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Trial record 3 of 27 for:    "Neurofibromatosis type 2"

Study of RAD001 for Treatment of NF2-related Vestibular Schwannoma

This study is ongoing, but not recruiting participants.
Sponsor:
Collaborator:
Novartis Pharmaceuticals
Information provided by (Responsible Party):
Marco Giovannini, M.D.,Ph.D., University of California, Los Angeles
ClinicalTrials.gov Identifier:
NCT01345136
First received: April 21, 2011
Last updated: November 4, 2014
Last verified: November 2014
  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: Endpoint Classification: Efficacy Study
Intervention Model: Single Group Assignment
Masking: 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 University of California, Los Angeles:

Primary Outcome Measures:
  • Vestibular schwannoma volume [ Time Frame: 1 year (12 months) ] [ Designated as safety issue: No ]
    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) ] [ Designated as safety issue: No ]
    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) ] [ Designated as safety issue: Yes ]
    Determine the number of study subjects with adverse events by grade of severity


Estimated Enrollment: 25
Study Start Date: December 2012
Estimated Study Completion Date: February 2017
Estimated Primary Completion Date: February 2017 (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

Ages Eligible for Study:   16 Years to 65 Years
Genders Eligible for Study:   Both
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
Choosing to participate in a study is an important personal decision. Talk with your doctor and family members or friends about deciding to join a study. To learn more about this study, you or your doctor may contact the study research staff using the Contacts provided below. For general information, see Learn About Clinical Studies.

Please refer to this study by its ClinicalTrials.gov identifier: NCT01345136

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

No publications provided

Responsible Party: Marco Giovannini, M.D.,Ph.D., Professor in Residence, University of California, Los Angeles
ClinicalTrials.gov Identifier: NCT01345136     History of Changes
Other Study ID Numbers: RAD001
Study First Received: April 21, 2011
Last Updated: November 4, 2014
Health Authority: United States: Food and Drug Administration

Keywords provided by University of California, Los Angeles:
Neurofibromatosis Type 2
Vestibular schwannoma
Hearing

Additional relevant MeSH terms:
Neurofibromatosis 2
Neurilemmoma
Neurofibromatoses
Neurofibromatosis 1
Neuroma, Acoustic
Cranial Nerve Diseases
Cranial Nerve Neoplasms
Ear Diseases
Genetic Diseases, Inborn
Heredodegenerative Disorders, Nervous System
Neoplasms
Neoplasms by Histologic Type
Neoplasms by Site
Neoplasms, Germ Cell and Embryonal
Neoplasms, Nerve Tissue
Neoplastic Syndromes, Hereditary
Nerve Sheath Neoplasms
Nervous System Diseases
Nervous System Neoplasms
Neurocutaneous Syndromes
Neurodegenerative Diseases
Neuroectodermal Tumors
Neuroendocrine Tumors
Neurofibroma
Neuroma
Neuromuscular Diseases
Otorhinolaryngologic Diseases
Otorhinolaryngologic Neoplasms
Peripheral Nervous System Diseases
Peripheral Nervous System Neoplasms

ClinicalTrials.gov processed this record on November 20, 2014