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

NCT05031780 · Status: ACTIVE_NOT_RECRUITING · Phase: PHASE2/PHASE3 · Type: INTERVENTIONAL · Enrollment: 286

Last updated 2026-04-23

No results posted yet for this study

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.

Conditions

Interventions

DRUG

Mitapivat

Mitapivat tablets

OTHER

Mitapivat-matching placebo

Placebo to match 50 mg or 100 mg tablets

OTHER

Mitapivat-matching placebo

Placebo to match 100 mg tablets

Sponsors & Collaborators

  • Agios Pharmaceuticals, Inc.

    lead INDUSTRY

Study Design

Allocation
RANDOMIZED
Purpose
TREATMENT
Masking
QUADRUPLE
Model
SEQUENTIAL

Eligibility

Min Age
16 Years
Sex
ALL
Healthy Volunteers
No

Timeline & Regulatory

Start
2022-02-11
Primary Completion
2025-10-30
Completion
2030-02-28
FDA Drug
Yes

Countries

  • United States
  • Belgium
  • Brazil
  • Canada
  • France
  • Germany
  • Israel
  • Italy
  • Kenya
  • Lebanon
  • Netherlands
  • Nigeria
  • Oman
  • Saudi Arabia
  • Turkey (Türkiye)
  • United Kingdom

Study Locations

More Related Trials

Entities

Diseases

Read the full study record

This page highlights key information. For complete eligibility criteria, study locations, investigator contacts, and the full protocol, visit the original record on ClinicalTrials.gov.

View NCT05031780 on ClinicalTrials.gov