Trial record 8 of 18 for:
"Acoustic neuroma"
Efficacy and Safety Study of RAD001 in the Growth of the Vestibular Schwannoma(s) in Neurofibromatosis 2 (NF2) Patients (AFINF2)
This study is ongoing, but not recruiting participants.
Sponsor:
Assistance Publique - Hôpitaux de Paris
Information provided by (Responsible Party):
Assistance Publique - Hôpitaux de Paris
ClinicalTrials.gov Identifier:
NCT01490476
First received: November 30, 2011
Last updated: February 13, 2013
Last verified: February 2013
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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.
| Condition | Intervention | Phase |
|---|---|---|
|
Neurofibromatosis 2 |
Drug: RAD001 |
Phase 2 |
| Study Type: | Interventional |
| Study Design: | Endpoint Classification: Safety/Efficacy Study Intervention Model: Single Group Assignment Masking: Open Label Primary Purpose: Treatment |
| Official Title: | A Single Arm, Single Center, 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 Assistance Publique - Hôpitaux de Paris:
Primary Outcome Measures:
- effect of RAD001 on the VS growth by MRI [ Time Frame: 1 year ] [ Designated as safety issue: No ]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 Outcome Measures:
- Effect of RAD001 on the volume of other intracranial tumors (MRI) and on hearing function (audiogram) [ Time Frame: 1 year and 2 years after inclusion in the study ] [ Designated as safety issue: No ]To determine if RAD001 has an effect on the volume of other intracranial tumors, to assess efficacy of RAD001 on hearing function, to determine whether RAD001 modulates signaling pathways in tumors removed during the course of the study.
| Enrollment: | 10 |
| Study Start Date: | January 2012 |
| Estimated Study Completion Date: | March 2014 |
| Estimated Primary Completion Date: | December 2013 (Final data collection date for primary outcome measure) |
| Arms | Assigned Interventions |
|---|---|
|
Experimental: RAD001
Patient with Neurofibromatosis Type 2 and Vestibular Schwannoma treated with RAD001.
|
Drug: RAD001
10 mg per os / day or 05mg per os / day with 12 month
Other Name: Afinitor / rapamycin
|
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; 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.
Eligibility| Ages Eligible for Study: | 15 Years and older |
| Genders Eligible for Study: | Both |
| Accepts Healthy Volunteers: | No |
Criteria
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
Contacts and Locations
Please refer to this study by its ClinicalTrials.gov identifier: NCT01490476
Locations
| France | |
| Hôpital Beaujon, 100 boulevard du Général Leclerc | |
| Clichy, France, 92110 | |
Sponsors and Collaborators
Assistance Publique - Hôpitaux de Paris
Investigators
| Principal Investigator: | Michel Kalamarides, Professor | Assistance Publique - Hôpitaux de Paris |
More Information
No publications provided
| Responsible Party: | Assistance Publique - Hôpitaux de Paris |
| ClinicalTrials.gov Identifier: | NCT01490476 History of Changes |
| Other Study ID Numbers: | P101202, 2011-002228-42 |
| Study First Received: | November 30, 2011 |
| Last Updated: | February 13, 2013 |
| Health Authority: | France: Ministry of Health |
Keywords provided by Assistance Publique - Hôpitaux de Paris:
|
Neurofibromatosis Type 2 with Vestibular Schwannoma Vestibular Schwannoma Peripheral Nervous System Diseases Otorhinolaryngologic Diseases Vestibulocochlear Nerve Diseases |
Additional relevant MeSH terms:
|
Neurilemmoma Neurofibromatosis 2 Neuroma, Acoustic Neurofibromatoses Neuroendocrine Tumors Neuroectodermal Tumors Neoplasms, Germ Cell and Embryonal Neoplasms by Histologic Type Neoplasms Nerve Sheath Neoplasms Neoplasms, Nerve Tissue Neuroma 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 22, 2013