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Carcinoma, Basal Cell

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Neoplasms, Basal Cell

This is a Phase 2B single-site, open-label, nonrandomized 24-week study of the efficacy and safety of vismodegib (150 mg PO daily) in subjects with high risk and/or locally advanced basal cell carcinoma (BCC). A total of 36 subjects with infiltrative/morpheaform, nodular, or superficial BCC will be enrolled in the study.

Biopsies will be performed on all participants at baseline, week 12 and week 24.

vismodegib (150 mg PO daily)

Scott Fosko, MD

The purpose of this study is to investigate the safety and effectiveness of oral vismodegib therapy in the treatment of different 'histologic subtypes' of basal cell skin cancer (BCC). The term 'histologic subtype' refers to how the cells and tumor tissue looks under the microscope. Three different 'histologic subtypes' of basal cell skin cancer (infiltrative/morpheaform, nodular and superficial) will be examined in this study.

The purpose of this study is to investigate the safety and effectiveness of oral vismodegib therapy in the treatment of different 'histologic subtypes' of basal cell skin cancer (BCC). The term 'histologic subtype' refers to how the cells and tumor tissue looks under the microscope. Three different 'histologic subtypes' of basal cell skin cancer (infiltrative/morpheaform, nodular and superficial) will be examined in this study. Each subtype has a characteristic look under the microscope, which is related to how the tumor will behave and grow.

ERIVEDGE (oral vismodegib capsule) has been approved for use in the United States for treatment of metastatic BCC tumors (mBCC), tumors that have spread further into the skin, bones or other tissues, or spread to other parts of the body and locally advanced basal cell skin cancer (laBCC), cancers that have come back after surgery or that the healthcare provider thinks cannot be treated with surgery or radiation. It works by blocking the signal, called Hedgehog, which basal cell skin cancer cells need to grow. It has been given to about 800 people during clinical trials.

Data from previous studies is mostly based on a subtype of BCC made up of little round collections of cancer cells, called "Nodular". There is almost no data on the use of vismodegib in other subtypes of BCC that that tend to extend deep into the skin ("Infiltrative" subtype), or spread widely near the surface of the skin ("Superficial" subtype).

A total of 36 subjects will be enrolled in the study. All study participants will receive oral vismodegib treatment.

At the Week 12 visit, skin biopsies will be performed to give the investigators more information on how the tumor is responding to vismodegib. If there is no evidence of tumor on the biopsy, the subject will be eligible to end treatment early and enter the Observation period. During this time the subject will be followed clinically every 3 months for up to 1 year.

For all other subjects, if any evidence of tumor is seen on biopsy at week 12, the subject will continue treatment for the full 24 weeks. At week 24 visit, skin biopsies will be performed again to see if there is any tumor left. If there is no evidence of tumor on the biopsy, the subject will be eligible to end treatment early and enter the Observation period. If there is tumor left, the subject will be referred for surgery or other standard of care treatment to remove the tumor.

Study Evaluating the Efficacy of Oral Vismodegib in Various Histologic Subtypes

ML28485:Phase 2B Single-site,Open-label,Nonrandomized Study Evaluating Efficacy of Oral Vismodegib in Various Histologic Subtypes (Infiltrative/Morpheaform,Nodular and Superficial)of High Risk and/or Locally Advanced Basal Cell Carcinoma

The efficacy of vismodegib was defined as the number of tumor biopsies with positive pathology after 24 weeks. Subjects had one target lesion and up to 3 additional non-target lesions. A cumulative total of 65 tumors was measured after 24 weeks of treatment. Histopathological subtypes were categorized primarily as infiltrative, nodular and superficial.

The safety of Vismodegib was evaluated by monitoring adverse effects. All adverse events, expected and unexpected, were recorded and categorized using the Common Terminology Criteria for Adverse Events (CTCAE) guide. This is a set of criteria from the National Cancer Institute (NCI) used to standardize classification of adverse effects of drugs. Grade 1 events are defined as mild, grade 2 as moderate, grade 3 as severe; grade 4 as life-threatening and grade 5 as death.

Onset of efficacy was measured by tumor surface area reduction or increase. Subjects had one target lesion and up to 3 additional non-target lesions. Surface area of a cumulative total of 65 tumors (27 target lesions and 38 non-target lesions) was measured after 24 weeks of treatment. A complete response was defined as 100% reduction in tumor surface area. Partial response was defined as greater than 50% reduction in tumor surface area. Stable disease was defined as less than 50% reduction in tumor surface area and Progressive disease was defined as greater than 20% increase in tumor surface area.

Genentech, Inc.

Mayo Clinic

Principal Investigator

This is a Phase 2B single-site, open-label, nonrandomized 24-week study of the efficacy and safety of vismodegib (150 mg PO daily) in subjects with high risk and/or locally advanced basal cell carcinoma.

vismodegib (150 mg PO daily): Biopsies will be performed on all participants at baseline, week 12 and week 24.

Open Label Oral Vismodegib

Age, Continuous

Sex: Female, Male

Race (NIH/OMB)

United States

Region of Enrollment

Dr. Scott Fosko

Infiltrative

Nodular

Superficial

Keratotic

A total of 65 tumors were categorized into histopathologic subtype where 24 of 65 were infiltrative, 29 of 65 were nodular, 10 of 65 were superficial and 2 out of 65 were keratotic.

Efficacy of Vismodegib

Grade 1

Grade 2

Grade 3

Grade 4

Grade 5

This is a Phase 2B single-site, open-label, nonrandomized 24-week study of the efficacy and safety of vismodegib (150 mg PO daily) in subjects with high risk and/or locally advanced basal cell carcinoma. vismodegib (150 mg PO daily): Biopsies will be performed on all participants at baseline, week 12 and week 24.

Safety of Vismodegib

Complete response

Partial response

Stable Disease

Progressive Disease

Onset of Efficacy of Vismodegib

This is a Phase 2B single-site, open-label, nonrandomized 24-week study of the efficacy and safety of vismodegib (150 mg PO daily) in subjects with high risk and/or locally advanced basal cell carcinoma (BCC).

vismodegib (150 mg PO daily): Biopsies will be performed on all participants at baseline, week 12 and week 24.

Study title	NCT ID	Conditions	Interventions	Eligibility	Organization	Link
Study Evaluating the Efficacy of Oral Vismodegib in Various Histologic Subtypes	NCT01700049	Basal Cell Carc...	vismodegib (150...	18 Years -	Mayo Clinic
Study Evaluating the Efficacy of Oral Vismodegib in Various Histologic Subtypes	NCT01700049	Basal Cell Carc...	vismodegib (150...	18 Years -	Mayo Clinic

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