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A Study Evaluating the Efficacy and Safety of Mitapivat (AG-348) in Participants With Sickle Cell Disease (RISE UP)
NCT05031780 · View on ClinicalTrials.gov ↗
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 Studied
Interventions
- DRUG Mitapivat
- OTHER Mitapivat-matching placebo
Study Locations (20)
California
- University of California San Diego — La Jolla
- UCLA Health — Los Angeles
- Children's Hospital Oakland — Oakland
Massachusetts
- Massachusetts General Hospital — Boston
- Boston Children's Hospital — Boston
- Boston Medical Center & Boston University School of Medicine — Boston
District of Columbia
- Children's National Hospital — Washington D.C.
- MedStar Washington Hospital Center — Washington D.C.
Maryland
- National Heart Lung and Blood Institute — Bethesda
- Kaiser Permanente - Largo Medical Center — Largo
Michigan
- University of Michigan — Ann Arbor
- Children's Hospital of Michigan — Detroit
Mississippi
- Southern Specialty Clinic — Flowood
- Mississippi Center for Advanced Medicine — Madison
Connecticut
- University of Connecticut Health Center — Farmington
Florida
- Sylvester Comprehensive Cancer Center-Miami — Miami
Trial Details
| Field | Value |
|---|---|
| Enrollment Target | 286 participants |
| Start Date | 2022-02-11 |
| Est. Completion | 2030-02 |
| Phase | Phase 2 |
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Full Details on ClinicalTrials.gov ↗What the Registry Record Tells You About NCT05031780
The ClinicalTrials.gov registry entry for NCT05031780 describes a study currently listed as active not recruiting. It is categorized as Phase 2, which is the standard way researchers label where a study sits along the investigational pathway from early safety work through later efficacy and post-marketing evaluation. The registered enrollment target is 286 participants, a figure that helps gauge the scale of data the investigators plan to collect. The listed sponsor is Agios Pharmaceuticals, which has 15 total studies on file at ClinicalTrials.gov, and sponsors are the parties responsible for study design, oversight, and regulatory filings.
The record links to 1 condition, with Sickle Cell Disease appearing as the primary indexed condition, and to 2 interventions — of which Mitapivat is the first listed. Interventions can include drugs, devices, procedures, behavioral programs, or observational arms, and each is tracked as a separate registry field so that downstream queries can filter accurately. When a trial lists multiple interventions, it usually reflects a multi-arm design or a comparison protocol rather than a single treatment being tested in isolation. The brief summary published in the registry is the clearest source of protocol intent and should be read before drawing conclusions from any sidebar tags.
Geographic footprint matters for practical reasons: NCT05031780 reports 20 study locations spanning 12 distinct geographic areas — top geographies include California, Massachusetts, District of Columbia. A larger site network tends to correlate with broader recruitment capacity, but it does not imply anything about study quality, and site-level enrollment status can diverge from the overall registry status shown above. Every data point on this page comes from the public ClinicalTrials.gov dataset and is reproduced here for reference only; it is not a medical recommendation, an endorsement of the sponsor, or an invitation to enroll. Verify current status, eligibility criteria, and contact details directly at ClinicalTrials.gov, and discuss any participation decision with your own healthcare provider.
Frequently Asked Questions
What is clinical trial NCT05031780 about?
NCT05031780 is a clinical study titled "A Study Evaluating the Efficacy and Safety of Mitapivat (AG-348) in Participants With Sickle Cell Disease (RISE UP)". 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 t...
What is the current status of trial NCT05031780?
This trial is currently active not recruiting. It is a Phase 2 study. The enrollment target is 286 participants. The study started on 2022-02-11. Estimated completion is 2030-02.
What conditions does trial NCT05031780 study?
This clinical trial studies the following conditions: Sickle Cell Disease. These conditions were identified from the trial registry and reflect the primary focus areas of the research.
What interventions are being tested in trial NCT05031780?
The interventions under investigation include: Mitapivat (DRUG), Mitapivat-matching placebo (OTHER). Each intervention is being evaluated for safety and efficacy as part of this clinical study.
Who is sponsoring clinical trial NCT05031780?
This trial is sponsored by Agios Pharmaceuticals, which has 15 total clinical trials registered on ClinicalTrials.gov. The sponsor is responsible for the study's design, funding, and regulatory compliance.
Where is trial NCT05031780 being conducted?
This trial has 20 study locations across California, Connecticut, District of Columbia, Florida, Illinois. Contact the study sites directly through ClinicalTrials.gov for enrollment availability.
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