The Alberta Diabetes Institute (ADI) at the University of Alberta is set to lead a clinical trial of new drug setmelanotide for the treatment of obesity associated with rare genetic disorders in Canada.
Scheduled to start by the end of this month, the international study is being funded by Rhythm Pharmaceuticals, the company that developed setmelanotide as a melanocortin-4 receptor (MC4R) agonist.
MC4R is believed to stimulate a biological pathway that regulates weight in humans. Variants in the MC4R pathway could lead to excessive hunger (hyperphagia) and severe early-onset obesity.
Setmelanotide is intended to act as a potential replacement therapy to restore lost MC4 pathway activity in order to regain weight and appetite regulation in patients with rare genetic conditions.
The new study will assess the drug over one year in patients with Bardet-Biedl syndrome or Alström syndrome, which are rare genetic diseases characterised by obesity.
Participants will initially be randomised to receive setmelanotide or placebo for 14 weeks before receiving treatment with the drug for 38 weeks.
The trial’s primary measure is the proportion of patients who lose 10% of their body mass or more after 52 weeks.
ADI clinical scientist Andrea Haqq said: “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.
“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 institute will act as the coordinating centre and will enrol and oversee additional sites in Canada.
Apart from the trial, ADI is also conducting a genetic study to analyse the DNA of children and adults who have obesity and hyperphagia.
Individuals who are identified to have obesity linked to their genetic disorders, other than Bardet-Biedl syndrome or Alström syndrome, could be enrolled into the setmelanotide trial in the future.