Classical Homocystinuria research hub

A Phase 3, Parallel-group Treatment, Blinded, Randomized, Placebo-controlled Study to Assess the Efficacy and Safety of Pegtibatinase Administered Subcutaneously in Addition to Standard of Care in Participants with Classical Homocystinuria due to Cystathionine Beta Synthase Deficiency (HARMONY)

This study is looking into a new treatment called Pegtibatinase for people with a rare genetic condition called Classical Homocystinuria. Researchers want to see if this new medicine, given as a shot under the skin, can help lower harmful levels of a substance in the blood called tHcy.