The University of Nebraska Medical Center is one of more than 60 sites nationally that will participate in a trial testing the safety and effectiveness of a drug for the treatment of monkeypox.
The drug, called tecovirimat or TPOXX, already is being given to patients with monkeypox, said Dr. Sara Bares, an associate professor in UNMC’s infectious diseases division.
But doctors are being very careful when they prescribe it, she said, because good effectiveness data is not available. The drug can be obtained only through the federal Centers for Disease Control and Prevention, under the agency’s expanded access investigational new drug protocol.
The drug is approved by the Food and Drug Administration for use in smallpox. The smallpox virus is in the same family as monkeypox but causes more severe illness.
Researchers involved in the study, called STOMP, or Study of Tecovirimat for Human Monkeypox Virus, want to determine whether the drug is in fact effective in treating monkeypox and, if so, who would benefit most, Bares said. They also want to see how much resistance the virus poses to the drug.
Once they gather the data, she said, they could seek to have the drug approved by the FDA, which would allow patients to get it more easily.
The researchers hope to enroll about 500 people. The phase 3 trial is being led by the AIDS Clinical Trials Group, a national network. It’s sponsored by the National Institute of Allergy and Infectious Diseases, part of the National Institutes of Health. UNMC is the only study site listed in Nebraska.
One challenge will be to enroll enough participants. Reports of new cases in the U.S. have slowed considerably since peaking in August. Nebraska has had 31 confirmed cases out of 28,302 nationwide. Worldwide, more than 77,000 cases have been reported.
“We’re facing this paradox where cases are beginning to trend down everywhere,†Bares said.
Two-thirds of participants will be randomly assigned to get the drug, and the other third will receive a placebo. Anyone who is suspected or confirmed with monkeypox can enroll within 14 days of illness. Those with a severe case can get the medication and still be enrolled.
Anecdotally, Bares said, the drug seems to slow the progression of disease.
“There’s some promise that it works,†Bares said, “but there’s also been some resistance reported in the literature.â€
One of the challenges of monkeypox is that patients have to isolate so long. They may remain contagious for longer than four weeks, which is a long time to be away from friends, family and work. “If this decreases the duration of infectiousness,†she said, “that would be a big win.â€
For information about participating at UNMC, contact Maureen Kubat at mo.kubat@unmc.edu or 402-559-4408; or Jennifer O’Neill at jloneill@unmc.edu or 402-559-4312.