Efficacy and Safety Study of RAD001 in the Growth of the Vestibular Schwannoma(s) in Neurofibromatosis 2 (NF2) Patients

NCT01490476 | Completed Phase 2

• 2 other identifiers: P1012022011-002228-42
• Study Type: interventional
• Target: 10
• Locations: 1 country
• Sites: 1

Brief 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.

Conditions

Neurofibromatosis 2

Keywords

Neurofibromatosis Type 2 with Vestibular Schwannoma; Vestibular Schwannoma; Peripheral Nervous System Diseases; Otorhinolaryngologic Diseases; Vestibulocochlear Nerve Diseases

Outcome Measures

Primary Outcomes (1)

• effect of RAD001 on the VS growth by MRI

  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

  1 year

Secondary Outcomes (1)

• Effect of RAD001 on the volume of other intracranial tumors (MRI) and on hearing function (audiogram)

  1, 2 and 4 years after inclusion in the study

Study Arms (1)

RAD001

EXPERIMENTAL

Patient with Neurofibromatosis Type 2 and Vestibular Schwannoma treated with RAD001.

Drug: RAD001

Interventions

RAD001 DRUG

10 mg per os / day or 05mg per os / day with 12 month

Also known as: Afinitor / rapamycin

RAD001

Eligibility Criteria

• Age: 15 Years+
• Sex: all
• Healthy Volunteers: No
• Age Groups: Child (0-17), Adult (18-64), Older Adult (65+)

You may qualify if:

• 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

You may not qualify if:

• 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.
• 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

Sponsors & Collaborators

• Assistance Publique - Hôpitaux de Paris: lead

Study Sites (1)

Hôpital Beaujon, 100 boulevard du Général Leclerc

Clichy, 92110, France

Location

Related Publications (1)

• 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.

  PMID: 25567352 BACKGROUND

MeSH Terms

Conditions

Neurofibromatosis 2; Neuroma, Acoustic; Peripheral Nervous System Diseases; Otorhinolaryngologic Diseases; Vestibulocochlear Nerve Diseases

Interventions

Everolimus; Sirolimus

Condition Hierarchy (Ancestors)

Neurilemmoma; Neuroendocrine Tumors; Neuroectodermal Tumors; Neoplasms, Germ Cell and Embryonal; Neoplasms by Histologic Type; Neoplasms; Neurofibromatoses; Neurofibroma; Nerve Sheath Neoplasms; Neoplasms, Nerve Tissue; Neuroma; Neoplastic Syndromes, Hereditary; Retrocochlear Diseases; Ear Diseases; Otorhinolaryngologic Neoplasms; Cranial Nerve Neoplasms; Cranial Nerve Diseases; Nervous System Diseases; Neurocutaneous Syndromes; Heredodegenerative Disorders, Nervous System; Neurodegenerative Diseases; Genetic Diseases, Inborn; Congenital, Hereditary, and Neonatal Diseases and Abnormalities; Nervous System Neoplasms; Neoplasms by Site; Peripheral Nervous System Neoplasms; Neuromuscular Diseases

Intervention Hierarchy (Ancestors)

Macrolides; Lactones; Organic Chemicals

Study Officials

• Michel Kalamarides, Professor

  Assistance Publique - Hôpitaux de Paris

  PRINCIPAL INVESTIGATOR

Study Design

• Study Type: interventional
• Phase: phase 2
• Allocation: NA
• Masking: NONE
• Purpose: TREATMENT
• Intervention Model: SINGLE GROUP
• Sponsor Type: OTHER
• Responsible Party: SPONSOR

Study Record Dates

• First Submitted: November 30, 2011
• First Posted: December 13, 2011
• Study Start: January 1, 2012
• Primary Completion: April 1, 2016
• Study Completion: January 1, 2017
• Last Updated: May 23, 2017

Record last verified: 2017-05

Locations

FR (1)