AIDS-Kaposi’s Sarcoma Study Changed Due to Drug Underperformance: Oral Etoposide Plus Antiretroviral Therapy Study Arm Discontinued

A clinical trial funded by the National Institute of Allergy and Infectious Diseases (NIAID) comparing three advanced Kaposi’s sarcoma chemotherapy regimens in combination with antiretroviral treatment (ART) for patients with AIDS will no longer enroll participants in the study arm testing the oral chemotherapy drug etoposide. The decision to discontinue this study arm was made because it under-performed compared to another arm testing the chemotherapy drug paclitaxel in combination with ART. No safety concerns have been found in any of the study’s three investigational treatment options.

The randomized, Phase 3 trial has enrolled 184 men and women in six countries across South America and Africa since it began in October 2013. The trial compared the clinical efficacy of two regimens, oral etoposide plus ART and intravenously delivered bleomycin and vincristine (BV) with ART, to intravenous paclitaxel plus ART for the initial treatment of advanced stage AIDS-Kaposi’s sarcoma. The oral strategy tested by the trial would have offered more flexible methods of treating AIDS-Kaposi’s sarcoma in areas where health care infrastructure cannot support the use of intravenous chemotherapy.

The decision to discontinue the etoposide arm of the trial was made following a March 10, 2016, recommendation to NIAID by the study’s data and safety monitoring board (DSMB), an independent group composed of clinical research experts, statisticians, ethicists and community representatives that meets at regular intervals throughout a study to review its safety and efficacy data.

While limited information was released to preserve the integrity of the blinded study, the DSMB found that trial participants who were randomized to the oral etoposide with ART arm were not improving as well as other participants. The DSMB recommended that participant enrollment into the etoposide plus ART arm halt, and that participants currently taking etoposide plus ART consult with their clinicians to determine their best treatment approach. The DSMB noted that some study participants may benefit from continuing to take etoposide plus ART. The additional arms of the study investigating the combination of the IV-delivered chemotherapy drugs BV with ART as well as the paclitaxel-ART arm will continue enrollment and follow up as originally planned. Participants, site investigators, institutional review boards and regulatory agencies are being notified of the DSMB recommendations and changes to the study.

The trial, conducted by the AIDS Clinical Trial Group with the AIDS Malignancy Consortium, is funded by NIAID and the National Cancer Institute, both part of the NIH. For more information on this trial, visit ClinicalTrials.gov.

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