Last Update: Jul 20, 2026 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 ClinicalTrials.gov Identifier: [NCT06546670](https://clinicaltrials.gov/ct2/show/NCT06546670) Novartis Reference Number:CITU512A12101 [See if you Pre-qualify](#trial-eligibility "See if you Pre-qualify") All compounds are either investigational or being studied for (a) new use(s). Efficacy and safety have not been established. There is no guarantee that they will become commercially available for the use(s) under investigation. ## Study 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). 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 will include Part 2A and 2B and may also include an extension part (Part 2C). Condition Sickle Cell Disease Phase Phase1, Phase2 Overall Status Recruiting Number of Participants161 Start Date Aug 15, 2024 Completion Date Feb 19, 2030 Gender All Age(s) 12 Years - 55 Years (Child, Adult) ## Interventions Drug ### ITU512 ITU512 is an investigational, oral, low molecular weight (LMW) compound. Drug ### Placebo An inactive substance that looks like and is given the same way as ITU512. The effect(s) of ITU512 will be evaluated against the placebo. Placebos are designed as a control and to have no real effect. ## Eligibility Criteria 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. Turkey (Türkiye) #### Novartis Investigative Site Recruiting Adana,Yuregir,01250,Turkey (Türkiye) #### Novartis Investigative Site Recruiting Ankara,Sihhiye-Altindag,06230,Turkey (Türkiye) United States #### University of Alabama Birmingham Recruiting Birmingham,Alabama,35233,United States ###### Emily Burke Phone: [+1 205 801 3411]() Email: ###### Julie Kanter-Washko #### Boston Childrens Hospital Recruiting Boston,Massachusetts,02215,United States ###### Matthew M Heeney ###### Nadia Tarhini Phone: [+1 617 355 4278]() Email: #### Lifespan Recruiting Providence,Rhode Island,02903,United States ###### Asher Dillman Phone: [+1 401 444 5328]() Email: ###### Patrick Mc Gann #### UT Health Science Center Recruiting Houston,Texas,77030,United States ###### Eyuel Baherey Email: ###### Modupe Idowu #### East Carolina University Recruiting Greenville,North Carolina,27858,United States ###### Darla Liles ###### Kelly Martin Email: #### Childrens Hospital Of Philadelphia Recruiting Philadelphia,Pennsylvania,19104,United States ###### Alexis A Thompson ###### Kevin Dougherty Phone: [+1 215 590 9959]() Email: ## Worldwide Contacts If the location of your choosing does not feature any contact detail, please reach out using the information below. #### Novartis Pharmaceuticals Phone: [ +41613241111](tel:+41613241111) Email: [](mailto:) #### Novartis Pharmaceuticals Phone: [ 1-888-669-6682](tel:1-888-669-6682) Email: