Perhaps we’re not quite going on a magic carpet ride, then again, that doesn’t sound like such a bad idea! But I wanted to take a moment to share a little about the world of trials and research.  Research can be carried out in a number of ways.  Investigator led trials occur where the investigator identifies need, develops protocol and runs the elements of the trial, including seeking any necessary funding.  For our purpose, it is often a doctor, perhaps someone working towards their PhD, who runs these.  At any given time there are a great many of these, big and small, occurring.  They can provide valuable insights into conditions.  It  can be difficult to attract researchers to rare conditions, but it does happen.  Protocols must have rigorous standards that can hold fast to the value of the results they yield, and all must be approved by a relevant ethics committee.

Clinical trials sponsored by pharmaceutical companies must uphold the same, possibly even stricter, protocol standards.  They are focused around developing new medications for use in patient populations.  The pharmaceutical company engage with medical centres who are resourced to carry out the trials.  An independent third party is also involved to ensure all obligations of both the pharmaceutical company and the trial centres are met. 

For a long time Australian’s have been crying out for PSC research in both arenas – and it’s great to see we are finally on the map.  We’re very excited to be talking to representatives from  a couple of companies running trials as well as a doctor doing her PhD in PSC outcomes.  Escient pharmaceuticals is one such company and they share a little about themselves and one of their current trials below.

“Escient Pharmaceuticals, Inc. is a biotechnology company focused on developing novel Mas-related G Protein-Coupled Receptor (MRGPR)-targeted drugs to address serious, underserved medical needs across a broad range of therapeutic indications. MRGPRX4 is a receptor that is activated by bile acids that may contribute to pruritus (itch) in patients with liver disease, such as primary sclerosing cholangitis. Pruritus can meaningfully impair the quality of life of patients with liver disease and there are few therapies available to treat this condition. Escient has initiated a Phase 1/1b clinical trial of EP547, a MRGPRX4-targeted product candidate to treat pruritus associated with liver and kidney disease.

The Phase 1/1b trial of EP547 is a randomized, double-blind, placebo-controlled study to evaluate the safety, tolerability, and pharmacokinetics of single and multiple doses of EP547 in healthy volunteers and patients with cholestatic or uremic pruritus. The study includes a one-week treatment period and is being conducted in Melbourne, Adelaide, and Auckland. Additional information about the Phase 1/1b trial can be found at The trial will inform future studies of longer treatment duration to assess the safety and efficacy of EP547 in patients with cholestatic pruritus as a result of primary biliary cholangitis or primary sclerosing cholangitis. “

It can be very rewarding participating in research. Being part of a clinical trial of any type brings you under the care of specialized and interested researchers, both at the level of the trial centre and the pharmaceutical companies.  This means you get great, and closely monitored care, while potentially advancing the researchers knowledge and perhaps even impacting future therapies.  So there are selfish, and altruistic motivations to being part of a clinical trial.

Let us know in the comments below if you’d like to know more about this trial, or other trials in general.