Table 3.
Clinical trials of OPB compounds, pyrimethamine, TTI-101: STAT3 SH2 domain inhibitors.
| NCT No. | Phase | Therapy | Cancer Types | Brief Description | Time | Ref. |
|---|---|---|---|---|---|---|
| OPB-31121 | ||||||
| NCT 00955812 |
I | Monotherapy | Advanced solid tumors (n = 14) |
Unfavorable pharmacokinetic profile and antitumor activity demonstrated, leading to discontinuation of compound development | 2009–2012 | [109] |
| OPB-111077 | ||||||
| NCT 01711034 |
I | Monotherapy | Advanced solid tumors (n = 145) |
Safety, tolerability and antitumor activity demonstrated, including one PR in DLBCL | 2012–2015 | [110] |
| NCT 01942083 |
I | Monotherapy | Advanced HCC (n = 33) |
Safety and tolerability demonstrated but no antitumor response was observed | 2013–2017 | [111] |
| NCT 02250170 |
I | Monotherapy | Advanced solid tumors (n = 47) |
To evaluate the safety, tolerability and antitumor activity | 2014–2019 | / |
| NCT 03197714 |
Ib | Monotherapy | Relapsed/refractory AML (n = 15) |
To evaluate the safety, tolerability and antitumor activity | 2017–2018 | / |
| NCT 03158324 |
IIa | Monotherapy | NPC/refractory tumors (n = 52) |
To evaluate the safety, tolerability and antitumor activity | 2017–2020 | / |
| NCT 03063944 |
I | Combination | AML (n = 12) |
To evaluate the safety, tolerability and antitumor activity in combination with decitabine | 2017–2023 | / |
| NCT 04049825 |
I | Combination | Relapsed/refractory DLBCL (n = 65) |
To evaluate the safety, tolerability and antitumor activity in combination with bendamustine and rituximab | 2019–2021 | / |
| OPB-51602 | ||||||
| NCT 01184807 |
I | Monotherapy | Refractory solid tumors (n = 51) |
Better tolerability for intermittent than continuous dosing; antitumor activity demonstrated, including 2 PRs in EGFR mutation-positive NSCLC prior anti-EGFR exposure | 2009–2013 | [112] |
| NCT 01423903 |
I | Monotherapy | Advanced cancers (n = 45) |
To evaluate the safety, tolerability, pharmacokinetic profile and antitumor activity | 2010–2013 | / |
| NCT 01344876 |
I | Monotherapy | Relapsed/refractory hematological malignancies (n = 20) |
Safety and tolerability demonstrated but long-term administration at higher doses was difficult with daily dosing and no antitumor response was observed, leading to termination of study | 2011–2014 | [113] |
| NCT 01867073 |
I | Monotherapy | Advanced solid tumors (n = 20) |
To evaluate the pharmacokinetic profile in relation to pSTAT3 expression in peripheral mononuclear blood cells and single nucleotide polymorphisms in patient tissues | 2011–2015 | / |
| NCT 02058017 |
I | Monotherapy | Advanced NPC (n = 9) |
Poor tolerability, leading to termination of study | 2013–2015 | [114] |
| Pyrimethamine | ||||||
| NCT 01066663 |
I & II | Monotherapy | Relapsed CLL/SLL (n = 26) |
To evaluate the safety, tolerability, pharmacokinetic profile and antitumor activity | 2010–2024 | / |
| TTI-101 | ||||||
| NCT 03195699 |
I | Monotherapy | Advanced cancers (n = 30) |
To evaluate the safety, tolerability and pharmacokinetic profile | 2017–2020 | / |