Overview
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).
Description
This is a global, randomized, Phase I/II study to assess the safety, tolerability, pharmacokinetics (PK), pharmacodynamics (PD), and preliminary food effect of single-agent 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 a first-in-human Phase I study (Part 1) in healthy participants, and a Phase II study (Part 2) in patients with SCD.
Part 1 will comprise of Part 1A, Part 1B, and Part 1C. Part 2 may include an extension part.
Eligibility
Key Inclusion Criteria:
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
Key Exclusion Criteria:
Part 1 (Healthy participants)
- QTcF ≥ 450 msec (as a mean value of triplicates)
- History of arrhythmias
- History of significant illness which has not resolved within two (2) weeks prior to initial dosing
- Women of child-bearing potential (WOCBP)
Part 2 (Sickle Cell Disease)
- Current use of hydroxyurea/hydroxycarbamide (HU/HC)
- QTcF ≥ 450 msec (as a mean value of triplicates)
- History of arrhythmias
Other protocol-defined inclusion/exclusion criteria may apply.