Testing a new medicine to reduce pain crises in sickle cell disease

This is a global study testing a new medication called etavopivat to see if it can reduce vaso-occlusive crises — the severe pain crises that happen when sickle cells block blood vessels — in adolescents and adults with sickle cell disease. Researchers also want to see if the medication can reduce damage to organs, help with exercise tolerance, and decrease fatigue. Half of participants will receive the medication, and half will receive a placebo (a pill with no active ingredient), decided by chance.

Sickle cell disease causes painful and potentially life-threatening crises when misshapen red blood cells block blood vessels. Currently available treatments don't work for everyone, and researchers are looking for new options that can prevent these crises and reduce long-term organ damage.