Hemoglobin (Hb) response, defined as a ≥1.0 g/dL increase in average Hb concentration from week 12 through week 24 compared with baseline
Key secondary endpoints
- Change from baseline in average Functional Assessment of Chronic Illness Therapy-Fatigue (FACIT-Fatigue) subscale score from week 12 through week 24
- Change from baseline in average Hb concentration from week 12 through week 24
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
- Hb concentration ≤10.0 g/dL
- Non–transfusion-dependent, defined as ≤5 red blood cell (RBC) units during the 24-week period before randomization, and no RBC transfusions ≤8 weeks before providing informed consent or during the screening period
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 NCT04770753
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.