Table 1.
Active clinical trials recruiting patients with relapsed RMS.
| Intervention | Class | Study Population | NCT | Phase |
|---|---|---|---|---|
| Vincristine/Irinotecan +/−Temozolomide (FaR-RMS) | Cytotoxic | RMS | 04625907 | 2 |
| Ganitumab + Dasatinib | IGF-1R antibody + multikinase inhibitor | RMS | 03041701 | 1/2 |
| Vinorelbine + Mocetinostat | HDAC inhibitor + cytotoxic | RMS | 04299113 | 1 |
| Prexasertib + Irinotecan | CHEK2 inhibitor + cytotoxic | RMS, DSRCT | 04095221 | 1/2 |
| Olaparib + Temozolomide | PARP inhibitor + cytotoxic | RMS, EWS | 01858168 | 1 |
| Eribulin mesylate | Cytotoxic | RMS, NRSTS, EWS | 03441360 | 2 |
| Eribulin mesylate + Irinotecan | Cytotoxic | Solid tumors | 03245450 | 1/2 |
| Nab-paclitaxel + Gemcitabine | Cytotoxic | RMS, NRSTS, OST, EWS | 02945800 | 2 |
| Nab-paclitaxel + Gemcitabine | Cytotoxic | Pediatric solid tumors | 03507491 | 1 |
| Regorafenib | Multikinase inhibitor | RMS, EWS, OST, LPS, MCS | 02048371 | 2 |
| Abemaciclib | CDK4/6 inhibitor | Pediatric solid tumors | 02644460 | 1 |
| Abemaciclib + Irinotecan or Irinotecan/Temozolomide | CDK4/6 inhibitor + cytotoxic | Pediatric solid tumors | 04238819 | 1 |
| Copanlisib | PI3K inhibitor | Pediatric solid tumors | 03458728 | 1/2 |
| Vorinostat + chemotherapy | HDAC inhibitor + cytotoxic | Solid tumors | 04308330 | 1 |
| Sirolimus + metronomic chemotherapy | mTOR inhibitor + cytotoxic | Pediatric solid tumors | 02574728 | 2 |
| High-dose alkylator chemotherapy + autologous transplant | Cytotoxic + cellular rescue | Solid tumors | 01505569 | 2 |
| Pediatric MATCH | Personalized | Pediatric solid tumors | Multiple | 2 |
| ESMART | Personalized | Pediatric malignancies | 02813135 | 1/2 |
| B7H3 CAR T Cells | Immunotherapy | Pediatric solid tumors | 04483778 | 1 |
| GD2 CAR T Cells | Immunotherapy | Pediatric solid tumors | 03635632 | 1 |
| EGFR CAR T Cells | Immunotherapy | Pediatric solid tumors | 03618381 | 1 |
| Allogeneic HSCT | Transplant | Pediatric solid tumors | 04530487 | 2 |
| Haploidentical HSCT + Zometa | Transplant | Pediatric malignancies | 02508038 | 1 |
| Reduced intensity haploidentical HSCT | Transplant | Solid tumors | 01804634 | 2 |
| Haploidentical NK cells | Transplant | RMS, EWS | 02409576 | 1/2 |
| Universal donor NK cells + ALT803 | Transplant | Malignancies | 02890758 | 1 |
| High intensity focused ultrasound (HIFU) | Local control | Pediatric solid tumors | 02076906 | 1 |
| HIFU + thermosensitive liposomal doxorubicin | Local control | Pediatric solid tumors | 02536183 | 1 |
| CLR-131 | Local control | Pediatric solid tumors | 03478462 | 1 |
CAR: chimeric antigen receptor; HSCT: hematopoietic stem cell transplant; NK: natural killer; IGF-1R: insulin-like growth factor receptor, type 1; HDAC: histone deacetylase; CHEK2: checkpoint kinase 2; PARP: poly-ADP ribose polymerase; CDK: cyclin dependent kinase; PI3K: phosphoinositide 3 kinase; mTOR: mammalian target of rapamycin; RMS: rhabdomyosarcoma; DSRCT: desmoplastic small round cell tumor; EWS: Ewing sarcoma; NRSTS: non-rhabdomyosarcoma soft tissue sarcoma; OST: osteosarcoma; LPS: liposarcoma, MCS: mesenchymal chondrosarcoma.