What side effects are associated with this type of intervention?

Common treatments for Neurofibromatosis, particularly using mTOR inhibitors like RAD001, can lead to side effects such as fatigue, anemia, nausea, and increased risk of infections like pneumonia. Other treatments, such as MEK inhibitors (e.g., trametinib), may cause cutaneous toxicity, including rash and paronychia. Targeted therapies can also result in ocular side effects, which are generally reversible. It is important for patients to discuss these potential side effects with their healthcare provider to manage and mitigate them effectively.

What prior FDA approvals exist?

Currently, there are no FDA-approved mTOR inhibitors specifically for the treatment of Neurofibromatosis type 2 (NF2). However, RAD001 (everolimus) is being studied for its potential to shrink or slow the growth of vestibular schwannomas in NF2 patients. Everolimus is an mTOR inhibitor that has been approved by the FDA for other types of tumors, indicating its potential utility in managing NF2-related tumors.

What other types of research exist?

Promising alternatives to RAD001 for Neurofibromatosis patients include bevacizumab, which has shown qualitative improvement in vision for optic pathway gliomas, and selumetinib, which is being tested in ongoing randomized trials for low-grade gliomas. Both treatments have demonstrated potential benefits in clinical settings.

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mTOR inhibitor

Everolimus for Acoustic Neuroma

Phase 2

Waitlist Available

Led By Marco Giovannini, MD, PhD

Research Sponsored by Jonsson Comprehensive Cancer Center

Eligibility Criteria Checklist

Specific guidelines that determine who can or cannot participate in a clinical trial

Must have

Diagnosis of NF2 by National Institutes of Health (NIH) criteria

Age ≥ 16 years

Must not have

Inability to tolerate periodic MRI scans or gadolinium contrast

Immunization with attenuated live vaccines within one week of study entry or during study period

Timeline

Screening 3 weeks

Treatment Varies

Follow Up 1 year, 1 month (13 months)

Awards & highlights

No Placebo-Only Group

Summary

This trial tests if RAD001 can shrink or slow the growth of tumors in NF2 patients and improve their hearing. RAD001 is a pill that blocks signals promoting tumor growth. It has shown effectiveness against various tumor types.

Who is the study for?

This trial is for individuals aged 16 or older with Neurofibromatosis type 2 (NF2) who have seen their vestibular schwannoma grow in the last year. They must be able to undergo MRI scans, not be pregnant or breastfeeding, and have good organ function. People can't join if they've had other cancers recently, severe medical conditions, or are sensitive to everolimus.

What is being tested?

The study tests RAD001 (everolimus), an oral medication approved for some tumors but not NF2-related ones. It aims to see if it can shrink or slow down tumor growth and improve hearing in NF2 patients.

What are the potential side effects?

While specific side effects aren't listed here, drugs like RAD001 may cause immune system suppression leading to infections, mouth sores, skin issues, fatigue, digestive problems and could affect blood sugar levels.

Eligibility Criteria

Inclusion Criteria

You may be eligible if you check “Yes” for the criteria below

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I have been diagnosed with NF2 according to NIH standards.

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I am 16 years old or older.

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My tumor has grown in the last year.

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I need help with my daily activities due to my health condition.

Exclusion Criteria

You may be eligible for the trial if you check “No” for criteria below:

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I cannot undergo MRI scans or use gadolinium contrast.

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I have not received any live vaccines within a week of joining the study or during it.

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I am currently being treated for a fungal infection.

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I do not have any severe or uncontrolled health conditions.

Timeline

Screening ~ 3 weeks3 visits

Treatment ~ Varies

Follow Up ~ 1 year, 1 month (13 months)

Screening ~ 3 weeks

Treatment ~ Varies

Follow Up ~1 year, 1 month (13 months)

This trial's timeline: 3 weeks for screening, Varies for treatment, and 1 year, 1 month (13 months) for reporting.

Treatment Details

Study Objectives

Study objectives can provide a clearer picture of what you can expect from a treatment.

Primary study objectives

Vestibular schwannoma volume

Secondary study objectives

Hearing

Number of adverse events

Awards & Highlights

No Placebo-Only Group

All patients enrolled in this study will receive some form of active treatment.

Trial Design

1Treatment groups

Experimental Treatment

Group I: RAD001 TreatmentExperimental Treatment1 Intervention

All subjects will be given RAD001 for 1 year (12 months).

0 / 8Eligibility criteria met

Research Highlights

Information in this section is not a recommendation. We encourage patients to speak with their healthcare team when evaluating any treatment decision.

Mechanism Of Action

Side Effect Profile

Prior Approvals

Other Research

The most common treatments for Neurofibromatosis, particularly NF2-related schwannomatosis, include mTOR inhibitors such as RAD001 (everolimus). These treatments work by inhibiting the mammalian target of rapamycin (mTOR) pathway, which is often aberrantly activated in NF2-related tumors. By blocking this pathway, mTOR inhibitors can slow down or halt the growth of tumors, such as vestibular schwannomas, and potentially improve symptoms like hearing loss. This is significant for Neurofibromatosis patients as it offers a targeted therapeutic approach that can manage tumor growth and associated symptoms, improving quality of life and reducing the need for surgical interventions.

EPH receptor signaling as a novel therapeutic target in NF2-deficient meningioma.Clinical and Histologic Analysis of the Efficacy of Topical Rapamycin Therapy Against Hypomelanotic Macules in Tuberous Sclerosis Complex.mTORC1 inhibition delays growth of neurofibromatosis type 2 schwannoma.