This is a global first-in-human randomized Phase I/II clinical study of ITU512, to assess the safety, tolerability, PK, PD and preliminary food effect of ITU512 in adult healthy participants, and safety, tolerability PK, PD, and efficacy of ITU512 in adolescent and adult patients with sickle cell disease (SCD). The study consists of Part 1 which is a first-in human Phase I study in healthy participants, and Part 2 which is a Phase II study in patients with SCD.
While the benefits of ITU512 on HbF induction have been demonstrated in preclinical studies, this will be the first clinical evaluation of the potential therapeutic effect of ITU512 in healthy participants and participants with SCD. The results of the study will be used to inform future development plans for ITU512 in SCD and β-globinopathies.
Expected impact:
ITU512 as a safe and efficacious oral option for SCD treatment. Participants with SCD may derive clinical benefit such as reduction in occurrence of vaso-occlusive crises, as ITU512 induces fetal hemoglobin.
