Trial · NCT00957229
To Determine The Efficacy and Safety of GDC-0449 in Patients With Basal Cell Nevus Syndrome (BCNS)
NCT00957229, Phase 2, Completed, UCSF Benioff Children's Hospital Oakland; results first posted 2021-01-11
3 sourcestrials-2026-11-12-0.5.0
Cite this record
ConductScience Library. To Determine The Efficacy and Safety of GDC-0449 in Patients With Basal Cell Nevus Syndrome (BCNS). Trials, record NCT00957229. Release trials-2026-11-12-0.5.0, 12 Nov 2026. https://conductscience.org/library/trials/NCT00957229
On this page
Trial
As registered on ClinicalTrials.gov, in the graph's capture of the registry.
- Title
- To Determine The Efficacy and Safety of GDC-0449 in Patients With Basal Cell Nevus Syndrome (BCNS)
- NCT number
- NCT00957229
- Status
- Completed
- Phases
- Phase 2
- Enrollment
- 41 participants, actual
- Lead sponsor
- UCSF Benioff Children's Hospital Oakland
- Conditions
- Basal Cell Nevus Syndrome, Gorlin Syndrome
- Interventions
- GDC-0449
- First posted
- 2009-08-12
- Last updated
- 2021-01-11
- Results first posted
- 2021-01-11
Source ClinicalTrials.gov
Registered outcomes3
Each outcome the trial registered, primary first, in ClinicalTrials.gov's words.
| List | Outcome | Time frame | Description |
|---|---|---|---|
| Primary | Number of New Surgically Eligible Basal-cell Carcinomas | 3 months of receiving study drug | Number of new surgically eligible basal-cell carcinomas per patient per year |
| Secondary | Change in Size of the Existing Carcinomas, Expressed as the Sum of Cumulative Diameters in Millimeters Over an 18 Month Period | Baseline and 18 months | |
| Secondary | Number of New Surgically Eligible Basal-cell Carcinomas After Stopping Vismodegib Treatment | 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. | The number of new surgically eligible basal-cell carcinomas per patient per month is reported. |
Source ClinicalTrials.gov
Outcomes3
Each outcome the trial posted results for, in ClinicalTrials.gov's words.
| Type | Outcome | Time frame | Population | Units | Measured as |
|---|---|---|---|---|---|
| PRIMARY | Number of New Surgically Eligible Basal-cell Carcinomas | 3 months of receiving study drug | events per patient year | MEAN | |
| SECONDARY | Change in Size of the Existing Carcinomas, Expressed as the Sum of Cumulative Diameters in Millimeters Over an 18 Month Period | Baseline and 18 months | Millimeters | NUMBER | |
| SECONDARY | Number of New Surgically Eligible Basal-cell Carcinomas After Stopping Vismodegib Treatment | 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. | 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). | carcinomas per patient per month | MEAN |
Source ClinicalTrials.gov
Results by group6
Each measurement the trial posted, group by group, in the words and numbers it posted.
| Outcome | Group | Category | Value | Spread | Units |
|---|---|---|---|---|---|
| Number of New Surgically Eligible Basal-cell Carcinomas | Sugar Pill | 34 | Standard Deviation 1.32 | events per patient year | |
| Number of New Surgically Eligible Basal-cell Carcinomas | GDC-0449 | 2 | Standard Deviation 0.12 | events per patient year | |
| Change in Size of the Existing Carcinomas, Expressed as the Sum of Cumulative Diameters in Millimeters Over an 18 Month Period | Sugar Pill | 55 | 95% Confidence Interval NA to NA | Millimeters | |
| Change in Size of the Existing Carcinomas, Expressed as the Sum of Cumulative Diameters in Millimeters Over an 18 Month Period | GDC-0449 | -60 | 95% Confidence Interval NA to NA | Millimeters | |
| Number of New Surgically Eligible Basal-cell Carcinomas After Stopping Vismodegib Treatment | Sugar Pill | 1.7 | Standard Deviation 1.5 | carcinomas per patient per month | |
| Number of New Surgically Eligible Basal-cell Carcinomas After Stopping Vismodegib Treatment | GDC-0449 | 0.06 | Standard Deviation 0.12 | carcinomas per patient per month |
Source ClinicalTrials.gov
Participant flow12
How many started, completed or left each group, period by period.
| Period | Milestone | Group | Participants | Units counted |
|---|---|---|---|---|
| Study Start | STARTED | Sugar Pill | 15 | |
| Study Start | STARTED | GDC-0449 | 26 | |
| Study Start | COMPLETED | Sugar Pill | 14 | |
| Study Start | COMPLETED | GDC-0449 | 23 | |
| Study Start | NOT COMPLETED | Sugar Pill | 1 | |
| Study Start | NOT COMPLETED | GDC-0449 | 3 | |
| Reallocated to or Continued Vismodegib | STARTED | Sugar Pill | 14 | |
| Reallocated to or Continued Vismodegib | STARTED | GDC-0449 | 23 | |
| Reallocated to or Continued Vismodegib | COMPLETED | Sugar Pill | 11 | |
| Reallocated to or Continued Vismodegib | COMPLETED | GDC-0449 | 21 |
1 to 10 of 12Source ClinicalTrials.gov
See all 12 participant flowAdverse events52
Each adverse event the trial posted, group by group: the participants affected out of those at risk.
| Type | Event | Organ system | Group | Affected | At risk | Events |
|---|---|---|---|---|---|---|
| other | Hair loss | Skin and subcutaneous tissue disorders | Sugar Pill | 1 | 15 | |
| other | Hair loss | Skin and subcutaneous tissue disorders | GDC-0449 | 40 | 40 | |
| other | Muscle cramps | Musculoskeletal and connective tissue disorders | Sugar Pill | 1 | 15 | |
| other | Muscle cramps | Musculoskeletal and connective tissue disorders | GDC-0449 | 40 | 40 | |
| other | Dysgeusia | Gastrointestinal disorders | Sugar Pill | 1 | 15 | |
| other | Dysgeusia | Gastrointestinal disorders | GDC-0449 | 37 | 40 | |
| other | Gastrointestinal upset | Gastrointestinal disorders | Sugar Pill | 2 | 15 | |
| other | Gastrointestinal upset | Gastrointestinal disorders | GDC-0449 | 26 | 40 | |
| other | Weight loss | Metabolism and nutrition disorders | Sugar Pill | 1 | 15 | |
| other | Weight loss | Metabolism and nutrition disorders | GDC-0449 | 25 | 40 |
1 to 10 of 52Source ClinicalTrials.gov
See all 52 adverse eventsAdverse event totals6
The totals the trial posted for each group.
| Total | Group | Affected | At risk |
|---|---|---|---|
| Total, all-cause mortality | Sugar Pill | 0 | 15 |
| Total, all-cause mortality | GDC-0449 | 2 | 40 |
| Total, other adverse events | Sugar Pill | 12 | 15 |
| Total, other adverse events | GDC-0449 | 40 | 40 |
| Total, serious adverse events | Sugar Pill | 2 | 15 |
| Total, serious adverse events | GDC-0449 | 21 | 40 |
Source ClinicalTrials.gov
Papers3
The papers the trial lists on ClinicalTrials.gov.
Grants0
The grants NIH RePORTER links to the trial, newest funded first: NIH's, and some of AHRQ's, CDC's, FDA's and VA's. Each shows its title, principal investigators, organization and funder in its newest year.
None in this release.
Trial comparisons2
Comparisons of the trial's two groups, calculated by ConductScience from the counts it posted.
| Comparison | Source |
|---|---|
| Sugar Pill vs GDC-0449: deaths from any cause | Trial comparisons |
| Sugar Pill vs GDC-0449: serious adverse events | Trial comparisons |
Tests0
The ConductCognition tests this trial's registered outcomes name, as ScienceGraph matched them in trial-tests-2026-11-12-0.2.1.
None in this release.
Data dates
The day each source was copied for this release.
Sources 3
- ClinicalTrials.gov's posted results, through CTTI's AACTFreely available. Acknowledgement requested
- ClinicalTrials.govNo license stated
- NIH RePORTER's clinical studies and project filesPublic domain
Each source's terms
- ClinicalTrials.gov's posted results, through CTTI's AACTClinicalTrials.gov data via Aggregate Analysis of ClinicalTrials.gov (AACT), CTTI. CTTI makes its materials freely available and requests acknowledgement. https://aact.ctti-clinicaltrials.org/documentation/299
- ClinicalTrials.govnone stated
- NIH RePORTER's clinical studies and project filesPublic domain (NIH RePORTER)
ClinicalTrials.gov's data carry an international copyright outside the United States: https://clinicaltrials.gov/about-site/terms-conditions.
For software
Read this trial from software.
The record and each of its lists are JSON, twenty rows a page. No key.
GET https://conductscience.org/api/trials/v1/NCT00957229
GET https://conductscience.org/api/trials/v1/NCT00957229/events?page=2