Novel agents inducing HbF
| Name . | Mechanism . | Trial phase, type, NTC# . | Primary end points . | Status . | Results and comments . |
|---|---|---|---|---|---|
| Decitabine + THU | DNA methyltransferase1 inhibitor | 1, single blinded, NCT01685515 | AEs grade ≥3 | Completed | All SCD genotypes |
| Well tolerated; HbF 4%-9% increase and doubling of F cells in higher-dose cohorts; increase in platelet counts noted54 | |||||
| Metformin | FOXO3 inducer | 1, open label, NCT02981329 | Change in HbF % or Hb | Recruiting | Age 12-40 y |
| INCB059872 | LSD1 inhibitor | 1, open label, NCT03132324 | Safety and tolerability, HbF levels | Recruiting |
| Name . | Mechanism . | Trial phase, type, NTC# . | Primary end points . | Status . | Results and comments . |
|---|---|---|---|---|---|
| Decitabine + THU | DNA methyltransferase1 inhibitor | 1, single blinded, NCT01685515 | AEs grade ≥3 | Completed | All SCD genotypes |
| Well tolerated; HbF 4%-9% increase and doubling of F cells in higher-dose cohorts; increase in platelet counts noted54 | |||||
| Metformin | FOXO3 inducer | 1, open label, NCT02981329 | Change in HbF % or Hb | Recruiting | Age 12-40 y |
| INCB059872 | LSD1 inhibitor | 1, open label, NCT03132324 | Safety and tolerability, HbF levels | Recruiting |
Unless otherwise specified, study included only patients with HbSS or HbSβ0.
AE, adverse event.