Efficacy and Safety of Patidegib Gel 2% for Preventing Basal Cell Carcinomas on the Face of Adults With Gorlin Syndrome
This study is testing a gel called Patidegib (2%) for adults who have a condition called Gorlin syndrome. People with Gorlin syndrome often get many skin cancers, called basal cell carcinomas (BCCs), especially on their face. The main goal of this study is to find out if applying Patidegib gel to the face twice a day can help prevent new BCCs from developing. It also aims to see if the gel is safe for people to use over a 12-month period. Some people will use the active Patidegib gel, while others will use a gel that looks the same but doesn't contain the active medicine. The researchers will then compare the number of new BCCs in both groups.
במבט חטוף
על מה המחקר הזה?
This study is about helping adults who have a condition called Gorlin syndrome. People with Gorlin syndrome are more likely to develop certain types of skin cancer, called basal cell carcinomas (BCCs), especially on their face. These BCCs can be a real problem, often requiring treatments like surgery.
The study is testing a new gel, called Patidegib, to see if it can help prevent these new BCCs from forming. Half of the people taking part will use the Patidegib gel on their face twice a day for a year. The other half will use a 'dummy' gel, which looks exactly the same but doesn't contain the active medicine. This helps researchers compare the effects accurately. Everything is carefully monitored, and any new spots on the face that look like BCCs will be checked by experts.
The main idea is to find a way to reduce the number of skin cancers people with Gorlin syndrome get, which could potentially improve their quality of life and reduce the need for multiple treatments. This is a "Phase 3" study, which means it’s a big step towards potentially making this treatment available if it proves safe and effective.
נקודות עיקריות
- Tests a gel (Patidegib) to prevent new skin cancers (BCCs) in Gorlin syndrome.
- Compares active gel to a 'dummy' gel over 12 months.
- Involves applying gel twice daily and regular clinic visits.
- Aims to reduce BCCs and improve quality of life.
- Participants must be 18+ with Gorlin syndrome and facial BCCs.
מי עשוי להיות זכאי?
To join this study, you need to be an adult, at least 18 years old. You must have been diagnosed with Gorlin syndrome and have a specific genetic change (called a PTCH1 mutation) confirmed by a doctor. You also need to have quite a few BCCs on your face – at least 10 – when you start the study. It's important that you're willing to only use the study gel on your face and avoid other skin creams or treatments, unless your study doctor says they are okay.
There are also some reasons why you wouldn't be able to join. For example, if you've recently used strong skin treatments on your face or other medications that might interfere with the study's results. If you're allergic to anything in the gel, or have another serious health condition that isn't under control, you wouldn't be able to participate. Also, if you've had certain types of cancer in the last five years, or if a previous similar treatment for Gorlin syndrome didn't work for you, you might not be eligible.
האם מחקר זה יכול להתאים לך?
ענה על השאלות המהירות הללו כדי לבדוק אם אתה עשוי להיות זכאי. זהו מדריך בלבד — צוות המחקר מקבל את ההחלטה הסופית.
- האם אתה לפחות בן 18?
- Do you have a confirmed diagnosis of Gorlin syndrome?
- Do you have at least 10 basal cell carcinomas on your face?
- Are you able to use only the study gel on your face for one year, unless prescribed by the study doctor?
- Have you not had certain other cancers or specific treatments recently?
מה כרוך בהשתתפות?
If you join the study, you'll be given either the active Patidegib gel or a 'dummy' gel. You won't know which one you're receiving, and neither will your study doctor. You'll need to apply the gel to your face twice a day every day for 12 months. Throughout this year, you'll have regular visits to the clinic where study staff will check your skin for any new spots, photograph your face, and assess how the gel is working. They will also ask about any side effects you might be experiencing and perform some health checks. It's important to attend all appointments so the researchers can track your progress and safety. There will be ongoing checks of your skin to record any new BCCs that appear.
סיכונים ותועלות פוטנציאליים
Locations (40)
- Northwest Arkansas Clinical Trials Centerמיקוד מאומתRogers, United States
- Center for Dermatology Clinical Researchמיקוד מאומתFremont, United States
- University of California Los Angeles (UCLA) - Center for Inflammatory Bowel Diseases - Westwood Locationמיקוד מאומתלוס אנג'לס, ארצות הברית
- The Dermatology Center of Newportמיקוד מאומתניופורט ביץ', ארצות הברית
- Stanford University - Lucille Packard's Children's Hospitalמיקוד מאומתרדווד סיטי, ארצות הברית
- אוניברסיטת יילמיקוד מאומתניו הייבן, ארצות הברית
- Dermatology Associates of Tallahasseeמיקוד מאומתטלהאסי, ארצות הברית
- University of South Florida Healthמיקוד מאומתטמפה, ארצות הברית
- The University of Chicago Medicineמיקוד מאומתשיקגו, ארצות הברית
- The Johns Hopkins Hospitalמיקוד מאומתבולטימור, ארצות הברית
- University of Michigan Health - Michigan Medicine - University Hospitalמיקוד מאומתאן ארבור, ארצות הברית
- Minnesota Clinical Study Centerמיקוד מאומתNew Brighton, United States
שאלות נפוצות
What is Gorlin syndrome?
Gorlin syndrome is a rare genetic condition that can make people more likely to develop certain health issues, including a type of skin cancer called basal cell carcinoma (BCC).
What are basal cell carcinomas (BCCs)?
BCCs are the most common type of skin cancer. They usually grow slowly and rarely spread to other parts of the body, but can cause damage if left untreated.
What is a 'dummy' gel?
A 'dummy' gel looks exactly like the active medicine but doesn't contain any real drug. This helps researchers fairly compare the active gel's effects.
כמה זמן אהיה במחקר?
You'll be in the study for a total of 12 months, during which you'll apply the gel twice daily and attend regular check-up appointments.
Can I stop the study if I want to?
Yes, you can leave the study at any time, for any reason, and your regular medical care will not be affected.
כיצד לגלות עוד
תמיד התייעץ עם רופא המשפחה או המומחה שלך לפני שתחליט להשתתף במחקר.
דיון
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