Patidegib topical gel phase 3 trial.
- A trial to evaluate patidegib gel in preventing BCCs on the face will start soon.
- Participants need to
- have had 10 or more BCCs in the previous two years
- have two BCCs on the face at the time of the trial
- There is a 50:50 chance of getting patidegib or a placebo.
- Participants will attend the trial centre several times over the year of the trial.
- We hope that participants will receive patidegib after the trial, until it is licensed.

What is patidegib?
Patidegib is the topical gel which has already shown some preliminary benefits in shrinking BCCs in people with Gorlin Syndrome in a Phase 2 trial. The gel also seems to be effective in preventing new BCCs from appearing. The gel seems to have very few side effects to date.
The next trial is taking place in the US, Canada, and across Europe. This trial is being developed by two pharmaceutical companies, PellePharm and LeoPharma. Various hospitals are going through the process of registering with the trial which will start in late winter in the US and in spring in Europe. Across the world, 150 people will be recruited into the study. Once it is up and running, we will provide a link so that you can see how many places are left.
What would I do to join the trial?
To be in the trial you need to be an adult (18 or over) with Gorlin Syndrome and have had ten or more BCCs over the past ten years and 2 BCCs on the face at the time of recruitment. Gorlin Syndrome Group argued to the pharmaceutical companies that needing to have 2 BCCs on the face at the start of the trial could dissuade patients from having surgery. The pharmaceutical companies decided not to budge on this. There are a few other conditions that need to be met before you join.
Before you join the trial, you, or another individual able to do so must give informed consent to take part in the study. Even after you’ve been evaluated and even once you’ve started, you can still pull out of the study. Very few people did so during the Phase 2 trial.
The trial is a double-blind vehicle (placebo)-controlled trial. Participants will be using patidegib or a placebo gel twice a day, for a year. There are monthly appointments where you will be checked over and given a new supply of the drug. Every three months you will have a longer appointment to take detailed pictures of your face. You will be asked to give detailed information about how you are feeling and how you are functioning during the trial. The trial has been carefully designed, with input from people with Gorlin syndrome, to make sure collects information about how patidegib (or placebo) affects the quality of our lives.
After the trial is completed, we anticipate that there will be an opportunity for all study participants to continue the gel containing the active drug for at least another year. This is called an ‘Open-Label Access Extension’ and during this period you will still be under the observation of the trial team.
What would I need to think about before joining the trial?
Reasons for doing the trial include the 50:50 chance of getting access to a promising new topical gel. You will be under close observation, so that if any BCCs you have at the start are not getting better, they will be very promptly treated. In addition, another important reason for helping in the study is that, as a community, we get to know more about safe, effective treatments.
On the other hand, reasons for not doing the study may be that you are anxious about the risk of side effects with a new drug. The Phase 2 study showed that temporary reddening of the skin was the most frequent side effect and only happened in a minority of people.
You may also decide that the trial is not for you because you can’t afford the time or the inconvenience. Being realistic, each session could take half a day, once travel and time at the hospital are taken into account. PellePharm and LeoPharma have been listening to the comments of people who took part in the Phase 2 trial, and PellePharm will do their best to ensure that appointments are planned in advance and reimbursement for travel costs happen very smoothly.
Will patidegib be available on the prescription after the trial is finished?
Finally, it’s worth bearing in mind that even if the trial shows that patidegib is effective in preventing BCCs in people with Gorlin Syndrome, NICE will not necessarily recommend its use in the UK. NICE is less likely to recommend patidegib if the gel causes severe side effects (unlikely), it doesn’t work very well (possible) or that the pharmaceutical companies decide to market it at a very high price (likely). This could also be a problem in counties where health insurance is required to pay form treatments: If you don’t have the right insurance, you may never get the drug. On the other hand, pharmaceutical companies argue that new drugs are expensive because it costs hundreds of millions of pounds to do a single trial and that they have to provide hefty rewards for their investors.
For more information about how NICE makes decisions, please see the posting on Vismodegib in the UK (https://gorlingroup.org/vismodegib-in-the-uk/).
You can find more information about clinical trials at the Cancer Research UK website https://www.cancerresearchuk.org/about-cancer/find-a-clinical-trial/ Your dermatologist will also be able to give you full information about the trial.