A Phase I/II Clinical Study to Investigate the Safety, Tolerability, Pharmacokinetics, Pharmacodynamics, and Efficacy of ITU512 in Healthy Participants and Patients With Sickle Cell Disease
The purpose of this study is to evaluate the safety, tolerability, pharmacokinetics (PK), and preliminary food effect of ITU512 as well as the fetal hemoglobin (HbF)-inducing capacity of ITU512. This will be the first evaluation of the potential therapeutic effect of ITU512 in healthy participants and patients with sickle cell disease (SCD).
⁃ Part 1 (Healthy participants)
• Healthy male participants and female participants of non-childbearing potential between 18-55 years of age
• In good health as determined by the investigator's assessment of medical history, physical examination, vital signs, ECG, and laboratory tests
• Participants must weigh at least 50 kg at screening and first baseline (admission) and must have a body mass index (BMI) within the range of 18.0-32.0 kg/m2 inclusive.
⁃ Part 2 (Sickle Cell Disease)
⁃ \- Male and female participants with a diagnosis of sickle cell disease