Clinical trial · Interventional
Study of RAD001 for Treatment of NF2-related Vestibular Schwannoma
A Single Arm, Monocenter Phase II Trial of RAD001 as Monotherapy in the Treatment of Neurofibromatosis Type 2 - Related Vestibular Schwannoma
NCT01345136CI-TRIAL-00073970terminatedPhase 2ClinicalTrials.gov clinicaltrialsProvenance
- Source
- ClinicalTrials.gov
- Retrieved
- Sep 8, 2026
- Layer
- normalized (units and labels harmonized; values unchanged)
- Run
- ING-CLINICALTRIALS-20260908-000001
Why stopped (as posted): slow accrual
Summary
Brief summary (as posted)
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.
Conditions
Conditions (2)
Free-text conditions as registered, with the CancerIndex entity they were reconciled to and the match type.
| Condition (as posted) | Mapped entity | Match | Confidence |
|---|---|---|---|
| Neurofibromatosis Type 2 | — | UNRESOLVED | — |
| Neuroma, Acoustic | Vestibular Schwannoma | ALIAS | 0.90 |
Interventions
Interventions (1)
| Intervention | Type | Mapped drug | Match |
|---|---|---|---|
| RAD001, everolimus | Drug | Everolimus | ALIAS |
Design
Arms and outcomes
Arms (1)
- type
- EXPERIMENTAL
- label
- RAD001 Treatment
- description
- All subjects will be given RAD001 for 1 year (12 months).
- interventionNames
- Drug: RAD001, everolimus
Primary outcomes (1)
- measure
- Vestibular schwannoma volume
- timeFrame
- 1 year (12 months)
- description
- Determine the effect of RAD001 on change in vestibular schwannoma volume (mm3) by MRI from baseline to 1 year.
Secondary outcomes (2)
- measure
- Hearing
- timeFrame
- 1 year (12 months)
- description
Eligibility
Eligibility (as posted)
- Sex
- All
- Minimum age
- 16 Years
- Maximum age
- 65 Years
Show eligibility criteria text
Inclusion Criteria: * Diagnosis of NF2 by National Institutes of Health (NIH) criteria * Age ≥ 16 years * Progressive VS growth during the previous 12 months. * WHO performance status \> or = 2 * Adequate bone marrow, liver and renal function. * For women of childbearing potential, no pregnancy or breast-feeding * Willingness and ability to comply with scheduled visits, drug administration plan, laboratory tests, other study procedures, and study restrictions. * Willingness to provide informed consent Exclusion Criteria: * Inability to tolerate periodic MRI scans or gadolinium contrast. * Inability to tolerate periodic audiologic testing or to understand a language with established scoring for word recognition testing. * Inability to adequately perform volumetric measurement of at least 1 target lesionNote: Patients with cochlear or auditory brainstem implants may participate if a target lesion can be accurately assessed. * Radiation therapy for the target lesion in the 60 months preceding inclusion in the study. * Patients currently receiving anticancer therapies or who have received anticancer therapies within 4 weeks of the start of study drug. * Immunization with attenuated live vaccines within one week of study entry or during study period. * Presence of a fungal infection requiring systemic antifungal treatment at enrollment * Other malignancies within the past 3 years except for adequately treated carcinoma of the cervix or basal or squamous cell carcinomas of the skin. * Patients who have any severe and/or uncontrolled medical conditions. * Patients with a known hypersensitivity to everolimus or other types of rapamycin or to its excipients. * Patients unwilling to or unable to comply with the protocol
References
Publications (3)
- BACKGROUNDGiovannini 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. PMID 24414536
- BACKGROUNDGoutagny 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. PMID 25567352
- DERIVEDNghiemphu 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. PMID 38372904