Phase 3 study · Agios · NCT07656415
Agios's phase 3 mitapivat study for sickle cell disease
About 159 people ages 12 and up are planned, and each gets mitapivat or a placebo tablet twice a day for up to 52 weeks. Groups are picked by chance and hidden from participants and study doctors, and those who finish may get mitapivat for up to 52 more weeks.
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Hear when Agios's phase 3 mitapivat study opens near you
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Check the main requirements privately
These questions restate the study's public listing in plain words. Your answers stay on this device and are never sent. You can print a one-page summary to go over with your hematologist.
1. Are you, or the person you care for, 12 or older?
Parents, guardians and caregivers can answer these questions for a teen they care for. For anyone under 18, the listing says a parent gives permission and the teen agrees too.2. Have you had at least 1 red blood cell transfusion in the past 12 months?
3. Do you get red blood cell transfusions on a regular schedule, sometimes called chronic transfusion therapy?
Transfusions given when anemia gets worse or during a pain crisis are allowed. The listing also requires no transfusion in the 60 days before consent or during screening.4. Is your hemoglobin usually between 5.5g/dL and 10.5g/dL?
The study measures this itself at least twice, 7 or more days apart, during screening. Your hematologist's office can tell you your latest hemoglobin number.5. Have you had 10 or fewer sickle cell pain crises in the past 12 months?
6. Have you taken voxelotor, crizanlizumab or L-glutamine in the past 90 days?
Crizanlizumab is sold under the brand name Adakveo, and L-glutamine under the brand name Endari. The listing counts the 90 days back from the day each person is assigned to a group.7. Have you ever had gene therapy, or a bone marrow or stem cell transplant?
The study team also checks for these, which are easier to go over with your hematologist:
- A documented sickle cell disease diagnosis, such as HbSS, HbSC, sickle beta thalassemia or another sickle cell type
- Signs of red blood cell breakdown on screening labs, such as hemoglobin under 8g/dL or a high reticulocyte count, indirect bilirubin or LDH
- A steady hydroxyurea dose for at least 90 days before randomization, or at least 90 days off hydroxyurea before consent
- A hospital stay for a pain crisis or other vaso-occlusive event in the 14 days before consent or during screening
- Active, uncontrolled heart or lung disease in the past 6 months, cirrhosis or other listed liver and gallbladder problems, or an eGFR under 30mL/min/1.73m2
- Strong CYP3A4 inhibitor or inducer medicines, anabolic steroids or blood cell stimulating medicines within the listed time frames, or an earlier mitapivat study
Sites and contact
US sites from the public ClinicalTrials.gov record, refreshed every hour. Only the study team decides who can join.
- Indiana Hemophilia & Thrombosis CenterIndianapolis, Indiana · Recruiting
- The Cancer and Hematology Centers (CHC)Grand Rapids, Michigan · Recruiting
- Southern Specialty ClinicFlowood, Mississippi · Recruiting
- Icahn School of Medicine at Mount SinaiNew York, New York · Recruiting
- University of Texas Health Science Center at HoustonHouston, Texas · Recruiting
Every site's own contact and the full eligibility list are on the official ClinicalTrials.gov listing for NCT07656415.
Questions people ask about Agios's phase 3 mitapivat study
Is Agios's phase 3 mitapivat study enrolling now?
ClinicalTrials.gov lists it as recruiting, as of its last update there on 2026-10-28. Only the study team can confirm whether a particular site is still taking people.
Where are the US sites for Agios's phase 3 mitapivat study?
The listing names 5 US sites, in Indiana, Michigan, Mississippi, New York, Texas. The full list is in the sites section of this page.
Who can join Agios's phase 3 mitapivat study?
About 159 people ages 12 and up are planned, and each gets mitapivat or a placebo tablet twice a day for up to 52 weeks. The main listed requirements are in the pre-check on this page, starting with: ages 12 and older. Only the study team decides who can join.
How do I contact the Agios's phase 3 mitapivat study team?
The listing names this contact: Agios Medical Affairs, 08332288474, medinfo@agios.com. Each site may also list its own contact on ClinicalTrials.gov.
Is Trial Friend part of this study?
No. Trial Friend is independent of this study and its sponsor, Agios. This page restates the public listing in plain words, and nothing here is medical advice; talk with your hematologist about whether a study is right for you.
From the public ClinicalTrials.gov record NCT07656415, last updated there 2026-10-28. Questions checked against the listing on November 10, 2026. Trial Friend is independent of this study and its sponsor, and is not a medical provider. Nothing here is medical advice; talk with your hematologist about whether a study is right for you.