What is ITU512 studying for sickle cell disease?

ITU512 is being studied to evaluate its safety, how it is processed by the body, and its potential to increase fetal hemoglobin levels in people with sickle cell disease. Researchers aim to learn if ITU512 may offer a new treatment approach. The study is sponsored by Novartis Pharmaceuticals and includes both healthy participants and patients with sickle cell disease.

Who may be eligible for this sickle cell disease clinical trial?

Participants aged 18 to 55 years may be eligible if they are healthy adults or have sickle cell disease. Eligible participants must be male or females unable to have children and meet health criteria including body weight and heart health. People using hydroxyurea or hydroxycarbamide are not eligible. Please review eligibility criteria for more details.

Is there any cost to participate in this sickle cell disease clinical trial?

Study-related care, including the study drug or procedure and all trial visits, is provided at no cost. However, personal travel and related expenses may vary and are not guaranteed to be covered by the study. Participants should plan accordingly for any travel or accommodation costs.

Actively Recruiting

Phase 1

Phase 2

Age: 12Years - 55Years

All Genders

Healthy Volunteers

ID06546670

Phase III Study of ITU512 to Assess Safety and Effects in Healthy Adults and Sickle Cell Disease Patients

Led by Novartis Pharmaceuticals · Updated on 2026-07-20

161

Participants Needed

Research Sites

94 weeks

Total Duration

AI-Summary

What this Trial Is About

Researchers are evaluating ITU512, an investigational oral drug, in healthy adults and patients with sickle cell disease SCD to study its safety, tolerability, how it is processed by the body pharmacokinetics, and its potential to increase fetal hemoglobin levels. This Phase III study is the first to assess ITU512s possible therapeutic effects in these groups, aiming to better understand its impact on SCD. The study is divided into two parts Part 1, a first-in-human Phase I trial involving healthy participants, and Part 2, a Phase II trial including patients with SCD. Part 1 includes three subparts 1A, 1B, 1C to examine safety, dosage, and food effects in healthy adults. Part 2 has two subparts 2A, 2B plus an optional extension 2C focusing on safety, tolerability, pharmacokinetics, pharmacodynamics, and efficacy in adolescents and adults with SCD. Participants may receive ITU512 or placebo in a randomized, double-blind crossover design. During the study, participants undergo various assessments including medical exams, vital signs, ECGs, and blood and urine tests to monitor drug levels and effects on fetal hemoglobin and other blood parameters. Safety is closely tracked with attention to adverse events and dose adjustments. The study includes up to 5 months of active treatment and follow-up, with detailed monitoring of outcomes such as adverse events, dose interruptions, and fetal hemoglobin percentage. Participant involvement includes scheduled visits for dosing and evaluations throughout the study timeline.

CONDITIONS

Brief Title

A Phase I/II Study of ITU512 in Healthy Participants and Patients With Sickle Cell Disease

Who Can Participate

Age: 12Years - 55Years

All Genders

Healthy Volunteers

Eligibility Criteria

You may qualify if you...

Healthy male participants and female participants of non-childbearing potential between 18-55 years of age
In good health based on medical history, physical exam, vital signs, ECG, and lab tests
Weigh at least 50 kg and have a body mass index between 18.0 and 32.0 kg/m2
Male and female participants with a diagnosis of sickle cell disease

You will not qualify if you...

QTcF interval of 450 msec or higher (average of three readings) in healthy participants and patients
History of arrhythmias
Significant illness unresolved within two weeks before initial dosing in healthy participants
Women who can become pregnant (women of child-bearing potential) in healthy participants
Current use of hydroxyurea or hydroxycarbamide in patients with sickle cell disease
Other protocol-defined exclusion criteria may apply

Research Team

Novartis Pharmaceuticals

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