TKI 258 in Von Hippel-Lindau Syndrome (VHL)

Von Hippel-Lindau Syndrome Clinical Trial

Official title: A Pilot Trial of TKI 258 (Dovitinib) in Von Hippel-Lindau Syndrome

Status Terminated

Clinical Trial Summary

The goal of this clinical research study is to learn if dovitinib can safely be given to patients who have VHL with a measurable hemangioblastoma (tumor of the central nervous system). The effects of this drug on the disease will also be studied.

Clinical Trial Description

The Study Drug:

Dovitinib is designed to perform several anti-tumor functions, including cutting off the blood supply to tumors.

Study Drug Administration:

If you are found to be eligible to take part in this study, you will take 5 dovitinib capsules by mouth each day on Days 1-5, 8-12, 15-19, and 22-26 of each 28-day cycle.

You should take the dovitinib capsules with about a cup (8 ounces) of water and at least 1 hour before breakfast or at least 2 hours following breakfast.

If you have any side effects from the drug, tell the study doctor right away. The study doctor may then lower the dose or keep the dose level the same.

If you miss a dose of dovitinib on Days 1-4 (or 8-11, 15-18, or 22-25), you should not make up the dose on the same day. You should continue taking the drug as scheduled the following day. If you miss a dose on Day 5 (or 12, 19, or 26), you should skip the dose, rest 2 days, and begin dosing again as scheduled on Day 8 (or 15, 22, or 1 of the next cycle). The study doctor will tell you about any additional steps that should be taken if you miss a dose.

Every 4 weeks on this study is called a study "cycle."

Study Visits:

On Day 1 of Cycle 1, you will have an ECG.

On Day 14 (+/- 3 days) of Cycles 1 and 2:

-Blood (about 3 teaspoons) will be drawn for routine tests. This blood testing may be done at your local doctor's office and faxed to MD Anderson. You will be provided instructions about how to fax laboratory test results.

On Day 1 of Cycles 3 and Beyond (+/- 3 days), blood (about 3 teaspoons) will be drawn for routine tests.

Every 8 weeks (+/- 3 days):

• Any changes to your medical history since your last visit will be recorded.

• You will have a physical exam, including measurement of your vital signs and weight.

• You will be asked about any drugs or treatments you may be receiving.

• Your performance status will be recorded.

• You will be asked about any side effects that you have had since your last visit.

At the End of Cycles 2 and 4:

• You will have CT scans and MRI scans to check the status of the disease.

• If the doctors know or suspect that VHL is affecting your eyes, you will have an eye exam.

Length of Study:

You may continue taking the study drugs for as long as you are benefiting. You will be taken off study if the disease gets worse or intolerable side effects occur.

Early Withdrawal /End of Treatment Visit:

• About 30 days after you stop taking study drug, you will have the following procedures:

• You will have a physical exam, including measurement of your vital signs and weight.

• Your performance status will be recorded.

• You will be asked about any drugs or treatments you may be receiving.

• You will be asked about any side effects that you may have had since your last visit.

• Blood (about 3 teaspoons) will be drawn for routine tests.

• You will have CT scans and MRI scans to check the status of the disease.

• If the doctors know or suspect that VHL is affecting your eyes, you will have an eye exam.

About 24 weeks after your last dose of study drug, you will have the following procedures:

• You will have a physical exam, including measurement of your vital signs and weight.

• You will have follow-up imaging scans to check the status of the disease.

• Blood (about 3 teaspoons) will be collected for routine tests.

• You will be asked about any drugs or treatments you may be receiving.

• You will be asked about any side effects that you may have had since your last visit.

This is an investigational study. Dovitinib is not FDA approved or commercially available. It is currently being used for research purposes only.

Up to 25 patients will take part in this study. All will be enrolled at MD Anderson. ;

Study Design

Endpoint Classification: Safety/Efficacy Study, Intervention Model: Single Group Assignment, Masking: Open Label, Primary Purpose: Treatment

Related Conditions & MeSH terms

• Syndrome
• Von Hippel-Lindau Disease
• Von Hippel-Lindau Syndrome

• NCT number: NCT01266070
• Study type: Interventional
• Source: M.D. Anderson Cancer Center
• Status: Terminated
• Phase: Phase 2
• Start date: November 2012
• Completion date: December 2015