A Phase I/II Study of ITU512 in Healthy Participants and Patients With Sickle Cell Disease
Recruiting · NCT06546670 · Interventional (participants receive a specific treatment) · Lead sponsor: Novartis Pharmaceuticals
View the official record on ClinicalTrials.gov →Interventions studied
ITU512Placebo
What this trial is about
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 take part
Age range
12 Years to 55 Years
Sex
All (male and female)
Healthy volunteers
Yes - healthy volunteers may be accepted
Phase
Phase 1, Phase 2
Study type
Interventional (participants receive a specific treatment)
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
- ther protocol-defined inclusion/exclusion criteria may apply.
Where it is running
10 locations listed across 7 US states.
- University of Alabama Birmingham - Birmingham, Alabama, United States
- Quotient Sciences Sea View - Miami, Florida, United States
- Boston Childrens Hospital - Boston, Massachusetts, United States
- Levine Cancer Insitute Carolinas Healthcare System - Charlotte, North Carolina, United States
- East Carolina University - Greenville, North Carolina, United States
- Childrens Hospital Of Philadelphia - Philadelphia, Pennsylvania, United States
- Lifespan - Providence, Rhode Island, United States
- UT Health Science Center - Houston, Texas, United States
- Novartis Investigative Site - Ankara, Sihhiye-Altindag, Turkey (Türkiye)
- Novartis Investigative Site - Adana, Yuregir, Turkey (Türkiye)