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 (10)
Facility
City
Region
Status
University of Alabama Birmingham
Birmingham
Alabama
Recruiting
Quotient Sciences Sea View
Miami
Florida
Completed
Boston Childrens Hospital
Boston
Massachusetts
Recruiting
Levine Cancer Insitute Carolinas Healthcare System
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.
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