Study of RAD001 for Treatment of NF2-related Vestibular Schwannoma

Summary

The purpose of the study is to determine if RAD001 treatment will shrink or slow the growth of the vestibular schwannoma(s) in Neurofibromatosis 2 (NF2) patients. Secondary objectives include determining if RAD001 treatment will improve hearing ability in NF2 patients. RAD001 is an oral drug that is approved by Food and Drug Administration (FDA) for other types of tumors, it is not approved by the FDA for treatment of NF2 related tumors.

Timeline

Start

2015-07-01

Primary completion

2024-02-06

Completion

2024-02-06

Outcome

Missed primary endpoint

Stopped (Enrollment): “slow accrual”

paper There was no VS RR and 3 of 8 (37.5%) participants had stable disease. PMID 38372904 ↗

Publications

• Background Giovannini M, Bonne NX, Vitte J, Chareyre F, Tanaka K, Adams R, Fisher LM, Valeyrie-Allanore L, Wolkenstein P, Goutagny S, Kalamarides M. mTORC1 inhibition delays growth of neurofibromatosis type 2 schwannoma. Neuro Oncol. 2014 Apr;16(4):493-504. doi: 10.1093/neuonc/not242. Epub 2014 Jan 10.
• Background Goutagny S, Raymond E, Esposito-Farese M, Trunet S, Mawrin C, Bernardeschi D, Larroque B, Sterkers O, Giovannini M, Kalamarides M. Phase II study of mTORC1 inhibition by everolimus in neurofibromatosis type 2 patients with growing vestibular schwannomas. J Neurooncol. 2015 Apr;122(2):313-20. doi: 10.1007/s11060-014-1710-0. Epub 2015 Jan 8.
• Nghiemphu PL, Vitte J, Dombi E, Nguyen T, Wagle N, Ishiyama A, Sepahdari AR, Cachia D, Widemann BC, Brackmann DE, Doherty JK, Kalamarides M, Giovannini M. Imaging as an early biomarker to predict sensitivity to everolimus for progressive NF2-related vestibular schwannoma. J Neurooncol. 2024 Apr;167(2):339-348. doi: 10.1007/s11060-024-04596-4. Epub 2024 Feb 19.

Drugs

Evaluation	Drug	Modality	Dose	Route
Subject	Everolimus	Small molecule	3 mg/m2	Oral
Subject	Everolimus	Small molecule	10 mg	Oral

Indications