Vismodegib in Basal Cell Carcinomas (BCC) Chemoprevention

• Sponsor: University of Arizona (Other)
• Overall Status: Terminated
• CT.gov ID: NCT02067104
• Collaborator: (none)
• Enrollment: 9
• Location: 1
• Arms: 2
• Actual Duration (Months): 34.4
• Patients Per Site Per Month: 0.3

Study Details

Study Description

Brief Summary

This study is a single site double blinded Phase II study to evaluate the chemopreventative effectiveness of vismodegib in the treatment of subjects at high risk for developing basal cell carcinomas (BCC).

Condition or Disease	Intervention/Treatment	Phase
Basal Cell Carcinomas	Drug: Vismodegib; Drug: Placebo	Phase 2

Detailed Description

The primary objective of this study will be to determine if vismodegib pulse therapy will significantly reduce the incidence of newly diagnosed basal cell carcinomas (BCC) in high risk individuals when compared with placebo, as measured by the incidence of biopsy confirmed BCC over a 24 month period.

The secondary objective will be to determine that there will be no difference in the incidence of newly diagnosed squamous cell carcinomas (SCC) in the same subjects receiving vismodegib treatment when compared with placebo, as measured by the incidence of biopsy confirmed SCC over the same 24 month period.

Study Design

• Study Type: Interventional
• Actual Enrollment: 9 participants
• Allocation: Randomized
• Intervention Model: Parallel Assignment
• Masking: Quadruple (Participant, Care Provider, Investigator, Outcomes Assessor)
• Primary Purpose: Prevention
• Official Title: A Placebo Controlled Double-Blind Study to Evaluate the Efficacy of Vismodegib as Chemoprevention of New Bcc Development in High Risk Subjects
• Actual Study Start Date: Feb 17, 2014
• Actual Primary Completion Date: Dec 31, 2016
• Actual Study Completion Date: Dec 31, 2016

Arms and Interventions

Arm	Intervention/Treatment
Placebo Comparator: Placebo receive 150 mg of oral placebo daily for a period of 2 months	Drug: Placebo; Pulse therapy pattern of 2 months of daily treatment and 2 months of no treatment for 24 months.
Experimental: Vismodegib receive 150 mg of vismodegib daily for a period of 2 months	Drug: Vismodegib; Pulse therapy pattern of 2 months of daily treatment and 2 months of no treatment for 24 months.; Other Names: Erivedge

Outcome Measures

Primary Outcome Measures

1. The Effect of Vismodegib Pulse Therapy on the Incidence of Newly Diagnosed Basal Cell Carcinomas (BCC) [24 Months]

   Measured by the incidence of biopsy confirmed BCC over a 24 month period

Secondary Outcome Measures

1. The Incidence of Newly Diagnosed Squamous Cell Carcinomas (SCC) in the Same Subjects Receiving Vismodegib Treatment When Compared With Placebo [24 Months]

   Measured by the incidence of biopsy confirmed SCC over the same 24 month period

Eligibility Criteria

Criteria

• Ages Eligible for Study: 18 Years and Older
• Sexes Eligible for Study: All
• Accepts Healthy Volunteers: No

Inclusion Criteria:

• Signed and dated informed consent document indicating that the subject (or legally acceptable representative) has been informed of all pertinent aspects

• Comply with scheduled visits, treatment plan, laboratory tests, and other study procedures.

• At least 18 years of age at the time of informed consent.

• History of 3 or more biopsy confirmed BCCs in the preceding 2 years, calculated from 2 years prior to the screening visit.

• No active skin cancers.

• Women of reproductive potential must agree to use two forms of acceptable contraception

• Male subjects must agree to use condoms at all times, even after a vasectomy, during sexual intercourse with female partners of reproductive potential during treatment with vismodegib and for 2 months after the last dose to avoid exposing a pregnant partner and the unborn fetus to vismodegib.

• Male patients must agree not to donate sperm during the study and for 2 months after discontinuation of vismodegib

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

• Ability to understand and the willingness to sign a written informed consent document in English

Exclusion Criteria:

• Women who are pregnant or lactating, or planning pregnancy while enrolled in the study or for 7 months after the last dose of the study drug.

• Uncontrolled intercurrent illness including, but not limited to, ongoing or active infection, symptomatic congestive heart failure, unstable angina pectoris, cardiac arrhythmia, or psychiatric illness/social situations that would limit compliance with study requirements. Subjects with clinically stable chronic medical conditions including, but not limited to, controlled hypertension, diabetes mellitus type II, hypercholesterolemia, or osteoarthritis, will be allowed to enter the study.

• Inability or unwillingness to swallow capsules.

• Have a history of alcohol of substance abuse, unless in full remission for greater than 6 months prior to the screening visit (Day 0) when the consent form is signed.

• Known to be infected with human immunodeficiency virus (HIV), hepatitis B or hepatitis C viruses.

• Currently receiving vismodegib, biologics or chemotherapy

• Currently undergoing treatment with photodynamic therapy, topical chemotherapy agents including Imiquimod, fluorouracil

• Subjects who have Gorlins syndrome

• Subjects who have received any type of solid organ transplant

• Subjects taking immunosuppressive medications at the screening visit. (Day 0)

• Participation in other study using an investigational or experimental therapy or procedure within 4 weeks or 5 half-lives (whichever is longer) before the screening visit and/or during study participation. Subjects cannot participate in studies of other investigational or experimental therapies or procedures at any time during their participation in this study.

• 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 and, in the judgment of the investigator, would make the subject inappropriate for entry into this study.

• Subjects unable or unwilling to comply with the study visit schedule and requirements of the study

• Subjects unable to speak and read the English language

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

Contacts and Locations

Locations

	Site	City	State	Country	Postal Code
1	University of Arizona Cancer Center	Tucson	Arizona	United States	85724

Sponsors and Collaborators

• University of Arizona

Investigators

• Principal Investigator: Clara Curiel-Lewandroski, University of Arizona

Study Documents (Full-Text)

None provided.

More Information

Publications

None provided.

• Responsible Party: University of Arizona
• ClinicalTrials.gov Identifier: NCT02067104
• Other Study ID Numbers: 1401183410; ML28585
• First Posted: Feb 20, 2014
• Last Update Posted: Aug 14, 2019
• Last Verified: Jul 1, 2019

• Studies a U.S. FDA-regulated Drug Product: Yes
• Studies a U.S. FDA-regulated Device Product: No
• Additional relevant MeSH terms: Carcinoma; Carcinoma, Basal Cell

Study Results

Participant Flow

Arm/Group Title	Placebo	Vismodegib
Arm/Group Description	receive 150 mg of oral placebo daily for a period of 2 months Placebo: Pulse therapy pattern of 2 months of daily treatment and 2 months of no treatment for 24 months.	receive 150 mg of vismodegib daily for a period of 2 months Vismodegib: Pulse therapy pattern of 2 months of daily treatment and 2 months of no treatment for 24 months.
Period Title: Overall Study
STARTED	4	5
COMPLETED	0	0
NOT COMPLETED	4	5

Baseline Characteristics

Arm/Group Title	Placebo	Vismodegib	Total
Arm/Group Description	receive 150 mg of oral placebo daily for a period of 2 months Placebo: Pulse therapy pattern of 2 months of daily treatment and 2 months of no treatment for 24 months.	receive 150 mg of vismodegib daily for a period of 2 months Vismodegib: Pulse therapy pattern of 2 months of daily treatment and 2 months of no treatment for 24 months.	Total of all reporting groups
Overall Participants	4	5	9
Age (Count of Participants)
<=18 years	0 0%	0 0%	0 0%
Between 18 and 65 years	2 50%	2 40%	4 44.4%
>=65 years	2 50%	3 60%	5 55.6%
Sex: Female, Male (Count of Participants)
Female	1 25%	1 20%	2 22.2%
Male	3 75%	4 80%	7 77.8%
Region of Enrollment (participants) [Number]
United States	4 100%	5 100%	9 100%

Outcome Measures

1. Primary Outcome

Title	The Effect of Vismodegib Pulse Therapy on the Incidence of Newly Diagnosed Basal Cell Carcinomas (BCC)
Description	Measured by the incidence of biopsy confirmed BCC over a 24 month period
Time Frame	24 Months

Outcome Measure Data

Analysis Population Description

Early study termination due to low accrual. No data analyzed; no results.

Arm/Group Title	Placebo	Vismodegib
Arm/Group Description	receive 150 mg of oral placebo daily for a period of 2 months Placebo: Pulse therapy pattern of 2 months of daily treatment and 2 months of no treatment for 24 months.	receive 150 mg of vismodegib daily for a period of 2 months Vismodegib: Pulse therapy pattern of 2 months of daily treatment and 2 months of no treatment for 24 months.
Measure Participants	0	0

2. Secondary Outcome

Title	The Incidence of Newly Diagnosed Squamous Cell Carcinomas (SCC) in the Same Subjects Receiving Vismodegib Treatment When Compared With Placebo
Description	Measured by the incidence of biopsy confirmed SCC over the same 24 month period
Time Frame	24 Months

Outcome Measure Data

Analysis Population Description

Early study termination due to low accrual. No data analyzed; no results.

Arm/Group Title	Placebo	Vismodegib
Arm/Group Description	receive 150 mg of oral placebo daily for a period of 2 months Placebo: Pulse therapy pattern of 2 months of daily treatment and 2 months of no treatment for 24 months.	receive 150 mg of vismodegib daily for a period of 2 months Vismodegib: Pulse therapy pattern of 2 months of daily treatment and 2 months of no treatment for 24 months.
Measure Participants	0	0

Adverse Events

Arm/Group Title	Placebo		Vismodegib
Arm/Group Description	receive 150 mg of oral placebo daily for a period of 2 months Placebo: Pulse therapy pattern of 2 months of daily treatment and 2 months of no treatment for 24 months.		receive 150 mg of vismodegib daily for a period of 2 months Vismodegib: Pulse therapy pattern of 2 months of daily treatment and 2 months of no treatment for 24 months.
All Cause Mortality
	Placebo		Vismodegib
	Affected / at Risk (%)	# Events	Affected / at Risk (%)	# Events
Total	0/4 (0%)		0/5 (0%)
Serious Adverse Events
	Placebo		Vismodegib
	Affected / at Risk (%)	# Events	Affected / at Risk (%)	# Events
Total	0/4 (0%)		0/5 (0%)
Other (Not Including Serious) Adverse Events
	Placebo		Vismodegib
	Affected / at Risk (%)	# Events	Affected / at Risk (%)	# Events
Total	4/4 (100%)		5/5 (100%)
Gastrointestinal disorders
Taste disturbance	1/4 (25%)		3/5 (60%)
Musculoskeletal and connective tissue disorders
Leg cramps	3/4 (75%)		2/5 (40%)
Skin and subcutaneous tissue disorders
Scale in ear	0/4 (0%)		1/5 (20%)

Limitations/Caveats

[Not Specified]

More Information

Certain Agreements

All Principal Investigators ARE employed by the organization sponsoring the study.

There is NOT an agreement between Principal Investigators and the Sponsor (or its agents) that restricts the PI's rights to discuss or publish trial results after the trial is completed.