- enrolling by invitationPHASE3
A Long-term Extension Study to Assess the Long-term Safety and Efficacy of Pegtibatinase Treatment in Participants ≥5 to ≤65 Years of Age With Classical Homocystinuria (HCU) (ENSEMBLE)
This study looks at a new treatment called pegtibatinase for Homocystinuria (HCU) in people aged 5 to 65. It's a long-term study to see how safe and effective the treatment is over time, especially for those already in other related studies.
United States · Australia · Belgium - recruitingPHASE3
A Study to Investigate Efficacy and Safety of Pegtibatinase Compared With Placebo in Participants ≥12 to ≤65 Years of Age With Classical Homocystinuria (HCU) Due to Cystathionine Beta Synthase Deficiency Receiving Standard of Care Treatment
This study is looking at a new medicine called pegtibatinase for people aged 12-65 with a rare condition called Homocystinuria (HCU). It aims to see if the drug is effective and safe compared to a dummy treatment.
United States · Australia · Belgium