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Kaposi’s Sarcoma Clinical Trial
Frequently Asked Questions |
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Clinical Trial of PTC299 in Patients with HIV-Related
Kaposi’s Sarcoma Frequently Asked Questions
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What is PTC299?
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What information is currently available about the safety and efficacy of PTC299?
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What is the purpose and design of the PTC299 clinical trial in patients with HIV-related KS?
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Why should patients consider participating in the trial?
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Who will be eligible to participate in the trial?
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Will there be any cost to participate in the trial?
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What is PTC299? |
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PTC299 is an investigational new drug, which means it is being tested as a potential treatment for certain types of cancers and it has not yet been approved for sale by the U.S. Food and Drug Administration (FDA). PTC299 is currently being investigated in clinical trials in patients with HIV-related Kaposi’s sarcoma (KS), metastatic breast cancer, neurofibromatosis type 2, and other tumors. Additional clinical trials in patients with other types of tumors are planned.
Researchers have come to understand that KS tumor cells make high levels of a protein called vascular endothelial growth factor (VEGF). VEGF stimulates formation of new blood vessels that feed tumors with oxygen and nutrients, helping them to grow. Inhibiting VEGF may slow new blood vessel formation and may slow tumor growth. Drugs targeting new blood vessel formation in tumors are currently available to treat cancers of the brain, lung, breast, colon and rectum, liver, and kidney.
PTC299 may offer a new approach to antitumor therapy in KS. PTC299 is designed to block tumor-produced VEGF and not the VEGF necessary to maintain the growth and function of normal blood vessels. In addition, unlike some available drugs that block or bind to VEGF and must be taken by intravenous injection, PTC299 is taken by mouth.
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What information is currently available about the safety and efficacy of PTC299? |
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Short duration, early phase clinical trials in healthy volunteers and patients with cancer have evaluated the safety of PTC299. Results from these trials show that symptoms observed in people receiving PTC299 have been infrequent, mostly mild to moderate, and not usually considered to be caused by PTC299, even at higher doses than those being used in the PTC299 clinical trial in KS.
The available efficacy information for PTC299 has been derived from animal experiments. In these animal studies, PTC299 decreases the amount of VEGF made by tumors. In addition, PTC299 slows or stops the growth of tumors or causes them to shrink.
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What is the purpose and design of the PTC299 clinical trial in patients with HIV-related KS? |
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This study is testing PTC299 to learn more about the safety and activity of the drug at different doses. In addition, the study is testing if PTC299 lowers levels of VEGF in the blood and makes Kaposi’s sarcoma tumors get smaller. The study will also measure the effect of PTC299 on HIV viral load and T cells.
Participants in this study will take PTC299 by mouth twice per day. Each participant will take the drug in 28-day treatment cycles. Cycles of treatment can continue for up to 48 weeks. Patients will continue taking their usual treatments for HIV infection.
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Why should patients consider participating in the trial? |
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Patients may consider participating in the trial to try an oral medication. While no benefit can be guaranteed, it is possible that PTC299 may slow the growth of KS or decrease the size of KS tumors.
As with other clinical trials, study participants have the opportunity to have their disease closely monitored by cancer experts and contribute to medical research and the broader understanding of HIV-related KS.
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Who will be eligible to participate in the trial? |
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To be eligible for this trial, patients must be at least 18 years old, be HIV-positive, and have a diagnosis of KS. Participants must also discontinue other therapies for KS, such as chemotherapy, at least four weeks prior to enrollment. Additional eligibility requirements will be explained at the trial site.
Before being considered for enrollment in the trial, a patient must also be informed about the trial by the medical researchers and must sign a document called an informed consent form, indicating a willingness to participate and to follow the study requirements. This document explains the trial in great detail to allow patients to assess the risks and benefits of participation. A copy of the informed consent form is provided to each patient.
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Will there be any cost to participate in the trial? |
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Participants will not pay for PTC299 or laboratory tests that are needed to complete the trial. However, some costs associated with routine medical care may be billed to participants’ insurance providers.
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