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Zafgen Clinical Trial Will Close Experimental Arm Following Death

Open-label extension portion in patients with Prader-Willi syndrome will continue

A trial studying the medication beloranib that was halted last week due to a patient death will close its experimental arm but continue the open-label extension portion of the study.

According to the Boston Globe, Zafgen Inc. said last week that the FDA had ordered a “partial clinical hold” on the trial in order to screen patients for thrombotic diseases. The company also confirmed that the dead patient was taking its experimental drug, not a placebo.

Zafgen confirmed the death on October 14 but provided few other details. In a conference call October 16, Zafgen officials said the patient who died was 23 years old, obese, and died about two weeks ago, but they said the cause of death was unknown. People with Prader-Willi syndrome are often obese because the illness causes feelings of chronic hunger. The FDA imposed the partial clinical hold orally, the company said.

Dr. Dennis Kim, the company’s chief medical officer, said the FDA would allow patients to resume the trial as soon as Zafgen obtained informed consent from them and screened them for blood-clot disorders, a process he estimated would take no more than two weeks. He said three-quarters of patients in the trial — one of two important trials planned by the company to show the drug’s effectiveness — had already completed the six-month treatment regimen.

Dr. Thomas Hughes, Zafgen’s chief executive, said the company reported the patient’s death within a week of learning about it, as required by FDA regulations. A company spokeswoman said “it would have been inappropriate to discuss this event publicly while the company was in initial dialogue with the FDA.” Hughes added that the company had actually expected “one or two deaths” during the trial based on the known mortality rate of people with Prader-Willi syndrome.

On October 22, according to The Wall Street Journal, Zafgen stated that it would end the randomized portion of the trial studying beloranib as a treatment for Prader-Willi syndrome and another treating patients with severe obesity complicated by type-2 diabetes. The company said it plans to report the results of the clinical trial in the first three months of 2016.

Source: Boston Globe, October 16, 2015; and The Wall Street Journal, October 22, 2015.

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