Trial Outcomes & Findings for Phase II Study of Radiation Therapy and Vismodegib for Advanced Head/Neck Basal Cell Carcinoma (NCT NCT01835626)
NCT ID: NCT01835626
Last Updated: 2021-05-03
Results Overview
The local-regional control rate at 12 months from protocol therapy completion, defined as absence of progressive disease within the irradiated planning tumor volumes (PTV) for patients with locally advanced basal cell carcinoma in the head and neck.
Recruitment status
COMPLETED
Study phase
PHASE2
Target enrollment
24 participants
Primary outcome timeframe
Up to 12 months after completing therapy
Results posted on
2021-05-03
Participant Flow
Participant milestones
| Measure |
Vismodegib and Radiation Therapy
150mg Vismodegib will be taken once a day, daily. Radiation therapy will be started after the patient has completed taking vismodegib for 12 weeks. The patient will take daily vismodegib through the completion of radiation therapy. The patient will receive radiation once a day, Monday through Friday, for 7 weeks. Each radiation treatment may take up to 30 minutes. After 12 weeks, they will be evaluated again to make sure they are still eligible to participate in the study. If they are eligible to continue, they will continue taking vismodeib daily as before for another 7 weeks while they receive radiation therapy.
|
|---|---|
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Overall Study
STARTED
|
24
|
|
Overall Study
COMPLETED
|
17
|
|
Overall Study
NOT COMPLETED
|
7
|
Reasons for withdrawal
Withdrawal data not reported
Baseline Characteristics
Phase II Study of Radiation Therapy and Vismodegib for Advanced Head/Neck Basal Cell Carcinoma
Baseline characteristics by cohort
| Measure |
Vismodegib and Radiation Therapy
n=24 Participants
150mg Vismodegib will be taken once a day, daily. Radiation therapy will be started after the patient has completed taking vismodegib for 12 weeks. The patient will take daily vismodegib through the completion of radiation therapy. The patient will receive radiation once a day, Monday through Friday, for 7 weeks. Each radiation treatment may take up to 30 minutes. After 12 weeks, they will be evaluated again to make sure they are still eligible to participate in the study. If they are eligible to continue, they will continue taking vismodeib daily as before for another 7 weeks while they receive radiation therapy.
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|---|---|
|
Age, Customized
50-59 years old
|
5 Participants
n=99 Participants
|
|
Age, Customized
60-69 years old
|
8 Participants
n=99 Participants
|
|
Age, Customized
70-79 years old
|
4 Participants
n=99 Participants
|
|
Age, Customized
80-89 years old
|
6 Participants
n=99 Participants
|
|
Age, Customized
90-99 years old
|
1 Participants
n=99 Participants
|
|
Sex: Female, Male
Female
|
6 Participants
n=99 Participants
|
|
Sex: Female, Male
Male
|
18 Participants
n=99 Participants
|
|
Ethnicity (NIH/OMB)
Hispanic or Latino
|
0 Participants
n=99 Participants
|
|
Ethnicity (NIH/OMB)
Not Hispanic or Latino
|
15 Participants
n=99 Participants
|
|
Ethnicity (NIH/OMB)
Unknown or Not Reported
|
9 Participants
n=99 Participants
|
|
Race (NIH/OMB)
American Indian or Alaska Native
|
0 Participants
n=99 Participants
|
|
Race (NIH/OMB)
Asian
|
1 Participants
n=99 Participants
|
|
Race (NIH/OMB)
Native Hawaiian or Other Pacific Islander
|
0 Participants
n=99 Participants
|
|
Race (NIH/OMB)
Black or African American
|
0 Participants
n=99 Participants
|
|
Race (NIH/OMB)
White
|
14 Participants
n=99 Participants
|
|
Race (NIH/OMB)
More than one race
|
0 Participants
n=99 Participants
|
|
Race (NIH/OMB)
Unknown or Not Reported
|
9 Participants
n=99 Participants
|
|
Region of Enrollment
United States
|
24 participants
n=99 Participants
|
PRIMARY outcome
Timeframe: Up to 12 months after completing therapyThe local-regional control rate at 12 months from protocol therapy completion, defined as absence of progressive disease within the irradiated planning tumor volumes (PTV) for patients with locally advanced basal cell carcinoma in the head and neck.
Outcome measures
| Measure |
Vismodegib and Radiation Therapy
n=17 Participants
150mg Vismodegib will be taken once a day, daily. Radiation therapy will be started after the patient has completed taking vismodegib for 12 weeks. The patient will take daily vismodegib through the completion of radiation therapy. The patient will receive radiation once a day, Monday through Friday, for 7 weeks. Each radiation treatment may take up to 30 minutes. After 12 weeks, they will be evaluated again to make sure they are still eligible to participate in the study. If they are eligible to continue, they will continue taking vismodeib daily as before for another 7 weeks while they receive radiation therapy.
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|---|---|
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Percentage of Patients With Local-regional Control Rate
|
100 percentage
|
SECONDARY outcome
Timeframe: From treatment start up to 12 months after completing therapyEstimate of the probability of PFS, with failure defined as any disease recurrence or death due to any cause with each duration measured from the time of first treatment with vismodegib to 12 months after completion of study treatment
Outcome measures
| Measure |
Vismodegib and Radiation Therapy
n=17 Participants
150mg Vismodegib will be taken once a day, daily. Radiation therapy will be started after the patient has completed taking vismodegib for 12 weeks. The patient will take daily vismodegib through the completion of radiation therapy. The patient will receive radiation once a day, Monday through Friday, for 7 weeks. Each radiation treatment may take up to 30 minutes. After 12 weeks, they will be evaluated again to make sure they are still eligible to participate in the study. If they are eligible to continue, they will continue taking vismodeib daily as before for another 7 weeks while they receive radiation therapy.
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|---|---|
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Progression-Free Survival (PFS)
|
1.00 probability of progression-free survival
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SECONDARY outcome
Timeframe: From treatment start up to 12 months after completing therapyEstimate of the probability of OS, with failure defined as any disease recurrence or death due to any cause with each duration measured from the time of first treatment with vismodegib to 12 months after completion of study treatment
Outcome measures
| Measure |
Vismodegib and Radiation Therapy
n=17 Participants
150mg Vismodegib will be taken once a day, daily. Radiation therapy will be started after the patient has completed taking vismodegib for 12 weeks. The patient will take daily vismodegib through the completion of radiation therapy. The patient will receive radiation once a day, Monday through Friday, for 7 weeks. Each radiation treatment may take up to 30 minutes. After 12 weeks, they will be evaluated again to make sure they are still eligible to participate in the study. If they are eligible to continue, they will continue taking vismodeib daily as before for another 7 weeks while they receive radiation therapy.
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|---|---|
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Overall Survival (OS)
|
1.00 probability of survival
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SECONDARY outcome
Timeframe: up to 12 months after completing therapyThe percentage of any adverse events (CTCAE, v. 4.0) assessed to be definitely, probably, or possibly related to vismodegib or its combination with radiation therapy at any point during protocol therapy or during the followup period.
Outcome measures
| Measure |
Vismodegib and Radiation Therapy
n=24 Participants
150mg Vismodegib will be taken once a day, daily. Radiation therapy will be started after the patient has completed taking vismodegib for 12 weeks. The patient will take daily vismodegib through the completion of radiation therapy. The patient will receive radiation once a day, Monday through Friday, for 7 weeks. Each radiation treatment may take up to 30 minutes. After 12 weeks, they will be evaluated again to make sure they are still eligible to participate in the study. If they are eligible to continue, they will continue taking vismodeib daily as before for another 7 weeks while they receive radiation therapy.
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|---|---|
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Percentage of Patients by Treatment-Related Adverse Events by Event Description
Grade 4 eye disorder
|
4 percentage of patients
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Percentage of Patients by Treatment-Related Adverse Events by Event Description
Grade 3 hyponatremia
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13 percentage of patients
|
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Percentage of Patients by Treatment-Related Adverse Events by Event Description
Grade 3 oral mucositis
|
8 percentage of patients
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|
Percentage of Patients by Treatment-Related Adverse Events by Event Description
Grade 3 Gastrointestinal disorder
|
8 percentage of patients
|
|
Percentage of Patients by Treatment-Related Adverse Events by Event Description
Grade 3 Dermatologic disorder
|
8 percentage of patients
|
|
Percentage of Patients by Treatment-Related Adverse Events by Event Description
Grade 3 Abnormal Liver function
|
4 percentage of patients
|
|
Percentage of Patients by Treatment-Related Adverse Events by Event Description
Grade 3 Anemia
|
4 percentage of patients
|
|
Percentage of Patients by Treatment-Related Adverse Events by Event Description
Grade 3 Hypertension
|
4 percentage of patients
|
|
Percentage of Patients by Treatment-Related Adverse Events by Event Description
Grade 2 Dermatitis
|
33 percentage of patients
|
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Percentage of Patients by Treatment-Related Adverse Events by Event Description
Grade 2 Dysgeusia
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33 percentage of patients
|
|
Percentage of Patients by Treatment-Related Adverse Events by Event Description
Grade 2 Myalgia
|
25 percentage of patients
|
|
Percentage of Patients by Treatment-Related Adverse Events by Event Description
Grade 2 Fatigue
|
21 percentage of patients
|
|
Percentage of Patients by Treatment-Related Adverse Events by Event Description
Grade 2 Oral mucositis
|
21 percentage of patients
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|
Percentage of Patients by Treatment-Related Adverse Events by Event Description
Grade 2 Weight Loss
|
17 percentage of patients
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SECONDARY outcome
Timeframe: up to 12 months after completing therapyPopulation: Only 1 patient experienced an adverse event (Grade 4 Eye Disorder) not related to disease progression
The percentage of patients experiencing Grade 4-5 adverse events classified using CTCAE v.4.1 assessed to be definitely, probably, or possibly related to the induction or concurrent treatment components of the protocol regimen (that is not definitely related to disease progression) at any point during protocol therapy or during the followup period
Outcome measures
| Measure |
Vismodegib and Radiation Therapy
n=17 Participants
150mg Vismodegib will be taken once a day, daily. Radiation therapy will be started after the patient has completed taking vismodegib for 12 weeks. The patient will take daily vismodegib through the completion of radiation therapy. The patient will receive radiation once a day, Monday through Friday, for 7 weeks. Each radiation treatment may take up to 30 minutes. After 12 weeks, they will be evaluated again to make sure they are still eligible to participate in the study. If they are eligible to continue, they will continue taking vismodeib daily as before for another 7 weeks while they receive radiation therapy.
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|---|---|
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Percentage of Patients by Adverse Event Not Related to Disease Progression
|
6 percentage of participants
|
SECONDARY outcome
Timeframe: up to 6 months from treatment startFeasibility of administering concurrent vismodegib with radiation therapy was assessed by the number of patients discontinuing treatment due to toxicity during the concurrent administration of vismodegib and radiation therapy (\<75% of planned radiation therapy delivered)
Outcome measures
| Measure |
Vismodegib and Radiation Therapy
n=24 Participants
150mg Vismodegib will be taken once a day, daily. Radiation therapy will be started after the patient has completed taking vismodegib for 12 weeks. The patient will take daily vismodegib through the completion of radiation therapy. The patient will receive radiation once a day, Monday through Friday, for 7 weeks. Each radiation treatment may take up to 30 minutes. After 12 weeks, they will be evaluated again to make sure they are still eligible to participate in the study. If they are eligible to continue, they will continue taking vismodeib daily as before for another 7 weeks while they receive radiation therapy.
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|---|---|
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Number of Patients Discontinuing Treatment Due to Toxicity
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5 Participants
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SECONDARY outcome
Timeframe: Up to 12 months after completing therapyResponse rate (as per RECIST) of the primary site and regionally involved areas following all treatment components at 3 months after the completion of protocol therapy will be reported as a percentage of the total sample.
Outcome measures
| Measure |
Vismodegib and Radiation Therapy
n=17 Participants
150mg Vismodegib will be taken once a day, daily. Radiation therapy will be started after the patient has completed taking vismodegib for 12 weeks. The patient will take daily vismodegib through the completion of radiation therapy. The patient will receive radiation once a day, Monday through Friday, for 7 weeks. Each radiation treatment may take up to 30 minutes. After 12 weeks, they will be evaluated again to make sure they are still eligible to participate in the study. If they are eligible to continue, they will continue taking vismodeib daily as before for another 7 weeks while they receive radiation therapy.
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|---|---|
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Clinical Response Rate
|
88 percentage of patients
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SECONDARY outcome
Timeframe: Up to 12 months after completing therapyProportion of patients with a decrease of BCC within the irradiated planning tumor volumes (PTV) in patients who complete initial combined therapy, indicating a clinical response to vismodegib and radiation therapy
Outcome measures
| Measure |
Vismodegib and Radiation Therapy
n=17 Participants
150mg Vismodegib will be taken once a day, daily. Radiation therapy will be started after the patient has completed taking vismodegib for 12 weeks. The patient will take daily vismodegib through the completion of radiation therapy. The patient will receive radiation once a day, Monday through Friday, for 7 weeks. Each radiation treatment may take up to 30 minutes. After 12 weeks, they will be evaluated again to make sure they are still eligible to participate in the study. If they are eligible to continue, they will continue taking vismodeib daily as before for another 7 weeks while they receive radiation therapy.
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|---|---|
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Percentage of Patients With a Decrease of Basal Cell Carcinoma (BCC)
|
88 percentage of patients
|
Adverse Events
Vismodegib and Radiation Therapy
Serious events: 4 serious events
Other events: 24 other events
Deaths: 1 deaths
Serious adverse events
| Measure |
Vismodegib and Radiation Therapy
n=24 participants at risk
150mg Vismodegib will be taken once a day, daily. Radiation therapy will be started after the patient has completed taking vismodegib for 12 weeks. The patient will take daily vismodegib through the completion of radiation therapy. The patient will receive radiation once a day, Monday through Friday, for 7 weeks. Each radiation treatment may take up to 30 minutes. After 12 weeks, they will be evaluated again to make sure they are still eligible to participate in the study. If they are eligible to continue, they will continue taking vismodeib daily as before for another 7 weeks while they receive radiation therapy.
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|---|---|
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Metabolism and nutrition disorders
Hyponatremia
|
8.3%
2/24 • Number of events 2 • Up to 12 months
|
|
Eye disorders
Corneal ulcer
|
4.2%
1/24 • Number of events 1 • Up to 12 months
|
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Gastrointestinal disorders
Gastric hemorrhage
|
4.2%
1/24 • Number of events 1 • Up to 12 months
|
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Vascular disorders
Abdominal Aortic Aneurysm
|
4.2%
1/24 • Number of events 1 • Up to 12 months
|
Other adverse events
| Measure |
Vismodegib and Radiation Therapy
n=24 participants at risk
150mg Vismodegib will be taken once a day, daily. Radiation therapy will be started after the patient has completed taking vismodegib for 12 weeks. The patient will take daily vismodegib through the completion of radiation therapy. The patient will receive radiation once a day, Monday through Friday, for 7 weeks. Each radiation treatment may take up to 30 minutes. After 12 weeks, they will be evaluated again to make sure they are still eligible to participate in the study. If they are eligible to continue, they will continue taking vismodeib daily as before for another 7 weeks while they receive radiation therapy.
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|---|---|
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Nervous system disorders
Dysgeusia
|
79.2%
19/24 • Number of events 30 • Up to 12 months
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Nervous system disorders
Headache
|
16.7%
4/24 • Number of events 4 • Up to 12 months
|
|
Nervous system disorders
Peripheral sensory neuropathy
|
16.7%
4/24 • Number of events 5 • Up to 12 months
|
|
Nervous system disorders
Dizziness
|
8.3%
2/24 • Number of events 2 • Up to 12 months
|
|
Nervous system disorders
Memory impairment
|
8.3%
2/24 • Number of events 4 • Up to 12 months
|
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Musculoskeletal and connective tissue disorders
Myalgia
|
75.0%
18/24 • Number of events 22 • Up to 12 months
|
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Musculoskeletal and connective tissue disorders
Superficial soft tissue fibrosis
|
8.3%
2/24 • Number of events 2 • Up to 12 months
|
|
Musculoskeletal and connective tissue disorders
Trismus
|
8.3%
2/24 • Number of events 2 • Up to 12 months
|
|
General disorders
Fatigue
|
79.2%
19/24 • Number of events 26 • Up to 12 months
|
|
General disorders
General disorders and administration site conditions - other
|
16.7%
4/24 • Number of events 12 • Up to 12 months
|
|
General disorders
Edema face
|
8.3%
2/24 • Number of events 2 • Up to 12 months
|
|
General disorders
Facial pain
|
8.3%
2/24 • Number of events 2 • Up to 12 months
|
|
Gastrointestinal disorders
Mucositis oral
|
33.3%
8/24 • Number of events 16 • Up to 12 months
|
|
Gastrointestinal disorders
Dry mouth
|
20.8%
5/24 • Number of events 8 • Up to 12 months
|
|
Gastrointestinal disorders
Nausea
|
20.8%
5/24 • Number of events 6 • Up to 12 months
|
|
Gastrointestinal disorders
Constipation
|
16.7%
4/24 • Number of events 4 • Up to 12 months
|
|
Gastrointestinal disorders
Diarrhea
|
8.3%
2/24 • Number of events 3 • Up to 12 months
|
|
Gastrointestinal disorders
Dysphagia
|
8.3%
2/24 • Number of events 2 • Up to 12 months
|
|
Investigations
Weight loss
|
41.7%
10/24 • Number of events 13 • Up to 12 months
|
|
Investigations
Alanine aminotransferase increased
|
8.3%
2/24 • Number of events 2 • Up to 12 months
|
|
Investigations
Creatinine increased
|
8.3%
2/24 • Number of events 3 • Up to 12 months
|
|
Skin and subcutaneous tissue disorders
Alopecia
|
37.5%
9/24 • Number of events 9 • Up to 12 months
|
|
Skin and subcutaneous tissue disorders
Pruritus
|
16.7%
4/24 • Number of events 5 • Up to 12 months
|
|
Skin and subcutaneous tissue disorders
Pain of skin
|
8.3%
2/24 • Number of events 2 • Up to 12 months
|
|
Injury, poisoning and procedural complications
Dermatitis radiation
|
45.8%
11/24 • Number of events 18 • Up to 12 months
|
|
Metabolism and nutrition disorders
Anorexia
|
29.2%
7/24 • Number of events 9 • Up to 12 months
|
|
Metabolism and nutrition disorders
Hypokalemia
|
8.3%
2/24 • Number of events 2 • Up to 12 months
|
|
Respiratory, thoracic and mediastinal disorders
Epistaxis
|
16.7%
4/24 • Number of events 5 • Up to 12 months
|
|
Respiratory, thoracic and mediastinal disorders
Cough
|
12.5%
3/24 • Number of events 3 • Up to 12 months
|
|
Respiratory, thoracic and mediastinal disorders
Nasal Congestion
|
8.3%
2/24 • Number of events 2 • Up to 12 months
|
|
Eye disorders
Blurred vision
|
8.3%
2/24 • Number of events 2 • Up to 12 months
|
|
Eye disorders
Dry eye
|
8.3%
2/24 • Number of events 2 • Up to 12 months
|
|
Vascular disorders
Hypertension
|
16.7%
4/24 • Number of events 5 • Up to 12 months
|
|
Vascular disorders
Hypotension
|
8.3%
2/24 • Number of events 2 • Up to 12 months
|
|
Cardiac disorders
Palpitations
|
8.3%
2/24 • Number of events 2 • Up to 12 months
|
|
Psychiatric disorders
Depression
|
8.3%
2/24 • Number of events 2 • Up to 12 months
|
|
Eye disorders
Eye disorders - Other
|
8.3%
2/24 • Number of events 3 • Up to 12 months
|
|
Metabolism and nutrition disorders
Hyponatremia
|
12.5%
3/24 • Number of events 9 • Up to 12 months
|
Additional Information
Results disclosure agreements
- Principal investigator is a sponsor employee
- Publication restrictions are in place