A Phase 3, Double-Blind, Randomized, Placebo-Controlled, Multicenter Study to Evaluate the Effect of Mitapivat on Transfusion Burden in Subjects With Sickle Cell Disease

Agios Pharmaceuticals, Inc.

Summary

The primary objective of this study is to determine the effect of mitapivat versus placebo on the need for transfusions in subjects with SCD.

Eligibility

Age range: 12+ years
Sex: All
Healthy volunteers: No

Detailed criteria

Inclusion Criteria: * Age ≥12 years. * Documented diagnosis of SCD (hemoglobin SS (HbSS), combined heterozygosity for hemoglobins S and C (HbSC), sickle cell hemoglobin (HbS)/β0-thalassemia, HbS/β+-thalassemia, or other sickle cell syndrome variants). * No more than 10 SCPCs in the 12 months before providing informed assent/consent. * At least 1 transfusion of packed RBCs in the 12 months before informed assent/consent. * Hb ≥5.5 and ≤10.5 g/dL. Hb concentration must be based on an average of at least 2 Hb concentration measurements (separated by ≥7 days) collected during the Screening Period…

Interventions

Drug
Mitapivat Matched Placebo
Tablets
Drug
Mitapivat
Tablets

Locations (3)

Indiana Hemophilia & Thrombosis Center
Indianapolis, Indiana
The Cancer and Hematology Centers (CHC)
Grand Rapids, Michigan
Southern Specialty Clinic
Flowood, Mississippi

A Phase 3, Double-Blind, Randomized, Placebo-Controlled, Multicenter Study to Evaluate the Effect of Mitapivat on Transfusion Burden in Subjects With Sickle Cell Disease

Summary

Eligibility

Interventions

Locations (3)

Compensation

Run this study?

Details

Conditions