Recent trials of DOACS in children
| Study . | Design . | Indications . | Population . | Interventions . | Status . |
|---|---|---|---|---|---|
| VTE treatment | |||||
| EINSTEIN Jr | Phase 3, noninferiority, open label, RCT | Acute VTE treatment and secondary prevention | Children ≤17 y with acute VTE N=500 | Rivaroxaban (n=335) vs SoC (n=165) Treatment period: 3 mo (1 mo for children <2 y with CVC-associated VTE) | Published |
| DIVERSITY | Phase 2b/3 noninferiority, open label, RCT | Acute VTE treatment and secondary prevention | Children <18 y with acute VTE N=267 | Dabigatran (n=177) vs SoC (n=90) Treatment period: 3 mo followed by 1-mo observation period | Published |
| NCT02197416 | Phase 3, single arm, open-label | Extended secondary prevention | Children <18 y with VTE s/p SoC for ≥3 mo, or completed the DIVERSITY study with persistent VTE risk factor(s) N=203 | Dabigatran (n=203) Treatment period: ≤12 mo with an additional 28 d of observation | Published |
| Hokusai VTE PEDIATRICS | Phase 3, noninferiority, open-label, RCT | Acute VTE treatment and secondary prevention | Children <18 y with acute VTE N=290 | Edoxaban vs SoC Treatment period: 3 mo, followed by extension period of 9 mo using edoxaban (1-y postenrollment) | Not published |
| NCT02464969 CANINES | Phase 4, open-label, RCT | Acute VTE treatment | Children <18 y with acute VTE N=250 (target) | Apixaban vs SoC Treatment period: 3 mo (12 wk) | Ongoing |
| Thromboprophylaxis | |||||
| UNIVERSE | Phase 3 open-label, active controlled, 2-part study | TE prevention after Fontan procedure | Children of 2 to 8 y with single ventricle physiology status after the Fontan procedure N=112 | Part A: rivaroxaban (n=12) Part B: rivaroxaban (n=66) vs ASA (n=34) | Published |
| ENNOBLE-ATE | Phase 3, open-label, RCT | TE prevention various cardiac diseases | Children <18 y with cardiac disease at risk for TE complications N=168 | Edoxaban (n=110) vs SoC (n=58) Treatment period: 3 mo, followed by extension period of 9 mo using edoxaban (1-y postenrollment) | Preprint |
| SAXOPHONE | Phase 2, open-label, RCT | TE prevention various cardiac diseases | Children 29 days to <18 y with heart disease at risk for TE complications N=192 | Apixaban (n=129) vs SoC (n=63) Treatment period: ≤12 mo | Completed; not published |
| PREVAPIX-ALL | Phase 3, superiority, open-label, RCT | Primary VTE prevention | Children ≥1 to <18 y with ALL or B or T cell LL N=512 | Apixaban (n=256) vs no systemic anticoagulation (n=256) Treatment period: from randomization through day 28 of induction | Completed; not published |
| Study . | Design . | Indications . | Population . | Interventions . | Status . |
|---|---|---|---|---|---|
| VTE treatment | |||||
| EINSTEIN Jr | Phase 3, noninferiority, open label, RCT | Acute VTE treatment and secondary prevention | Children ≤17 y with acute VTE N=500 | Rivaroxaban (n=335) vs SoC (n=165) Treatment period: 3 mo (1 mo for children <2 y with CVC-associated VTE) | Published |
| DIVERSITY | Phase 2b/3 noninferiority, open label, RCT | Acute VTE treatment and secondary prevention | Children <18 y with acute VTE N=267 | Dabigatran (n=177) vs SoC (n=90) Treatment period: 3 mo followed by 1-mo observation period | Published |
| NCT02197416 | Phase 3, single arm, open-label | Extended secondary prevention | Children <18 y with VTE s/p SoC for ≥3 mo, or completed the DIVERSITY study with persistent VTE risk factor(s) N=203 | Dabigatran (n=203) Treatment period: ≤12 mo with an additional 28 d of observation | Published |
| Hokusai VTE PEDIATRICS | Phase 3, noninferiority, open-label, RCT | Acute VTE treatment and secondary prevention | Children <18 y with acute VTE N=290 | Edoxaban vs SoC Treatment period: 3 mo, followed by extension period of 9 mo using edoxaban (1-y postenrollment) | Not published |
| NCT02464969 CANINES | Phase 4, open-label, RCT | Acute VTE treatment | Children <18 y with acute VTE N=250 (target) | Apixaban vs SoC Treatment period: 3 mo (12 wk) | Ongoing |
| Thromboprophylaxis | |||||
| UNIVERSE | Phase 3 open-label, active controlled, 2-part study | TE prevention after Fontan procedure | Children of 2 to 8 y with single ventricle physiology status after the Fontan procedure N=112 | Part A: rivaroxaban (n=12) Part B: rivaroxaban (n=66) vs ASA (n=34) | Published |
| ENNOBLE-ATE | Phase 3, open-label, RCT | TE prevention various cardiac diseases | Children <18 y with cardiac disease at risk for TE complications N=168 | Edoxaban (n=110) vs SoC (n=58) Treatment period: 3 mo, followed by extension period of 9 mo using edoxaban (1-y postenrollment) | Preprint |
| SAXOPHONE | Phase 2, open-label, RCT | TE prevention various cardiac diseases | Children 29 days to <18 y with heart disease at risk for TE complications N=192 | Apixaban (n=129) vs SoC (n=63) Treatment period: ≤12 mo | Completed; not published |
| PREVAPIX-ALL | Phase 3, superiority, open-label, RCT | Primary VTE prevention | Children ≥1 to <18 y with ALL or B or T cell LL N=512 | Apixaban (n=256) vs no systemic anticoagulation (n=256) Treatment period: from randomization through day 28 of induction | Completed; not published |
SoC, standard of care; TE, venous thromboembolism.