A Multicenter, Randomized, Double blind, Vehicle-controlled, Phase 3 Efficacy and Safety Study of Patidegib Gel 2% for the Reduction of Disease Burden of Persistently Developing Basal Cell Carcinomas (BCCs) in Subjects with Gorlin Syndrome
Gorlin syndrome is a rare genetic condition that causes people to develop many basal cell carcinomas, a common type of skin cancer, especially on the face. Managing these spots often means having repeated surgeries, which can cause scarring and distress. This Phase 3 trial is testing whether an experimental skin gel called Patidegib 2% can prevent new skin cancers from forming or help shrink existing ones. Participants will apply either the active Patidegib gel or a dummy gel (with no active medicine) to their face daily for 12 months. Neither the participants nor their doctors will know who gets which gel. The study aims to find out if this treatment can reduce the need for surgery, improve skin symptoms, and enhance daily quality of life for people living with Gorlin syndrome.
Överblick
Vad handlar denna studie om?
Gorlin syndrome, also known as nevoid basal cell carcinoma syndrome, is a rare inherited condition. People who have it often develop numerous basal cell carcinomas (BCCs), usually starting from a young age. While BCC is a slow-growing type of skin cancer that rarely spreads to other parts of the body, having dozens or even hundreds of them on the face can be very challenging. Currently, the most common treatment is surgery to cut them out, which can lead to visible scarring and repeated hospital visits.
This clinical study is evaluating a new topical treatment called Patidegib gel 2%. The gel is designed to block specific signals in skin cells that cause these tumours to grow. By applying the medicine directly to the skin, researchers hope it will work right where it is needed without causing the wider side effects often seen with pills used for advanced skin cancers.
Researchers are running this trial to find out if Patidegib gel can significantly cut down the number of new facial skin cancers that appear over one year. They also want to see whether it can shrink existing spots so they no longer need surgical removal, ease skin pain and itching, and ultimately improve day-to-day quality of life for people living with this demanding condition.
Viktiga punkter
- Tests a daily skin gel (Patidegib 2%) for adults with Gorlin syndrome.
- Aims to prevent new facial skin cancers and reduce the need for surgery.
- Compares the active drug against a dummy gel over 12 months.
- Neither you nor your doctor will choose or know which gel you receive.
- Participation includes regular clinic visits, skin checks, and questionnaires.
Vem kan vara berättigad?
This trial is open to adults aged 18 and older who have been diagnosed with Gorlin syndrome and regularly develop basal cell carcinomas on their face. Both men and women can join.
To take part, participants will need to have active facial skin spots that a doctor can monitor throughout the study. People must also be willing to apply the study gel as directed every day for a full year and attend regular hospital check-ups.
Certain health factors might prevent someone from joining. For instance, people who are currently using other specific treatments for skin cancer, those with severe medical conditions, or women who are pregnant or planning to become pregnant may not be able to participate. A study doctor will review each person's medical history to make sure taking part is safe for them.
Kan denna studie passa dig?
Svara på dessa snabba frågor för att se om du kan vara berättigad. Detta är endast en vägledning — forskarteamet fattar det slutgiltiga beslutet.
- I am aged 18 or older.
- I have been diagnosed with Gorlin syndrome.
- I regularly develop basal cell carcinomas (BCCs) on my face.
- I am willing and able to apply a gel to my face daily for 12 months.
- I can attend regular hospital visits for skin checks and assessments.
Vad innebär deltagande?
Taking part in this study lasts for about 12 months. You will be randomly assigned by a computer to receive either the active Patidegib gel or a vehicle gel, which looks identical but contains no active medicine. You will apply the gel to your face every day as instructed by the study team. You will have regular clinic visits over the year so doctors can examine your skin, count any new or changing spots, and take measurements or photographs. You will also be asked to complete brief questionnaires about any skin itching, pain, and how the condition affects your daily life. The team will closely check your skin for any redness or irritation, and you will be monitored for general health and safety throughout.
Potentiella risker och fördelar
Platser (6)
- —ObekräftadNederländerna
- —ObekräftadFrankrike
- —ObekräftadItalien
- —ObekräftadSpanien
- —ObekräftadPolen
- —ObekräftadTyskland
Vanliga frågor
What is Gorlin syndrome?
Gorlin syndrome is a rare genetic condition that causes various health features, most notably the development of multiple basal cell carcinoma skin cancers, often from an early age.
What is the difference between the active gel and the vehicle gel?
The active gel contains Patidegib, the medicine being tested. The vehicle gel has the same base ingredients and appearance, but contains no active drug, acting as a comparison.
Will I know which gel I am receiving?
No. This is a double-blind trial, which means neither you nor your study doctor will know which gel you receive until the study is over, ensuring fair and unbiased results.
Can I continue having skin surgeries if I need them during the study?
Yes. Your safety comes first. The study doctors will monitor your skin closely and arrange appropriate care or removal if any spots require urgent treatment.
Do I have to pay to take part in this clinical trial?
No, clinical trial medications and study visits are provided free of charge, and travel expenses are often reimbursed.
Hur du får reda på mer
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Diskussion
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