Phase III Study of ITU512 to Assess Safety and Effects in Healthy Adults and Sickle Cell Disease Patients

What this Trial Is About

Researchers are evaluating ITU512, an investigational oral drug, in healthy adults and patients with sickle cell disease SCD to study its safety, tolerability, how it is processed by the body pharmacokinetics, and its potential to increase fetal hemoglobin levels. This Phase III study is the first to assess ITU512s possible therapeutic effects in these groups, aiming to better understand its impact on SCD. The study is divided into two parts Part 1, a first-in-human Phase I trial involving healthy participants, and Part 2, a Phase II trial including patients with SCD. Part 1 includes three subparts 1A, 1B, 1C to examine safety, dosage, and food effects in healthy adults. Part 2 has two subparts 2A, 2B plus an optional extension 2C focusing on safety, tolerability, pharmacokinetics, pharmacodynamics, and efficacy in adolescents and adults with SCD. Participants may receive ITU512 or placebo in a randomized, double-blind crossover design. During the study, participants undergo various assessments including medical exams, vital signs, ECGs, and blood and urine tests to monitor drug levels and effects on fetal hemoglobin and other blood parameters. Safety is closely tracked with attention to adverse events and dose adjustments. The study includes up to 5 months of active treatment and follow-up, with detailed monitoring of outcomes such as adverse events, dose interruptions, and fetal hemoglobin percentage. Participant involvement includes scheduled visits for dosing and evaluations throughout the study timeline.

A Phase I/II Study of ITU512 in Healthy Participants and Patients With Sickle Cell Disease