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A Phase I/II Study of ITU512 in Healthy Participants and Patients With Sickle Cell Disease
Sponsor: Novartis Pharmaceuticals
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).
Official title: 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
Key Details
Gender
All
Age Range
12 Years - 55 Years
Study Type
INTERVENTIONAL
Enrollment
161
Start Date
2024-08-15
Completion Date
2029-11-12
Last Updated
2026-01-29
Healthy Volunteers
Yes
Conditions
Interventions
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.
Locations (1)
Quotient Sciences Sea View
Miami, Florida, United States