Table 1.
Ongoing clinical studies of PK activators in SCD
| Medication | Clinicaltrials.gov identifier | Study design | Study population (age) | N | Anticipated completion |
|---|---|---|---|---|---|
| Mitapivat | NCT04610866 | Ph 1, open-label, single center | HbSS (18-70) | 15 | 2/2028 |
| Mitapivat | NL8517 (The Netherlands) EudraCT 2010-003438-18 (ESTIMATE) |
Ph 2, open-label, single center | HbSS or HbSβ0 (≥18) | 10 | |
| Mitapivat |
NCT05031780 EudraCT 2021-001674-34 (RISE UP) |
Ph 2/3, randomized, double-blind, placebo-controlled, multicenter | SCD, any (≥16) | 69 (Ph 2) 198 (Ph 3) |
11/2029 |
| AG-946 | NCT04536792 | Ph 1, randomized, double-blind (healthy volunteers), open-label (SCD), multicenter | Healthy volunteers SCD, any (18-70) |
64 | 3/2024 |
| Etavopivat | NCT04624659 (HIBISCUS) | Ph 2/3, randomized, double-blind, placebo-controlled, multicenter | SCD, any (12-65) | 344 | 12/2026 |
| Etavopivat | NCT04987489 | Ph 2, open-label, multiple-cohort, multicenter | Cohort A: SCD, any, on chronic transfusions (12-65) Cohort B: thalassemia, any, on chronic transfusions (12-65) Cohort C: thalassemia, any, nontransfused (12-65) |
60 | 7/2025 |