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

NCT06546670 | Recruiting Phase 1 DMC FDA Drug

• 2 other identifiers: CITU512A121012024-515696-35-00
• Study Type: interventional
• Target: 161
• Locations: 1 country
• Sites: 1

Brief Summary

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).

Conditions

Sickle Cell Disease

Keywords

ITU512; Low molecular weight; Small molecule; Sickle cell disease

Outcome Measures

Primary Outcomes (6)

• 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.

  Up to approximately 60 days

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

  Number of participants with dose discontinuation due to AEs

  Up to 30 days

• 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.

  Up to 5 months

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

  Number of participants with dose interruptions or reductions of ITU512

  Up to 4 months

• Part 2A, Part 2B: Dose intensity

  Dose intensity of ITU512 is computed as the ratio of actual cumulative dose received and actual duration of exposure

  Up to 4 months

• Part 2B: Fetal hemoglobin (HbF)%

  Assessment of fetal hemoglobin expression by measuring fetal hemoglobin (HbF)% by high-performance liquid chromatography (HPLC) assay

  Month 4

Secondary Outcomes (13)

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

  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)

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

  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)

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

  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)

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

  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)

• Part 2A, Part 2B: Plasma concentrations of ITU512

  From pre-dose up to 4, 6 or 8 hours post-dose on Day 1 at Month 1 and Month 2

Study Arms (6)

Part 1A

EXPERIMENTAL

Part 1A in healthy participants

Drug: ITU512; Drug: Placebo

Part 1B

EXPERIMENTAL

Part 1B in healthy participants

Drug: ITU512; Drug: Placebo

Part 1C

EXPERIMENTAL

Part 1C in healthy participants

Drug: ITU512

Part 2A

EXPERIMENTAL

Part 2A in patients with sickle cell disease

Drug: ITU512; Drug: Placebo

Part 2B

EXPERIMENTAL

Part 2B in patients with sickle cell disease

Drug: ITU512; Drug: Placebo

Part 2C

EXPERIMENTAL

Optional extension in patients with sickle cell disease

Drug: ITU512

Interventions

ITU512 DRUG

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

Part 1A; Part 1B; Part 1C; Part 2A; Part 2B; Part 2C

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.

Part 1A; Part 1B; Part 2A; Part 2B

Eligibility Criteria

• Age: 12 Years - 55 Years
• Sex: all
• Healthy Volunteers: Yes
• Age Groups: Child (0-17), Adult (18-64)

You may qualify if:

• 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

You may not qualify if:

• 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

Sponsors & Collaborators

• Novartis Pharmaceuticals: lead

Study Sites (1)

Quotient Sciences Sea View

Miami, Florida, 33126, United States

RECRUITING

MeSH Terms

Conditions

Anemia, Sickle Cell

Condition Hierarchy (Ancestors)

Anemia, Hemolytic, Congenital; Anemia, Hemolytic; Anemia; Hematologic Diseases; Hemic and Lymphatic Diseases; Hemoglobinopathies; Genetic Diseases, Inborn; Congenital, Hereditary, and Neonatal Diseases and Abnormalities

Central Study Contacts

Novartis Pharmaceuticals

CONTACT

1-888-669-6682; novartis.email@novartis.com

Novartis Pharmaceuticals

CONTACT

+41613241111

Study Design

• Study Type: interventional
• Phase: phase 1
• Allocation: RANDOMIZED
• Masking: DOUBLE
• Who Masked: PARTICIPANT, INVESTIGATOR
• Purpose: TREATMENT
• Intervention Model: CROSSOVER
• Sponsor Type: INDUSTRY
• Responsible Party: SPONSOR

Study Record Dates

• First Submitted: July 16, 2024
• First Posted: August 9, 2024
• Study Start: August 15, 2024
• Primary Completion (Estimated): May 1, 2028
• Study Completion (Estimated): November 12, 2029
• Last Updated: January 29, 2026

Record last verified: 2026-01

Data Sharing

• IPD Sharing: Will share

Locations

US (1)