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Clinical Trials in the USA / NCT06546670
Recruiting Phase 1/2

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

NCT06546670 · tracked via the Priya Life Science USA tracker
Sponsor
Novartis Pharmaceuticals
Phase
Phase 1/2
Started
2024-08-15
Last updated
2026-10-27

Condition(s) studied

Sickle Cell Disease

Investigational drug(s) / intervention(s)

ITU512Placebo

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

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.

Study summary

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).

Eligibility

Sex
ALL
Min age
12 Years
Max age
55 Years
Healthy volunteers
Accepted
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.

Primary outcome measure(s)

  • Part 1A, Part 1B, Part 1C: Incidence of AEs and SAEs — Up to approximately 60 days
    Number of participants with adverse events (AEs) and serious adverse events (SAEs), including changes in vital signs, electrocardiograms (ECGs) and laboratory values qualifying and reported as AEs.
  • Part 1A, Part 1B , Part 1C: Dose discontinued due to AE — Up to 30 days
    Number of participants with dose discontinuation due to AEs
  • Part 2A, Part 2B: Incidence of AEs and SAEs — Up to 5 months
    Number of participants with adverse events (AEs) and serious adverse events (SAEs), including changes in vital signs, electrocardiograms (ECGs) and laboratory values qualifying and reported as AEs.
  • Part 2A, Part 2B: Dose interruptions and reductions — Up to 4 months
    Number of participants with dose interruptions or reductions of ITU512
  • Part 2A, Part 2B: Dose intensity — Up to 4 months
    Dose intensity of ITU512 is computed as the ratio of actual cumulative dose received and actual duration of exposure
  • Part 2B: Fetal hemoglobin (HbF)% — Month 4
    Assessment of fetal hemoglobin expression by measuring fetal hemoglobin (HbF)% by high-performance liquid chromatography (HPLC) assay

Trial sites (14)

FacilityCityRegionStatus
University of Alabama Birmingham Birmingham Alabama Recruiting
Quotient Sciences Sea View Miami Florida Completed
Boston Childrens Hospital Boston Massachusetts Recruiting
Icahn School Of Med At Mount Sinai New York New York Recruiting
Levine Cancer Insitute Carolinas Healthcare System Charlotte North Carolina Recruiting
East Carolina University Greenville North Carolina Recruiting
Childrens Hospital Of Philadelphia Philadelphia Pennsylvania Recruiting
Lifespan Providence Rhode Island Recruiting
UT Health Science Center Houston Texas Recruiting
Novartis Investigative Site Ribeirão Preto São Paulo Recruiting
Novartis Investigative Site São Paulo São Paulo Recruiting
Novartis Investigative Site São Paulo Brazil Recruiting
Novartis Investigative Site Ankara Sihhiye Altindag Recruiting
Novartis Investigative Site Adana Yuregir Recruiting

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Other trials for the same condition

A Study Evaluating Gene Therapy With BB305 Lentiviral Vector in Sickle Cell Disease Active, not recruiting
Long-term Follow-up of Subjects with Sickle Cell Disease Treated with Ex Vivo Gene Therapy Enrolling by invitation
A Study to Investigate the Effect of Mitapivat on Transfusion Burden in Subjects With Sickle Cell Disease (SCD) Recruiting
A Study of Intravenous L-Citrulline in Patients Aged 6-21 Years Old With Sickle Cell Disease Presenting to Emergency Departments in Acute Vaso-Occlusive Crisis Starting soon
A Long-term Follow-up Study in Patients Who Received BEAM-101 Recruiting
Official registry record

This page summarises publicly available registry data for informational purposes — not medical advice. Eligibility is determined by each study team; patients should discuss participation with their clinician.

View NCT06546670 on ClinicalTrials.gov ↗ ← All trials in the USA