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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

Phase 2
18 Years
90 Years
Open (Enrolling)
Basal Cell Carcinoma

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Trial Information

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
the tumor.

Inclusion Criteria

Inclusion Criteria

1. A signed and data informed consent

2. Willing to comply with scheduled visits, treatment plan, laboratory tests, and other
study procedures

3. 18-90 years of age at time of informed consent

4. Have one or more clinically suspicious lesions for BCC at Pre-Study screening Visit
that has:

1. a diameter ≥ 6 mm if located on the "mask areas" of face (central face, eyelids,
eyebrows, periorbital,nose,lips,chin,mandible,preauricular and postauricular
skin/sulci,temple,ear),genitalia,hands,or feet

2. a diameter ≥ 10 mm if located on cheeks,forehead,scalp,or neck

3. a diameter ≥ 20 mm if located on trunk and extremities

or has a lesion suspicious for locally advanced BCC defined as a lesion that:

4. is ≥ 10 mm,

5. has recurred following surgery or surgical resection would result in substantial
deformity, and

6. has been deemed not appropriate for radiation.

5. Have a histologically-confirmed BCC prior to first dose of study drug

6. Have an Eastern Cooperative Oncology Group performance status of 2 or less at

7. Female of reproductive potential must use 2 effective methods to avoid pregnancy
during therapy and for 7 months after completing therapy

8. Male patients must use effective measures to avoid pregnancy in their partner at all
times during treatment and for 2 months after the last dose

9. Agree not to donate blood or blood products during the study and for 7 months after
the last dose

10. Subjects with Basal Cell Nevus Syndrome are eligible for enrollment

Exclusion Criteria:

1. Women who are pregnant, lactating, or planning pregnancy while in the study

2. History of prior treatment with vismodegib or any Hh Pathway Inhibitor

3. Evidence of clinically significant and unstable diseases or conditions; Subjects with
clinically stable chronic medical conditions will be allowed to enter the study

4. Any dermatological disease at treatment site that the investigator thinks may be
exacerbated by treatment with vismodegib or cause difficulty with examination

5. The target lesion identified at Pre-study Screening visit has been determined to be
mBCC by radiological assessment prior to first dose of study drug

6. Inability or unwillingness to swallow capsules

7. History of infection requiring hospitalization, IV antimicrobial therapy, or is
otherwise judged to be clinically significant by the investigator within 4 wks prior
to first dose of study drug

8. History of infection requiring antimicrobial therapy within 2 wks prior to first dose
of study drug

9. History of alcohol or substance abuse, unless in full remission for greater than 6
months prior to first dose of study drug

10. Known to be infected with human immunodeficiency virus, hepatitis B or hepatitis C

11. Participation in other study using an investigational or experimental therapy or
procedure within 4 weeks or 5 half-lives (whichever is longer) before the study
begins and/or during study participation

12. Other severe acute or chronic medical or psychiatric condition or laboratory
abnormality that may increase the risk associated with study participation or
investigational product administration or may interfere with the interpretation of
study results in the judgment of the investigator

13. Subjects who are study site staff members or who are Sponsor employees directly
involved in the conduct of the trial

14. A subject who, in the opinion of the investigator or sponsor, will be uncooperative
or unable to comply with study procedures

Type of Study:


Study Design:

Endpoint Classification: Safety/Efficacy Study, Intervention Model: Single Group Assignment, Masking: Open Label, Primary Purpose: Treatment

Outcome Measure:


Outcome Description:

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

Outcome Time Frame:

Week 24

Safety Issue:


Principal Investigator

Scott W Fosko, MD

Investigator Role:

Principal Investigator

Investigator Affiliation:

Saint Louis University Dermatology


United States: Institutional Review Board

Study ID:




Start Date:

April 2013

Completion Date:

January 2016

Related Keywords:

  • Basal Cell Carcinoma
  • advanced BCC
  • high risk
  • locally advanced
  • Carcinoma
  • Carcinoma, Basal Cell



Saint Louis University Dermatology Saint Louis, Missouri  63104