Basal Cell Carcinoma of the SkinEarly Phase 1NCT01631331
Vismodegib in Treating Patients With Basal Cell Carcinoma (BCC)
A Early Phase 1 study testing vismodegib and Mohs surgery in people with Basal Cell Carcinoma of the Skin and Recurrent Skin Cancer, run by Stanford University at 1 site in United States.
Trial summary
The purpose of this study is to learn about the effect of vismodegib on sporadic basal cell carcinoma (BCCs) prior to surgical removal. Read more about this study
- Who is it for?
- Ages 18 years and older
- Conditions the study is for
- Basal Cell Carcinoma of the Skin, Recurrent Skin Cancer
- What is being tested?
- vismodegib and Mohs surgery
- Type of treatment
- Surgery or procedure
- Could I receive a placebo?
- No, everyone receives an active treatment
- How many people
- 15 participants
- Where
- 1 siteUnited States
- Study dates
- Jun 2012to 31 May 2016
- Phase
- Early Phase 1
- Are there results?
- Yes, posted in the registrySummary in the doctors' view
Key requirements
Who can take part, as listed by the sponsor. Only the study team can confirm whether you are eligible, so take this list to your doctor.
- Ages 18 years and older
- All sexes
- No healthy volunteers
- Adult, Older adult
Who may be able to join
- Study patients must have at least one BCC, > 5 mm, eligible for Mohs surgical removal; patients with BCCs that have been treated before (recurrent BCCs, BCCs that failed other chemotherapy) are eligible for this trial, if they meet size criteria
- No Eastern Cooperative Oncology Group (ECOG) or Karnofsky performance status will be employed
- Normal hepatic function: aspartate aminotransferase (AST) and alanine aminotransferase (ALT) =< 2 x the upper limit of normal (ULN)
- Normal renal function : normal serum creatinine defined as <= 2.5 mg/dL
- Clinically acceptable complete blood count (CBC)
- Ability to understand and the willingness to sign a written informed consent document
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- The patient is willing to forego surgical treatment of BCCs by up to 6 months, except when the principal investigator (PI) believes that delay in treatment potentially might compromise the health of the subject
- Documented negative serum pregnancy test for women of childbearing potential, with agreement to the use of two acceptable methods of contraception during the study and for 7 months after discontinuation of vismodegib
- For men with female partners of childbearing potential, agreement to use a latex, non-latex, or any other male condom and to advise their female partners to use an additional acceptable method of birth control during the study and for 2 months after discontinuation of study drug
- Be willing to not donate blood or semen for three months following discontinuation of study medications
Who cannot join
- The patient has a history of invasive cancer within the past five years excluding non-melanoma skin cancer, stage I cervical cancer, ductal carcinoma in situ of the breast, or chronic lymphocytic leukemia (CLL) stage 0
- The subject has uncontrolled systemic disease, including known human immunodeficiency virus (HIV) positive patients:
- The patient has history of congestive heart failure
- The patient has clinically important history of liver disease, including viral or hepatitis, current alcohol abuse, or cirrhosis
- The patient has any condition or situation which in the investigator's opinion may put the patient at significant risk, could confound the study results, or could interfere significantly with the subject's participation in the study; this includes history of other skin conditions or disease, metabolic dysfunction, physical examination finding, or clinical laboratory finding giving reasonable suspicion of a disease or condition that contraindicates use of an investigational drug or that might affect interpretation of the results of the study or render the patient at high risk from treatment complications
- The patient has a history of hypersensitivity to any of the ingredients in the study medication formulations
- The patient is willing to abstain from application of non-study topical medications to the skin for the duration of the study, including prescription and over the counter preparations; for example, topical preparations containing corticosteroids or vitamin A derivatives are not allowed
- Pregnant or nursing patients will be excluded from the study
Trial locations and contacts
1 site in 1 country.
About this study
Official title: A Pilot Study to Investigate the Off Label Use of Vismodegib as an Adjuvant to Surgery for Basal Cell Carcinoma Tumors (BCCs)
The purpose of this study is to learn about the effect of vismodegib on sporadic basal cell carcinoma (BCCs) prior to surgical removal.
Read the detailed description
PRIMARY OBJECTIVES:
- The percent reduction in surgical defect area/size surrounding BCC tumor pre and post-vismodegib.
SECONDARY OBJECTIVES:
- Recurrence rate post treatment II. Safety, tolerability and percent drop-out after 3 vs. 6 months of vismodegib in otherwise healthy patients.
OUTLINE:
Patients receive vismodegib orally (PO) once daily (QD) for up to 3 months if the initial BCC size is < 2 cm and superficial or for up to 6 months if the initial BCC size is >= 2 cm or non-superficial. After completion of vismodegib treatment, patients undergo Mohs surgery.
After completion of study treatment, patients are followed up for an average of 24 months.
- Being tested
- vismodegib, Mohs surgery
- Medical subject headings
- Carcinoma, Basal Cell, Skin Neoplasms
What participants receive
What participants receive. In a randomised study each person is assigned to one group by chance.
vismodegibDrug
Also called Erivedge, GDC-0449, Hedgehog antagonist GDC-0449
Given PO
Mohs surgeryProcedure or surgery
Undergo Mohs surgery
| Study group | Role | What they receive |
|---|---|---|
| Treatment (vismodegib and Mohs surgery) | Experimental | Patients receive vismodegib PO daily for 3-6 months based on the size of basal cell carcinoma and then undergo Mohs surgery. Receives: Drug: vismodegib; Procedure: Mohs surgery |
What is being measured
The primary outcome is the main question the study is designed to answer.
Percent Change in Surgical Defect Area After the Treatment Period Using Calipers and Photographs Was Calculated
At baseline, we selected 1 to 2 tumors per patient for surgery (13 target tumors selected). At baseline,1 Mohs surgeon measured the estimated surgical defect area around the target tumor. For tumors to be excised by Mohs we defined estimated surgical defect as the tumor size plus a 2-mm circumferential margin, presuming tumor clearance after a Mohs stage-1 excision. For the tumor undergoing standard (non-Mohs) excision, we used tumor size plus a standard 4-mm margin11 for the estimated surgical defect. On the day of the surgery, we measured the surgical defect area as the final tumor-free defect after the Mohs procedure or non-Mohs excision immediately before closure. We used the Image J software program (National Institutes of Health, Bethesda, MD) to calculate tumor area (cm2). Only target tumors are included in this analysis.
Measured: average of 4 months
Secondary outcomes (3)
Number of Tumors Demonstrating Histologic Cure
Determination of histologic cure (no residual BCC on the first piece of excised tissue) post serial sectioning of paraffin embedded Mohs specimens
Measured: Average of 4 months
Tumor Recurrence Rate of Treated BCCs
Recurrence rate of BCCs during a 22 month average (range 12 to 28 months) follow up period.
Measured: average of 22 months
Tumor Size Measurements Before and After Short Term Vismodegib Treatment
We measured the length and width of all tumors (target and non-target) before and after vismodegib treatment.
Measured: 4 months (average)
Timeline
From the start of the study to its completion; the mark is today. Estimated dates are the sponsor’s plan.
- First posted29 Jun 2012 (estimated)
- Study startJun 2012
- Primary completion17 Sep 2015
- Study completion31 May 2016
Resources
- Official record on ClinicalTrials.govThe registry entry this page is built from, with its full change history.
- Posted resultsParticipant flow, outcome measures and adverse events reported by the sponsor.
- Publications (1)Papers the sponsor has linked to this study.
- Other trials for Basal Cell Carcinoma of the SkinStudies of the same condition, open and under way.
- More trials run by Stanford UniversityThe sponsor's other registered studies.
- What the trial phases meanHow a treatment moves from first tests in people to approval.
More details
For doctors and researchers: how the study is designed, who runs it, and its registry IDs.
Study design
- Study type
- Interventional
- Phase
- Early Phase 1
- Primary purpose
- Treatment
- Allocation
- Not applicable
- Intervention model
- Single group
- Masking
- None (open label)
- Enrolment
- 15 participants (actual)
Sponsor and investigators
- Lead sponsor
- Stanford University
- Sponsor type
- University, hospital or other
- Responsible party
- Principal investigator, Jean Yuh Tang, Stanford University
- Investigators
- Jean Tang, Principal investigator, Stanford University
IDs, dates and publications
- ClinicalTrials.gov ID
- NCT01631331
- Sponsor's protocol ID
- IRB-24313
- Other IDs
- NCI-2012-01055, SKIN0012
- Status last verified
- Aug 2017
- First submitted
- 25 Jun 2012
- Last updated
- 8 Dec 2017
- Results posted
- Yes, on ClinicalTrials.gov
- Data monitoring committee
- Yes
- FDA-regulated drug
- Yes
- FDA-regulated device
- No
- Individual data sharing
- No
Publications
Ally MS, Aasi S, Wysong A, Teng C, Anderson E, Bailey-Healy I, Oro A, Kim J, Chang AL, Tang JY. An investigator-initiated open-label clinical trial of vismodegib as a neoadjuvant to surgery for high-risk basal cell carcinoma. J Am Acad Dermatol. 2014 Nov;71(5):904-911.e1. doi: 10.1016/j.jaad.2014.05.020. Epub 2014 Jun 11.
PubMed 24929884
Study at a glance
From the registry record, last updated by the sponsor on 8 Dec 2017. Recruitment status is as the sponsor reported it: confirm with the study team.
- Official title
- A Pilot Study to Investigate the Off Label Use of Vismodegib as an Adjuvant to Surgery for Basal Cell Carcinoma Tumors (BCCs)
- ClinicalTrials.gov ID
- NCT01631331
- Acronym / sponsor ID
- IRB-24313
- Phase
- Early Phase 1
- Study type
- Interventional
- Recruitment status
- Completed
- Enrolment
- 15 participants (actual)
- Lead sponsor
- Stanford UniversityUniversity, hospital or other
- Collaborators
- National Cancer Institute (NCI), University Hospitals Cleveland Medical Center
- Study start
- Jun 2012
- Primary completion
- 17 Sep 2015 (actual)
- Study completion
- 31 May 2016 (actual)
- First posted
- 29 Jun 2012 (estimated)
- Results
- Posted 2 Oct 2017
Clinical summary
The research question as the sponsor registered it. This describes what the study plans to test, not what it found.
The purpose of this study is to learn about the effect of vismodegib on sporadic basal cell carcinoma (BCCs) prior to surgical removal.
Detailed description
PRIMARY OBJECTIVES:
- The percent reduction in surgical defect area/size surrounding BCC tumor pre and post-vismodegib.
SECONDARY OBJECTIVES:
- Recurrence rate post treatment II. Safety, tolerability and percent drop-out after 3 vs. 6 months of vismodegib in otherwise healthy patients.
OUTLINE:
Patients receive vismodegib orally (PO) once daily (QD) for up to 3 months if the initial BCC size is < 2 cm and superficial or for up to 6 months if the initial BCC size is >= 2 cm or non-superficial. After completion of vismodegib treatment, patients undergo Mohs surgery.
After completion of study treatment, patients are followed up for an average of 24 months.
Study design
- Primary purpose
- Treatment
- Allocation
- Not applicable
- Intervention model
- Single group
- Masking
- None (open label)
- Number of arms
- 1
| Arm | Type | Receives |
|---|---|---|
| Treatment (vismodegib and Mohs surgery) | Experimental | Drug: vismodegib, Procedure: Mohs surgeryPatients receive vismodegib PO daily for 3-6 months based on the size of basal cell carcinoma and then undergo Mohs surgery. |
Who may qualify
- Ages
- 18 years and older
- Sex
- All
- Healthy volunteers
- Not accepted
- Age groups
- Adult, Older adult
Key inclusion criteria (10 listed)
- Study patients must have at least one BCC, > 5 mm, eligible for Mohs surgical removal; patients with BCCs that have been treated before (recurrent BCCs, BCCs that failed other chemotherapy) are eligible for this trial, if they meet size criteria
- No Eastern Cooperative Oncology Group (ECOG) or Karnofsky performance status will be employed
- Normal hepatic function: aspartate aminotransferase (AST) and alanine aminotransferase (ALT) =< 2 x the upper limit of normal (ULN)
- Normal renal function : normal serum creatinine defined as <= 2.5 mg/dL
- Clinically acceptable complete blood count (CBC)
Key exclusion criteria (8 listed)
- The patient has a history of invasive cancer within the past five years excluding non-melanoma skin cancer, stage I cervical cancer, ductal carcinoma in situ of the breast, or chronic lymphocytic leukemia (CLL) stage 0
- The subject has uncontrolled systemic disease, including known human immunodeficiency virus (HIV) positive patients:
- The patient has a history of hypersensitivity to any of the ingredients in the study medication formulations
- The patient is willing to abstain from application of non-study topical medications to the skin for the duration of the study, including prescription and over the counter preparations; for example, topical preparations containing corticosteroids or vitamin A derivatives are not allowed
- Pregnant or nursing patients will be excluded from the study
Eligibility is decided by the study team after screening; this overview does not establish that a patient qualifies. Full inclusion and exclusion criteria.
Interventions
| Intervention | Type | Arms |
|---|---|---|
| vismodegibAlso: Erivedge, GDC-0449, Hedgehog antagonist GDC-0449Given PO | Drug | Treatment (vismodegib and Mohs surgery) |
| Mohs surgeryUndergo Mohs surgery | Procedure or surgery | Treatment (vismodegib and Mohs surgery) |
Primary outcome
Percent Change in Surgical Defect Area After the Treatment Period Using Calipers and Photographs Was Calculated
Time frame: average of 4 months
At baseline, we selected 1 to 2 tumors per patient for surgery (13 target tumors selected). At baseline,1 Mohs surgeon measured the estimated surgical defect area around the target tumor. For tumors to be excised by Mohs we defined estimated surgical defect as the tumor size plus a 2-mm circumferential margin, presuming tumor clearance after a Mohs stage-1 excision. For the tumor undergoing standard (non-Mohs) excision, we used tumor size plus a standard 4-mm margin11 for the estimated surgical defect. On the day of the surgery, we measured the surgical defect area as the final tumor-free defect after the Mohs procedure or non-Mohs excision immediately before closure. We used the Image J software program (National Institutes of Health, Bethesda, MD) to calculate tumor area (cm2). Only target tumors are included in this analysis.
3 secondary outcomes
Number of Tumors Demonstrating Histologic Cure
Time frame: Average of 4 months
Tumor Recurrence Rate of Treated BCCs
Time frame: average of 22 months
Tumor Size Measurements Before and After Short Term Vismodegib Treatment
Time frame: 4 months (average)
Results and safety
Loading the posted results from ClinicalTrials.gov…
Research links
- Official record on ClinicalTrials.govLast updated by the sponsor 8 Dec 2017.
- Posted resultsParticipant flow, baseline, all outcome measures and adverse events.
- Results publication: PubMed 24929884Ally MS, Aasi S, Wysong A, Teng C, Anderson E, Bailey-Healy I, Oro A, Kim J, Chang AL, Tang JY. An investigator-initiated open-label clinical trial of vismodegib as a neoadjuvant to surgery for high-risk basal cell carcinoma. J Am Acad Dermatol. 2014 Nov;71(5):904-911.e1. doi: 10.1016/j.jaad.2014.05.020. Epub 2014 Jun 11.
- Individual participant dataSharing planned: No
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Referral and contact
AzTrials does not enrol participants or take referrals. Contact the study team or a site listed below; they decide who can take part. This study is not recruiting at the moment.
Jean Tang principal investigator
Stanford University
1 site in 1 country: see every site and its status.
Questions about this trial
What is this clinical trial studying?
A Early Phase 1 study testing vismodegib and Mohs surgery in people with Basal Cell Carcinoma of the Skin and Recurrent Skin Cancer, run by Stanford University at 1 site in United States.
The full registered description is in the About this study section and the official record.
Who may be eligible to take part?
The sponsor lists 10 inclusion and 8 exclusion criteria. Eligibility is decided by the study team after screening, so read the full criteria and contact a listed site.
Is this clinical trial currently recruiting?
The record says: finished, as of the sponsor's last update on 8 Dec 2017. Status can change between updates, so confirm with the study contact before making plans.
Where is the study taking place?
The record lists 1 site in United States. Whether a site is taking part right now should be confirmed with that site. See every site.
What are the possible benefits and risks?
They depend on the study and the treatment, and no study can promise a benefit. The study team explains the known risks during informed consent, before anyone joins; the record itself does not list them.
What does this phase mean?
Early Phase 1 (sometimes called Phase 0) studies give a very small amount of a treatment to a few people, mainly to see how the body handles it.
Not every study follows the same path, and some studies (observational studies, device studies) have no phase.
Are results available for this study?
Yes. Results are posted in the registry (first posted 2 Oct 2017). A summary is in the For doctors view, and the full results are on ClinicalTrials.gov.
How can a doctor refer a patient or contact the research team?
The record lists no contact. The official record names the sponsor, who can be asked directly. AzTrials does not enrol participants or take referrals.
What does the primary outcome measure?
Percent Change in Surgical Defect Area After the Treatment Period Using Calipers and Photographs Was Calculated (time frame: average of 4 months). It is what the study was designed to measure, not a finding.
Where can I read the original study information?
On the ClinicalTrials.gov record for NCT01631331, which also lists 1 linked publication.
Does a listing mean the treatment is approved or proven to work?
No. A listing means the study is registered. It does not show that the treatment is safe, works, or is approved by a regulator.
How often is this information updated?
AzTrials copies changed records from ClinicalTrials.gov every night and reloads the whole registry every week. The sponsor last updated this record on 8 Dec 2017.