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Study of RAD001 for Treatment of NF2-related Vestibular Schwannoma

This study is ongoing, but not recruiting participants.
Sponsor:
ClinicalTrials.gov Identifier:
NCT01345136
First Posted: April 29, 2011
Last Update Posted: November 17, 2017
The safety and scientific validity of this study is the responsibility of the study sponsor and investigators. Listing a study does not mean it has been evaluated by the U.S. Federal Government. Read our disclaimer for details.
Collaborator:
Novartis Pharmaceuticals
Information provided by (Responsible Party):
Jonsson Comprehensive Cancer Center
  Purpose

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.


Condition Intervention Phase
Neurofibromatosis Type 2 Neuroma, Acoustic Drug: RAD001, everolimus Phase 2

Study Type: Interventional
Study Design: Intervention Model: Single Group Assignment
Masking: None (Open Label)
Primary Purpose: Treatment
Official Title: A Single Arm, Monocenter Phase II Trial of RAD001 as Monotherapy in the Treatment of Neurofibromatosis Type 2 - Related Vestibular Schwannoma

Resource links provided by NLM:


Further study details as provided by Jonsson Comprehensive Cancer Center:

Primary Outcome Measures:
  • Vestibular schwannoma volume [ Time Frame: 1 year (12 months) ]
    Determine the effect of RAD001 on change in vestibular schwannoma volume (mm3) by MRI from baseline to 1 year.


Secondary Outcome Measures:
  • Hearing [ Time Frame: 1 year (12 months) ]
    Determine the effects of RAD001 treatment on hearing changes (from baseline to 1 year in the ear with the growing vestibular schwannoma.

  • Number of adverse events [ Time Frame: 1 year, 1 month (13 months) ]
    Determine the number of study subjects with adverse events by grade of severity


Enrollment: 4
Actual Study Start Date: July 1, 2015
Estimated Study Completion Date: February 1, 2019
Estimated Primary Completion Date: February 1, 2018 (Final data collection date for primary outcome measure)
Arms Assigned Interventions
Experimental: RAD001 Treatment
All subjects will be given RAD001 for 1 year (12 months).
Drug: RAD001, everolimus
Adults: 10 mg p.o. daily dose, age 16 - 17: 3.0 mg/m2 p.o. daily
Other Names:
  • Everolimus
  • Afinitor

Detailed Description:

This protocol is a Phase II, open-label, efficacy and safety study of single-agent RAD001 in patients with NF2. During the study, subjects will receive continuous daily oral treatment with RAD001 for up to 1 year or until tumor progression.

Primary Objective: 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 Objectives: To determine whether RAD001 has an effect on the volume of other intracranial tumors, and to assess the effect of RAD001 on hearing function in patients with NF2 (when applicable).

  Eligibility

Information from the National Library of Medicine

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Ages Eligible for Study:   16 Years to 65 Years   (Child, Adult)
Sexes Eligible for Study:   All
Accepts Healthy Volunteers:   No
Criteria

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
  Contacts and Locations
Information from the National Library of Medicine

To learn more about this study, you or your doctor may contact the study research staff using the contact information provided by the sponsor.

Please refer to this study by its ClinicalTrials.gov identifier (NCT number): NCT01345136


Locations
United States, California
University of California Los Angeles
Los Angeles, California, United States, 90095-7286
Sponsors and Collaborators
Jonsson Comprehensive Cancer Center
Novartis Pharmaceuticals
Investigators
Principal Investigator: Marco Giovannini, MD, PhD University of California, Los Angeles
  More Information

Publications:
Responsible Party: Jonsson Comprehensive Cancer Center
ClinicalTrials.gov Identifier: NCT01345136     History of Changes
Other Study ID Numbers: 13-001492
First Submitted: April 21, 2011
First Posted: April 29, 2011
Last Update Posted: November 17, 2017
Last Verified: November 2017

Keywords provided by Jonsson Comprehensive Cancer Center:
Neurofibromatosis Type 2
Vestibular schwannoma
Hearing

Additional relevant MeSH terms:
Neurofibromatoses
Neurofibroma
Neuroma
Neurilemmoma
Neuroma, Acoustic
Neurofibromatosis 2
Nerve Sheath Neoplasms
Neoplasms, Nerve Tissue
Neoplasms by Histologic Type
Neoplasms
Neoplastic Syndromes, Hereditary
Neurocutaneous Syndromes
Nervous System Diseases
Heredodegenerative Disorders, Nervous System
Neurodegenerative Diseases
Genetic Diseases, Inborn
Peripheral Nervous System Neoplasms
Nervous System Neoplasms
Peripheral Nervous System Diseases
Neuromuscular Diseases
Neuroendocrine Tumors
Neuroectodermal Tumors
Neoplasms, Germ Cell and Embryonal
Cranial Nerve Neoplasms
Neoplasms by Site
Vestibulocochlear Nerve Diseases
Retrocochlear Diseases
Ear Diseases
Otorhinolaryngologic Diseases
Otorhinolaryngologic Neoplasms