A Single Arm, Single Center, Phase II Trial of RAD001 as Monotherapy in the Treatment of Neurofibromatosis Type 2 - Related Vestibular Schwannoma
15 Years
N/A
Both
Neurofibromatosis 2
Trial Information
A Single Arm, Single Center, Phase II Trial of RAD001 as Monotherapy in the Treatment of Neurofibromatosis Type 2 - Related Vestibular Schwannoma
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; to assess the effect of RAD001 on hearing function in patients with NF2
(when applicable); to determine whether RAD001 modulates signaling pathways in intracranial
NF2 tumors removed during the course of the study.
All patients will be treated with RAD001 10 mg p.o. daily dose (5mg if 15-17 years old with
cutaneous surface area <1.5m2) for one year except in case of unacceptable toxicity, death,
or discontinuation from the study for any other reason.
At 12 months an extension for another one year of RAD001 treatment will be discussed in case
of response. All patients will have a follow-up visit (including MRI) scheduled at 12 months
after the last dose of the study drug.
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
Type of Study:
Interventional
Study Design:
Endpoint Classification: Safety/Efficacy Study, Intervention Model: Single Group Assignment, Masking: Open Label, Primary Purpose: Treatment
Outcome Measure:
effect of RAD001 on the VS growth by MRI
Outcome 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
Outcome Time Frame:
1 year
Safety Issue:
No
Principal Investigator
Michel Kalamarides, Professor
Investigator Role:
Principal Investigator
Investigator Affiliation:
Assistance Publique - Hôpitaux de Paris
Authority:
France: Ministry of Health
Study ID:
P101202
NCT ID:
NCT01490476
Start Date:
January 2012
Completion Date:
March 2014
Related Keywords:
- Neurofibromatosis 2
- Neurofibromatosis Type 2 with Vestibular Schwannoma
- Vestibular Schwannoma
- Peripheral Nervous System Diseases
- Otorhinolaryngologic Diseases
- Vestibulocochlear Nerve Diseases
- Neurilemmoma
- Neurofibromatoses
- Neurofibromatosis 1
- Osteitis Fibrosa Cystica
- Neurofibromatosis 2
- Neuroma, Acoustic
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