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RecruitingPhase 1 / Phase 2

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

Who can join

As published by the study on ClinicalTrials.gov. "Not stated" means the study hasn't said — ask the study team.

Age
12 Years to 55 Years
Sex
All sexes
Healthy volunteers
Accepted
Compensation
Not stated

Other requirements are listed under Eligibility below. Only the study team can confirm whether you can join.

What to expect

No one can promise a trial will help you personally — that is what the research is trying to find out.

What participation involves

Not stated in the study description — ask the study team about visits and procedures.

Compensation
Not stated
  • Study-related exams and procedures are typically provided at no cost. This is how many trials work in general — confirm the details for this study with the study team.
  • Participants are monitored closely by the research team throughout the study.
  • Some participants may receive access to an investigational treatment, which is still being studied.
  • Your participation helps researchers learn whether this approach could help future patients.

About this study

In plain language

This study tests ITU512 in healthy adults and people with sickle cell disease. It checks safety, how the body handles the drug, and how food affects it. It also checks whether ITU512 raises fetal hemoglobin, a protein in red blood cells made before birth that carries oxygen.

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).

Compensation is read from the study's own description and may be missing — always confirm with the study team.

Eligibility

Who can join

  • Part 1: Healthy men and women ages 18–55. Women must not be able to become pregnant.
  • Part 1: A study doctor must find you in good health based on your health history, exam, heart test, and lab tests.
  • Part 1: You must weigh at least 50 kg (about 110 pounds) at screening and the first admission visit.
  • Part 1: Your body mass index (BMI), a measure based on height and weight, must be 18–32.
  • Part 2: Men and women with sickle cell disease may join.
  • Other study rules may apply.

Who can't join

  • Both parts: A heart test shows a QTcF of 450 milliseconds or more, averaged over 3 readings. This measures how long the heart takes to reset between beats.
  • Both parts: A history of an uneven heartbeat.
  • Part 1: A significant illness that has not cleared up at least 2 weeks before the first dose.
  • Part 1: Women who can become pregnant.
  • Part 2: Currently taking hydroxyurea, also called hydroxycarbamide.
  • Other study rules may apply.

Simplified for readability. The official criteria below are what the study team uses.

Official eligibility 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.

Study sites (9)

AL

FL

MA

NC

NY

PA

RI

TX