Evaluating ITU512 for Sickle Cell Disease and Healthy Participants
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
This study is testing a new drug called ITU512 to see if it is safe and effective for people with sickle cell disease and healthy adults.
Quick facts
| Phase | Phase1; Phase2 |
|---|---|
| Study type | Interventional |
| Enrollment | 161 (estimated) |
| Ages | 12 Years to 55 Years |
| Sex | All |
| Sponsor | Novartis Industry-sponsored |
| Locations | 3 sites (Birmingham, Alabama and 2 other locations) |
| Trial ID | NCT06546670 on ClinicalTrials.gov |
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.
Who should consider this trial
Good fit: Ideal candidates include male and female patients diagnosed with sickle cell disease.
Not a fit: Patients currently using hydroxyurea or those with significant cardiac issues may not benefit from this study.
Why it matters
Potential benefit: If successful, this study could provide a new treatment option for patients with sickle cell disease.
How similar studies have performed: Other studies have shown promise in using similar approaches to increase fetal hemoglobin levels in sickle cell disease, but this specific compound is novel.
Eligibility criteria
Show full inclusion / exclusion 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.
Where this trial is running
Birmingham, Alabama and 2 other locations
- University of Alabama Birmingham — Birmingham, Alabama, United States (Recruiting)
- Quotient Sciences Sea View — Miami, Florida, United States (Completed)
- Boston Childrens Hospital — Boston, Massachusetts, United States (Recruiting)
Study contacts
- Study coordinator: Novartis Pharmaceuticals
- Email: novartis.email@novartis.com
- Phone: 1-888-669-6682
How to participate
- Review the eligibility criteria above with your treating physician.
- Visit the official trial page on ClinicalTrials.gov for the most current contact information and recruitment status.
- Contact the listed study coordinator or principal investigator to request pre-screening. Pre-screening is free and never obligates you to enroll.