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Study of Sonidegib for Patients with Nevoid Basal Cell Carcinoma Syndrome and Sporadic Basal Cell Carcinoma

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What is this trial about?

A plain-language summary of the goals, design and what participants do

This clinical trial is focused on studying the effects of a medication called Sonidegib on two types of skin conditions: Nevoid Basal Cell Carcinoma Syndrome (also known as Gorlin syndrome) and sporadic Basal Cell Carcinoma (BCC). These conditions involve the development of skin cancer, with Gorlin syndrome being a genetic disorder that leads to multiple skin cancers, while sporadic BCC occurs randomly and is not inherited. The treatment being tested is Odomzo 200 mg hard capsules, which contain the active substance Sonidegib.

The purpose of the study is to observe how well patients with these conditions respond to the treatment over a period of 40 weeks. Initially, participants will take Sonidegib daily for 16 weeks. After this period, the dosing schedule will change to a less frequent, pulsed schedule for the remaining 24 weeks. This approach aims to assess the treatment's effectiveness and how well patients can adhere to the medication regimen.

The study will monitor various outcomes, including the rate at which participants discontinue the treatment due to side effects, the overall response rate of the skin cancer to the treatment, and the duration of response. Additionally, the study will evaluate the quality of life of participants using a questionnaire called the EORTC QLQ C30 at different stages of the treatment. The trial is expected to conclude by June 2027.

The research process

The trial runs in 3 steps – from screening to follow-up. Each step says what happens and what the team monitors.

Step 1
Initial treatment phase
The treatment begins with the administration of Odomzo 200 mg hard capsules, which contain the active substance sonidegib.
The medication is taken orally once daily for a duration of 16 weeks.
This phase aims to assess the initial response to the treatment in patients with Nevoid Basal Cell Carcinoma Syndrome or sporadic Basal Cell Carcinoma.
Step 2
Pulsed dosing phase
Following the initial 16 weeks, the treatment continues with a pulsed dosing schedule.
This phase lasts for an additional 24 weeks.
The objective is to maintain the treatment effects while potentially reducing side effects.
Step 3
Evaluation and monitoring
Throughout the trial, regular evaluations are conducted to monitor the treatment's effectiveness and any side effects.
The primary goal is to achieve a discontinuation rate of less than 54% due to adverse effects by the end of the 40-week period.
Secondary assessments include overall response rate, progression-free survival, and duration of response, as well as quality of life scores.

Who can join the trial?

7 criteria

Be willing and able to provide written informed consent for the trial. This means you agree to participate and understand what the trial involves.
Be at least 18 years old and no older than 50 years old on the day you sign the informed consent.
Have one of the following conditions:
- Established diagnosis of Nevoid Basal Cell Carcinoma Syndrome (NBCCS), a genetic condition that can cause skin cancer.
- Common or sporadic Basal Cell Carcinoma (BCC), a type of skin cancer.
- Have a clinical stage IIB according to the EADO Classification. This means having a very high number of common BCCs (more than 10) or multiple complex BCCs (more than 5) in cases that seem to occur randomly or in Gorlin syndrome. If at least one of the BCCs is more severe, the classification may change.
- Be considered difficult to treat, meaning surgery or radiation therapy (RT) is not suitable after evaluation by a team of specialists, including a dermatologist and a medical oncologist. This makes you eligible for HHI therapy, a type of treatment for BCC.
- Have an Eastern Cooperative Oncology Group (ECOG) performance status of 0 or 1. This is a scale that measures your ability to perform daily activities, where 0 means fully active and 1 means restricted in physically strenuous activity but able to do light work.

Who cannot join the trial?

5 criteria

Patients who are not diagnosed with Nevoid Basal Cell Carcinoma Syndrome (a genetic condition that increases the risk of developing skin cancer) cannot participate.
Patients who are not in the age range specified for the study cannot participate.
Patients who do not meet the specific health conditions required for the study cannot participate.
Patients who are not able to follow the treatment plan as required by the study cannot participate.
Patients who are part of a vulnerable population, which means they might need special protection or care, cannot participate.

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Verified sites

All sites with verified contact details – recruitment status may not be available; ask directly

Azienda Ospedaliera Universitaria Citta' Della Salute E Della Scienza Di Torino
Verified
Turin, Italy
Azienda Ospedaliera Universitaria Federico II Di Napoli
Verified
Naples, Italy
Istituto Europeo Di Oncologia S.r.l.
Responsive
Milan, Italy

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Investigated drugs

Sonidegib is a medication used in this clinical trial to treat patients with Nevoid Basal Cell Carcinoma Syndrome (Gorlin syndrome) and those with sporadic Basal Cell Carcinoma. The trial involves an initial treatment period of 16 weeks where patients take sonidegib daily. After this period, the treatment continues with a pulsed dosing schedule for an additional 24 weeks. Sonidegib works by targeting specific pathways in the body that are involved in the growth of cancer cells, helping to slow down or stop their growth.

Sonidegib

What is already known about the treatment

Sonidegib – Sonidegib is administered orally in the form of a capsule. It is currently being studied in clinical trials for its effectiveness in treating Nevoid Basal Cell Carcinoma Syndrome (NBCCS) and sporadic Basal Cell Carcinoma (BCC). The medication works by inhibiting the Hedgehog signaling pathway, which is involved in the growth of cancer cells. Sonidegib is classified as a Hedgehog pathway inhibitor and is being evaluated for its ability to reduce tumor size and prevent cancer progression.

Investigated diseases

Nevoid Basal Cell Carcinoma Syndrome – This is a genetic condition that leads to the development of multiple basal cell carcinomas, which are a type of skin cancer. People with this syndrome often have a higher number of these skin cancers at a younger age compared to the general population. The syndrome can also cause other abnormalities, such as jaw cysts, skeletal abnormalities, and distinctive facial features. It is caused by mutations in a specific gene that affects cell growth. The condition is inherited in an autosomal dominant pattern, meaning a single copy of the altered gene in each cell is sufficient to cause the disorder. Over time, individuals with this syndrome may develop additional health issues related to the various abnormalities associated with the condition.