Last updated 11 days ago
A Phase I/II Study of ITU512 in Healthy Participants and Patients With Sickle Cell Disease
161 patients around the world
Available in Brazil, United States
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).
Novartis Pharmaceuticals
161Patients around the world
This study is for people with
Rare diseases
Sickle cell disease
Requirements for the patient
To 55 Years
All Gender
Medical requirements
- 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.
- 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.
SponsorNovartis Pharmaceuticals
Study typeInterventional
Conditions
Sickle cell disease
Requirements
To 55 YearsAll Gender
Unique study IDclinicaltrials.gov
NCT06546670
