UAB - Center for Clinical and Translational Science

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

Purpose

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).

Condition

Sickle Cell Disease

Eligibility

Eligible Ages: Between 12 Years and 55 Years
Eligible Sex: All
Accepts Healthy Volunteers: Yes

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 Other protocol-defined inclusion/exclusion criteria may apply.

Study Design

Phase: Phase 1/Phase 2
Study Type: Interventional
Allocation: Randomized
Intervention Model: Crossover Assignment
Primary Purpose: Treatment
Masking: Double (Participant, Investigator)

Arm Groups

Arm	Description	Assigned Intervention
Experimental Part 1A	Part 1A in healthy participants	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.
Experimental Part 1B	Part 1B in healthy participants	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.
Experimental Part 1C	Part 1C in healthy participants	Drug: ITU512 ITU512 is an investigational, oral, low molecular weight (LMW) compound.
Experimental Part 2A	Part 2A in patients with sickle cell disease	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.
Experimental Part 2B	Part 2B in patients with sickle cell disease	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.
Experimental Part 2C	Optional extension in patients with sickle cell disease	Drug: ITU512 ITU512 is an investigational, oral, low molecular weight (LMW) compound.

Recruiting Locations

University of Alabama Birmingham
Birmingham, Alabama 35233

Contact:
Emily Burke
205-975-4020
eduke@uabmc.edu

More Details

Status: Recruiting
Sponsor: Novartis Pharmaceuticals

Study Contact

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

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).