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

Phases

Phase 1

Study type

Interventional

Source

PACTR

Registry ID

PACTR202508611698393

Enrollment

161

Registered

2025-08-01

Start date

2024-08-15

Completion date

Unknown

Last updated

2025-07-18

For informational purposes only — not medical advice. Sourced from public registries and may not reflect the latest updates. Terms

Conditions

Haematological Disorders

Interventions

None listed

Eligibility

Sex/Gender

All

Inclusion criteria

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

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

Design outcomes

Primary

Measure	Time frame
Part 1A, Part 1B, Part 1C: Incidence of AEs and SAEs;Part 1A, Part 1B , Part 1C: Dose discontinued due to AE;Part 2: Incidence of AEs and SAEs;Part 2: Dose interruptions and reductions;Part 2: Dose intensity;Part 2: HbF% evaluated from central laboratory assessments at Month 4	—

Secondary

Measure	Time frame
Part 2: Change from baseline in total hemoglobin (Hb);Part 1A, Part 1B, Part 2: Change from baseline in Fridericia-corrected Holter QT interval (QTcF);Part 1C: Area under the plasma concentration-time curve from time 0 up to the time of the last quantifiable concentration (AUClast) of ITU512;Part 1C: Area under the plasma concentration-time curve from time 0 up to infinity (AUCinf) of ITU512;Part 1C: Maximum plasma concentration (Cmax) of ITU512;Part 1C: Time to maximum plasma concentration (Tmax) of ITU512;Part 1A, Part 1B: Area under the plasma concentration-time curve (AUC) of ITU512;Part 1A, Part 1B: Maximum plasma concentration (Cmax) of ITU512;Part 1A, Part 1B: Time to maximum plasma concentration (Tmax) of ITU512;Part 1A, Part 1B: Renal clearance (CLr);Part 2: Plasma concentrations of ITU512;Part 2: Urine concentrations of ITU512;Part 2: Fetal hemoglobin (HbF)%	—

Measure

Time frame

Part 2: Change from baseline in total hemoglobin (Hb);Part 1A, Part 1B, Part 2: Change from baseline in Fridericia-corrected Holter QT interval (QTcF);Part 1C: Area under the plasma concentration-time curve from time 0 up to the time of the last quantifiable concentration (AUClast) of ITU512;Part 1C: Area under the plasma concentration-time curve from time 0 up to infinity (AUCinf) of ITU512;Part 1C: Maximum plasma concentration (Cmax) of ITU512;Part 1C: Time to maximum plasma concentration (Tmax) of ITU512;Part 1A, Part 1B: Area under the plasma concentration-time curve (AUC) of ITU512;Part 1A, Part 1B: Maximum plasma concentration (Cmax) of ITU512;Part 1A, Part 1B: Time to maximum plasma concentration (Tmax) of ITU512;Part 1A, Part 1B: Renal clearance (CLr);Part 2: Plasma concentrations of ITU512;Part 2: Urine concentrations of ITU512;Part 2: Fetal hemoglobin (HbF)%

—

Countries

Ghana, Kenya, Uganda

Contacts

Public ContactNovartis Pharmaceuticals Novartis Pharmaceuticals

Trial Director

novartis.email@novartis.com+41613241111

Outcome results