Study of RAD001 for Treatment of NF2-related Vestibular Schwannoma
This study is currently recruiting participants.
Verified December 2012 by House Research Institute
Sponsor:
House Research Institute
Collaborator:
Novartis Pharmaceuticals
Information provided by (Responsible Party):
Marco Giovannini, MD, PhD, House Research Institute
ClinicalTrials.gov Identifier:
NCT01345136
First received: April 21, 2011
Last updated: December 7, 2012
Last verified: December 2012
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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 House Research Institute:
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: | 10 |
| Study Start Date: | December 2012 |
| Estimated Study Completion Date: | January 2015 |
| Estimated Primary Completion Date: | January 2015 (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:
|
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
Please refer to this study by its ClinicalTrials.gov identifier: NCT01345136
Contacts
| Contact: Marco Giovannini, MD, PhD | 213-989-6708 | mgiovannini@hei.org |
Locations
| United States, California | |
| House Research Institute | Recruiting |
| Los Angeles, California, United States, 90057 | |
| Principal Investigator: Marco Giovannini, MD, PhD | |
Sponsors and Collaborators
House Research Institute
Novartis Pharmaceuticals
Investigators
| Principal Investigator: | Marco Giovannini, MD, PhD | House Research Institute |
More Information
No publications provided
| Responsible Party: | Marco Giovannini, MD, PhD, Director, Division of Clinical and Translational Research, House Research Institute |
| ClinicalTrials.gov Identifier: | NCT01345136 History of Changes |
| Other Study ID Numbers: | RAD001 |
| Study First Received: | April 21, 2011 |
| Last Updated: | December 7, 2012 |
| Health Authority: | United States: Food and Drug Administration |
Keywords provided by House Research Institute:
|
Neurofibromatosis Type 2 Vestibular schwannoma Hearing |
Additional relevant MeSH terms:
|
Neurilemmoma Neurofibromatosis 2 Neuroma Neuroma, Acoustic Neurofibromatoses Neuroendocrine Tumors Neuroectodermal Tumors Neoplasms, Germ Cell and Embryonal Neoplasms by Histologic Type Neoplasms Nerve Sheath Neoplasms Neoplasms, Nerve Tissue Peripheral Nervous System Neoplasms Nervous System Neoplasms Nervous System Diseases |
Peripheral Nervous System Diseases Neuromuscular Diseases Neurofibroma Neoplasms by Site Neoplastic Syndromes, Hereditary Vestibulocochlear Nerve Diseases Retrocochlear Diseases Ear Diseases Otorhinolaryngologic Diseases Otorhinolaryngologic Neoplasms Cranial Nerve Neoplasms Cranial Nerve Diseases Neurocutaneous Syndromes Heredodegenerative Disorders, Nervous System Neurodegenerative Diseases |
ClinicalTrials.gov processed this record on May 23, 2013