Table 2.
Ongoing clinical trials of targeted agents in MB
| Agent | Target(s) | Phase: Statusa | Study Population (Nplanned) | Regimen | NCT ID |
|---|---|---|---|---|---|
| GDC-0449 (vismodegib) | SMO | 2: ANR | Adult pts with R/R MB (n = 50) | GDC-0449 | NCT00939484 |
| 2: CR | Pts aged 3–21 years with R/R MB (n = 50) | GDC-0449 | NCT01239316 | ||
| 1/2: CR | Adult pts with MB with activated SHH (n = 38) | TMZ ± GDC-0449 | NCT01601184 | ||
| 1: ANR | Pts aged 3–21 years with R/R MB (n = 30) | GDC-0449 | NCT00822458 | ||
| 2: CR | Pts aged 3–21 years with newly diagnosed MB stratified based on clinical risk and molecular subgroup (WNT, SHH, non-WNT/non-SHH) (n = 350) | For SHH group: craniospinal radiation and chemotherapy (cisplatin, vincristine, cyclophosphamide) followed by GDC-0449 + maintenance chemotherapy | NCT01878617 | ||
| LDE225 | SMO | 3: CR | Pediatric or adult pts with Hh-activated, relapsed MB (n = 109) | LDE225 or TMZ | NCT01708174 |
| 1/2: CR | Pediatric or adult pts with R/R MB (n = 91) | LDE225 | NCT01125800 | ||
| 1: ANR | Adult pts with advanced solid tumors, including MB (n = 100) | LDE225 | NCT00880308 | ||
| 1: CR | East Asian adult pts with advanced solid tumors, including MB (n = 44) | LDE225 | NCT01208831 | ||
| LEQ506 | SMO | 1: ANR | Adult pts with advanced solid tumors, including R/R MB (n = 71) | LEQ506 | NCT01106508 |
| Veliparib | PARP | 1: ANR | Pts aged ≤ 21 years with R/R CNS tumors, including MB (n = 24) | ABT-888 + TMZ | NCT00946335 |
| Sirolimus | mTOR | 1: CR | Pediatric or adult pts aged ≤30 years with R/R solid tumors, including MB (n = 24) | Sirolimus + daily celecoxib, plus low-dose etoposide alternating with cyclophosphamide | NCT01331135 |
| Antiangiogenic agents, including bevacizumab | VEGF, VEGFR | 2: CR | Pts aged ≤21 years with R/R MB or CNS primitive neuroectodermal tumors (n = 108) | TMZ + irinotecan hydrochloride ± BEV | NCT01217437 |
| 2: ANR | Pts aged ≤21 years with R/R or progressive malignant gliomas, diffuse/intrinsic brainstem gliomas, MB, ependymomas, or low-grade gliomas (n = 140) | BEV + irinotecan | NCT00381797 | ||
| 2: CR | Pediatric pts with recurrent or progressive MB (n = 40) | BEV + intrathecal etoposide and cytarabine + thalidomide, celecoxib, and fenofibric acid, with alternating cycles of daily low-dose etoposide and cyclophosphamide | NCT01356290 | ||
| 1: CR | Pts aged 18 months to 23 years with R/R CNS tumors, including MB (n = 30) | BEV + irinotecan + TMZ | NCT00876993 | ||
| Vorinostat | HDAC | 1: CR | Pediatric pts aged ≤ 4 years treated with surgery for embryonal CNS tumors, including MB (n = 62) | Vorinostat, isotretinoin + combination chemotherapy | NCT00867178 |
Abbreviations: ANR, active, not recruiting; BEV, bevacizumab; CNS, central nervous system; CR, currently recruiting; GD2, ganglioside G2; HDAC, histone deacetylase; Hh, hedgehog; ID, identifier; mAb, monoclonal antibody; MB, medulloblastoma; mTOR, mammalian target of rapamycin; NCT, national clinical trial; PARP, poly(ADP-ribose) polymerase; pts, patients; R/R, recurrent/refractory; SHH, sonic hedgehog; SMO, smoothened; TMZ, temozolomide; VEGF, vascular endothelial growth factor; VEGFR, vascular endothelial growth factor receptor.
aCurrent status of each trial based on a search of ClinicalTrials.gov conducted on July 26, 2013.