In short
There are currently 2 ongoing clinical trials investigating treatments for Basal Cell Naevus Syndrome (also known as Gorlin Syndrome, Nevoid Basal Cell Carcinoma Syndrome). These studies are testing medications that target the pathways involved in the development of basal cell carcinomas, offering hope for better management of this genetic condition.
Clinical trial locations
- France
- Germany
- Italy
- Netherlands
- Poland
- Spain
Study of Sonidegib for Patients with Nevoid Basal Cell Carcinoma Syndrome and Sporadic Basal Cell Carcinoma
This trial is investigating Sonidegib, a medication that works by blocking specific pathways in the body responsible for cancer cell growth. The study focuses on patients with both the inherited form of the condition and those with sporadic basal cell carcinoma.
Who can participate:
- Adults between 18 and 50 years of age
- People with a confirmed diagnosis of the syndrome or common basal cell carcinoma
- Patients with very high numbers of skin cancers (more than 10 common types or more than 5 complex types)
- Those for whom surgery or radiation therapy is not suitable, as determined by a team of specialists
- Individuals who are fully active or only slightly restricted in physically demanding activities
Who cannot participate:
- Patients without a confirmed diagnosis
- Those outside the specified age range
- Individuals who do not meet the specific health requirements
- People unable to follow the treatment plan
- Members of vulnerable populations who may need special protection
How the trial works: Participants take Sonidegib capsules (200 mg) once daily for the first 16 weeks. After this initial phase, the treatment continues with a less frequent, pulsed dosing schedule for an additional 24 weeks. This approach aims to maintain treatment benefits while potentially reducing side effects. The trial monitors how well patients respond to treatment, how long the response lasts, and the impact on quality of life. The study is expected to conclude by June 2027.
Investigational drug: Sonidegib (brand name Odomzo) is a medication that targets the Hedgehog signaling pathway involved in cancer cell growth, helping to slow down or stop the development of skin cancers.
Study on Patidegib Gel for Reducing Basal Cell Carcinomas in Adults with Gorlin Syndrome
This trial is testing Patidegib Gel 2%, a topical treatment applied directly to the skin. The study aims to evaluate whether this gel can effectively reduce the number of new basal cell carcinomas that develop on the face of adults living with this condition.
Who can participate:
- Adults aged 18 years or older
- People with a confirmed PTCH1 gene mutation, which is linked to the syndrome
- Patients with at least 10 basal cell carcinomas on their face at the start of the study
- Those willing to avoid using any other topical medications on their face during the trial, unless prescribed by the study doctor
Who cannot participate:
- Individuals without a confirmed diagnosis
- Those outside the specified age range
- People with certain health conditions or taking specific treatments that might interfere with the study
- Members of vulnerable populations
How the trial works: Participants are randomly assigned to use either Patidegib Gel 2% or a placebo gel (vehicle gel without active medication). The gel is applied to the face twice daily for 12 months. Throughout the study, researchers monitor the number of new skin cancers that develop, changes in the size of existing lesions, and any side effects. The trial also assesses quality of life and the overall safety of the treatment.
Investigational drug: Patidegib Gel is a topical medication that inhibits the Hedgehog signaling pathway involved in the growth of basal cell carcinomas. By blocking this pathway, the gel aims to prevent the development of new skin cancers in people with this genetic condition.
Summary
The two ongoing clinical trials for Basal Cell Naevus Syndrome represent different approaches to managing this genetic condition. One study focuses on an oral medication taken as capsules, while the other tests a gel applied directly to the skin. Both treatments work by targeting the same biological pathway responsible for cancer cell growth.
The oral Sonidegib trial is currently available only in Italy and focuses on a specific age group of adults between 18 and 50 years. In contrast, the Patidegib Gel trial is more widely available across six European countries: Poland, France, Germany, Italy, Netherlands, and Spain, and is open to all adults aged 18 and older.
Both studies reflect ongoing efforts to develop better treatment options for people living with this challenging condition. The topical gel approach may be particularly appealing to patients who prefer a treatment applied to the skin rather than taking daily capsules, while the oral medication may suit those with more severe or widespread disease.
