NCT06546670 · Novartis Pharmaceuticals
A Phase I/II Study of ITU512 in Healthy Participants and Patients With Sickle Cell Disease
What this study is about
The purpose of this study is to evaluate the safety, tolerability, how the drug moves through the body (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).
View original scientific description
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).
Who can participate
This study lists these criteria on ClinicalTrials.gov. A study coordinator reviews eligibility during screening — this page does not determine whether you qualify.
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.
Where
- Birmingham, Alabama
- Miami, Florida
- Boston, Massachusetts
- Greenville, North Carolina
- Providence, Rhode Island
- Houston, Texas
Frequently asked questions
What is a clinical trial?
A clinical trial is a research study that tests new medical treatments, drugs, devices, or procedures to determine their safety and effectiveness. Trials are carefully designed and monitored to protect participants while advancing medical knowledge.
Is it safe to participate?
Clinical trials follow strict safety guidelines and ethical standards. Trials must be reviewed and approved, and participants are closely monitored by medical professionals throughout the study. You can withdraw at any time if you choose.
Will I be compensated?
Many clinical trials offer compensation for your time, travel expenses, and inconvenience. The specific compensation varies by study and will be discussed during the screening process. All study-related medical care is typically provided at no cost to participants.
Will I receive a placebo instead of treatment?
When effective treatment exists, participants typically receive either the standard treatment plus the study intervention, or the standard treatment plus placebo. You would not be denied effective care. Placebos are primarily used when no proven treatment is available, or in addition to standard care. Your trial consent form will clearly explain what treatments you may receive.
Can I leave a trial if I change my mind?
Absolutely. Participation in clinical trials is completely voluntary. You have the right to withdraw from the study at any time, for any reason, without penalty or loss of benefits to which you are otherwise entitled.
How long does a clinical trial last?
Trial duration varies widely depending on the study design and purpose. Some trials last just a few weeks, while others may continue for months or years. The study coordinator will provide specific timeline information during your screening call.
Data: ClinicalTrials.gov · synced Jun 30, 2026 · Source of record for eligibility and locations