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

Study Results

Results pending

The study team has not published outcome measurements, participant flow, or safety data for this trial yet. Check back later for updates.

Basic Information

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Recruitment Status

RECRUITING

Clinical Phase

PHASE1/PHASE2

Total Enrollment

161 participants

Study Classification

INTERVENTIONAL

Study Start Date

2024-08-15

Study Completion Date

2029-11-12

Brief Summary

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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).

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Detailed Description

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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

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Sickle Cell Disease

Study Design

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Allocation Method

RANDOMIZED

Intervention Model

CROSSOVER

Primary Study Purpose

TREATMENT

Blinding Strategy

DOUBLE

Participants Investigators

Study Groups

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Part 1A

Part 1A in healthy participants

Group Type EXPERIMENTAL

ITU512

Intervention Type DRUG

ITU512 is an investigational, oral, low molecular weight (LMW) compound.

Placebo

Intervention Type DRUG

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.

Part 1B

Part 1B in healthy participants

Group Type EXPERIMENTAL

ITU512

Intervention Type DRUG

ITU512 is an investigational, oral, low molecular weight (LMW) compound.

Placebo

Intervention Type DRUG

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.

Part 1C

Part 1C in healthy participants

Group Type EXPERIMENTAL

ITU512

Intervention Type DRUG

ITU512 is an investigational, oral, low molecular weight (LMW) compound.

Part 2A

Part 2A in patients with sickle cell disease

Group Type EXPERIMENTAL

ITU512

Intervention Type DRUG

ITU512 is an investigational, oral, low molecular weight (LMW) compound.

Placebo

Intervention Type DRUG

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.

Part 2B

Part 2B in patients with sickle cell disease

Group Type EXPERIMENTAL

ITU512

Intervention Type DRUG

ITU512 is an investigational, oral, low molecular weight (LMW) compound.

Placebo

Intervention Type DRUG

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.

Part 2C

Optional extension in patients with sickle cell disease

Group Type EXPERIMENTAL

ITU512

Intervention Type DRUG

ITU512 is an investigational, oral, low molecular weight (LMW) compound.

Interventions

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ITU512

ITU512 is an investigational, oral, low molecular weight (LMW) compound.

Intervention Type 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.

Intervention Type DRUG

Eligibility Criteria

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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

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

Minimum Eligible Age

12 Years

Maximum Eligible Age

55 Years

Eligible Sex

ALL

Accepts Healthy Volunteers

Yes