Clinical Trial

Primary endpoint

Transfusion reduction response, defined as a ≥50% reduction in transfused red blood cell (RBC) units with a reduction of ≥2 units of transfused RBCs in any consecutive 12-week period through week 48 compared with baseline

Key secondary endpoints

• ≥33% reduction in transfused RBC units from week 13 through week 48 compared with baseline
• ≥50% reduction in transfused RBC units in any consecutive 24-week period through week 48 compared with baseline
• ≥50% reduction in transfused RBC units from week 13 through week 48 compared with baseline

Trial design

Key inclusion criteria

• ≥18 years of age at the time of providing informed consent
• Diagnosis of β-thalassemia with or without α-globin gene mutations, HbE/β-thalassemia, or α-thalassemia/HbH disease
• Transfusion-dependent, defined as 6 to 20 RBC units transfused and a ≤6-week transfusion-free period during the 24-week period before randomization

Key exclusion criteria

• Pregnant or breastfeeding
• Documented history of homozygous or heterozygous HbS or HbC
• Certain prior or current therapies
• Significant medical condition that confers an unacceptable risk to participating in the study and/or could confound the interpretation of the study data in the opinion of the investigator

For more information and study locations, visit ClinicalTrials.gov.

ENERGIZE-T (NCT04770779)

For additional information, or to find out about becoming a study site, visit Agios.com.

Mitapivat is not approved by health authorities for the treatment of thalassemia.