Clinical trial · Interventional
Efficacy and Safety Study of RAD001 in the Growth of the Vestibular Schwannoma(s) in Neurofibromatosis 2 (NF2) Patients
A Single Arm, Single Center, Phase II Trial of RAD001 as Monotherapy in the Treatment of Neurofibromatosis Type 2 - Related Vestibular Schwannoma
NCT01490476CI-TRIAL-00027744AFINF2completedPhase 2ClinicalTrials.gov clinicaltrialsProvenance
- Source
- ClinicalTrials.gov
- Retrieved
- Sep 8, 2026
- Layer
- normalized (units and labels harmonized; values unchanged)
- Run
- ING-CLINICALTRIALS-20260908-000001
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.
Conditions
Conditions (1)
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 2 | — | UNRESOLVED | — |
Interventions
Interventions (1)
| Intervention | Type | Mapped drug | Match |
|---|---|---|---|
| RAD001 | Drug | Everolimus | ALIAS |
Design
Arms and outcomes
Arms (1)
- type
- EXPERIMENTAL
- label
- RAD001
- description
- Patient with Neurofibromatosis Type 2 and Vestibular Schwannoma treated with RAD001.
- interventionNames
- Drug: RAD001
Primary outcomes (1)
- measure
- effect of RAD001 on the VS growth by MRI
- timeFrame
- 1 year
- description
- To determine whether RAD001 has an effect on the VS growth in patients with NF2 at a rate sufficient to submit the drug for further testing
Secondary outcomes (1)
- measure
- Effect of RAD001 on the volume of other intracranial tumors (MRI) and on hearing function (audiogram)
- timeFrame
- 1, 2 and 4 years after inclusion in the study
Eligibility
Eligibility (as posted)
- Sex
- All
- Minimum age
- 15 Years
Show eligibility criteria text
Inclusion Criteria: * Diagnosis of NF2 by National Institutes of Health (NIH) criteria * Age ≥ 15 years * Progressive VS growth during the previous 12 months. Evidence of disease progression defined by progressive VS during the previous 12 months (\>20% increase in volume) in subjects who are at elevated risk for surgical complications (eg, deafness, lower cranial nerve injury, facial weakness) or who refuse surgery * 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. * Patients receiving chronic, systemic treatment with corticosteroids or another immunosuppressive agent. Topical or inhaled corticosteroids are allowed. * 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 (1)
- 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