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. 2018 Nov 30;2018(1):493–506. doi: 10.1182/asheducation-2018.1.493

Table 3.

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

Unless otherwise specified, study included only patients with HbSS or HbSβ0.

AE, adverse event.