Trial Outcomes & Findings for To Determine The Efficacy and Safety of GDC-0449 in Patients With Basal Cell Nevus Syndrome (BCNS) (NCT NCT00957229)
NCT ID: NCT00957229
Last Updated: 2021-01-11
Results Overview
Number of new surgically eligible basal-cell carcinomas per patient per year
Recruitment status
COMPLETED
Study phase
PHASE2
Target enrollment
41 participants
Primary outcome timeframe
3 months of receiving study drug
Results posted on
2021-01-11
Participant Flow
Participant milestones
| Measure |
Sugar Pill
placebo pill by mouth once daily
GDC-0449: capsule, 150 mg, one pill daily, 18 months
|
GDC-0449
vismodegib 150MG by mouth once daily
GDC-0449: capsule, 150 mg, one pill daily, 18 months
|
|---|---|---|
|
Study Start
STARTED
|
15
|
26
|
|
Study Start
COMPLETED
|
14
|
23
|
|
Study Start
NOT COMPLETED
|
1
|
3
|
|
Reallocated to or Continued Vismodegib
STARTED
|
14
|
23
|
|
Reallocated to or Continued Vismodegib
COMPLETED
|
11
|
21
|
|
Reallocated to or Continued Vismodegib
NOT COMPLETED
|
3
|
2
|
Reasons for withdrawal
| Measure |
Sugar Pill
placebo pill by mouth once daily
GDC-0449: capsule, 150 mg, one pill daily, 18 months
|
GDC-0449
vismodegib 150MG by mouth once daily
GDC-0449: capsule, 150 mg, one pill daily, 18 months
|
|---|---|---|
|
Study Start
Adverse Event
|
0
|
3
|
|
Study Start
Disease Progression
|
1
|
0
|
|
Reallocated to or Continued Vismodegib
Death
|
1
|
1
|
|
Reallocated to or Continued Vismodegib
Adverse Event
|
2
|
1
|
Baseline Characteristics
To Determine The Efficacy and Safety of GDC-0449 in Patients With Basal Cell Nevus Syndrome (BCNS)
Baseline characteristics by cohort
| Measure |
Sugar Pill
n=15 Participants
placebo pill by mouth once daily
GDC-0449: capsule, 150 mg, one pill daily, 18 months
|
GDC-0449
n=26 Participants
vismodegib 150MG by mouth once daily
GDC-0449: capsule, 150 mg, one pill daily, 18 months
|
Total
n=41 Participants
Total of all reporting groups
|
|---|---|---|---|
|
Age, Continuous
|
53 years
STANDARD_DEVIATION 8 • n=5 Participants
|
54 years
STANDARD_DEVIATION 8 • n=7 Participants
|
54 years
STANDARD_DEVIATION 8 • n=5 Participants
|
|
Sex: Female, Male
Female
|
6 Participants
n=5 Participants
|
8 Participants
n=7 Participants
|
14 Participants
n=5 Participants
|
|
Sex: Female, Male
Male
|
9 Participants
n=5 Participants
|
18 Participants
n=7 Participants
|
27 Participants
n=5 Participants
|
|
Region of Enrollment
United States
|
15 participants
n=5 Participants
|
26 participants
n=7 Participants
|
41 participants
n=5 Participants
|
PRIMARY outcome
Timeframe: 3 months of receiving study drugNumber of new surgically eligible basal-cell carcinomas per patient per year
Outcome measures
| Measure |
Sugar Pill
n=15 Participants
placebo pill by mouth once daily
GDC-0449: capsule, 150 mg, one pill daily, 18 months
|
GDC-0449
n=26 Participants
vismodegib 150MG by mouth once daily
GDC-0449: capsule, 150 mg, one pill daily, 18 months
|
|---|---|---|
|
Number of New Surgically Eligible Basal-cell Carcinomas
|
34 events per patient year
Standard Deviation 1.32
|
2 events per patient year
Standard Deviation 0.12
|
SECONDARY outcome
Timeframe: Baseline and 18 monthsOutcome measures
| Measure |
Sugar Pill
n=15 Participants
placebo pill by mouth once daily
GDC-0449: capsule, 150 mg, one pill daily, 18 months
|
GDC-0449
n=26 Participants
vismodegib 150MG by mouth once daily
GDC-0449: capsule, 150 mg, one pill daily, 18 months
|
|---|---|---|
|
Change in Size of the Existing Carcinomas, Expressed as the Sum of Cumulative Diameters in Millimeters Over an 18 Month Period
|
55 Millimeters
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.
|
-60 Millimeters
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.
|
SECONDARY outcome
Timeframe: 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.Population: 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).
The number of new surgically eligible basal-cell carcinomas per patient per month is reported.
Outcome measures
| Measure |
Sugar Pill
n=5 Participants
placebo pill by mouth once daily
GDC-0449: capsule, 150 mg, one pill daily, 18 months
|
GDC-0449
n=5 Participants
vismodegib 150MG by mouth once daily
GDC-0449: capsule, 150 mg, one pill daily, 18 months
|
|---|---|---|
|
Number of New Surgically Eligible Basal-cell Carcinomas After Stopping Vismodegib Treatment
|
1.7 carcinomas per patient per month
Standard Deviation 1.5
|
0.06 carcinomas per patient per month
Standard Deviation 0.12
|
Adverse Events
Sugar Pill
Serious events: 2 serious events
Other events: 12 other events
Deaths: 0 deaths
GDC-0449
Serious events: 21 serious events
Other events: 40 other events
Deaths: 2 deaths
Serious adverse events
| Measure |
Sugar Pill
n=15 participants at risk
placebo pill by mouth once daily
GDC-0449: capsule, 150 mg, one pill daily, 18 months
|
GDC-0449
n=40 participants at risk
vismodegib 150MG by mouth once daily
GDC-0449: capsule, 150 mg, one pill daily, 18 months
|
|---|---|---|
|
Cardiac disorders
Cardiac stent for blocked artery
|
6.7%
1/15
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.
|
0.00%
0/40
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.
|
|
Reproductive system and breast disorders
Hysterectomy
|
6.7%
1/15
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.
|
0.00%
0/40
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.
|
|
Musculoskeletal and connective tissue disorders
Muscle cramps
|
0.00%
0/15
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.
|
5.0%
2/40
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.
|
|
Metabolism and nutrition disorders
Weight loss
|
0.00%
0/15
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.
|
15.0%
6/40
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.
|
|
Infections and infestations
Pneumonia
|
0.00%
0/15
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.
|
5.0%
2/40
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.
|
|
Injury, poisoning and procedural complications
Reactions to antibiotic
|
0.00%
0/15
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.
|
5.0%
2/40
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.
|
|
Cardiac disorders
Chest pain
|
0.00%
0/15
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.
|
5.0%
2/40
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.
|
|
Cardiac disorders
Atrial flutter
|
0.00%
0/15
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.
|
2.5%
1/40
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.
|
|
Gastrointestinal disorders
Colitis
|
0.00%
0/15
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.
|
2.5%
1/40
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.
|
|
Gastrointestinal disorders
Oesophagitis
|
0.00%
0/15
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.
|
2.5%
1/40
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.
|
|
Musculoskeletal and connective tissue disorders
Hip replacement surgery
|
0.00%
0/15
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.
|
2.5%
1/40
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.
|
|
Musculoskeletal and connective tissue disorders
Knee replacement surgery
|
0.00%
0/15
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.
|
2.5%
1/40
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.
|
|
Gastrointestinal disorders
Mesenteric cyst
|
0.00%
0/15
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.
|
2.5%
1/40
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.
|
|
Psychiatric disorders
Suicide attempt
|
0.00%
0/15
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.
|
2.5%
1/40
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.
|
Other adverse events
| Measure |
Sugar Pill
n=15 participants at risk
placebo pill by mouth once daily
GDC-0449: capsule, 150 mg, one pill daily, 18 months
|
GDC-0449
n=40 participants at risk
vismodegib 150MG by mouth once daily
GDC-0449: capsule, 150 mg, one pill daily, 18 months
|
|---|---|---|
|
Skin and subcutaneous tissue disorders
Hair loss
|
6.7%
1/15
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.
|
100.0%
40/40
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.
|
|
Musculoskeletal and connective tissue disorders
Muscle cramps
|
6.7%
1/15
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.
|
100.0%
40/40
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.
|
|
Gastrointestinal disorders
Dysgeusia
|
6.7%
1/15
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.
|
92.5%
37/40
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.
|
|
Gastrointestinal disorders
Gastrointestinal upset
|
13.3%
2/15
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.
|
65.0%
26/40
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.
|
|
Metabolism and nutrition disorders
Weight loss
|
6.7%
1/15
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.
|
62.5%
25/40
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.
|
|
General disorders
Fatigue
|
0.00%
0/15
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.
|
47.5%
19/40
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.
|
|
Respiratory, thoracic and mediastinal disorders
Common cold
|
20.0%
3/15
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.
|
20.0%
8/40
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.
|
|
Skin and subcutaneous tissue disorders
Acne
|
13.3%
2/15
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.
|
17.5%
7/40
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.
|
|
Respiratory, thoracic and mediastinal disorders
Runny nose
|
0.00%
0/15
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.
|
17.5%
7/40
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.
|
|
Skin and subcutaneous tissue disorders
Rash
|
0.00%
0/15
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.
|
12.5%
5/40
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.
|
|
Vascular disorders
Dizziness
|
0.00%
0/15
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.
|
10.0%
4/40
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.
|
|
Gastrointestinal disorders
Nausea
|
6.7%
1/15
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.
|
10.0%
4/40
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.
|
Additional Information
Ervin H Epstein Jr., MD
Children's Hospital of Oakland Research Institute, Oakland
Phone: 510-450-5688
Email: [email protected]
Results disclosure agreements
- Principal investigator is a sponsor employee
- Publication restrictions are in place