Table 2 Clinical trials of various diseases by targeting protein lipidation
Lipidation | Drug | Mechanism | Disease or patients | Treatment | Phase | NCT number |
|---|---|---|---|---|---|---|
N-myristoylation | PCLX-001 | Pan-NMT inhibitor | Relapsed/refractory B-cell non-Hodgkin lymphoma and advanced solid malignancies | Monotherapy | I | NCT04836195 |
Asciminib (ABL001) | Target the myristoyl pocket of the BCR-ABL1 tyrosine kinase | Ph+ ALL or CML | Asciminib in combination with Dasatinib, Prednisone, and Blinatumomab | I | NCT03595917 | |
CML | Monotherapy of imatinib or combination with asciminib; combination of asciminib with TKI | II | NCT04216563 | |||
CML | Continued treatment of asciminib | IV | NCT04877522 | |||
Newly diagnosed CML-CP | Monotherapy or combination with one TKI | II | NCT05143840 | |||
Newly diagnosed, previously untreated Ph+ CML-CP | Monotherapy of asciminib or nilotinib; or one selected TKI | III | NCT05456191 NCT04971226 | |||
CML-CP previously treated with one prior ATP-binding site TKI | Monotherapy | II | NCT05384587 | |||
Relapsed CML previously attempted to discontinue imatinib | Asciminib combination with imatinib | II/ III | NCT05413915 NCT04838041 | |||
CML-CP previously treated with two or more TKIs | Monotherapy of asciminib; or best available treatment; or bosutinib | II/III | NCT04795427 NCT04948333 NCT03106779 | |||
CML-CP without T315I mutation previously treated with two prior TKIs; CML-CP with T315I mutation with previously treated with at least prior TKIs | Monotherapy | III | NCT04666259 | |||
S-prenylation | Pravastatin | Inhibition of FPP and GGPP biosynthesis | HGPS | A combination of zoledronic acid and pravastatin | II | NCT00731016 |
Zoledronic acid | Inhibition of the FPPS | Progeria; HGPS | A combination of pravastatin and lonafarnib and zoledronic acid | II | NCT00879034 NCT00916747 | |
Lonafarnib | FTase inhibitor | HDV infection | Monotherapy | II 11 | NCT01495585 | |
HDV infection | Lonafarnib in combination with ritonavir | II/III | NCT02527707 NCT02511431 NCT05229991 | |||
HDV infection | Lonafarnib With or Without Ritonavir | II | NCT02968641 NCT02430181 | |||
HDV infection | A combination of Lonafarnib, ritonavir and peginterferon lambda-1a | II | NCT03600714 NCT05953545 | |||
BMS-214662 | FTase inhibitor | acute leukaemia, MDS, or CML | Monotherapy | I | NCT00006213 | |
Solid tumours | Monotherapy | I | NCT00005973 NCT00004877 | |||
Advanced solid tumours | Monotherapy; BMS-214662 in combination with trastuzumab; BMS-214662 in combination with paclitaxel | I | NCT00004877 NCT00022529 NCT00006018 | |||
S-prenylation | Lonafarnib | FTase inhibitor | HGPS | monotherapy | II | NCT00425607 |
Progeria | Lonafarnib in combination with and everolimus | I/II | NCT02579044 | |||
MDS or CML | Monotherapy | III | NCT00109538 | |||
Chronic or accelerated phase CML | Monotherapy; Lonafarnib in combination with Gleevec | I/II | NCT00038597 NCT00047502 | |||
Metastatic breast cancer | Monotherapy | II | NCT00773474 | |||
Advanced breast cancer | Lonafarnib in combination with Herceptin Plus Paclitaxel; anastrozole in combination with lonafarnib or monotherapy of anastrozole | II | NCT00068757 NCT00081510 | |||
Recurrent or progressive brain tumours in children | Monotherapy | I | NCT00015899 | |||
HNSCC | Monotherapy | I/II | NCT00038584 NCT00073450 | |||
Advanced or recurrent HNSCC | Lonafarnib in combination with Fenretinide | I | NCT00102635 | |||
Advanced ovarian carcinoma | Monotherapy | II | NCT00281515 | |||
Glioblastoma multiforme | Lonafarnib in combination with temozolomide | I | NCT00102648 | |||
Recurrent glioblastoma multiforme | Lonafarnib in combination with temozolomide | II | NCT00038493 | |||
Recurrent primary supratentorial gliomas | A combination of lonafarnib and temozolomide | I | NCT00083096 | |||
Grade 3 & 4 malignant gliomas | Lonafarnib in combination with Temodar | I | NCT00612651 | |||
Metastatic colorectal cancer | Monotherapy or conventional surgery | I | NCT00005030 | |||
Stage 3b or 4 NSCLC | Lonafarnib in combination with, paclitaxel and carboplatin | III | NCT00050336 | |||
Advanced cancer of the urinary tract | Lonafarnib in combination with gemcitabine | II | NCT00006351 | |||
Advanced malignancies | Lonafarnib in combination with docetaxel; Lonafarnib in combination with fluorouracil, and leucovorin | I | NCT00288444 NCT00003956 | |||
Tipifarnib | FTase inhibitor | Leukaemia | Monotherapy | I/II | NCT00022451 NCT00004009 NCT02807272 | |
AML | Monotherapy; Tipifarnib in combination with chemotherapy; | I/II/III | NCT00124644 NCT00027872 NCT01361464 NCT00354146 NCT00093418 NCT00048503 NCT00093990 NCT00093470 | |||
Advanced haematologic cancer | Monotherapy | I | NCT00005967 | |||
AML | Tipifarnib in combination with Etoposide | I/II | NCT00112853 NCT00005989 | |||
CML-CP | Tipifarnib in combination with Imatinib mesylate | I | NCT00040105 | |||
Myeloproliferative disorders | Monotherapy | I/II | NCT02210858 NCT02779777 | |||
Multiple myeloma | Monotherapy or Tipifarnib in combination with Bortezomib or PS-341 | I/II | NCT00012350 NCT00972712 NCT00361088 |
