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 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. The histologic subtype can also help give your doctor information on
how quickly your basal cell skin cancer will grow, if it will come back or if it will spread
to other parts of your body.
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). We know that the subtype of BCC 'can affect response to other types of treatment.
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 us more information on how
your tumor is responding to vismodegib. If there is no evidence of tumor on the biopsy, you
will be eligible to end treatment early and enter the Observation period. During this time
you 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, you 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,
you will be eligible to end treatment early and enter the Observation period. If there is
tumor left, you will be referred for surgery or other standard of care treatment to remove
Endpoint Classification: Safety/Efficacy Study, Intervention Model: Single Group Assignment, Masking: Open Label, Primary Purpose: Treatment
Efficacy of vismodegib in treatment of target lesion(s) by clinical and histologic response evaluation. Clinical Response: Comparison of # of target lesions in each group with overall response rate and best overall response rate Comparison of maximum diameter and lesion area of target lesion(s) post-biopsy and at End of Treatment Comparison of dimensions of tumor within "debulking" specimen at surgery visit with dimensions of pre-biopsy tumor Histologic response with evaluation of: Residual tumor: Evidence of any histologic change in residual tumor tissue compared to pre-treatment biopsy specimens and estimate of changes in cellular density and composition Area of Tumor Clearance with assessment of histologic clearance Comparison of deepest "invasion index" on specimens Histologic subtypes observed on pathologic specimens with location and depth of specific tumor cells Percentage of each histologic subtype in tumor specimen on biopsy
Scott W Fosko, MD
Saint Louis University Dermatology
United States: Institutional Review Board
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