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NCT06546670PHASE1, PHASE2RecruitingAccepts Healthy Volunteers

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

Novartis Pharmaceuticals

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

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.

Interventions

DRUG

ITU512

DRUG

Placebo

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Conditions

Sickle Cell Disease

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)