University of Alberta clinical researcher leading genetic study with drug trial to help patients with rare genetic disorders lose weight.
Credit: University of Alberta
The Alberta Diabetes Institute (ADI) will be at the centre of a new clinical trial for an anti-obesity drug targeting people with rare genetic conditions that contribute to their obesity. Led by Andrea Haqq, clinical scientist at the ADI and associate professor in the University of Alberta’s Department of Pediatrics, the drug trial will also be tied to a genetic study already underway attempting to create a more comprehensive picture of the genes that are linked to obesity.
“One thing that people don’t always recognize about obesity, especially childhood obesity, is that it is very complex. It’s not as simple as eating too much,” said Haqq. “One of those complex components is the genetic influences on obesity, which actually account for 60 to 70 per cent of what sets our body mass index (BMI).”
The clinical trial, sponsored by Rhythm Pharmaceuticals and expected to begin in late July, will test the effects of the drug setmelanotide on participants with obesity who have either Bardet-Biedl syndrome or Alström syndrome, two rare genetic disorders that include obesity as a symptom. In studies of adults with obesity with other rare genetic disorders, setmelanotide was found to have a profound effect on weight loss–nearly 50 per cent in some cases.
“What’s really exciting about this drug is that this is one of the first anti-obesity drugs that doesn’t have the cardiovascular side-effects that previous drugs had,” she said. “As we gain more experience with it, I’m hopeful this trial will make a big splash and lead to other trials for other populations.”
The trial will run over the course of a year. After a two-to-three-week screening phase, participants will be part of a 14-week double blind test with the drug and a placebo before moving to a 38-week treatment with the drug. The primary measure at the end of the study will be the proportion of participants who lose 10 per cent of their body mass or more after 52 weeks, said Haqq.
Along with the drug trial, Haqq is also conducting a genetic screening study to analyze the DNA of children and adults who are obese, and have been identified as having hyperphagia, or excessive hunger. Through the study, Haqq’s team is seeking to add to the list of genes known to be associated with obesity. It’s hoped the findings will help physicians better recognize the physical signs of genetic disorders contributing to a patient’s obesity, including symptoms, medical history and family history. Participants who have other genetic disorders linked to their obesity identified as part of the genetic study could be added to the anti-obesity drug trial in future expansions, said Haqq.
The ADI is the primary Canadian coordinating centre for the drug trial, responsible for recruiting and overseeing other sites across Canada as the trial expands. Haqq will also be coordinating her findings with counterparts in the United States and the United Kingdom.
“We’re fortunate to have very skilled research coordinators with a lot of experience in running these types of trials,” she said. “The ADI also has everything we need in one location so we can do all our work right here, and patients have only one place to come to.”