Kaposi’s sarcoma is the most common cancer in people infected with the human immunodeficiency virus (HIV). Kaposi’s sarcoma tumors make high levels of a protein called vascular endothelial growth factor (VEGF). VEGF causes new blood vessels to grow. These new blood vessels help tumors to grow by feeding them with nutrients and oxygen.

Inhibiting VEGF may slow the growth of tumors by preventing new blood vessels from forming. Drugs targeting new blood vessel formation in tumors are currently available to treat other types of cancer, such as those of the brain, lung, breast, colon and rectum, kidney, and liver.

PTC Therapeutics has developed an oral investigational drug called PTC299. PTC299 is designed to block tumor-produced VEGF and not the VEGF necessary to maintain the growth and function of normal blood vessels.

In animal studies, PTC299 has been shown to decrease tumor VEGF and slow or stop the growth of tumors or cause them to shrink. Short duration, early phase clinical trials in healthy adult volunteers and patients with cancer have evaluated the safety of PTC299. Results from these trials show that adverse events observed in people receiving PTC299 at high doses have been infrequent, mostly mild to moderate, and not usually considered to be caused by PTC299.

New Clinical Trial of Oral PTC299 in Patients with HIV-Related Kaposi’s Sarcoma

The AIDS Malignancy Consortium (AMC), a treatment group funded by the National Cancer Institute, and PTC Therapeutics are testing PTC299 in adults with HIV-related Kaposi’s sarcoma at medical centers in the United States.

This study is testing PTC299 to learn more about the safety and activity of the drug at different doses. 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 take PTC299 by mouth twice per day. Each participant takes the drug in 28-day treatment cycles. Cycles of treatment can continue for up to 48 weeks as long as PTC299 appears to be safely offering tumor control. Participants will continue taking their usual treatments for HIV infection.

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.

Requirements for Participating in This Study

To be eligible for the trial, participants must:
• be at least 18 years old
• be HIV-positive
• have a diagnosis of Kaposi’s sarcoma
• discontinue other therapies for Kaposi’s sarcoma, such as chemotherapy, at least four weeks prior to enrollment
• be willing and able to participate in study procedures

Other eligibility requirements will be explained at the trial site.

Additional information about this ataluren clinical trial can be found at www.clinicaltrials.gov or by contacting Patient and Professional Advocacy at (866) 282-5873 (toll free in the United States), (908) 912-9256 or PatientInfo@ptcbio.com.