Actively Recruiting
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
Led by Novartis Pharmaceuticals · Updated on 2026-06-02
161
Participants Needed
5
Research Sites
94 weeks
Total Duration
On this page
AI-Summary
What this Trial Is About
Researchers are studying ITU512, an oral investigational drug, to evaluate its safety, tolerability, how it is processed in the body, and its ability to increase fetal hemoglobin (HbF) levels. This study includes both healthy adults and adolescents or adults with sickle cell disease (SCD). It is a global, randomized Phase I/II trial and represents the first time ITU512 is tested for potential therapeutic effects in these groups. The study is divided into two main parts: Part 1 involves healthy participants and consists of three subparts (1A, 1B, and 1C), focusing on safety, tolerability, pharmacokinetics, and preliminary food effects of ITU512. Part 2 is conducted in patients with sickle cell disease and includes Parts 2A and 2B, assessing safety, tolerability, pharmacokinetics, pharmacodynamics, and efficacy of ITU512, with an optional extension (Part 2C) for further evaluation. Participants will undergo various assessments such as monitoring adverse events, dose adjustments, and measuring fetal hemoglobin percentages up to several months. Blood and urine samples will be collected to study drug concentration and effects on hemoglobin. Safety will be closely monitored through ECGs, vital signs, laboratory tests, and physical exams. The total participation time varies by study part, with some assessments lasting up to five months.
CONDITIONS
Brief Title
A Phase I/II Study of ITU512 in Healthy Participants and Patients With Sickle Cell Disease
Who Can Participate
Eligibility Criteria
You may qualify if you...
- Healthy male participants and female participants of non-childbearing potential between 18 and 55 years of age
- Good health confirmed by medical history, physical exam, vital signs, ECG, and lab tests
- Body weight of at least 50 kg and body mass index (BMI) between 18.0 and 32.0 kg/m2 inclusive
- Male and female participants diagnosed with sickle cell disease
You will not qualify if you...
- QTcF interval of 450 milliseconds or more (mean of triplicates)
- History of arrhythmias or heart rhythm problems
- Significant illness not resolved within two weeks before dosing
- Women who are of child-bearing potential
- Current use of hydroxyurea or hydroxycarbamide in sickle cell disease patients
AI-Screening
AI-Powered Screening
Complete this quick 3-step screening to check your eligibility
Your Study Journey
Duration - 2 to 4 weeks
Participants are screened for eligibility to participate in the trial.
1 visit (in-person)
Duration - Up to approximately 5 months depending on the study part
Participants receive the investigational drug ITU512 or placebo to assess safety, tolerability, pharmacokinetics, pharmacodynamics, and efficacy.
Multiple visits during treatment depending on study part
Duration - Up to 60 days for healthy participants and up to 5 months for patients with sickle cell disease
Participants are monitored for adverse events and other safety outcomes after treatment ends.
Visits scheduled according to study protocol for safety monitoring
Trial Site Locations
Total: 5 locations
1
University of Alabama Birmingham
Birmingham, Alabama, United States, 35233
Actively Recruiting
2
Quotient Sciences Sea View
Miami, Florida, United States, 33126
Completed
3
Boston Childrens Hospital
Boston, Massachusetts, United States, 02215
Actively Recruiting
4
Lifespan
Providence, Rhode Island, United States, 02903
Actively Recruiting
5
UT Health Science Center
Houston, Texas, United States, 77030
Actively Recruiting
Research Team
N
Novartis Pharmaceuticals
N
Novartis Pharmaceuticals
How is the study designed?
Study Type
INTERVENTIONAL
Masking
DOUBLE
Allocation
RANDOMIZED
Model
CROSSOVER
Primary Purpose
TREATMENT
Number of Arms
6
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