Recruiting
Phase 1
Phase 2

ITU512

Sponsor:

Novartis Pharmaceuticals

Code:

NCT06546670

Conditions

Sickle Cell Disease

Eligibility Criteria

Sex: All

Age: 12 - 55

Healthy Volunteers: Accepted

Interventions

ITU512

Placebo

Study Details

Brief 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).

Conditions

Sickle Cell Disease

Study ID

NCT06546670

Start date

Aug 15, 2024

Status verified date

Jul, 2026

Completion date

Feb 19, 2030

Anticipated

Primary completion date

May 1, 2028

Anticipated

Eligibility Criteria

Eligibility Criteria

Sex: All

Age: 12 - 55

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.

Study Design

Enrollment

161 participants

Anticipated

Allocation

Randomized

Intervention Model

Crossover

Primary purpose

Treatment

Interventions and Outcome Measures

Arms

experimental: Part 1A

Part 1A in healthy participants

experimental: Part 1B

Part 1B in healthy participants

experimental: Part 1C

Part 1C in healthy participants

experimental: Part 2A

Part 2A in patients with sickle cell disease

experimental: Part 2B

Part 2B in patients with sickle cell disease

experimental: Part 2C

Optional extension in patients with sickle cell disease

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.

Primary outcome measure

  • Part 1A, Part 1B, Part 1C: Incidence of AEs and SAEs [ Time Frame: Up to approximately 60 days ]
  • Part 1A, Part 1B , Part 1C: Dose discontinued due to AE [ Time Frame: Up to 30 days ]
  • Part 2A, Part 2B: Incidence of AEs and SAEs [ Time Frame: Up to 5 months ]
  • Part 2A, Part 2B: Dose interruptions and reductions [ Time Frame: Up to 4 months ]
  • Part 2A, Part 2B: Dose intensity [ Time Frame: Up to 4 months ]
  • Part 2B: Fetal hemoglobin (HbF)% [ Time Frame: Month 4 ]

Central Contacts and Locations

Central contacts

Novartis Pharmaceuticals

1-888-669-6682novartis.email@novartis.com

Novartis Pharmaceuticals

+41613241111

Locations

University of Alabama Birmingham

Recruiting

Birmingham, Alabama, United States, 35233

Contacts

Emily Burke

+1 205 801 3411eduke@uabmc.edu

Principal Investigator:

Julie Kanter-Washko

Boston Childrens Hospital

Recruiting

Boston, Massachusetts, United States, 02215

Contacts

Nadia Tarhini

+1 617 355 4278nadia.tarhini@childrens.harvard.edu

Principal Investigator:

Matthew M Heeney

East Carolina University

Recruiting

Greenville, North Carolina, United States, 27858

Contacts

Kelly Martin

martinke21@ecu.edu

Principal Investigator:

Darla Liles

Childrens Hospital Of Philadelphia

Recruiting

Philadelphia, Pennsylvania, United States, 19104

Contacts

Kevin Dougherty

+1 215 590 9959doughertk8@chop.edu

Principal Investigator:

Alexis A Thompson

Lifespan

Recruiting

Providence, Rhode Island, United States, 02903

Contacts

Asher Dillman

+1 401 444 5328adillmann@brownhealth.org

Principal Investigator:

Patrick Mc Gann

UT Health Science Center

Recruiting

Houston, Texas, United States, 77030

Contacts

Eyuel Baherey

Eyuel.L.Baherey@uth.tmc.edu

Principal Investigator:

Modupe Idowu

More Information

Sponsor

Novartis Pharmaceuticals

Last update posted

Jul 20, 2026

Last verified

Jul, 2026

Keywords

  • ITU512
  • Low molecular weight
  • Small molecule
  • Sickle cell disease

Trial information was received from ClinicalTrials.gov and was last updated on 2026-09-08. This information was provided to ClinicalTrials.gov by Novartis Pharmaceuticals on 2026-07-20.