Gorlin syndrome is being studied every day by doctors and scientists who are searching for new ways to help patients with Gorlin syndrome and who hope that, one day, Gorlin syndrome can be cured. These researchers need the participation of patients with Gorlin syndrome so that they can learn more about the syndrome and determine what treatment options are best. You or a loved one may be asked to participate in a clinical trial by your doctor to help advance what is already known about Gorlin syndrome. Or, you may actively seek out a clinical trial on your own, allowing you to gain access to cutting edge therapy options that are still being studied and are not widely available yet.
What is a clinical trial?
A Clinical trial is a type of research study. In this type of study, doctors and scientists ask individuals to participate so they can determine whether a new treatment, test, or prevention strategy is better than other currently available options. To accomplish this, a new treatment is studied voluntarily in people that the treatment is designed to help. Those who participate are then followed over time to determine whether the new treatment is better than existing treatments.
Why should I participate in a clinical trial?
Clinical trials have a number of benefits for patients with Gorlin syndrome. Here are some reasons you may consider when thinking about participating in a Clinical trial:
- Clinical trials can help you! Before a treatment becomes mainstream, it starts by being studied in a clinical trial to make sure that it is the best treatment available. Patients with Gorlin syndrome may benefit from trials that specifically study treatments for Gorlin syndrome and/or certain symptoms or manifestations of Gorlin syndrome. Clinical Trials are especially helpful for patients with rare diseases, like Gorlin syndrome, because there may not be many mainstream treatment options available, so Clinical Trials can provide individuals with more attention and help with associated medical problems that don’t have a mainstream solution at this time.
- Clinical Trials can help advance science and help others with Gorlin syndrome! Many of the standard treatments or tests that have been offered to help manage your Gorlin syndrome were initially identified through Clinical Trials. By participating in Clinical Trials you help doctors and industry determine how best to help patients with Gorlin syndrome so that other people like you get the best treatment possible. Additionally, as doctors and scientists learn more about Gorlin syndrome through research, they become closer to identifying a cure for Gorlin syndrome.
Should I be scared about being experimented on?
When you hear about a new Clinical Trial or are asked to participate, a normal reaction may be fear that you might be hurt as part of someone’s experiment. Fortunately, both the government and hospitals take great care to make sure that trials are safe prior to allowing you to participate in them. All trials with experimental medications have to be approved by the Food and Drug Administration (FDA) prior to being conducted. Furthermore, all studies that evaluate whether an existing therapy is helpful for patients with Gorlin have to be approved by what is called an Institutional Review Board (IRB). An Institutional Review Board is a group of experts who review studies to make sure that they are safe and ethical prior to allowing them to be conducted. Many times, therapies have been proven to work in labs or in animals prior to being tried in humans. Throughout the duration of the study, researchers will continuously evaluate your safety and discontinue the trial if it does not appear to be safe. Plus, you can leave a trial at any point if you feel uncomfortable.
How do I find Clinical Trials that may help me?
In general, there are two ways that you can find out about clinical trials that might benefit you:
- Your doctor may recommend that you participate in a specific clinical trial designed to help you;
- You may seek out a clinical trial that is designed to help you. Below you will find a list of clinical trials that are designed to help patients with Gorlin syndrome or that are designed to help with common symptoms of Gorlin syndrome. You can also go to clinicaltrials.gov to find additional trials.
What do I do if I find a Clinical Trial that might I might be interested in?
Once you find a Clinical Trial that you might be interested in participating, it is important that you contact the people conducting the study to see if you are eligible to participate and to find out how you go about participating. It is important to make sure you find out what you must do if you participate in the clinical trial and to make sure that these are things you are willing to do. For example, some trials may require you to go to a set number of follow-up appointments and some may require that you get frequent bloodwork. Make sure that you are willing to fulfill these requirements prior to agreeing to participate in a trial. Be sure to note, that by asking for more information about a clinical trial does not commit you to participate in the study. Even once you agree to participate in a study, you can leave the study at any time if you feel uncomfortable or no longer wish to participate.
Gorlin Syndrome Treatment Trials
Below are links to various experimental treatment trials. These trials help determine which treatments are effective, and what can still be improved. Click the links below to learn more about each trial.
- Nivolumab Alone or Plus Ipilimumab for Patients With Locally-Advanced Unresectable or Metastatic Basal Cell Carcinoma
- Study of Patidegib Topical Gel, 2% for the Reduction of Disease Burden of Persistently Developing Basal Cell Carcinomas (BCCs) in Subjects with Basal Cell Nevus Syndrome
- Vitamin D as a Nutritional Neoadjuvant During Photodynamic Therapy of Basal Cell Carcinoma
- Open-label Trial of SUBA®-Itraconazole (SUBA-Cap) in Subjects With Basal Cell Carcinoma Nevus Syndrome (BCCNS)
- Photodynamic Therapy Using Blue Light or Red Light in Treating Basal Cell Carcinoma in Patients With Basal Cell Nevus Syndrome
- An Observational Study of the Effectiveness and Safety of Erivedge® in Patients With Advanced Basal Cell Carcinoma
- An Observational Study to Determine the Clinical Cure Rate of Therapeutic up Front Shave Removal of Basal Cell Carcinoma of the Skin With Long Term Follow up to Evaluate Recurrence
- An Observational Study of Treatment Patterns and Effectiveness and Safety Outcomes in Advanced Basal Cell Carcinoma and Basal Cell Carcinoma Nevus Syndrome Patients(RegiSONIC)
- Study Assessing the Feasibility of a Surgery and Chemotherapy-Only in Children With Wnt Positive Medulloblastoma
- Study of Vismodegib in Combination With Temozolomide Versus Temozolomide Alone in Patients WithMedulloblastomas With an Activation of the Sonic Hedgehog Pathway
- Metronomic and Targeted Anti-angiogenesis Therapy for Children With Recurrent/Progressive Medulloblastoma(MEMMAT)