Summary of ongoing and completed clinical trials of pyruvate kinase activators in sickle cell disease
| . | Trial . | Subjects . | Study design . | Status . |
|---|---|---|---|---|
| Mitapivat (AG-348) | NCT0400016529 | N = 17, age ≥18, HbSS | Phase 1, open-label, multiple ascending dose study | Completed |
| ESTIMATE (EudraCT 2019-003438-18)30,31 | N = 9, age ≥16, HbSS, HbS/β0- or HbS/β+-thalassemia | Phase 2, open-label, multiple ascending dose phase, followed by fixed-dose extension study | Ongoing | |
| NCT04610866 | N = 15, age 18-70, HbSS | Phase 1/2, open-label extension study | Ongoing | |
| RISE UP (NCT05031780) | N = 267, age ≥16, any SCD genotype | Phase 2/3, randomized, placebo-controlled, double-blind, followed by open-label extension study | Ongoing | |
| Etavopivat (FT-4202) | NCT0381569534-36 | N = 130, healthy volunteers and patients with SCD (any genotype) age 12-65 | Phase 1, randomized, placebo-controlled, double-blind, single ascending and multiple ascending dose study | Completed |
| NCT04987489 | N = 60, age 12-65, patients with thalassemia and any SCD genotype | Phase 2, open-label study | Ongoing | |
| NCT05725902 | N = 12, age 12-21, HbSS or HbS/β0 | Phase 2, open-label study | Ongoing | |
| HIBISCUS (NCT04624659) | N = 344, age 12-65, any SCD genotype | Phase 2/3, randomized, placebo-controlled, double-blind, followed by open-label extension study | Ongoing | |
| HIBISCUS-KIDS (PACTR202209604592389) | N = 50, age 12 to <18, any SCD genotype | Phase 1/2, open-label, single-arm, followed by extension study | Ongoing | |
| AG-946 | NCT04536792 | N = 64, age 18-70, healthy volunteers and patients with SCD (any genotype) | Phase 1, open-label, single ascending and multiple ascending dose study | Ongoing |
| . | Trial . | Subjects . | Study design . | Status . |
|---|---|---|---|---|
| Mitapivat (AG-348) | NCT0400016529 | N = 17, age ≥18, HbSS | Phase 1, open-label, multiple ascending dose study | Completed |
| ESTIMATE (EudraCT 2019-003438-18)30,31 | N = 9, age ≥16, HbSS, HbS/β0- or HbS/β+-thalassemia | Phase 2, open-label, multiple ascending dose phase, followed by fixed-dose extension study | Ongoing | |
| NCT04610866 | N = 15, age 18-70, HbSS | Phase 1/2, open-label extension study | Ongoing | |
| RISE UP (NCT05031780) | N = 267, age ≥16, any SCD genotype | Phase 2/3, randomized, placebo-controlled, double-blind, followed by open-label extension study | Ongoing | |
| Etavopivat (FT-4202) | NCT0381569534-36 | N = 130, healthy volunteers and patients with SCD (any genotype) age 12-65 | Phase 1, randomized, placebo-controlled, double-blind, single ascending and multiple ascending dose study | Completed |
| NCT04987489 | N = 60, age 12-65, patients with thalassemia and any SCD genotype | Phase 2, open-label study | Ongoing | |
| NCT05725902 | N = 12, age 12-21, HbSS or HbS/β0 | Phase 2, open-label study | Ongoing | |
| HIBISCUS (NCT04624659) | N = 344, age 12-65, any SCD genotype | Phase 2/3, randomized, placebo-controlled, double-blind, followed by open-label extension study | Ongoing | |
| HIBISCUS-KIDS (PACTR202209604592389) | N = 50, age 12 to <18, any SCD genotype | Phase 1/2, open-label, single-arm, followed by extension study | Ongoing | |
| AG-946 | NCT04536792 | N = 64, age 18-70, healthy volunteers and patients with SCD (any genotype) | Phase 1, open-label, single ascending and multiple ascending dose study | Ongoing |