date 2022-02-11
Primary Completion 2025-10-30 (estimated)
Study Completion 2030-02 (estimated)
Updated on ClinicalTrials.gov 2026-06-23