A Phase I/II Study of ITU512 in Healthy Participants and Patients With Sickle Cell Disease
Start Date
8/15/2024
Completion Date
2/19/2030
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).
Detailed Description
This is a global, randomized, Phase I/II study to assess the safety, tolerability, pharmacokinetics (PK), pharmacodynamics (PD), and preliminary food effect of single-agent ITU512 in adult healthy participants, and safety, tolerability, PK, PD, and efficacy of ITU512 in adolescent and adult patients with sickle cell disease (SCD). The study consists of a first-in-human Phase I study (Part 1) in healthy participants, and a Phase II study (Part 2) in patients with SCD. Part 1 will comprise of Part 1A, Part 1B, and Part 1C. Part 2 will include Part 2A and 2B and may also include an extension part (Part 2C).
Eligibility Criteria
Age Range: 12 years to 55 years
Interventions
ITU512
Placebo
Conditions
Locations
University of Alabama Birmingham
Birmingham, Alabama 35233
United States
Quotient Sciences Sea View
Miami, Florida 33126
United States
Boston Childrens Hospital
Boston, Massachusetts 02215
United States
East Carolina University
Greenville, North Carolina 27858
United States
Childrens Hospital Of Philadelphia
Philadelphia, Pennsylvania 19104
United States
Lifespan
Providence, Rhode Island 02903
United States
UT Health Science Center
Houston, Texas 77030
United States
Novartis Investigative Site
Ankara, Sihhiye-Altindag 06230
Turkey (Türkiye)
Novartis Investigative Site
Adana, Yuregir 01250
Turkey (Türkiye)