A Phase 2/3, Double-Blind, Randomized, Placebo-Controlled, Multicenter Study to Evaluate the Efficacy and Safety of Mitapivat in Subjects With Sickle Cell Disease

About the study

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.

Study point of contact

Agios Medical Affairs
[email protected]


11 United States sites

4 Belgium sites

3 France sites

3 Israel sites

2 Lebanon sites

1 Netherlands site

1 Italy site


> 16 Years




Phase 2/Phase 3

Study type







participation requirements

Age 16 years or older (18 years or older [France and Germany]); participants age 16 or 17 years must physically have completed puberty;
Documented diagnosis of sickle cell disease (SCD) (HbSS, HbSC [combined heterozygosity for hemoglobins S and C], HbS/beta 0- thalassemia, HbS/ beta plus thalassemia, or other sickle cell syndrome variants);
At least 2 sickle cell pain crises (SCPCs) and no more than 10 SCPCs in the past 12 months;
Hemoglobin at least 5.5 and 10.5 gram per deciliter (g/dL) at the most. Hemoglobin concentration must be based on an average of at least 2 Hb concentration measurements (separated by ≥7 days) collected during the Screening Period;
If taking hydroxyurea, the hydroxyurea dose must be stable for at least 90 days before starting study drug;
Women capable of becoming pregnant and men with partners who are women that are capable of becoming pregnant must agree to use 2 forms of contraception.

participation restrictions

Pregnant, breastfeeding, or parturient;
Receiving regularly scheduled transfusions;
Hepatobiliary disorders including but not limited to significant liver disease or gallbladder disease;
Severe kidney disease;
Prior exposure to gene therapy or prior bone marrow or stem cell transplantation;
Currently receiving treatment for SCD (eg, voxelotor, crizanlizumab, L-glutamine), with the exception of hydroxyurea. The last dose of such therapies must have been administered at least 90 days before starting study drug;
Currently receiving treatment with hematopoietic stimulating agents; the last dose must have been administered at least 90 days before starting study drug;
Received treatment on another investigational trial within 90 days prior to start of study drug or plans to participate in another investigational drug trial;
Taking medications that are strong inhibitors of CYP3A4/5 or strong inducers of CYP3A4 that cannot be stopped in an acceptable timeframe before starting study drug (timeframe will be discussed with your doctor).


  • La Jolla, California, United States, University of California San Diego
  • Oakland, California, United States, Children's Hospital Oakland
  • Largo, Maryland, United States, Kaiser Permanente - Largo Medical Center
  • Ann Arbor, Michigan, United States, University of Michigan
  • Detroit, Michigan, United States, Children's Hospital of Michigan
  • Las Vegas, Nevada, United States, Cure 4 The Kids Foundation, A Division of Roseman University of Health Sciences
  • Greenville, North Carolina, United States, East Carolina University - Brody School of Medicine
  • Hershey, Pennsylvania, United States, Penn State Health Milton S. Hershey Medical Center
  • Philadelphia, Pennsylvania, United States, Penn Medicine - University of Pennsylvania Health System
  • Philadelphia, Pennsylvania, United States, St. Christopher's Hospital for Children
  • Seattle, Washington, United States, Seattle Cancer Care Alliance, University of Washington
  • Anderlecht, Brussels, Belgium, Hôpital Erasme
  • Edegem, Brussels, Belgium, Universitair Ziekenhuis Antwerpen
  • Liège, Belgium, CHR de la Citadelle
  • Liège, Belgium, Clinique CHC MontLégia
  • Bordeaux, Gironde, France, Hôpital Pellegrin, CHU de Bordeaux
  • Toulouse, Haute-Garonne, France, Institut Universitaire du Cancer de Toulouse - Oncopole
  • Créteil, Val-de-Marne, France, CHU Hôpital Henri Mondor
  • Afula, Israel, HaEmek Medical Center
  • Haifa, Ḥeifā, Israel, Rambam Medical Center
  • Safed, Ḥeifā, Israel, Ziv Medical Center
  • Napoli, Campania, Italy, A.O.R.N. "A. Cardarelli"
  • Tarablus, Liban Nord, Lebanon, Nini Hospital
  • Sidon, Lebanon, Hammoud Hospital University Medical Center
  • Rotterdam, Zuid-Holland, Netherlands, Erasmus MC
Last updated 2022-09-21