Trial Outcomes & Findings for A Study Evaluating the Efficacy and Safety of Vismodegib (GDC-0449) in Operable Basal Cell Carcinoma (NCT NCT01201915)

NCT ID: NCT01201915

Last Updated: 2014-06-24

Results Overview

Complete histologic clearance was defined as the absence of histological evidence of basal cell carcinoma at the target tumor site. Histological examination was performed by an independent pathologist on specimens collected within 2 weeks of the end of treatment period, ie, at 12 weeks after Baseline in Cohort 1, at 36 weeks after Baseline in Cohort 2, and at 20 weeks after Baseline in Cohort 3.

Recruitment status

COMPLETED

Study phase

PHASE2

Target enrollment

74 participants

Primary outcome timeframe

Baseline to Week 12 (Cohort 1), Baseline to Week 36 (Cohort 2), Baseline to Week 20 (Cohort 3)

Results posted on

2014-06-24

Participant Flow

Participant milestones

Participant milestones
Measure	Cohort 1: Vismodegib 150 mg Participants received vismodegib 150 mg orally daily for 12 weeks.	Cohort 2: Vismodegib 150 mg Participants received vismodegib 150 mg orally daily for 12 weeks.	Cohort 3: Vismodegib 150 mg Participants received vismodegib 150 mg orally daily for 8 weeks, followed by 4 weeks with no treatment, followed by a second 8-week vismodegib treatment period.
Overall Study STARTED	24	25	25
Overall Study COMPLETED	16	14	19
Overall Study NOT COMPLETED	8	11	6

Reasons for withdrawal

Reasons for withdrawal
Measure	Cohort 1: Vismodegib 150 mg Participants received vismodegib 150 mg orally daily for 12 weeks.	Cohort 2: Vismodegib 150 mg Participants received vismodegib 150 mg orally daily for 12 weeks.	Cohort 3: Vismodegib 150 mg Participants received vismodegib 150 mg orally daily for 8 weeks, followed by 4 weeks with no treatment, followed by a second 8-week vismodegib treatment period.
Overall Study Adverse Event	2	1	1
Overall Study Lost to Follow-up	0	4	0
Overall Study Physician Decision	3	0	0
Overall Study Subject Decision	3	3	5
Overall Study Target Lesion Progression	0	3	0

Baseline Characteristics

A Study Evaluating the Efficacy and Safety of Vismodegib (GDC-0449) in Operable Basal Cell Carcinoma

Baseline characteristics by cohort

Baseline characteristics by cohort
Measure	Cohort 1: Vismodegib 150 mg n=24 Participants Participants received vismodegib 150 mg orally daily for 12 weeks.	Cohort 2: Vismodegib 150 mg n=25 Participants Participants received vismodegib 150 mg orally daily for 12 weeks.	Cohort 3: Vismodegib 150 mg n=25 Participants Participants received vismodegib 150 mg orally daily for 8 weeks, followed by 4 weeks with no treatment, followed by a second 8-week vismodegib treatment period.	Total n=74 Participants Total of all reporting groups
Age, Continuous	60.5 years STANDARD_DEVIATION 11.2 • n=99 Participants	65.2 years STANDARD_DEVIATION 13.3 • n=107 Participants	65.1 years STANDARD_DEVIATION 11.8 • n=206 Participants	63.6 years STANDARD_DEVIATION 12.1 • n=7 Participants
Sex: Female, Male Female	5 Participants n=99 Participants	3 Participants n=107 Participants	8 Participants n=206 Participants	16 Participants n=7 Participants
Sex: Female, Male Male	19 Participants n=99 Participants	22 Participants n=107 Participants	17 Participants n=206 Participants	58 Participants n=7 Participants

PRIMARY outcome

Timeframe: Baseline to Week 12 (Cohort 1), Baseline to Week 36 (Cohort 2), Baseline to Week 20 (Cohort 3)

Population: Efficacy evaluable population: All participants who were treated with at least 1 dose of study drug.

Outcome measures

Outcome measures
Measure	Cohort 1: Vismodegib 150 mg n=24 Participants Participants received vismodegib 150 mg orally daily for 12 weeks.	Cohort 2: Vismodegib 150 mg n=25 Participants Participants received vismodegib 150 mg orally daily for 12 weeks.	Cohort 3: Vismodegib 150 mg n=25 Participants Participants received vismodegib 150 mg orally daily for 8 weeks, followed by 4 weeks with no treatment, followed by a second 8-week vismodegib treatment period.
Percentage of Participants With Complete Histologic Clearance	42.0 Percentage of participants Interval 22.1 to 63.4	16.0 Percentage of participants Interval 4.5 to 36.1	44.0 Percentage of participants Interval 24.4 to 65.1

SECONDARY outcome

Timeframe: Baseline to the end of the study (up to 12 weeks for Cohort 1; up to 36 weeks for Cohort 2, up to 20 weeks for Cohort 3)

Population: Efficacy evaluable population: All participants who were treated with at least 1 dose of study drug. Only participants who achieved complete clinical clearance were included in the analysis.

Time to complete clinical clearance was defined as the time from the first treatment with vismodegib until complete clinical clearance as determined by the investigator.

Outcome measures

Outcome measures
Measure	Cohort 1: Vismodegib 150 mg n=10 Participants Participants received vismodegib 150 mg orally daily for 12 weeks.	Cohort 2: Vismodegib 150 mg n=9 Participants Participants received vismodegib 150 mg orally daily for 12 weeks.	Cohort 3: Vismodegib 150 mg n=18 Participants Participants received vismodegib 150 mg orally daily for 8 weeks, followed by 4 weeks with no treatment, followed by a second 8-week vismodegib treatment period.
Time to Complete Clinical Clearance	59.5 Days Interval 28.0 to 80.0	84.0 Days Interval 27.0 to 120.0	60.0 Days Interval 55.0 to 86.0

Adverse Events

Cohort 1: Vismodegib 150 mg

Serious events: 0 serious events

Other events: 24 other events

Deaths: 0 deaths

Cohort 2: Vismodegib 150 mg

Serious events: 3 serious events

Other events: 25 other events

Deaths: 0 deaths

Cohort 3: Vismodegib 150 mg

Serious events: 3 serious events

Other events: 24 other events

Deaths: 0 deaths

Serious adverse events

Serious adverse events
Measure	Cohort 1: Vismodegib 150 mg n=24 participants at risk Participants received vismodegib 150 mg orally daily for 12 weeks.	Cohort 2: Vismodegib 150 mg n=25 participants at risk Participants received vismodegib 150 mg orally daily for 12 weeks.	Cohort 3: Vismodegib 150 mg n=25 participants at risk Participants received vismodegib 150 mg orally daily for 8 weeks, followed by 4 weeks with no treatment, followed by a second 8-week vismodegib treatment period.
Cardiac disorders Atrial fibrillation	0.00% 0/24 Safety population: All participants who were treated with at least 1 dose of study drug.	0.00% 0/25 Safety population: All participants who were treated with at least 1 dose of study drug.	4.0% 1/25 • Number of events 1 Safety population: All participants who were treated with at least 1 dose of study drug.
Gastrointestinal disorders Small intestinal haemorrhage	0.00% 0/24 Safety population: All participants who were treated with at least 1 dose of study drug.	0.00% 0/25 Safety population: All participants who were treated with at least 1 dose of study drug.	4.0% 1/25 • Number of events 1 Safety population: All participants who were treated with at least 1 dose of study drug.
Hepatobiliary disorders Hepatitis	0.00% 0/24 Safety population: All participants who were treated with at least 1 dose of study drug.	0.00% 0/25 Safety population: All participants who were treated with at least 1 dose of study drug.	4.0% 1/25 • Number of events 1 Safety population: All participants who were treated with at least 1 dose of study drug.
Neoplasms benign, malignant and unspecified (incl cysts and polyps) Bladder cancer	0.00% 0/24 Safety population: All participants who were treated with at least 1 dose of study drug.	4.0% 1/25 • Number of events 1 Safety population: All participants who were treated with at least 1 dose of study drug.	0.00% 0/25 Safety population: All participants who were treated with at least 1 dose of study drug.
Neoplasms benign, malignant and unspecified (incl cysts and polyps) Liposarcoma	0.00% 0/24 Safety population: All participants who were treated with at least 1 dose of study drug.	4.0% 1/25 • Number of events 1 Safety population: All participants who were treated with at least 1 dose of study drug.	0.00% 0/25 Safety population: All participants who were treated with at least 1 dose of study drug.
Nervous system disorders Ischaemic stroke	0.00% 0/24 Safety population: All participants who were treated with at least 1 dose of study drug.	4.0% 1/25 • Number of events 1 Safety population: All participants who were treated with at least 1 dose of study drug.	0.00% 0/25 Safety population: All participants who were treated with at least 1 dose of study drug.
Gastrointestinal disorders Haemorrhoidal haemorrhage	0.00% 0/24 Safety population: All participants who were treated with at least 1 dose of study drug.	0.00% 0/25 Safety population: All participants who were treated with at least 1 dose of study drug.	4.0% 1/25 Safety population: All participants who were treated with at least 1 dose of study drug.

Other adverse events

Additional Information

Medical Communications

Genentech, Inc.

Phone: 800 821-8590

Email: [email protected]

Results disclosure agreements

Principal investigator is a sponsor employee The Study being conducted under this Agreement is part of the Overall Study. Investigator is free to publish in reputable journals or to present at professional conferences the results of the Study, but only after the first publication or presentation that involves the Overall Study. The Sponsor may request that Confidential Information be deleted and/or the publication be postponed in order to protect the Sponsor's intellectual property rights.
Publication restrictions are in place

Restriction type: OTHER

Measure	Cohort 1: Vismodegib 150 mg n=24 participants at risk Participants received vismodegib 150 mg orally daily for 12 weeks.	Cohort 2: Vismodegib 150 mg n=25 participants at risk Participants received vismodegib 150 mg orally daily for 12 weeks.	Cohort 3: Vismodegib 150 mg n=25 participants at risk Participants received vismodegib 150 mg orally daily for 8 weeks, followed by 4 weeks with no treatment, followed by a second 8-week vismodegib treatment period.
Gastrointestinal disorders Nausea	20.8% 5/24 Safety population: All participants who were treated with at least 1 dose of study drug.	8.0% 2/25 Safety population: All participants who were treated with at least 1 dose of study drug.	24.0% 6/25 Safety population: All participants who were treated with at least 1 dose of study drug.
Gastrointestinal disorders Constipation	0.00% 0/24 Safety population: All participants who were treated with at least 1 dose of study drug.	12.0% 3/25 Safety population: All participants who were treated with at least 1 dose of study drug.	12.0% 3/25 Safety population: All participants who were treated with at least 1 dose of study drug.
Gastrointestinal disorders Diarrhoea	8.3% 2/24 Safety population: All participants who were treated with at least 1 dose of study drug.	0.00% 0/25 Safety population: All participants who were treated with at least 1 dose of study drug.	12.0% 3/25 Safety population: All participants who were treated with at least 1 dose of study drug.
Gastrointestinal disorders Dyspepsia	8.3% 2/24 Safety population: All participants who were treated with at least 1 dose of study drug.	0.00% 0/25 Safety population: All participants who were treated with at least 1 dose of study drug.	8.0% 2/25 Safety population: All participants who were treated with at least 1 dose of study drug.
General disorders Fatigue	20.8% 5/24 Safety population: All participants who were treated with at least 1 dose of study drug.	12.0% 3/25 Safety population: All participants who were treated with at least 1 dose of study drug.	28.0% 7/25 Safety population: All participants who were treated with at least 1 dose of study drug.
Infections and infestations Upper Respiratory Tract Infection	0.00% 0/24 Safety population: All participants who were treated with at least 1 dose of study drug.	12.0% 3/25 Safety population: All participants who were treated with at least 1 dose of study drug.	0.00% 0/25 Safety population: All participants who were treated with at least 1 dose of study drug.
Metabolism and nutrition disorders Decreased Appetite	12.5% 3/24 Safety population: All participants who were treated with at least 1 dose of study drug.	0.00% 0/25 Safety population: All participants who were treated with at least 1 dose of study drug.	16.0% 4/25 Safety population: All participants who were treated with at least 1 dose of study drug.
Musculoskeletal and connective tissue disorders Muscle Spasms	79.2% 19/24 Safety population: All participants who were treated with at least 1 dose of study drug.	76.0% 19/25 Safety population: All participants who were treated with at least 1 dose of study drug.	72.0% 18/25 Safety population: All participants who were treated with at least 1 dose of study drug.
Nervous system disorders Dysgeusia	37.5% 9/24 Safety population: All participants who were treated with at least 1 dose of study drug.	52.0% 13/25 Safety population: All participants who were treated with at least 1 dose of study drug.	60.0% 15/25 Safety population: All participants who were treated with at least 1 dose of study drug.
Nervous system disorders Ageusia	41.7% 10/24 Safety population: All participants who were treated with at least 1 dose of study drug.	20.0% 5/25 Safety population: All participants who were treated with at least 1 dose of study drug.	28.0% 7/25 Safety population: All participants who were treated with at least 1 dose of study drug.
Nervous system disorders Hypogeusia	8.3% 2/24 Safety population: All participants who were treated with at least 1 dose of study drug.	0.00% 0/25 Safety population: All participants who were treated with at least 1 dose of study drug.	0.00% 0/25 Safety population: All participants who were treated with at least 1 dose of study drug.
Reproductive system and breast disorders Amenorrhoea	0.00% 0/24 Safety population: All participants who were treated with at least 1 dose of study drug.	8.0% 2/25 Safety population: All participants who were treated with at least 1 dose of study drug.	0.00% 0/25 Safety population: All participants who were treated with at least 1 dose of study drug.
Respiratory, thoracic and mediastinal disorders Cough	0.00% 0/24 Safety population: All participants who were treated with at least 1 dose of study drug.	0.00% 0/25 Safety population: All participants who were treated with at least 1 dose of study drug.	12.0% 3/25 Safety population: All participants who were treated with at least 1 dose of study drug.
Respiratory, thoracic and mediastinal disorders Nasal Congestion	0.00% 0/24 Safety population: All participants who were treated with at least 1 dose of study drug.	0.00% 0/25 Safety population: All participants who were treated with at least 1 dose of study drug.	8.0% 2/25 Safety population: All participants who were treated with at least 1 dose of study drug.
Skin and subcutaneous tissue disorders Alopecia	33.3% 8/24 Safety population: All participants who were treated with at least 1 dose of study drug.	68.0% 17/25 Safety population: All participants who were treated with at least 1 dose of study drug.	72.0% 18/25 Safety population: All participants who were treated with at least 1 dose of study drug.
Skin and subcutaneous tissue disorders Madarosis	12.5% 3/24 Safety population: All participants who were treated with at least 1 dose of study drug.	8.0% 2/25 Safety population: All participants who were treated with at least 1 dose of study drug.	12.0% 3/25 Safety population: All participants who were treated with at least 1 dose of study drug.
Neoplasms benign, malignant and unspecified (incl cysts and polyps) Squamous Cell Carcinoma	0.00% 0/24 Safety population: All participants who were treated with at least 1 dose of study drug.	0.00% 0/25 Safety population: All participants who were treated with at least 1 dose of study drug.	8.0% 2/25 Safety population: All participants who were treated with at least 1 dose of study drug.
Skin and subcutaneous tissue disorders Hair Colour Changes	0.00% 0/24 Safety population: All participants who were treated with at least 1 dose of study drug.	8.0% 2/25 Safety population: All participants who were treated with at least 1 dose of study drug.	0.00% 0/25 Safety population: All participants who were treated with at least 1 dose of study drug.
Skin and subcutaneous tissue disorders Actinic Keratosis	0.00% 0/24 Safety population: All participants who were treated with at least 1 dose of study drug.	8.0% 2/25 Safety population: All participants who were treated with at least 1 dose of study drug.	0.00% 0/25 Safety population: All participants who were treated with at least 1 dose of study drug.
Ear and labyrinth disorders Vertigo	8.3% 2/24 Safety population: All participants who were treated with at least 1 dose of study drug.	0.00% 0/25 Safety population: All participants who were treated with at least 1 dose of study drug.	0.00% 0/25 Safety population: All participants who were treated with at least 1 dose of study drug.
Respiratory, thoracic and mediastinal disorders Bronchitis	0.00% 0/24 Safety population: All participants who were treated with at least 1 dose of study drug.	8.0% 2/25 Safety population: All participants who were treated with at least 1 dose of study drug.	0.00% 0/25 Safety population: All participants who were treated with at least 1 dose of study drug.
Musculoskeletal and connective tissue disorders Myalgia	0.00% 0/24 Safety population: All participants who were treated with at least 1 dose of study drug.	8.0% 2/25 Safety population: All participants who were treated with at least 1 dose of study drug.	8.0% 2/25 Safety population: All participants who were treated with at least 1 dose of study drug.
Gastrointestinal disorders Vomiting	0.00% 0/24 Safety population: All participants who were treated with at least 1 dose of study drug.	0.00% 0/25 Safety population: All participants who were treated with at least 1 dose of study drug.	12.0% 3/25 Safety population: All participants who were treated with at least 1 dose of study drug.
Investigations Weight Decreased	0.00% 0/24 Safety population: All participants who were treated with at least 1 dose of study drug.	0.00% 0/25 Safety population: All participants who were treated with at least 1 dose of study drug.	12.0% 3/25 Safety population: All participants who were treated with at least 1 dose of study drug.
General disorders Influenza Like Illness	0.00% 0/24 Safety population: All participants who were treated with at least 1 dose of study drug.	0.00% 0/25 Safety population: All participants who were treated with at least 1 dose of study drug.	8.0% 2/25 Safety population: All participants who were treated with at least 1 dose of study drug.
Neoplasms benign, malignant and unspecified (incl cysts and polyps) Skin neoplasms malignant and unspecified (Excl melanoma)	0.00% 0/24 Safety population: All participants who were treated with at least 1 dose of study drug.	8.0% 2/25 Safety population: All participants who were treated with at least 1 dose of study drug.	0.00% 0/25 Safety population: All participants who were treated with at least 1 dose of study drug.