Active Surveillance for Basal Cell Carcinoma

Age: 65+

Sex: Any

Trial Phase: Academic

Sponsor: University of Michigan Rogel Cancer Center

Must not be taking: Immunosuppressive medications

Disqualifiers: Immunocompromised, Relocation, Genetic syndrome, others

No Placebo Group All trial participants will receive the active study treatment (no placebo)

Approved in 5 Jurisdictions This treatment is already approved in other countries

Compensation: Varies

Number of Visits: 2

200 Participants Needed

Trial Summary

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

The trial protocol does not specify if you need to stop taking your current medications. However, if you are on immunosuppressive medications like prednisone > 10 mg daily, you may be excluded from participating.

Will I have to stop taking my current medications?

The trial information does not specify whether you need to stop taking your current medications. However, if you are on immunosuppressive medications like prednisone over 10 mg daily, you may not be eligible to participate.

What data supports the idea that Active Surveillance for Basal Cell Carcinoma (also known as: Active Surveillance, Watchful Waiting, Observation) is an effective treatment?

The available research does not provide specific data supporting Active Surveillance as an effective treatment for Basal Cell Carcinoma. Instead, it highlights surgery as the standard treatment due to its ability to control the cancer and low risk of it coming back. Other treatments like radiation and certain drugs are used for more advanced cases. Active Surveillance might be considered for low-risk cases, but the research does not provide specific outcomes or data for its effectiveness compared to these other treatments. ^{1 2 3 4 5}

What data supports the effectiveness of the treatment Active Surveillance, Watchful Waiting, Observation for basal cell carcinoma?

The research does not provide direct evidence for the effectiveness of active surveillance for basal cell carcinoma, but it mentions that basal cell carcinoma rarely metastasizes and has low mortality, suggesting that less aggressive management like active surveillance might be appropriate for certain low-risk cases. ^{1 2 3 4 5}

What safety data exists for active surveillance of basal cell carcinoma?

The provided research does not directly address safety data for active surveillance, watchful waiting, or observation of basal cell carcinoma. The studies focus on the epidemiology, risk factors, genetic mutations, and treatment options for basal cell carcinoma, but do not specifically evaluate the safety of non-interventional management strategies like active surveillance. ^{1 4 6 7 8}

Is Active Surveillance a promising treatment for Basal Cell Carcinoma?

Active Surveillance, also known as Watchful Waiting, is a promising treatment for Basal Cell Carcinoma because it allows doctors to monitor the cancer without immediate surgery or other treatments. This approach can be suitable for patients who may not need aggressive treatment right away, especially if the cancer is not causing problems or if the patient has other health concerns. ^{1 4 9 10 11}

How does active surveillance differ from other treatments for basal cell carcinoma?

Active surveillance, also known as watchful waiting, is unique because it involves closely monitoring basal cell carcinoma without immediate treatment, which can be suitable for patients who may not live long enough to benefit from more aggressive treatments. This approach contrasts with standard treatments like surgery or topical therapies, which actively remove or treat the cancer. ^{1 4 9 10 11}

What is the purpose of this trial?

This clinical trial evaluates whether active surveillance (AS) is a safe and comfortable alternative to standard of care (SOC) treatment for elderly patients with low-risk basal cell carcinoma (LR-BCC). Basal cell carcinoma is a type of slow-growing skin cancer that has a very low risk of spreading inside the body (metastasis) or death. Basal cell skin cancers that are smaller across than a nickel in size and located on the trunk or limbs are particularly low risk to overall health. Active surveillance - watching and not treating unless the cancer worsens - has been shown to be a generally safe way to manage LR-BCC. Despite this, many doctors do not feel comfortable discussing this option with patients due to a lack of studies comparing it to standard of care treatment. Standard of care treatment for LR-BCC can include "scrape and burn" (electrodesiccation and curettage), surgical resection, Mohs surgery, and other approaches. These treatments can carry risks like post-operative bleeding and wound infection, and they do not always improve tumor-related quality of life. Active surveillance may be a safe and comfortable alternative to SOC treatment for elderly patients with LR-BCC.

Research Team

Allison Billi

Principal Investigator

University of Michigan Rogel Cancer Center

Eligibility Criteria

This trial is for elderly patients with low-risk basal cell carcinoma (LR-BCC), a type of skin cancer. It's specifically for those whose cancers are smaller than a nickel and located on the trunk or limbs. Patients must be comfortable with active surveillance, which means monitoring the cancer without immediate treatment.

Inclusion Criteria

I have been diagnosed with a low-risk basal cell carcinoma smaller than 2cm, not on my hands, feet, or face.

Willing and able to provide informed consent

Willing and able to comply with the protocol requirements

Exclusion Criteria

I am considered immunocompromised due to medication, HIV, or an organ transplant.

Individuals who expect to relocate and will be unable to return to UMichDD for clinical follow-up visit(s)

I have a genetic condition that increases my risk for basal cell carcinoma.

Timeline

Screening

Participants are screened for eligibility to participate in the trial

2-4 weeks

Educational Activity and Survey

Participants watch an educational video on active surveillance for low-risk basal cell carcinoma and complete a survey about their comfort with this approach.

1 visit

1 visit (in-person or virtual)

Active Surveillance or Standard of Care Treatment

Participants are randomized to either active surveillance with no treatment unless disease progression occurs, or standard of care treatment which may include electrodesiccation and curettage, surgical resection, or Mohs surgery.

1 year

Regular monitoring visits as needed

Follow-up

Participants complete a standard 1-year follow-up total body skin examination followed by a second survey to assess satisfaction and outcomes.

1 year

1 visit (in-person)

Treatment Details

Interventions

• Active Surveillance

Trial Overview The study is testing if watching and waiting (active surveillance) is as safe and comfortable as standard treatments like surgery for small, low-risk skin cancers in elderly patients. Participants will not receive immediate treatment unless their cancer worsens.

Participant Groups

Group I: Part A (educational activity, survey administration) Experimental Treatment 2 Interventions

Patients watch an educational video on AS for LR-BCC and complete a survey about comfort with AS for LR-BCC on study.

Group II: Part B arm 1 (active surveillance) Active Control 2 Interventions

Patients receive no treatment and undergo active surveillance over 1 year in the absence of disease progression. Patients may choose to undergo treatment at any point on study. Patients complete a standard 1-year follow-up total body skin examination followed by a second survey.

Group III: Part B arm 2 (SOC) Active Control 2 Interventions

Patients receive SOC treatment which typically includes either electrodesiccation and curettage, surgical resection, or Mohs surgery as agreed upon by patient and provider in the absence of disease progression. Patients complete a standard 1-year follow-up total body skin examination followed by a second survey.

Active Surveillance is already approved in United States, European Union, Canada, Japan for the following indications:

Approved in United States as Active Surveillance for:

• Papillary thyroid microcarcinoma (PTMC)

Approved in European Union as Active Surveillance for:

• Papillary thyroid microcarcinoma (PTMC)

Approved in Canada as Active Surveillance for:

• Papillary thyroid microcarcinoma (PTMC)

Approved in Japan as Active Surveillance for:

• Papillary thyroid microcarcinoma (PTMC)

Who Is Running the Clinical Trial?

University of Michigan Rogel Cancer Center

Lead Sponsor

Trials

303

Recruited

20,700+

Findings from Research

Basal cell carcinoma (BCC) is the most common skin cancer, primarily affecting light-skinned individuals, with risk factors including UV exposure and genetic mutations in the hedgehog pathway.

Most BCC cases can be effectively treated with standard surgery or topical treatments, while advanced cases may require radiation or systemic therapies, including hedgehog inhibitors like vismodegib and sonidegib.

NCBI (Pubmed)

pubmed.ncbi.nlm.nih.gov

Known and new facts on basal cell carcinoma. Seidl-Philipp, M., Frischhut, N., Höllweger, N., et al.[2021]

In a study of 34 patients with basal cell carcinoma (BCC) that had positive surgical margins, EGFR expression was found to be significantly higher in tumor tissues compared to healthy tissues, indicating its potential role in tumor behavior.

Patients with recurrent BCC lesions exhibited EGFR expression levels that were 6.66 times higher than those without recurrence, suggesting that EGFR overexpression may be linked to increased recurrence rates, particularly in infiltrative subtypes.

NCBI (Pubmed)

pubmed.ncbi.nlm.nih.gov

The role of EGFR overexpression on the recurrence of basal cell carcinomas with positive surgical margins. Biray Avci, C., Kaya, I., Ozturk, A., et al.[2019]

In a study of 1,832 basal cell carcinomas excised from 1,329 patients over six years, 14% of the lesions were found to be incompletely excised, highlighting a significant challenge in surgical treatment.

The study identified that the location of the tumor, particularly those on the nose, was a key predictor of incomplete excision, suggesting that surgical techniques may need to be adjusted based on tumor location to improve outcomes.

NCBI (Pubmed)

pubmed.ncbi.nlm.nih.gov

Risk and outcome analysis of 1832 consecutively excised basal cell carcinomas in a tertiary referral plastic surgery unit. Malik, V., Goh, KS., Leong, S., et al.[2010]

A study of 14,628 participants identified that 293 individuals developed a second basal cell carcinoma (BCC) within a median of 3 years, highlighting the significant risk of recurrence after an initial BCC diagnosis.

The developed prediction model, which uses 13 clinical characteristics, can categorize patients into three risk groups (7%, 12%, and 28% risk of a second BCC within 3 years), helping to identify those at higher risk for better monitoring and intervention.

NCBI (Pubmed)

pubmed.ncbi.nlm.nih.gov

Predicting the Risk of a Second Basal Cell Carcinoma. Verkouteren, JAC., Smedinga, H., Steyerberg, EW., et al.[2018]

Basal cell carcinomas (BCCs) are the most common skin cancers in fair-skinned adults over 50, primarily caused by UV exposure, and their management has been updated in recent guidelines to address varying prognoses and treatment options.

For difficult-to-treat BCCs, hedgehog pathway inhibitors have been developed, and ongoing trials suggest that immunotherapy may be an option for tumors resistant to these inhibitors, highlighting the importance of personalized treatment approaches.

NCBI (Pubmed)

pubmed.ncbi.nlm.nih.gov

Update in the Management of Basal Cell Carcinoma. Basset-Seguin, N., Herms, F.[2022]

A retrospective study in Dorset, UK, revealed a significant increase in basal cell carcinoma (BCC) cases, with 2455 patients in 2006 rising to 3797 in 2016, indicating a higher incidence rate than previously reported in the UK.

The most common sites for BCC were found to be on the head and neck, particularly the cheek, nose, and forehead, highlighting the need for improved monitoring and preventive strategies in healthcare systems.

NCBI (Pubmed)

pubmed.ncbi.nlm.nih.gov

Epidemiology of basal cell carcinoma: a 10-year comparative study. Devine, C., Srinivasan, B., Sayan, A., et al.[2022]

From 2001 to 2019, over 601,000 patients were diagnosed with basal cell carcinoma (BCC) in the Netherlands, with age-standardized incidence rates for first BCCs significantly increasing for both men and women, indicating a growing public health concern.

While younger patients (under 50) show signs of stabilizing incidence rates, older patients (50 and above) continue to experience rising rates, with predictions suggesting a further increase of over 30% in BCC cases in the next decade.

NCBI (Pubmed)

pubmed.ncbi.nlm.nih.gov

A nationwide study of the incidence and trends of first and multiple basal cell carcinomas in the Netherlands and prediction of future incidence. Schreuder, K., Hollestein, L., Nijsten, TEC., et al.[2022]

The study found that mutations in the PTCH gene, particularly those associated with UV exposure, are common in basal cell carcinoma (BCC) patients, occurring in 48% of cases, but these mutations do not vary significantly among different age groups or lesion counts.

Certain single-nucleotide polymorphisms (SNPs) in the PTCH gene, specifically IVS16-80G/C and IVS17+21G/A, may play a role in tumorigenesis for some BCC patients, although their distribution did not differ significantly across early-onset and regular BCC cases.

NCBI (Pubmed)

pubmed.ncbi.nlm.nih.gov

UV fingerprints predominate in the PTCH mutation spectra of basal cell carcinomas independent of clinical phenotype. Heitzer, E., Lassacher, A., Quehenberger, F., et al.[2020]

In a study of 89 patients with basal cell carcinoma (BCC) who chose watchful waiting (WW) for a median follow-up of 9 months, the majority (83%) opted for this approach due to personal factors like frailty or limited life expectancy, indicating that WW can be a suitable option for certain patients.

Only 47% of the tumors increased in size during the observation period, with tumor growth linked to specific BCC subtypes, suggesting that many patients may safely delay treatment without significant risk of rapid tumor progression.

NCBI (Pubmed)

pubmed.ncbi.nlm.nih.gov

Evaluation of Watchful Waiting and Tumor Behavior in Patients With Basal Cell Carcinoma: An Observational Cohort Study of 280 Basal Cell Carcinomas in 89 Patients. van Winden, MEC., Hetterschijt, CRM., Bronkhorst, EM., et al.[2022]

In a 10-year study of basal cell carcinoma (BCC), only 53 tumors were found in the groin or buttocks, representing a very rare occurrence (0.094% of all BCCs), with the most common subtype being nodular (49%).

The study found that these BCCs in nonexposed areas were not associated with genetic predispositions or immunosuppression, and their histological characteristics were not more aggressive than BCCs found in other body locations.

NCBI (Pubmed)

pubmed.ncbi.nlm.nih.gov

Basal cell carcinoma of the groin and buttocks: a clinical and histological analysis of a rare presentation of a common tumor. Tidwell, WJ., Sutton, A., Gilbertson, R., et al.[2019]

Surgery, particularly micrographically controlled surgery, remains the gold standard for treating basal cell carcinoma, while non-invasive methods and histological confirmation are important for accurate diagnosis and risk stratification.

For advanced or metastatic cases, effective treatments like hedgehog inhibitors and the PD-1 inhibitor cemiplimab are available, alongside preventive measures such as UV protection and oral medications like nicotinamide.

NCBI (Pubmed)

pubmed.ncbi.nlm.nih.gov

[Current Management of Basal Cell Carcinoma]. Krieter, M., Schultz, E.[2023]

References

1. Germany pubmed.ncbi.nlm.nih.gov

Known and new facts on basal cell carcinoma. [2021]

2. Netherlands pubmed.ncbi.nlm.nih.gov

The role of EGFR overexpression on the recurrence of basal cell carcinomas with positive surgical margins. [2019]

3. Netherlands pubmed.ncbi.nlm.nih.gov

Risk and outcome analysis of 1832 consecutively excised basal cell carcinomas in a tertiary referral plastic surgery unit. [2010]

4. United States pubmed.ncbi.nlm.nih.gov

Predicting the Risk of a Second Basal Cell Carcinoma. [2018]

5. Sweden pubmed.ncbi.nlm.nih.gov

Update in the Management of Basal Cell Carcinoma. [2022]

6. Scotland pubmed.ncbi.nlm.nih.gov

Epidemiology of basal cell carcinoma: a 10-year comparative study. [2022]

7. England pubmed.ncbi.nlm.nih.gov

A nationwide study of the incidence and trends of first and multiple basal cell carcinomas in the Netherlands and prediction of future incidence. [2022]

8. United States pubmed.ncbi.nlm.nih.gov

UV fingerprints predominate in the PTCH mutation spectra of basal cell carcinomas independent of clinical phenotype. [2020]

9. United States pubmed.ncbi.nlm.nih.gov

Evaluation of Watchful Waiting and Tumor Behavior in Patients With Basal Cell Carcinoma: An Observational Cohort Study of 280 Basal Cell Carcinomas in 89 Patients. [2022]

10. England pubmed.ncbi.nlm.nih.gov

Basal cell carcinoma of the groin and buttocks: a clinical and histological analysis of a rare presentation of a common tumor. [2019]

11. Germany pubmed.ncbi.nlm.nih.gov

[Current Management of Basal Cell Carcinoma]. [2023]