Approval Probability 71%
TA Base Rate26%
Adjusted LOA41%
ML RiskLOW_RISK
| NCT ID | Title | Phase | Status | Enrollment | Velocity | Design | Start | Completion | Last Updated | Sites | Countries |
|---|---|---|---|---|---|---|---|---|---|---|---|
| NCT06546670 | A Phase I/II Study of ITU512 in Healthy Participants and Patients With Sickle Cell Disease | PHASE1 | RECRUITING | 161 | — | — | Aug 15, 2024 | Feb 19, 2030 | Jun 2, 2026 | 5 | United States |
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.
Number of participants with dose discontinuation due to AEs
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.
Number of participants with dose interruptions or reductions of ITU512
Dose intensity of ITU512 is computed as the ratio of actual cumulative dose received and actual duration of exposure
Assessment of fetal hemoglobin expression by measuring fetal hemoglobin (HbF)% by high-performance liquid chromatography (HPLC) assay
| Arm | Type | Description |
|---|---|---|
| Part 1A | EXPERIMENTAL | Part 1A in healthy participants |
| Part 1B | EXPERIMENTAL | Part 1B in healthy participants |
| Part 1C | EXPERIMENTAL | Part 1C in healthy participants |
| Part 2A | EXPERIMENTAL | Part 2A in patients with sickle cell disease |
| Part 2B | EXPERIMENTAL | Part 2B in patients with sickle cell disease |
| Part 2C | EXPERIMENTAL | Optional extension in patients with sickle cell disease |
| Name | Type | Description |
|---|---|---|
| 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. |
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...