Table 2.
Active and Recently Completed Prospective Clinical Trials for NF2-Associated VS (http://clinicaltrials/gov)
| Drug | Mechanism of Action | Trial Design | Status |
|---|---|---|---|
| Bevacizumab (AvastinTM) | Anti-VEGF monoclonal antibody | Phase II, Multicenter (Johns Hopkins University, National Cancer Institute, Massachusetts General Hospital), USA | Completed, efficacious33 N = 14; Hearing improvement in 36%, tumor response in 43% [NCT01207687] |
| Phase II, Multicenter (Children’s Hospital in Los Angeles, Children’s National Medical Center, Children’s HealthCare of Atlanta, University of Chicago, Indiana University, National Cancer Institute, Children’s Hospital Boston and Massachusetts General Hospital, Washington University – St. Louis, New York University Medical Center, Cincinnati Children’s Hospital Medical Center, Children’s Hospital of Philadelphia, University of Utah), USA | Active, not recruiting N = 22 [NCT01767792] |
||
| Phase II, Multicenter (Northwestern University, Dana-Farber Cancer Institute, University of Virginia, University of Washington), USA | Active, not recruiting N = 50 [NCT01125046] |
||
| Axitinib (InlytaTM) | VEGFR1/2/3 inhibitor | Phase II, Multicenter, New York University, USA | Active, not recruiting N = 12 [NCT02129647] |
| Endostatin | VEGF expression inhibitor | Phase II, Beijing Tiantan Hospital, China | Complete, outcome not reported N = 20 [NCT02104323] |
| Aspirin | Cox 2 inhibitor | Phase II, Multicenter (Massachusetts Eye and Ear, Stanford University, Mayo Clinic, University of Iowa, University of Utah), USA | Active, recruiting N = 300 [NCT03079999] |
| Everolimus | Inhibits mTORC1 | Phase II, Hopital Beaujon, France | Completed, efficacious41 N = 10; Hearing stable, tumor response in 55% [NCT01490476] |
| Early Phase I, New York University, USA | Active, not recruiting N = 5 [NCT01880749] |
||
| Phase II, University of California Los Angeles, USA | Active, not recruiting N = 4 [NCT01345136] |
||
| Phase II, New York University, USA | Completed, ineffective42 [NCT01419639] |
||
| AR-42 | Histone deacetylase inhibitor | Early Phase 1, Multicenter (Stanford University, Johns Hopkins University, Massachusetts Eye and Ear, Mayo Clinic), USA | Active, not recruiting N = 5 [NCT02282917] |
| Crizotinib | FAK1 inhibitor | Phase II, University of Alabama, USA | Active, not recruiting N = 19 [NCT04283669] |
| Selumetinib | MEK1/2 inhibitor | Phase II, Cincinnati Children’s Hospital Medical Center, USA | Active, recruiting N = 34 [NCT03095248] |
| Lapatinib | EGFR/ ErbB2 inhibitor | Early Phase I, Multicenter (House Research Institute, Johns Hopkins Hospital, Massachusetts General Hospital, Washington University Medical Center, New York University, Weil Cornell Medical College, Presbyterian Hospital, Ohio State University Medical Center), USA | Completed N = 26 [NCT00863122] |
| Phase II, New York, University, USA | Completed, efficacious43 N = 17; Hearing improvement in 30.8%, tumor response in 23.5% (15% decline in tumor volume) [NCT00973739] |
||
| Icotinib | EGFR inhibitor | Phase II, Beijing Tiantan Hospital, China | Unknown N = 20 [NCT02934256] |
| Nilotinib (TasignaTM) | Bcr-Abl inhibitor | Phase II, Toronto Western Hospital, University Health Network, Canada | Terminated N = 2 [NCT01201538] |
Clinical trials that demonstrated efficacy are shown in green, trials that are ongoing or whose results have not been published yet are shown in yellow, and trials that were reported as ineffective or were terminated are shown in red.