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
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 full inclusion and exclusion criteria, as well as study locations, search ClinicalTrials.gov for NCT04770779
For more information and study locations, visit ClinicalTrials.gov.
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.