Table 2.
Clinical trials with innovative mAb in MDS.
| NCT Code | Trial | Target | Study Population | Efficacy Results | Ref. |
|---|---|---|---|---|---|
| NCT03248479 | Ongoing phase Ib, magrolimab + AZA | CD47 | treatment-naïve MDS from intermediate to very high | ORR 91%: CR 42%, mCR 24%; PR 3% |
[36] |
| NCT04313881 | Ongoing phase III, magrolimab + AZA vs. AZA + placebo | CD47 | Treatment-naïve HR-MDS | NA | N/A |
| NCT04417517 | Ongoing phase I/II, evorpacept + AZA | CD47 | R/R or ND HR-MDS | mCR: 3/10; cytogenic response: 2/10 SD: 2/10 |
[37] |
| NCT02530463 | Ongoing phase II, ipilimumab and/or nivolumab +/− AZA | CTLA-4 | HMA-failure MDS or untreated MDS | HMA-failure arm: ORR 36%, CR 9%, CRi 9%, mOS 11.4 months; frontline arm: ORR 67%, CR 33%, mOS 12% |
[38] |
| NCT03094637 | Ongoing phase II, pembrolizumab + AZA | PD-1 | HMA-failure or untreated INT1 or HR-MDS | HMA-failure arm: ORR 25%; frontline arm: ORR 76%, CR 18%, mCR 29% |
[39] |
| NCT03066648 | Ongoing phase Ib, sabatolimab + HMA | TIM-3 | High risk and very high risk MDS | ORR 56.9%, mDOR: 16.1 months |
[40] |
| NCT02363491 | Ongoing phase I/II, tomaralimab | TLR-2 | HMA-failure and transfusion-dependent LR-MDS patients | ORR: 50% | [41] |
| NCT03337451 | Ongoing phase I/II, tomaralimab | TLR-2 | HMA-failure and transfusion-dependent LR-MDS patients | ORR: 50% | [42] |
| NCT03214666 | Phase I/II, GTB-3550 | CD33 | HR-MDS | N/A | [43] |
| NCT03647800 | Ongoing Ib, APVO436 | CD123 | R/R MDS after HMA-failure | mCR: 50% | [28] |
| NCT03113643 | Ongoing phase Ib, tagraxofusp + AZA | CD123 | MDS | CR 50%, mCR: 25% | [44] |
AZA, azacytidine; MDS, myelodysplastic sindromes; ORR, overall response rate; CR, complete response; mCR, marrow CR; PR, partial response; HR, higher risk; SD, stable disease; R/R, relapsed/refractory; HMA, hypomethylating agents; INT1, intermediate 1; LR, lower risk; mDOR, median duration of response; ND, newly diagnosed.