Table 2.
Partial list of investigational drugs and/or strategies (and their molecular targets) that are currently under clinical evaluation. The list is limited to represent distinct drugs and strategies.
| Phase | NCT ID | Drugs / Strategy | Target |
|---|---|---|---|
| Phase 1 | NCT04049864 | DNA Vaccine | Phox2B, Survivin, MAGEA1/3, PRAME |
| Phase 1 | NCT00790413 | 131I-MIBG+CT+T-Cell−+Lymp+ +HSC+MSC+Rituximab | CD20 |
| Phase 1 | NCT02573896 | Auto-NKCs + Dinutuximab +/− Lenalidomide | GD2, TNFa, VEGF, bFGF |
| Phase 1 | NCT03294954 | GD2-CAR + IL15- Auto-NKTs + CT | GD2 |
| Phase 1 | NCT04106219 | LY3295668 Erbumine +/− CT | AURA |
| Phase 1 | NCT02298348 | Sorafenib + Topotecan, Cyclophosphamide | Tyrosine Kinase |
| Phase 1 | NCT04023331 | 67Cu-SARTATE PR-RT | SSTR |
| Phase 1 | NCT03966651 | 177Lu-DOTA0-Tyr3-Octreotate PR-RT | SSTR |
| Phase 1 | NCT02914405 | Nivolumab +Ch14.18/CHO | PD-1, GD2 |
| Phase 1 | NCT00492167 | beta-glucan + 3F8 | |
| Phase 1 | NCT04239040 | GVAX + Nivolumab, Ipilimumab | PD-1, CTLA-4 |
| Phase 1 | NCT01711554 | Lenalidomide +Dinutuximab +/− Isotretinoin | angiogenesis inhibitor |
| Phase 1 | NCT02311621 | CD171-CAR T cells expressing EGFRt | CD171, EGFR |
| Phase 1 | NCT03332667 | 131I-MIBG With Dinutuximab | GD2 |
| Phase 1 | NCT03107988 | Lorlatinib | ALK, ROS-1 |
| Phase 1 | NCT01953900 | iC9-GD2-CAR-VZV-CTLs | GD2 |
| Phase 1 | NCT02030964 | DFMO + Celecoxib + CT | ODC |
| Phase 1 | NCT02761915 | 1RG-CART | GD2 |
| Phase 1 | NCT02748135 | TB-403 | PLGF |
| Phase 1 | NCT03635632 | C7R-GD2.CART cells + CT | GD2 |
| Phase 1 | NCT00089245 | I131 MOAB 8H9 | glycoprotein 4Ig-B7-H3 |
| Phase 1 | NCT03478462 | CLR 131 | |
| Phase 1 | NCT02909777 | CUDC-907 | HDAC |
| Phase 1 | NCT04239092 | 9-ING-41 + Irinotecan | GSK-3β |
| Phase 1 | NCT03709680 | Palbociclib + Temozolomide + Irinotecan | CDK4, CDK6, DNA replication |
| Phase 1 | NCT03236857 | venetoclax + CT | BCl2 |
| Phase 1 | NCT04337177 | VAL-413 + Temozolomide | DNA Replication |
| Phase 1 | NCT04308330 | Vorinostat + Chemotherapy | HDAC |
| Phase 1 | NCT02536183 | Lyso-thermosensitive liposomal dox + MR-HIFU | |
| Phase 1 | NCT03618381 | EGFR806-EGFRt + CD19-Her2tG | EGFR |
| Phase 1 | NCT02508038 | Zoledronate + TCRαβ+/CD19+ depleted H-HSCT | |
| Phase 1 | NCT01331135 | Sirolimus | mTOR |
| Phase 1 | NCT01625351 | Sirolimus, Alemtuzumab | mTOR, CD52, |
| Phase 1 | NCT02644460 | Abemaciclib | CDK4, CDK6 |
| Phase 1 | NCT03434262 | Gemcitabine, Ribocicilib, Sonidegib, Trametinib | CyclinD1, CDK4/6, HH, MEK1, MEK2 |
| Phase 1 | NCT01121588 | Crizotinib (PF-02341066) | ALK, ROS1 |
| Phase 1 | NCT02085148 | Regorafenib + Vincristine + Irinotecan | RTK, VEGFR2-TIE2 |
| Phase 1 | NCT03936465 | BMS-986158 | BET |
| Phase 1 | NCT03507491 | Nab-paclitaxel + Gemcitabine | |
| Phase 1 | NCT04238819 | Abemaciclib + Temozolomide, Irinotecan | CDK4, CDK6 |
| Phase 1 | NCT02650401 | Entrectinib (Rxdx-101) | Trk, Ros1, Alk Fusions |
| Phase 1/2 | NCT00703222 | SNJB-JF-IL2 and SJNB-JF-Lptn + Dose Level 1 SKNLP | |
| Phase 1/2 | NCT02173093 | GD2Bi-aATC | GD2 |
| Phase 1/2 | NCT02139397 | DFMO + Bortezomib | ODC, proteasome inhibitor |
| Phase 1/2 | NCT00911560 | OPT-821 vaccine with GD2L and GD3L | GD2, GD3 |
| Phase 1/2 | NCT03860207 | Hu3F8-BsAb | GD2 |
| Phase 1/2 | NCT03923257 | PRRT-177Lu-DOTA tyr3-Octreotide | SSTR |
| Phase 1/2 | NCT04029688 | Idasanutlin + Venetoclax + CT | MDM2, BCl2 |
| Phase 1/2 | NCT02095132 | Adavosertib + Irinotecan | WEE1 |
| Phase 1/2 | NCT02124772 | Dabrafenib, Trametinib | B-RAF, MEK1, MEK2 |
| Phase 1/2 | NCT00788125 | Dasatinib + Ifosfamide + CT + surgery + RT | Bcr-ABL, Src |
| Phase 2 | NCT03363373 | GM-CSF + Naxitamab | GD2 |
| Phase 2 | NCT03503864 | Arsenic Trioxide + induction CT | |
| Phase 2 | NCT03373097 | GD2-CAR-T Cells +/− CT & lymphodepletion | GD2 |
| Phase 2 | NCT04301843 | DFMO + Etoposide | ODC, |
| Phase 2 | NCT02679144 | DFMO | ODC |
| Phase 2 | NCT04023331 | 67Cu-SARTATE PR-RT (Cohort expansion) | SSTR |
| Phase 2 | NCT02998983 | Racotumomab | anti-N-glycolyl GM3 Ab inducer |
| Phase 2 | NCT03033303 | Hu3F8/GM-CSF + Isotretinoin | GD2 |
| Phase 2 | NCT02559778 | Ceritinib + Dasatinib + Sorafenib + Vorinostat + DFMO | ODC, HDAC, ALK, VEGFR, PDGFR, RAF |
| Phase 2 | NCT02308527 | Bevacizumab + Temozolomide ± Irinotecan | VEGF-A, DNA replication |
| Phase 2 | NCT01467986 | Dasatinib + Rapamycin + Irinotecan + Temozolomide | Bcr-ABL, Src, mTOR, DNA replication |
| Phase 2 | NCT00107289 | lobenguane I 131 | |
| Phase 2 | NCT00601003 | Nifurtimox | |
| Phase 2 | NCT02035137 | 131I-MIBG +/− Viscristine, Irinotecan / Vorinostat | HDAC |
| Phase 2 | NCT02452554 | Lorvotuzumab Mertansine | CD56 binding |
| Phase 2 | NCT03273712 | PRRT-90Y-DOTA tyr3-Octreotide | SSTR |
| Phase 2 | NCT04320888 | Selpercatinib | RET |
| Phase 2 | NCT04195555 | Ivosidenib | IDH1 |
| Phase 2 | NCT04284774 | Tipifarnib | FTase |
| Phase 2 | NCT03698994 | Ulixertinib | ERK1/2 |
| Phase 2 | NCT03526250 | Palbociclib | CDK4, CDK6 |
| Phase 2 | NCT03220035 | Vemurafenib | B-RAF |
| Phase 2 | NCT03233204 | Olaparib | PARP |
| Phase 2 | NCT03213704 | Larotrectinib | TrkA, TrkB, TrkC |
| Phase 2 | NCT03213678 | Samotolisib | PI3K, mTOR, DNA-PK |
| Phase 2 | NCT03210714 | Erdafitinib | FGFR |
| Phase 2 | NCT03155620 | Ensartinib, Erdafitinib, Larotrectinib, Olaparib, Palbociclib, Samotolisib, Selpercatinib, Selumetinib Sulfate, Tazemetostat, Tipifarnib, Ulixertinib, Vemurafenib | ALK, FGFR, TrkA, TrkB, TrkC, PARP, CDK4/6, PI3K, mTOR, RET, MEK1, MEK2, EZH2, Ftase, ERK1/2, B-RAF |
| Phase 2 | NCT03213665 | Tazemetostat | EZH2 |
| Phase 2 | NCT02378428 | MIBG | NET Receptors |
| Phase 2 | NCT03458728 | BAY806946 | PI3K |
| Phase 2 | NCT02574728 | Sirolimus + Metronomic CT | mTOR |
| Phase 2/3 | NCT03275402 | 131I-omburtamab | B7-H3 |
| Phase 3 | NCT03126916 | Crizotinib/Iobenguane I131 + Auto-HSC, CT, Dinutuximab, EBRT, Isotretinoin, Sargramostim, Surgery, Vincristine | ALK, ROS-1, NET receptors |
| Phase 4 | NCT03975829 | Dabrafenib, Trametinib | B-RAF, MEK1, MEK2 |