Table 5.
Name | Mechanism | Trial phase, type, NTC# | Primary end points | Status | Results and comments |
---|---|---|---|---|---|
Unfractionated heparin* | Inhibits P-selectin in addition to anticoagulant effect | 2, open label randomized, NCT02098993 | Time to discharge | Recruiting | HbSS, HbSC, or HbSβ0 with ACS |
Tinzaparin* | Inhibits P-selectin in addition to anticoagulant effect | 3, randomized to 2 different dosing levels, NCT02580773 | Time to ACS resolution | Recruiting | HbSS, HbSC, or HbSβ0 with ACS |
Rivaroxiban | Direct oral factor Xa inhibitor | 2, NCT02072668 | VCAM1 and IL-6 levels | Enrolling patients by invitation | |
Apixiban | Direct oral factor Xa inhibitor | 3, RDBPC, NCT02179177 | Daily outpatient pain score | Completed | HbSS, HbSC, or HbSβ0 |
Ticagrelor | P2Y12 receptor antagonist | 2 (adults), RDBPC, NCT02482298 | Proportion of pain days (e-diary) | Completed | |
2 (pediatrics), NCT02214121 | PKs, PDs, safety | Completed | Age 2-18 y; safe and well tolerated69 | ||
1 (infants), NCT03492931 | PKs | Recruiting | Infants and toddlers age 0-24 mo |
Unless otherwise specified, study included only adult patients with HbSS or HbSβ0.
PD, pharmacodynamic; RDBPC, randomized double-blind placebo control.
Study drug administered during acute VOC are marked with an asterix