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To Determine The Efficacy and Safety of GDC-0449 in Patients With Basal Cell Nevus Syndrome (BCNS) (GDC-0449)

The safety and scientific validity of this study is the responsibility of the study sponsor and investigators. Listing a study does not mean it has been evaluated by the U.S. Federal Government. Read our disclaimer for details.
 
ClinicalTrials.gov Identifier: NCT00957229
Recruitment Status : Completed
First Posted : August 12, 2009
Results First Posted : January 11, 2021
Last Update Posted : January 11, 2021
Sponsor:
Collaborator:
Genentech, Inc.
Information provided by (Responsible Party):
UCSF Benioff Children's Hospital Oakland

Study Type Interventional
Study Design Allocation: Randomized;   Intervention Model: Crossover Assignment;   Masking: Triple (Participant, Investigator, Outcomes Assessor);   Primary Purpose: Treatment
Conditions Basal Cell Nevus Syndrome
Gorlin Syndrome
Intervention Drug: GDC-0449
Enrollment 41
Recruitment Details  
Pre-assignment Details  
Arm/Group Title Sugar Pill GDC-0449
Hide Arm/Group Description

placebo pill by mouth once daily

GDC-0449: capsule, 150 mg, one pill daily, 18 months

vismodegib 150MG by mouth once daily

GDC-0449: capsule, 150 mg, one pill daily, 18 months

Period Title: Study Start
Started 15 26
Completed 14 23
Not Completed 1 3
Reason Not Completed
Adverse Event             0             3
Disease Progression             1             0
Period Title: Reallocated to or Continued Vismodegib
Started 14 23
Completed 11 21
Not Completed 3 2
Reason Not Completed
Death             1             1
Adverse Event             2             1
Arm/Group Title Sugar Pill GDC-0449 Total
Hide Arm/Group Description

placebo pill by mouth once daily

GDC-0449: capsule, 150 mg, one pill daily, 18 months

vismodegib 150MG by mouth once daily

GDC-0449: capsule, 150 mg, one pill daily, 18 months

Total of all reporting groups
Overall Number of Baseline Participants 15 26 41
Hide Baseline Analysis Population Description
[Not Specified]
Age, Continuous  
Mean (Standard Deviation)
Unit of measure:  Years
Number Analyzed 15 participants 26 participants 41 participants
53  (8) 54  (8) 54  (8)
Sex: Female, Male  
Measure Type: Count of Participants
Unit of measure:  Participants
Number Analyzed 15 participants 26 participants 41 participants
Female
6
  40.0%
8
  30.8%
14
  34.1%
Male
9
  60.0%
18
  69.2%
27
  65.9%
Region of Enrollment  
Measure Type: Number
Unit of measure:  Participants
United States Number Analyzed 15 participants 26 participants 41 participants
15 26 41
1.Primary Outcome
Title Number of New Surgically Eligible Basal-cell Carcinomas
Hide Description Number of new surgically eligible basal-cell carcinomas per patient per year
Time Frame 3 months of receiving study drug
Hide Outcome Measure Data
Hide Analysis Population Description
[Not Specified]
Arm/Group Title Sugar Pill GDC-0449
Hide Arm/Group Description:

placebo pill by mouth once daily

GDC-0449: capsule, 150 mg, one pill daily, 18 months

vismodegib 150MG by mouth once daily

GDC-0449: capsule, 150 mg, one pill daily, 18 months

Overall Number of Participants Analyzed 15 26
Mean (Standard Deviation)
Unit of Measure: events per patient year
34  (1.32) 2  (0.12)
2.Secondary Outcome
Title Change in Size of the Existing Carcinomas, Expressed as the Sum of Cumulative Diameters in Millimeters Over an 18 Month Period
Hide Description [Not Specified]
Time Frame Baseline and 18 months
Hide Outcome Measure Data
Hide Analysis Population Description
[Not Specified]
Arm/Group Title Sugar Pill GDC-0449
Hide Arm/Group Description:

placebo pill by mouth once daily

GDC-0449: capsule, 150 mg, one pill daily, 18 months

vismodegib 150MG by mouth once daily

GDC-0449: capsule, 150 mg, one pill daily, 18 months

Overall Number of Participants Analyzed 15 26
Measure Type: Number
Number (95% Confidence Interval)
Unit of Measure: Millimeters
55 [1] 
(NA to NA)
-60 [1] 
(NA to NA)
[1]
The PI has left the institution and the only access to the data is from the publication. The records no longer exist. All efforts have been made to obtain the data.
3.Secondary Outcome
Title Number of New Surgically Eligible Basal-cell Carcinomas After Stopping Vismodegib Treatment
Hide Description The number of new surgically eligible basal-cell carcinomas per patient per month is reported.
Time Frame These five patients were given placebo for a mean of 7.4 months (SD 2.3). After receiving vismodegib for a mean of 13.8 months (SD 6.8) and then discontinuing vismodegib for a mean of 11.8 months (SD 7.9) the new basal-cell carcinoma rate is reported.
Hide Outcome Measure Data
Hide Analysis Population Description
The incidence of new surgically eligible basal-cell carcinomas after stopping vismodegib was analysed in an exploratory analysis of the patients who crossed over to vismodegib and were followed up for at least 6 months after stopping vismodegib (n=5).
Arm/Group Title Sugar Pill GDC-0449
Hide Arm/Group Description:

placebo pill by mouth once daily

GDC-0449: capsule, 150 mg, one pill daily, 18 months

vismodegib 150MG by mouth once daily

GDC-0449: capsule, 150 mg, one pill daily, 18 months

Overall Number of Participants Analyzed 5 5
Mean (Standard Deviation)
Unit of Measure: carcinomas per patient per month
1.7  (1.5) 0.06  (0.12)
Time Frame [Not Specified]
Adverse Event Reporting Description

Grade 1 and 2 events are listed if considered related to vismodegib treatment. All other graded events are listed.

The PI has left the institution and the only access to the data is from the publication, in which the data are reported as presented here. The records no longer exist. All efforts have been made to obtain the data.

 
Arm/Group Title Sugar Pill GDC-0449
Hide Arm/Group Description

placebo pill by mouth once daily

GDC-0449: capsule, 150 mg, one pill daily, 18 months

vismodegib 150MG by mouth once daily

GDC-0449: capsule, 150 mg, one pill daily, 18 months

All-Cause Mortality
Sugar Pill GDC-0449
Affected / at Risk (%) Affected / at Risk (%)
Total   0/15 (0.00%)   2/40 (5.00%) 
Hide Serious Adverse Events
Sugar Pill GDC-0449
Affected / at Risk (%) Affected / at Risk (%)
Total   2/15 (13.33%)   21/40 (52.50%) 
Cardiac disorders     
Cardiac stent for blocked artery  1  1/15 (6.67%)  0/40 (0.00%) 
Chest pain  1  0/15 (0.00%)  2/40 (5.00%) 
Atrial flutter  1  0/15 (0.00%)  1/40 (2.50%) 
Gastrointestinal disorders     
Colitis  1  0/15 (0.00%)  1/40 (2.50%) 
Oesophagitis  1  0/15 (0.00%)  1/40 (2.50%) 
Mesenteric cyst  1  0/15 (0.00%)  1/40 (2.50%) 
Infections and infestations     
Pneumonia  1  0/15 (0.00%)  2/40 (5.00%) 
Injury, poisoning and procedural complications     
Reactions to antibiotic  1 [1]  0/15 (0.00%)  2/40 (5.00%) 
Metabolism and nutrition disorders     
Weight loss  1  0/15 (0.00%)  6/40 (15.00%) 
Musculoskeletal and connective tissue disorders     
Muscle cramps  1  0/15 (0.00%)  2/40 (5.00%) 
Hip replacement surgery  1  0/15 (0.00%)  1/40 (2.50%) 
Knee replacement surgery  1  0/15 (0.00%)  1/40 (2.50%) 
Psychiatric disorders     
Suicide attempt  1  0/15 (0.00%)  1/40 (2.50%) 
Reproductive system and breast disorders     
Hysterectomy  1  1/15 (6.67%)  0/40 (0.00%) 
1
Term from vocabulary, CTCAE (3.0)
Indicates events were collected by systematic assessment
[1]
Reactions to antibiotics include ciprofloxacin and Bactrim
Hide Other (Not Including Serious) Adverse Events
Frequency Threshold for Reporting Other Adverse Events 0%
Sugar Pill GDC-0449
Affected / at Risk (%) Affected / at Risk (%)
Total   12/15 (80.00%)   40/40 (100.00%) 
Gastrointestinal disorders     
Dysgeusia  1  1/15 (6.67%)  37/40 (92.50%) 
Gastrointestinal upset  1  2/15 (13.33%)  26/40 (65.00%) 
Nausea  1  1/15 (6.67%)  4/40 (10.00%) 
General disorders     
Fatigue  1  0/15 (0.00%)  19/40 (47.50%) 
Metabolism and nutrition disorders     
Weight loss  1  1/15 (6.67%)  25/40 (62.50%) 
Musculoskeletal and connective tissue disorders     
Muscle cramps  1  1/15 (6.67%)  40/40 (100.00%) 
Respiratory, thoracic and mediastinal disorders     
Common cold  1  3/15 (20.00%)  8/40 (20.00%) 
Runny nose  1  0/15 (0.00%)  7/40 (17.50%) 
Skin and subcutaneous tissue disorders     
Hair loss  1  1/15 (6.67%)  40/40 (100.00%) 
Acne  1  2/15 (13.33%)  7/40 (17.50%) 
Rash  1  0/15 (0.00%)  5/40 (12.50%) 
Vascular disorders     
Dizziness  1  0/15 (0.00%)  4/40 (10.00%) 
1
Term from vocabulary, CTCAE (3.0)
Indicates events were collected by systematic assessment
Certain Agreements
All Principal Investigators ARE employed by the organization sponsoring the study.
Results Point of Contact
Layout table for Results Point of Contact information
Name/Title: Ervin H Epstein Jr., MD
Organization: Children's Hospital of Oakland Research Institute, Oakland
Phone: 510-450-5688
EMail: eepstein@chori.org
Layout table for additonal information
Responsible Party: UCSF Benioff Children's Hospital Oakland
ClinicalTrials.gov Identifier: NCT00957229    
Other Study ID Numbers: 2009-026
SHH-4685s ( Other Grant/Funding Number: Genentech )
First Submitted: August 5, 2009
First Posted: August 12, 2009
Results First Submitted: October 8, 2020
Results First Posted: January 11, 2021
Last Update Posted: January 11, 2021