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ITU512

Phase 1

Sickle Cell Disease | Small molecule | Hematology |Novartis AG|Last Updated: Jul 20, 2026

Success Probability

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Market & Valuation

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Trial Design

RandomizedDouble-BlindCONTROLLEDDMC
Total Trials1
Total Enrollment161

FDA Designations

No designations recorded

Clinical trial landscape

ITU512 · 1 trial · 1 indication

Phase 1 1
NCT06546670A Phase I/II Study of ITU512 in Healthy Participants and Patients With Sickle Cell DiseaseSickle Cell Disease
RECRUITING161 Analytics
PHASE1RECRUITING
A Phase I/II Study of ITU512 in Healthy Participants and Patients With Sickle Cell Disease
Sickle Cell DiseaseUnlock trial analytics

Study Endpoints

Primary Endpoints

Part 1A, Part 1B, Part 1C: Incidence of AEs and SAEs
Up to approximately 60 days

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.

Part 1A, Part 1B , Part 1C: Dose discontinued due to AE
Up to 30 days

Number of participants with dose discontinuation due to AEs

Part 2A, Part 2B: Incidence of AEs and SAEs
Up to 5 months

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.

Part 2A, Part 2B: Dose interruptions and reductions
Up to 4 months

Number of participants with dose interruptions or reductions of ITU512

Part 2A, Part 2B: Dose intensity
Up to 4 months

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

Part 2B: Fetal hemoglobin (HbF)%
Month 4

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

Secondary Endpoints

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)
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Study Design & Arms

AllocationRANDOMIZED
MaskingDOUBLE
ModelCROSSOVER
PurposeTREATMENT

Treatment Arms

ArmTypeDescription
Part 1AEXPERIMENTALPart 1A in healthy participants
Part 1BEXPERIMENTALPart 1B in healthy participants
Part 1CEXPERIMENTALPart 1C in healthy participants
Part 2AEXPERIMENTALPart 2A in patients with sickle cell disease
Part 2BEXPERIMENTALPart 2B in patients with sickle cell disease
Part 2CEXPERIMENTALOptional extension in patients with sickle cell disease

Interventions

NameTypeDescription
ITU512DRUGITU512 is an investigational, oral, low molecular weight (LMW) compound.
PlaceboDRUGAn 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.
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Eligibility Criteria

Age Range12 Years to 55 Years
SexALL
Healthy VolunteersYes
Study Sites9

Key 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 laborat...

Countries:United StatesTurkey (Türkiye)
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Recent Changes (Last 90 Days)

LOWJul 20, 2026NCT06546670lastUpdatePostDate: changed
LOWJul 20, 2026NCT06546670lastUpdatePostDate: changed
LOWJul 14, 2026NCT06546670lastUpdatePostDate: changed
LOWJul 14, 2026NCT06546670lastUpdatePostDate: changed
LOWJun 30, 2026NCT06546670lastUpdatePostDate: changed
LOWJun 30, 2026NCT06546670lastUpdatePostDate: changed
LOWJun 30, 2026NCT06546670lastUpdatePostDate: changed

Frequently asked questions about ITU512

What is ITU512 used for in Sickle Cell Disease?

ITU512 is an investigational small molecule being developed for the treatment of Sickle Cell Disease. It is currently in Phase 1 clinical development, with a Phase I/II study evaluating its safety and efficacy in healthy participants and patients with Sickle Cell Disease. The drug is not yet approved and remains under investigation.

Who makes ITU512?

ITU512 is being developed by Novartis AG, a multinational pharmaceutical company traded on the New York Stock Exchange under the ticker symbol NVS. The company is conducting a Phase I/II clinical trial of ITU512 in healthy participants and patients with Sickle Cell Disease.

What phase is ITU512 in?

ITU512 is in Phase 1 clinical development. The ongoing study, NCT06546670, is a Phase I/II trial that is currently recruiting participants. The drug is investigational and has not been approved by regulatory authorities for the treatment of Sickle Cell Disease.

What clinical trials is ITU512 in?

ITU512 is being studied in a single active clinical trial, NCT06546670, titled "A Phase I/II Study of ITU512 in Healthy Participants and Patients With Sickle Cell Disease." This randomized, double-blind, controlled trial is recruiting 161 participants in the United States and Turkey, and includes healthy volunteers.

Is ITU512 the same as any other drug?

No alternative names for ITU512 have been reported. The drug is identified solely by its code name ITU512 in clinical trial registrations and is being developed by Novartis AG for Sickle Cell Disease.