Health TrialsbyHealthPanel

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

ConditionSickle Cell Disease
Trial statusRecruiting
Trial phasePhase 1, Phase 2
Trial typeInterventional
Biological sexAll
Age12-55
SponsorNovartis Pharmaceuticals

About this trial

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).

Eligibility criteria

This trial accepts healthy volunteers

Qualifiers

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.

Disqualifiers

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)

Trial design

Design model

Crossover

Treatments tested in this trial

  • ITU512

    Drug

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

  • Placebo

    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.

Treatment groups

161 Participants
are divided into 6 treatment groups

6

Treatment groups

See each treatment group below.

Group A: Part 1AExperimental treatment 2 interventions
Group B: Part 1BExperimental treatment 2 interventions
Group C: Part 1CExperimental treatment 1 intervention
Group D: Part 2AExperimental treatment 2 interventions
Group E: Part 2BExperimental treatment 2 interventions
Group F: Part 2CExperimental treatment 1 intervention

Trial outcomes

Primary outcomes

1

Part 1A, Part 1B, Part 1C: Incidence of AEs and SAEs

Number of participants with adverse events (AEs) and serious adverse events (SAEs), including changes in vital signs, electrocardiograms (ECGs) and laboratory values qualifying and reported as AEs.

Time frame
Up to approximately 60 days
2

Part 1A, Part 1B , Part 1C: Dose discontinued due to AE

Number of participants with dose discontinuation due to AEs

Time frame
Up to 30 days
3

Part 2A, Part 2B: Incidence of AEs and SAEs

Number of participants with adverse events (AEs) and serious adverse events (SAEs), including changes in vital signs, electrocardiograms (ECGs) and laboratory values qualifying and reported as AEs.

Time frame
Up to 5 months
4

Part 2A, Part 2B: Dose interruptions and reductions

Number of participants with dose interruptions or reductions of ITU512

Time frame
Up to 4 months

Secondary outcomes

1

Part 1A, Part 1B: Area under the plasma concentration-time curve (AUC) of ITU512

Pharmacokinetic (PK) parameters calculated based on ITU512 plasma concentrations by non-compartmental methods

Time frame
From pre-dose up to 144 hours post-dose on Day 1 (Part 1A) and from pre-dose up to 24 hours post-dose on Day 1 and Day 10 (Part 1B)
2

Part 1A, Part 1B: Maximum plasma concentration (Cmax) of ITU512

PK parameters calculated based on ITU512 plasma concentrations by non-compartmental methods

Time frame
From pre-dose up to 144 hours post-dose on Day 1 (Part 1A) and from pre-dose up to 24 hours post-dose on Day 1 and Day 10 (Part 1B)
3

Part 1A, Part 1B: Time to maximum plasma concentration (Tmax) of ITU512

PK parameters calculated based on ITU512 plasma concentrations by non-compartmental methods

Time frame
From pre-dose up to 144 hours post-dose on Day 1 (Part 1A) and from pre-dose up to 24 hours post-dose on Day 1 and Day 10 (Part 1B)
4

Part 1A, Part 1B: Renal clearance (CLr)

PK parameters calculated based on ITU512 urine concentrations by non-compartmental methods. The renal clearance (CLr) may be determined based on AUC and amount of drug excreted into urine (Ae) available for the same time period.

Time frame
From pre-dose up to 48 hours post-dose on Day 1 (Part 1A) and from pre-dose up to 24 hours post-dose on Day 1 and Day 10 (Part 1B)

Other outcomes

Sponsors and contacts

Click on the lead sponsor to view all of their trials.

Novartis Pharmaceuticals

Lead sponsor

Source ClinicalTrials.gov
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