Thalidomide effectively and safely heals severe mouth ulcers (also called oral aphthous ulcers) in persons with HIV infection, according to an interim analysis of data from a placebo-controlled study supported by the National Institute of Allergy and Infectious Diseases (NIAID).
The study (ACTG 251), sponsored through NIAID's AIDS Clinical Trials Group, compares the effectiveness and safety of thalidomide with placebo for treating patients with severe oral and esophageal ulcers. In the second or maintenance phase of the study, doctors are evaluating thalidomide's ability to prevent recurrences of ulcers in the mouth or the esophagus. As a result of the findings of the interim analysis, current and newly enrolled study participants with oral ulcers will all receive open-label thalidomide rather than being randomized to receive either placebo or drug. Patients with esophageal ulcers will continue to be randomized. The maintenance phase of the study will continue as originally planned.
"Many patients with HIV infection suffer from these extremely painful mouth ulcers, making eating difficult and contributing to weight loss and debilitation," says Anthony S. Fauci, M.D., director of NIAID. "Thalidomide is the first treatment shown in a rigorous scientific study to heal these ulcers. As the study continues, we anticipate additional valuable information about the drug's effectiveness and long-term toxicity."
As planned in the study design, an Ad Hoc Interim Review Committee looked at data from patients with mouth ulcers who had received four weeks of treatment with either placebo or 200 mg of thalidomide taken by mouth each day. The committee found that the ulcers had healed in 14 of 23 patients receiving thalidomide compared with only one out of 22 patients receiving placebo. The effectiveness of thalidomide for patients with ulcers in the esophagus could not be evaluated because of limited patient enrollment. The committee also looked at the safety data available for the first 73 patients enrolled in either the mouth ulcer or esophageal ulcer part of the trial and found no significant differences in severe side effects that could be attributable to either thalidomide or placebo.
According to study chair Jeffrey M. Jacobson, M.D., of the Bronx Veterans Affairs Medical Center and Mt. Sinai School of Medicine in New York, ACTG 251 is continuing to enroll HIV-infected men and women at ACTG sites across the United States. Study plans call for 164 participants. Thalidomide is known to cause malformations in infants born to women taking the drug, and women of child-bearing age who participate in ACTG 251 are fully informed about this risk. The women must agree not to become pregnant either by abstaining from sexual intercourse or by using a hormonal contraceptive as well as two barrier-type contraceptives. They also must agree to frequent tests for pregnancy. Thalidomide for this study is provided by Andrulis Pharmaceutical Corporation.
For more information about this and other AIDS clinical trials, please call the AIDS Clinical Trials Information Service at 1-800-TRIALS-A (1-800-874-2572), Monday through Friday, 9:00 a.m. to 7:00 p.m. ET.
NIAID is a component of the National Institutes of Health (NIH), an agency of the U.S. Department of Health and Human Services. NIAID supports basic and applied research to prevent, diagnose and treat infectious diseases such as HIV/AIDS and other sexually transmitted infections, influenza, tuberculosis, malaria and illness from potential agents of bioterrorism. NIAID also supports research on transplantation and immune-related illnesses, including autoimmune disorders, asthma and allergies.
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