Evaluating ITU512 for Sickle Cell Disease and Healthy Participants

Is this trial a fit for you?

A plain-English reading of the study's rules. Only the study team can confirm whether you qualify.

What this trial studies

This study aims to assess the safety, tolerability, pharmacokinetics, and preliminary food effect of ITU512 in both healthy participants and patients with sickle cell disease (SCD). It consists of a Phase I component involving healthy adults and a Phase II component focusing on adolescents and adults diagnosed with SCD. The study will evaluate the potential therapeutic effects of ITU512, particularly its ability to induce fetal hemoglobin. Participants will be randomized to receive either ITU512 or a placebo.

Why it matters

Who can join: the full rules

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.

Where it's running

• University of Alabama Birmingham
  Birmingham, Alabama, United States Recruiting
• Boston Childrens Hospital
  Boston, Massachusetts, United States Recruiting
• Icahn School Of Med At Mount Sinai
  New York, New York, United States Recruiting
• Levine Cancer Insitute Carolinas Healthcare System
  Charlotte, North Carolina, United States Recruiting
• East Carolina University
  Greenville, North Carolina, United States Recruiting
• Childrens Hospital Of Philadelphia
  Philadelphia, Pennsylvania, United States Recruiting

Study contacts

• Study coordinator: Novartis Pharmaceuticals
• Email: novartis.email@novartis.com
• Phone: 1-888-669-6682

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Other studies for the same condition or treatment. Recruiting studies are listed first.

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How to take part

1. Review the eligibility criteria above with your treating physician.
2. Check the official listing on ClinicalTrials.gov for the latest contact details and recruitment status.
3. Contact the study coordinator or principal investigator to ask for pre-screening. Pre-screening is free and never obligates you to enroll.