NCT06546670

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

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

• Status: Recruiting
• Phase: Phase 1 / Phase 2
• Study type: Interventional
• Enrollment: 161 (estimated)
• Sponsor: Novartis Pharmaceuticals · Industry
• Sex: All
• Age: 12 Years – 55 Years
• Healthy volunteers: Accepted

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).

Conditions

• Sickle Cell Disease

Interventions

Type	Name	Description
DRUG	ITU512	ITU512 is an investigational, oral, low molecular weight (LMW) compound.
DRUG	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.

Timeline

• Start date: 2024-08-15
• Primary completion: 2028-05-01
• Completion: 2029-11-12
• First posted: 2024-08-09
• Last updated: 2026-01-29

Locations

1 site across 1 country: United States

Regulatory

• FDA-regulated drug study