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Patient with Neurofibromatosis Type 2 and Vestibular Schwannoma treated with RAD001.

10 mg per os / day or 05mg per os / day with 12 month

RAD001

Michel Kalamarides, Professor

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.

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 4 years 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. And determine the long term safety.

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.

If the patient is stable (decrease or increase lower than 20%), treatment should be stopped and the patient should be kept under quarterly continuous surveillance. Resumption of treatment will be discussed case by case basis if tumor regrowth over 20% relative to the tumor volume at the end of treatment.

All patients will have a follow-up visit (including MRI) scheduled at 24 months after enrollment.

We modify the protocol to extend the treatment by RAD001 for a period of two additional years for stable patients still on treatment two years after enrollment.

Efficacy and Safety Study of RAD001 in the Growth of the Vestibular Schwannoma(s) in Neurofibromatosis 2 (NF2) Patients

A Single Arm, Single Center, Phase II Trial of RAD001 as Monotherapy in the Treatment of Neurofibromatosis Type 2 - Related Vestibular Schwannoma

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

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 and to determine long term safety

Assistance Publique - Hôpitaux de Paris

Urinary Tract, Sexual Organs, and Pregnancy Conditions

Gland and Hormone Related Diseases

Testicular Neoplasms

Endocrine Gland Neoplasms

Genital Neoplasms, Male

Urogenital Neoplasms

Genital Diseases

Genital Diseases, Male

Urogenital Diseases

Male Urogenital Diseases

Endocrine System Diseases

Testicular Diseases

Gonadal Disorders

Cisplatin

Diagnosis of testicular cancer; History of any grade 3 or higher peripheral neuropathy after receiving standard dose cisplatin completed more than 1 year but within the last 5 years; Long-term persistence (\> 6 months) of grade 2 or higher peripheral neuropathy after completion of a cisplatin containing regimen. Interventions: blood sample collection and report of peripheral neuropathy after cisplatin therapy.

Diagnosis of testicular cancer; No history of neurotoxicity (grade 0-1) after completion of a standard cisplatin-containing chemotherapy regimen completed more than 1 year but within the last 5 years; Matched to a specified subject with neurotoxicity based on age (within 10 years), chemotherapy regimen or total cisplatin dosage. Interventions: blood sample collection and report of peripheral neuropathy after cisplatin therapy.

Six tubes of blood will be collected during one blood draw.

Blood sample collection

Twenty questions about the peripheral neuropathy the patient is currently experiencing, and twenty questions about the worst peripheral neuropathy the patient ever experienced after cisplatin treatment.

Report of peripheral neuropathy after cisplatin therapy

Costantine Albany, MD

This study targets patients with cisplatin-induced peripheral neuropathy and will allow us to: a) study peripheral neuropathy in diverse human sensory neurons in ways that were not possible previously; b) gain insight on druggable targets to treat or prevent this devastating side effect of chemotherapy; c) provide a human cellular model that can be used for screening of drugs to determine if they are neurotoxic. The combination of patient information and in vitro measurements provides a highly relevant and clinically useful model for studies aimed to impact treatment selection for the individual cancer patient.

Primary Objective-To establish a biobank of specimens that allows for the future creation of iPS cells with the potential to make human neurons for functional studies of specific genes/genetic variants.

Secondary Objectives-To develop a clinically relevant in vitro model of cisplatin induced peripheral neuropathy to be used to 1) screen new drugs for the treatment or prevention of peripheral neuropathy; 2) evaluate candidate genes for their role in cisplatin induced neuropathy.

Study Procedures-Subjects who wish to participate will undergo the following study procedures, which will be performed after the informed consent document is obtained:

1. Sample Collection-A trained phlebotomist will conduct a blood draw for the collection of SIX samples for peripheral blood mononuclear cell (PBMC) isolation and infectious disease testing.
2. Patient Self-Report of Toxicities-All patients in the study will be asked to fill out the Patient-reported Toxicity Form. This will take approximately 10 minutes to complete and consists of 20 questions related to patient symptoms describing toxicities experienced secondary to receiving cisplatin chemotherapy. They will be asked to fill out the form twice: (1) describing the peripheral neuropathy they currently are experiencing and (2) describing the worst peripheral neuropathy toxicity they experienced as a result of cisplatin therapy. These paper forms will be filled out at time of enrollment in the study.
3. Physician Report of Toxicities-The patient's physician or the designatee will complete an adverse event form at day of enrollment detailing the physician's assigned grade of toxicities (according to CTCAE version 4.3) experienced from cisplatin. Other information regarding the particularly severe neurotoxicity may also be collected from physicians (e.g. patient use of walker or wheelchair, patient application for disability based on neuropathy).
4. General Information- This information will be collected at the same time point as all of the other study procedures (i.e., Day One). This will include but may not be limited to: age, height, weight, patient-identified race and ethnicity, information about the chemotherapy regimen, total cisplatin dose administered and date of last cisplatin dose.
5. Cisplatin Dose-Total cumulative dose of cisplatin administered will be recorded. Outside records will be obtained where needed.
6. Follow-up and Duration of Follow-Up-There will be no subsequent patient follow-up after time of enrollment. Subjects may withdraw consent for specimen use by contacting the PI in writing.

Whole blood samples will be obtained from all patients so that peripheral blood mononuclear cells (PBMCs) can be isolated and stored for the eventual creation of iPS cells. iPS cells have the potential to make human neurons that the researchers would like to use for functional studies of specific genes/genetic variants. All samples will also be tested for infectious diseases (Hepatitis B, Hepatitis C, HIV 1 and HIV 2).

Establishing of Neuronal-like Cells From Patients With Cisplatin-Induced Peripheral Neuropathy

PBMCs will be used for the future creation of iPS cells with the potential to make human neurons

Costantine Albany

Clinical Faculty

Study title	NCT ID	Conditions	Interventions	Eligibility	Organization	Link
Efficacy and Safety Study of RAD001 in the Growth of the Vestibular Schwannoma(s) in Neurofibromatosis 2 (NF2) Patients	NCT01490476	Neurofibromatos...	RAD001	15 Years -	Assistance Publique - Hôpitaux de Paris
Establishing of Neuronal-like Cells From Patients With Cisplatin-Induced Peripheral Neuropathy	NCT02492360	Peripheral Nerv... Testicular Neop...	Blood sample co... Report of perip...	18 Years -	Indiana University

Spots Global Cancer Trial Database for peripheral nervous system diseases