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RECRUITINGPhase 1 / Phase 2INTERVENTIONAL

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

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

Important: This information is not medical advice. Talk to your doctor about whether a clinical trial is right for you.

About This Trial

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 May Be Eligible (Plain English)

Key Who May Qualify: 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 Who Should NOT Join This Trial: 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. Always talk to your doctor about whether this trial is right for you.

Original Eligibility Criteria

View original clinical language
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.

Treatments Being Tested

DRUG

ITU512

ITU512 is an investigational, oral, low molecular weight (LMW) compound.

DRUG

Placebo

An inactive substance that looks like and is given the same way as ITU512. The effect(s) of ITU512 will be evaluated against the placebo. Placebos are designed as a control and to have no real effect.

Locations (1)

Quotient Sciences Sea View
Miami, Florida, United States