Screening for the patidegib phase III trial

What is screening?

Screening in the process used to see who is suitable to participate in a clinical trial. Screening uses a checklist of inclusion and exclusion criteria. You can read more about these here. The criteria aim to do several things

If you go for screening, your research team will explain other criteria and details which apply to you.

• Make sure the participants actually have Gorlin Syndrome.
• Reduce risks to an absolute minimum. For example, although patidegib gel has been shown to be very unlikely to damage to unborn babies, the screening process makes sure no one can get pregnant just before, during, or for one year after the trial.
• Check nothing can confuse the science of the trial. For example, it is important that participants have not used any other medications on their faces for the three months leading up to the trial or during the trial. This is because these medications could also prevent BCCs appearing during the trial, which would confuse the comparison between patidegib and placebo.
• Make sure the disease is active on the face. Everyone with Gorlin Syndrome has a different experience. Some people get 10 BCCs on their faces every year whilst others get BCCs less often. The trial needs to focus on people who get a lot of BCCs. So, Pellepharm decided that only people who have had three BCCs on the face in the two years before the trial, including two on the face at the time of joining, will be able to join. However, Gorlin Syndrome Group argued that people with fewer BCCs on the face at the time of joining should be able to participate and, as a result, Pellepharm has said it will keep this under review.

If you go for screening, your research team will explain other criteria and details which apply to you.

What happens at the screening visit?

You will have had some information about the trial before hand and you will have a chance to go through this again with your trial doctor. If you agree to go ahead with the trial, you will sign a consent form, confirming you understand the potential risks and benefits. Next, you will be asked a long series of questions related to the inclusion and exclusion criteria, you’ll have a physical examination by the doctor, have some photographs taken and some blood tests done.

It could be a long session! Bear this in mind when you plan transport and snacks. Any friends or family that come with you may need to bring some entertainment.

What happens next?

The information from the screening visit will be reviewed centrally. So, if you are eligible to go ahead, you will be assigned to either placebo or patidegib gel. Remember, neither you nor your doctor will know!

Once you have been screened and accepted, you be able to start the trial within about 6 weeks of the screening visit. During this time, it is important to remember the inclusion criteria, for example about becoming pregnant or using other medications on your face.

It’s also important to understand that there are only 150 places on the trial. It’s possible you might be screened and be eligible, but all the places may have been taken.

Has screening started?

Yes – screening has started in the US. As of early April 2019, 9 people have been screened and 5 have ‘passed’. 2 people ‘failed’ screening because they had used another medication on their face. One person had insufficient BCCs on the face and one other person was doing another clinical trial.

The first person actually started the trial at the very end of March 2019.

Trial centres in Europe are signing up now. Each country must get Ethics Committee approval before starting screening. We hope that screening will start in Europe before summer 2019.