Patidegib Gel for Preventing Basal Cell Carcinoma

Not currently recruiting at 41 trial locations

Overseen By Patient Referral Support Service (US toll-free number)

Age: 18+

Sex: Any

Trial Phase: Phase 3

Sponsor: Sol-Gel Technologies, Ltd.

Must not be taking: Topical treatments, Systemic therapies

Disqualifiers: Uncontrolled systemic disease, Invasive cancer, others

Pivotal Trial (Near Approval) This treatment is in the last trial phase before FDA approval

Prior Safety Data This treatment has passed at least one previous human trial

Compensation: Varies

Number of Visits: Unknown

Duration: 12 months

What You Need to Know Before You Apply

What is the purpose of this trial?

This trial tests Patidegib Gel 2% to determine its effectiveness in preventing new basal cell carcinomas (a type of skin cancer) on the faces of individuals with Gorlin syndrome, a genetic condition. Participants will apply either Patidegib Gel or a placebo gel without active ingredients twice daily for a year. Researchers will compare the number of new skin cancers between the two groups after 12 months. The study seeks adults with at least 10 existing basal cell carcinomas on their face who are willing to stop using other facial skin products during the trial. As a Phase 3 trial, this study represents the final step before FDA approval, offering participants a chance to contribute to potentially groundbreaking treatment.

Do I need to stop my current medications for the trial?

The trial requires you to stop using any non-study topical medications on your face during the study, unless prescribed by the investigator. It does not specify about other medications, so you should discuss your current medications with the study team.

Is there any evidence suggesting that Patidegib Gel 2% is likely to be safe for humans?

Research has shown that Patidegib Gel 2% is generally safe for use. In earlier studies, individuals with Gorlin syndrome applied Patidegib Gel 2% to their skin. These studies did not report any serious side effects. Some participants experienced mild skin reactions, such as redness or irritation, but these were manageable and not serious. This suggests that Patidegib Gel 2% is safe for its intended use on the skin. ^{1 2 3 4 5}

Why do researchers think this study treatment might be promising?

Unlike the standard treatments for basal cell carcinoma, which often involve surgical procedures or topical medications with different active ingredients, Patidegib Gel stands out due to its unique mechanism of action. Patidegib is a topical gel that works by inhibiting the Hedgehog signaling pathway, which is crucial in the development of basal cell carcinoma. This non-invasive, topical application aims to prevent the formation of new cancerous lesions, offering a potentially simpler and less invasive option than surgery. Researchers are particularly excited about its potential to be used preventatively, reducing the need for frequent medical interventions.

What evidence suggests that Patidegib Gel 2% might be an effective treatment for preventing basal cell carcinoma?

Research has shown that Patidegib Gel 2% can reduce the number of new basal cell skin cancers (BCCs) when applied to the skin. In this trial, participants will receive either Patidegib Gel 2% or a Vehicle Gel as a placebo comparator. Earlier studies found that people using Patidegib Gel had fewer BCCs compared to those who did not. Specifically, one study found that the gel was significantly more effective at clearing BCCs, both visibly and under a microscope, after just three months. This suggests that Patidegib Gel 2% could be a promising way to prevent new BCCs, especially for individuals with Gorlin syndrome, which leads to frequent BCCs. ^{2 5 6 7 8}

Are You a Good Fit for This Trial?

This trial is for adults over 18 with Gorlin Syndrome, who have had at least 15 basal cell carcinomas (BCCs) on the face in the past two years and currently have a minimum of 10 BCCs. Participants must not use other facial skin medications during the trial.

Inclusion Criteria

My cancer has a PTCH1 mutation.

I have had 15 or more basal cell carcinomas on my face in the last 2 years.

I agree not to use any non-study face creams or medications during the trial.

Exclusion Criteria

Subject is known to have hypersensitivity to any of the ingredients in the IP formulation

I have not used any skin treatments or other medicines that could affect the study's treatment.

I have a disease that is not currently under control.

Timeline for a Trial Participant

Screening

Participants are screened for eligibility to participate in the trial

2-4 weeks

Treatment

Participants apply either Patidegib Gel 2% or Vehicle Gel to their face twice daily for 12 months

12 months

Regular visits for imaging and dermoscopic analysis

Follow-up

Participants are monitored for safety and effectiveness after treatment

4 weeks

What Are the Treatments Tested in This Trial?

Interventions

• Patidegib Gel 2%

Trial Overview The study tests if Patidegib Gel 2% can prevent new BCCs on the face of Gorlin Syndrome patients compared to a Vehicle Gel without active drug. It's applied twice daily for one year, and results are measured after this period.

How Is the Trial Designed?

Group I: Patidegib Gel 2% Experimental Treatment 1 Intervention

Patidegib Gel 2% (w/w), applied topically to the face twice daily for 12 months

Group II: Vehicle Gel Placebo Group 1 Intervention

Vehicle Gel, applied topically to the face twice daily for 12 months

Who Is Running the Clinical Trial?

Sol-Gel Technologies, Ltd.

Lead Sponsor

Trials

15

Recruited

4,300+

Premier Research Group plc

Industry Sponsor

Trials

65

Recruited

74,200+

John Ratliff

Premier Research Group plc

Chief Executive Officer since 2024

MBA

Dr. Milena Kanova-Petrova

Premier Research Group plc

Chief Medical Officer since 2024

MD

Published Research Related to This Trial

In the phase 2 BOLT trial, sonidegib demonstrated significant efficacy in treating locally advanced basal cell carcinoma (BCC), with 61% of patients achieving an objective response and a median duration of response of 26.1 months.

The most common side effects of sonidegib were muscle spasms (54.4%), hair loss (49.4%), and loss of taste (44.3%), but most were mild (grade 1 or 2), indicating that the drug is generally well-tolerated.

NCBI (Pubmed)

pubmed.ncbi.nlm.nih.gov

Sonidegib in the Treatment of Locally Advanced Basal Cell Carcinoma. Sanmartín, O., Llombart, B., Carretero Hernández, G., et al.[2021]

In a study of 33 patients with advanced basal cell carcinoma treated with hedgehog inhibitors, vismodegib resulted in a complete response in 33% and a partial response in another 33%, while sonidegib had a higher complete response rate of 42%.

Adverse events were the most common reason for discontinuation of therapy, highlighting the need for careful management of side effects and the exploration of additional treatment options, such as immunotherapy, to improve patient outcomes.

NCBI (Pubmed)

pubmed.ncbi.nlm.nih.gov

Eight Years of Real-Life Experience with Smoothened Inhibitors in a Swiss Tertiary Skin Referral Center. Grossmann, LE., Ramelyte, E., Nägeli, MC., et al.[2022]

Sonidegib and vismodegib, the only approved systemic treatments for advanced basal cell carcinoma (BCC), show similar efficacy in treating the disease, as demonstrated by comparable objective response rates and complete response rates in pivotal clinical studies.

Both drugs have similar safety profiles, with common side effects including muscle spasms, hair loss, and altered taste; however, they differ in their pharmacokinetics, with sonidegib reaching peak plasma concentration faster than vismodegib.

NCBI (Pubmed)

pubmed.ncbi.nlm.nih.gov

A Review of Hedgehog Inhibitors Sonidegib and Vismodegib for Treatment of Advanced Basal Cell Carcinoma. Migden, M., Farberg, AS., Dummer, R., et al.[2021]

Sonidegib, a Hedgehog pathway inhibitor, effectively induced remission in two patients with locally advanced basal cell carcinoma (BCC) within 7 months, demonstrating its efficacy in challenging cases where surgery or radiotherapy is not an option.

Mild adverse effects such as cramps, alopecia, ageusia, and weight loss were reported, aligning with the known safety profile of sonidegib, indicating it is generally well-tolerated in patients.

NCBI (Pubmed)

pubmed.ncbi.nlm.nih.gov

Experience with sonidegib in patients with advanced basal cell carcinoma: case reports. Puig, S., Serra-Guillén, C., Pérez-Pastor, G., et al.[2022]

In the Phase II BOLT study, sonidegib (200 mg daily) demonstrated a high objective response rate (ORR) of 71.2% in patients with locally advanced basal cell carcinoma (laBCC) over 42 months, indicating its efficacy as a treatment option.

The efficacy of sonidegib was maintained in patients taking select concomitant medications, with an ORR of 73.0%, suggesting that it can be safely administered alongside other treatments, except for strong cytochrome P450 3A4/5 inhibitors/inducers.

NCBI (Pubmed)

pubmed.ncbi.nlm.nih.gov

Concomitant medication use does not appear to alter long-term efficacy of sonidegib for advanced basal cell carcinoma: a post hoc analysis based on the 42-month BOLT study. Lear, JT., Robert, C., Grob, JJ., et al.[2023]

Sonidegib (LDE225) is an FDA-approved oral medication that acts as a Smoothened (SMO) antagonist, providing a new treatment option for patients with advanced basal cell carcinoma who cannot undergo surgery or radiotherapy.

The article reviews the pharmacology and pharmacokinetics of sonidegib, along with insights from the BOLT trial, highlighting its efficacy and the ongoing challenges faced with Hedgehog inhibitors.

NCBI (Pubmed)

pubmed.ncbi.nlm.nih.gov

Sonidegib for the treatment of advanced basal cell carcinoma: a comprehensive review of sonidegib and the BOLT trial with 12-month update. Chen, L., Silapunt, S., Migden, MR.[2019]

In a comparison of sonidegib and vismodegib for treating advanced basal cell carcinoma, sonidegib showed a lower overall incidence of adverse events (AEs) and a slower onset of these events, suggesting it may be a safer option for patients.

Common AEs included muscle spasms and alopecia, with sonidegib patients experiencing fewer severe side effects compared to vismodegib, although further studies are needed to confirm these findings due to the lack of direct head-to-head trials.

NCBI (Pubmed)

pubmed.ncbi.nlm.nih.gov

Key Clinical Adverse Events in Patients with Advanced Basal Cell Carcinoma Treated with Sonidegib or Vismodegib: A Post Hoc Analysis. Gutzmer, R., Loquai, C., Robert, C., et al.[2022]

Sonidegib is an effective treatment for advanced basal cell carcinoma (BCC), showing objective response rates of 43% for the 200 mg dose and 38% for the 800 mg dose in the BOLT clinical trials, which involved patients who could not undergo surgery or radiation.

The 200 mg dose of sonidegib resulted in fewer adverse events compared to the 800 mg dose, suggesting that lower doses may provide a better benefit-to-risk ratio for patients.

NCBI (Pubmed)

pubmed.ncbi.nlm.nih.gov

Differential pharmacology and clinical utility of sonidegib in advanced basal cell carcinoma. Wahid, M., Jawed, A., Dar, SA., et al.[2020]

Citations

1. clinicaltrials.gov clinicaltrials.gov/study/NCT06050122

NCT06050122 | Efficacy and Safety of Patidegib Gel 2% for ... The aim of this clinical study is to find out how well Patidegib Gel 2% works in preventing new basal cell carcinomas (BCCs) developing on the face of ...

2. pubmed.ncbi.nlm.nih.gov pubmed.ncbi.nlm.nih.gov/39545486/

Topical application of the Hedgehog inhibitor patidegib in ... The findings from this study suggest that patidegib gel applied to the skin could reduce the number of new BCC tumours.

3. clinicaltrials.gov clinicaltrials.gov/study/NCT03703310

NCT03703310 | Study of Patidegib Topical Gel, 2%, for the ... This is a global, multicenter, randomized, double-blind, stratified, vehicle-controlled study of the efficacy and safety of Patidegib Topical Gel, 2%, ...

4. bridgebio.com bridgebio.com/news/pellepharm-presents-updated-data-from-two-phase-2-studies/

PellePharm | New Data from Two Phase 2 Studies Use of patidegib topical gel 2% was significantly more effective in clinical and histologic clearance of BCCs after 3 months compared with the ...

5. clinicaltrials.stanford.edu clinicaltrials.stanford.edu/trials/s/NCT03703310.html

Study of Patidegib Topical Gel, 2%, for the Reduction of ... The primary endpoint is a comparison between the two treatment arms of the number of new BCCs that develop over the 12 month period. Official Title. A ...

6. academic.oup.com academic.oup.com/bjd/article-abstract/192/4/565/8024190

Patidegib: a novel, promising treatment for basal cell ... Seventeen patients with Gorlin syndrome with a total of 85 surgically eligible BCC (SEBs) were randomized 1 : 1 : 1 to patidegib topical gel 2%, ...

7. mayo.edu mayo.edu/research/clinical-trials/cls-20467341

A Study of Patidegib Topical Gel, 2%, for the Reduction of ... The purpose of this study is to evaluate the effectiveness and safety of Patidegib Topical Gel, 2%, applied topically twice daily to the face of adult ...

8. ir.sol-gel.com ir.sol-gel.com/news-releases/news-release-details/sol-gel-acquires-patidegib-phase-3-fda-breakthrough-designated

Sol-Gel Acquires Patidegib, a Phase 3, FDA-Breakthrough ... Sol-Gel acquires Patidegib, a Phase 3, FDA-Breakthrough-Designated Orphan Product Candidate to pursue potential market of over $300 million.