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Trial record 8 of 78 for:    vismodegib

Study Evaluating the Efficacy of Oral Vismodegib in Various Histologic Subtypes

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ClinicalTrials.gov Identifier: NCT01700049
Recruitment Status : Completed
First Posted : October 4, 2012
Results First Posted : May 23, 2019
Last Update Posted : May 23, 2019
Sponsor:
Collaborator:
Genentech, Inc.
Information provided by (Responsible Party):
Scott W. Fosko, Mayo Clinic

Study Type Interventional
Study Design Intervention Model: Single Group Assignment;   Masking: None (Open Label);   Primary Purpose: Treatment
Condition Basal Cell Carcinoma
Intervention Drug: vismodegib (150 mg PO daily)
Enrollment 28
Recruitment Details  
Pre-assignment Details  
Arm/Group Title Open Label Oral Vismodegib
Hide Arm/Group Description

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.

Period Title: Overall Study
Started 28
Completed 27
Not Completed 1
Reason Not Completed
Withdrawal by Subject             1
Arm/Group Title Open Label Oral Vismodegib
Hide Arm/Group Description

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.

Overall Number of Baseline Participants 28
Hide Baseline Analysis Population Description
[Not Specified]
Age, Continuous  
Median (Standard Deviation)
Unit of measure:  Years
Number Analyzed 28 participants
69.5  (13.0)
Sex: Female, Male  
Measure Type: Count of Participants
Unit of measure:  Participants
Number Analyzed 28 participants
Female
12
  42.9%
Male
16
  57.1%
Race (NIH/OMB)  
Measure Type: Count of Participants
Unit of measure:  Participants
Number Analyzed 28 participants
American Indian or Alaska Native
0
   0.0%
Asian
0
   0.0%
Native Hawaiian or Other Pacific Islander
0
   0.0%
Black or African American
1
   3.6%
White
27
  96.4%
More than one race
0
   0.0%
Unknown or Not Reported
0
   0.0%
Region of Enrollment  
Measure Type: Number
Unit of measure:  Participants
United States Number Analyzed 28 participants
28
1.Primary Outcome
Title Efficacy of Vismodegib
Hide Description 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.
Time Frame Week 24
Hide Outcome Measure Data
Hide Analysis Population Description
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.
Arm/Group Title Open Label Oral Vismodegib
Hide Arm/Group Description:

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.

Overall Number of Participants Analyzed 27
Overall Number of Units Analyzed
Type of Units Analyzed: Tumors
65
Count of Units
Unit of Measure: tumors
Infiltrative Number Analyzed 24 tumors
2
   8.3%
Nodular Number Analyzed 29 tumors
5
  17.2%
Superficial Number Analyzed 10 tumors
1
  10.0%
Keratotic Number Analyzed 2 tumors
1
  50.0%
2.Secondary Outcome
Title Safety of Vismodegib
Hide Description 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.
Time Frame Up to 18 months
Hide Outcome Measure Data
Hide Analysis Population Description
[Not Specified]
Arm/Group Title Open Label Oral Vismodegib
Hide Arm/Group Description:
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.
Overall Number of Participants Analyzed 28
Measure Type: Number
Unit of Measure: adverse events
Grade 1 133
Grade 2 48
Grade 3 8
Grade 4 1
Grade 5 2
3.Secondary Outcome
Title Onset of Efficacy of Vismodegib
Hide Description 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.
Time Frame Up to week 24
Hide Outcome Measure Data
Hide Analysis Population Description
[Not Specified]
Arm/Group Title Open Label Oral Vismodegib
Hide Arm/Group Description:

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.

Overall Number of Participants Analyzed 27
Overall Number of Units Analyzed
Type of Units Analyzed: Tumors
65
Count of Units
Unit of Measure: tumors
Complete response
13
  20.0%
Partial response
27
  41.5%
Stable Disease
24
  36.9%
Progressive Disease
1
   1.5%
Time Frame The study period during which all Adverse Events (AE) and Serious Adverse Events (SAE) must be reported begins after informed consent is obtained and initiation of study treatment and ends 30 days following the last administration of study treatment or study discontinuation/termination, whichever is earlier.
Adverse Event Reporting Description [Not Specified]
 
Arm/Group Title Open Label Oral Vismodegib
Hide Arm/Group Description

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.

All-Cause Mortality
Open Label Oral Vismodegib
Affected / at Risk (%)
Total   2/28 (7.14%)    
Show Serious Adverse Events Hide Serious Adverse Events
Open Label Oral Vismodegib
Affected / at Risk (%) # Events
Total   4/28 (14.29%)    
Cardiac disorders   
Myocardial infarction   1/28 (3.57%)  1
Congestive Heart Failure   1/28 (3.57%)  1
Gastrointestinal disorders   
Duodenal ulcer   1/28 (3.57%)  1
Gastrointestinal bleed   1/28 (3.57%)  1
General disorders   
Exacerbation of low back pain   1/28 (3.57%)  1
Nervous system disorders   
Neurogenic claudication   1/28 (3.57%)  1
Indicates events were collected by systematic assessment
Show Other (Not Including Serious) Adverse Events Hide Other (Not Including Serious) Adverse Events
Frequency Threshold for Reporting Other Adverse Events 4%
Open Label Oral Vismodegib
Affected / at Risk (%) # Events
Total   28/28 (100.00%)    
Blood and lymphatic system disorders   
Hyperlipidemia   1/28 (3.57%)  1
Cardiac disorders   
Elevated blood pressure   1/28 (3.57%)  1
Ear and labyrinth disorders   
Vertigo   2/28 (7.14%)  2
Tinnitus   1/28 (3.57%)  1
Eye disorders   
Blurry vision   2/28 (7.14%)  2
Gastrointestinal disorders   
Diarrhea   7/28 (25.00%)  7
Constipation   2/28 (7.14%)  2
Dyspepsia   2/28 (7.14%)  2
Gastroenteritis   2/28 (7.14%)  2
Esophagitis   1/28 (3.57%)  1
General disorders   
Dysgeusia/Loss of Taste   24/28 (85.71%)  24
Nausea   8/28 (28.57%)  8
Fatigue   7/28 (25.00%)  7
Swelling   4/28 (14.29%)  4
Abnormal hair growth   2/28 (7.14%)  2
Insomnia   2/28 (7.14%)  2
Scalp tenderness   2/28 (7.14%)  2
Headache   2/28 (7.14%)  2
Falls   2/28 (7.14%)  2
Vomiting   1/28 (3.57%)  1
Dehydration   1/28 (3.57%)  1
Decrease in eyelashes/eyebrows   1/28 (3.57%)  1
Stunted nail growth   1/28 (3.57%)  1
Bloating   1/28 (3.57%)  1
Dry mouth   1/28 (3.57%)  1
Tooth pain   1/28 (3.57%)  1
Numbness   1/28 (3.57%)  1
Rhinorrhea   1/28 (3.57%)  1
Visual hallucinations   1/28 (3.57%)  1
Burning sensation of tongue   1/28 (3.57%)  1
Halitosis   1/28 (3.57%)  1
Lower extremity edema/intermittent swelling   1/28 (3.57%)  1
Vitamin D deficiency   1/28 (3.57%)  1
Infections and infestations   
Upper respiratory infection   5/28 (17.86%)  5
Wound infection   2/28 (7.14%)  2
Urinary Tract Infection   2/28 (7.14%)  2
Viral syndrome   1/28 (3.57%)  1
Bronchitis   1/28 (3.57%)  1
Sinus infection   1/28 (3.57%)  1
Salivary gland infection   1/28 (3.57%)  1
Metabolism and nutrition disorders   
Decreased appetite/anorexia/weight loss   9/28 (32.14%)  9
Hypokalemia   1/28 (3.57%)  1
Musculoskeletal and connective tissue disorders   
Muscle spasms   23/28 (82.14%)  23
Joint aches   5/28 (17.86%)  5
Muscle aches   2/28 (7.14%)  2
Maxillary fibrous cyst   1/28 (3.57%)  1
Fracture   1/28 (3.57%)  1
Dislocation   1/28 (3.57%)  1
Sprain   1/28 (3.57%)  1
Gout   1/28 (3.57%)  1
Orthostatic tremor   1/28 (3.57%)  1
Reproductive system and breast disorders   
Vaginal bleeding   1/28 (3.57%)  1
Skin and subcutaneous tissue disorders   
Alopecia   20/28 (71.43%)  20
Itching/pruritus   4/28 (14.29%)  4
Comedones   2/28 (7.14%)  2
Actinic keratosis   2/28 (7.14%)  2
Rash   1/28 (3.57%)  1
Biopsy site inflammation   1/28 (3.57%)  1
Acne   1/28 (3.57%)  1
Rosacea flare   1/28 (3.57%)  1
Squamous cell carcinoma   1/28 (3.57%)  1
Surgical and medical procedures   
Eyelid reconstruction   1/28 (3.57%)  1
Vascular disorders   
Peripheral arterial disease   1/28 (3.57%)  1
Indicates events were collected by systematic assessment
Certain Agreements
All Principal Investigators ARE employed by the organization sponsoring the study.
Results Point of Contact
Name/Title: Dr. Scott Fosko
Organization: Mayo Clinic
Phone: 904-953-2303
Publications:
Responsible Party: Scott W. Fosko, Mayo Clinic
ClinicalTrials.gov Identifier: NCT01700049     History of Changes
Other Study ID Numbers: ML28485
22355 ( Other Identifier: Saint Louis University )
First Submitted: September 25, 2012
First Posted: October 4, 2012
Results First Submitted: March 21, 2019
Results First Posted: May 23, 2019
Last Update Posted: May 23, 2019