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

Novartis

Status and phase: Enrolling; Phase 2; Phase 1

Conditions: Sickle Cell Disease

Treatments: Drug: Placebo; Drug: ITU512

Study type: Interventional

Funder types: Industry

Identifiers

NCT06546670

CITU512A12101

2024-515696-35-00 (Registry Identifier)

Details and patient eligibility

About

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).

Full 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).

Enrollment: 161 estimated patients

Sex: All

Ages: 12 to 55 years old

Volunteers: Accepts Healthy Volunteers

Inclusion and exclusion criteria

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.

Trial design

Primary purpose: Treatment

Allocation: Randomized

Interventional model: Crossover Assignment

Masking: Double Blind

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161 participants in 6 patient groups

Part 1A

Experimental group

Description:

Part 1A in healthy participants

Treatment:

Drug: ITU512

Drug: Placebo

Part 1B

Experimental group

Description:

Part 1B in healthy participants

Treatment:

Drug: ITU512

Drug: Placebo

Part 1C

Experimental group

Description:

Part 1C in healthy participants

Treatment:

Drug: ITU512

Part 2A

Experimental group

Description:

Part 2A in patients with sickle cell disease

Treatment:

Drug: ITU512

Drug: Placebo

Part 2B

Experimental group

Description:

Part 2B in patients with sickle cell disease

Treatment:

Drug: ITU512

Drug: Placebo

Part 2C

Experimental group

Description:

Optional extension in patients with sickle cell disease

Treatment:

Drug: ITU512