Table 1.
Currently active clinical trials of GO in AML.
| Disease type and inclusion criteria | Drug or drug combination, other therapies | Outcome measures | (Estimated) enrollment | Clinical trials identifier | Trial status |
|---|---|---|---|---|---|
| R/R AML or APL | GO | Primary: access to GO and toxicity | 30 | NCT01869803 | Suspended (estimated completion mid 2020) |
| R/R AML in patients not eligible for curative therapy | GO + donor leukocyte infusions | Primary: ORR Secondary: PFS, OS, and DLT |
18 | NCT03374332 | Not yet recruiting (estimated completion early 2022) |
| De novo AML in elderly patients (60–80 years), favorable or intermediate-risk cytogenetics | Low-dose GO + cytarabine vs. idarubicin + cytarabine | Primary: EFS Secondary: efficacy, toxicity, and MRD level |
225 | NCT02473146 | Recruiting (estimated completion late 2020) |
| Adverse-risk AML in 1st CR AML in 2nd CR with MRD AML in 3rd CR R/R AML High-risk MDS ≤70 years |
GO + busulfan + cyclophosphamide, followed by aHSCT and ATG | Primary: ORR | 25 | NCT02221310 | Recruiting (estimated completion mid 2021) |
| AML in 1st CR with matched donor AML in 2nd CR MDS (≥10% CD33+ blasts) |
aHSCT + GO | Primary: graft failure, EFS, OS, and SAE Secondary: chimerism and GvHD |
26 | NCT02117297 | Recruiting (estimated completion late 2021) |
| Relapsed AML | AZA + GO | Primary: MTD Secondary: RTT |
50 | NCT00766116 | Active, not recruiting (completion late 2017) |
| AML with NPM-1 mutation | Idarubicin + etoposide + cytarabine + ATRA vs. idarubicin + etoposide + cytarabine + ATRA + GO | Primary: OS Secondary: CR, DOR, EFS, AE, and QOL |
588 | NCT00893399 | Recruiting (estimated completion mid 2020) |
| R/R AML | Observational: retrospective analysis of GO monotherapy | Primary: CR Secondary: OS, DOR, RFS, SAE, and aHSCT incidence |
300 | NCT03287128 | Recruiting (estimated completion early 2019) |
| De novo AML | Idarubicin + cytarabine + GO + G-CSF | Primary: CR Secondary: AE and OS |
40 | NCT01698879 | Active, not recruiting (completion late 2016) |
| APL in patients ≥10 years | Addition of GO to ATRA + arsenic trioxide | Primary: EFS | 100 | NCT01409161 | Recruiting (estimated completion late 2019) |
| AML, high-risk MDS in children ≤18 years | Addition of GO to cytarabine + mitoxantrone (or liposomal daunorubicin) during induction | Primary: DLT, EFS, RFS, and AE Secondary: AE, PK, CR, EFS, and OS |
700 | NCT02724163 | Recruiting (estimated completion late 2032) |
| R/R AML | PF-04518600 (OX-40 agonist mAb) vs. PF-04518600 + avelumab (anti-PD-L1 mAb) vs. PF-04518600 + AZA vs. PF-04518600 + utomilumab (4-1BB agonist mAb) vs. avelumab + utomilumab vs. PF-04518600, + avelumab + AZA vs. GO + glasdegib (smoothened inhibitor) vs. glasdegib + avelumab | Primary: AE and CR Secondary: DFS, MRD, and OS |
138 | NCT03390296 | Recruiting (estimated completion late 2023) |
| AML MDS JMML |
Fludarabine + busulfan, followed by aHSCT + GO | Primary: MTD Secondary: MRD, EFS, OS, and chimerism |
18 | NCT01020539 | Active, not recruiting (estimated completion early 2018) |
| AML in patients ≥60 years | “3 + 7” + GO vs. “3 + 7” vs. less-intensive therapy | Primary: OS, CR, CRi, toxicity, DOR, and supportive care requirements Secondary: MRD levels and tissue storage |
1,600 | NCT02272478 | Recruiting (estimated completion late 2020) |
| AML | “3 + 7” + aHSCT vs. “3 + 7” (high-dose daunorubicin) + aHSCT vs. “3 + 7” + GO + aHSCT | Primary: OS and DFS Secondary: OS |
657 | NCT00049517 | Active, not recruiting (estimated completion early 2019) |
“3 + 7”, daunorubicin (days 1–3) + cytarabine (days 1–7); AE, adverse events; aHSCT, allogeneic hematopoietic stem cell transplantation; AML, acute myeloid leukemia; APL, acute promyelocytic leukemia; ATG, anti-thymocyte globulin; ATRA, all-trans retinoic acid; AZA, 5-azacytidine; CML, chronic myeloid leukemia; CR, complete remission; CRi, complete remission with incomplete recovery; DFS, disease-free survival; DLT, dose-limiting toxicity; DOR, duration of response; G-CSF, granulocyte colony-stimulating factor; GO, gemtuzumab ozogamicin; GvHD, graft-versus-host disease; JMML, juvenile; mAb, monoclonal antibody; MDS, myelodysplastic syndrome; MRD, minimal residual disease; MTD, maximum tolerated dose; NPM-1, nucleophosmin-1; ORR, overall response rate; OS, overall survival; PFS, progression-free survival; PK, pharmacokinetics; QOL, quality of life; RTT, response to treatment; R/R, refractory/relapsed.
Data from http://ClinicalTrials.gov (Accessed: March 14, 2018).