Trial Outcomes & Findings for VISmodegib for ORbital and Periocular Basal Cell Carcinoma (NCT NCT02436408)
NCT ID: NCT02436408
Last Updated: 2021-10-28
Results Overview
The VAWS was developed for the purpose of this study. It is made up of standard ophthalmic exam points, as well as subjective assessment of tearing and overall patient satisfaction. The maximum score is 50 and a score of 21 or greater will be considered a good outcome.
Recruitment status
COMPLETED
Study phase
PHASE4
Target enrollment
35 participants
Primary outcome timeframe
Until end of study treatment (up to 15 months after start of study treatment); treatment duration ranged from 53 - 386 days.
Results posted on
2021-10-28
Participant Flow
Participant milestones
| Measure |
Vismodegib
150mg taken orally once daily
|
|---|---|
|
Overall Study
STARTED
|
35
|
|
Overall Study
COMPLETED
|
34
|
|
Overall Study
NOT COMPLETED
|
1
|
Reasons for withdrawal
| Measure |
Vismodegib
150mg taken orally once daily
|
|---|---|
|
Overall Study
Death
|
1
|
Baseline Characteristics
VISmodegib for ORbital and Periocular Basal Cell Carcinoma
Baseline characteristics by cohort
| Measure |
Vismodegib
n=35 Participants
150mg taken orally once daily
|
|---|---|
|
Age, Continuous
|
69 years
n=5 Participants
|
|
Sex: Female, Male
Female
|
17 Participants
n=5 Participants
|
|
Sex: Female, Male
Male
|
18 Participants
n=5 Participants
|
|
Ethnicity (NIH/OMB)
Hispanic or Latino
|
0 Participants
n=5 Participants
|
|
Ethnicity (NIH/OMB)
Not Hispanic or Latino
|
34 Participants
n=5 Participants
|
|
Ethnicity (NIH/OMB)
Unknown or Not Reported
|
1 Participants
n=5 Participants
|
|
Region of Enrollment
United States
|
35 participants
n=5 Participants
|
PRIMARY outcome
Timeframe: Until end of study treatment (up to 15 months after start of study treatment); treatment duration ranged from 53 - 386 days.The VAWS was developed for the purpose of this study. It is made up of standard ophthalmic exam points, as well as subjective assessment of tearing and overall patient satisfaction. The maximum score is 50 and a score of 21 or greater will be considered a good outcome.
Outcome measures
| Measure |
Vismodegib
n=34 Participants
150mg taken orally once daily
|
|---|---|
|
The Number of Patients With a Score of 21 or Greater by the Visual Assessment Weighted Score (VAWS).
|
34 Participants
|
SECONDARY outcome
Timeframe: Until end of study treatment (up to 15 months after start of study treatment); treatment duration ranged from 53 - 386 days.Treatment response will be determined per Response Evaluation Criteria in Solid Tumors (RECIST) protocol, using clinical measurements and/or tumor imaging measurements (determined by treating physician).
Outcome measures
| Measure |
Vismodegib
n=34 Participants
150mg taken orally once daily
|
|---|---|
|
Number of Patients With Progressive Disease (PD)
|
0 Participants
|
SECONDARY outcome
Timeframe: Until end of study treatment (up to 15 months after start of study treatment); treatment duration ranged from 53 - 386 days.Tolerance will be self-reported by patient. Treatment response will be determined per Response Evaluation Criteria in Solid Tumors (RECIST) protocol, using clinical measurements and/or tumor imaging measurements (determined by treating physician).
Outcome measures
| Measure |
Vismodegib
n=34 Participants
150mg taken orally once daily
|
|---|---|
|
Number of Patients With a Complete Response (CR), Partial Response (PR) or Stable Disease (SD) AND Good Tolerance of Vismodegib
|
7 Participants
|
SECONDARY outcome
Timeframe: Until end of study treatment (up to 15 months after start of study treatment); treatment duration ranged from 53 - 386 days.Tolerance will be self-reported by patient. Treatment response will be determined per Response Evaluation Criteria in Solid Tumors (RECIST) protocol, using clinical measurements and/or tumor imaging measurements (determined by treating physician).
Outcome measures
| Measure |
Vismodegib
n=34 Participants
150mg taken orally once daily
|
|---|---|
|
Number of Patients With a Complete Response (CR), Partial Response (PR) or Stable Disease (SD) AND Poor Tolerance of Vismodegib
|
27 Participants
|
Adverse Events
Vismodegib
Serious events: 9 serious events
Other events: 34 other events
Deaths: 2 deaths
Serious adverse events
| Measure |
Vismodegib
n=35 participants at risk
150mg taken orally once daily
|
|---|---|
|
Cardiac disorders
Acute coronary syndrome
|
2.9%
1/35 • Up to 15 months after start of study treatment; data was collected on study for 62 months total
|
|
Renal and urinary disorders
Acute kidney injury
|
2.9%
1/35 • Up to 15 months after start of study treatment; data was collected on study for 62 months total
|
|
Respiratory, thoracic and mediastinal disorders
Aspiration
|
2.9%
1/35 • Up to 15 months after start of study treatment; data was collected on study for 62 months total
|
|
Gastrointestinal disorders
Colitis
|
2.9%
1/35 • Up to 15 months after start of study treatment; data was collected on study for 62 months total
|
|
Gastrointestinal disorders
Gastrointestinal disorders - Other
|
2.9%
1/35 • Up to 15 months after start of study treatment; data was collected on study for 62 months total
|
|
Cardiac disorders
Heart failure
|
2.9%
1/35 • Up to 15 months after start of study treatment; data was collected on study for 62 months total
|
|
Metabolism and nutrition disorders
Hypoglycemia
|
2.9%
1/35 • Up to 15 months after start of study treatment; data was collected on study for 62 months total
|
|
Infections and infestations
Infections and infestations - Other
|
2.9%
1/35 • Up to 15 months after start of study treatment; data was collected on study for 62 months total
|
|
Infections and infestations
Lung infection
|
5.7%
2/35 • Up to 15 months after start of study treatment; data was collected on study for 62 months total
|
|
Gastrointestinal disorders
Nausea
|
5.7%
2/35 • Up to 15 months after start of study treatment; data was collected on study for 62 months total
|
|
Neoplasms benign, malignant and unspecified (incl cysts and polyps)
Neoplasms benign, malignant and unspecified (incl cysts and polyps) - Other
|
2.9%
1/35 • Up to 15 months after start of study treatment; data was collected on study for 62 months total
|
|
Respiratory, thoracic and mediastinal disorders
Pulmonary edema
|
2.9%
1/35 • Up to 15 months after start of study treatment; data was collected on study for 62 months total
|
|
Infections and infestations
Sepsis
|
2.9%
1/35 • Up to 15 months after start of study treatment; data was collected on study for 62 months total
|
|
Skin and subcutaneous tissue disorders
Skin and subcutaneous tissue disorders - Other
|
2.9%
1/35 • Up to 15 months after start of study treatment; data was collected on study for 62 months total
|
|
Vascular disorders
Vascular disorders - Other
|
2.9%
1/35 • Up to 15 months after start of study treatment; data was collected on study for 62 months total
|
|
Gastrointestinal disorders
Vomiting
|
5.7%
2/35 • Up to 15 months after start of study treatment; data was collected on study for 62 months total
|
Other adverse events
| Measure |
Vismodegib
n=35 participants at risk
150mg taken orally once daily
|
|---|---|
|
Blood and lymphatic system disorders
Anemia
|
5.7%
2/35 • Up to 15 months after start of study treatment; data was collected on study for 62 months total
|
|
Cardiac disorders
Sinus bradycardia
|
2.9%
1/35 • Up to 15 months after start of study treatment; data was collected on study for 62 months total
|
|
Ear and labyrinth disorders
Hearing impaired
|
2.9%
1/35 • Up to 15 months after start of study treatment; data was collected on study for 62 months total
|
|
Ear and labyrinth disorders
Tinnitus
|
2.9%
1/35 • Up to 15 months after start of study treatment; data was collected on study for 62 months total
|
|
Eye disorders
Blurred vision
|
2.9%
1/35 • Up to 15 months after start of study treatment; data was collected on study for 62 months total
|
|
Eye disorders
Cataract
|
5.7%
2/35 • Up to 15 months after start of study treatment; data was collected on study for 62 months total
|
|
Eye disorders
Dry eye
|
2.9%
1/35 • Up to 15 months after start of study treatment; data was collected on study for 62 months total
|
|
Eye disorders
Eye disorders - Other
|
22.9%
8/35 • Up to 15 months after start of study treatment; data was collected on study for 62 months total
|
|
Eye disorders
Eye pain
|
2.9%
1/35 • Up to 15 months after start of study treatment; data was collected on study for 62 months total
|
|
Eye disorders
Eyelid function disorder
|
8.6%
3/35 • Up to 15 months after start of study treatment; data was collected on study for 62 months total
|
|
Eye disorders
Watering eyes
|
8.6%
3/35 • Up to 15 months after start of study treatment; data was collected on study for 62 months total
|
|
Gastrointestinal disorders
Abdominal pain
|
5.7%
2/35 • Up to 15 months after start of study treatment; data was collected on study for 62 months total
|
|
Gastrointestinal disorders
Constipation
|
17.1%
6/35 • Up to 15 months after start of study treatment; data was collected on study for 62 months total
|
|
Gastrointestinal disorders
Diarrhea
|
8.6%
3/35 • Up to 15 months after start of study treatment; data was collected on study for 62 months total
|
|
Gastrointestinal disorders
Gastroesophageal reflux disease
|
14.3%
5/35 • Up to 15 months after start of study treatment; data was collected on study for 62 months total
|
|
Gastrointestinal disorders
Nausea
|
31.4%
11/35 • Up to 15 months after start of study treatment; data was collected on study for 62 months total
|
|
Gastrointestinal disorders
Vomiting
|
11.4%
4/35 • Up to 15 months after start of study treatment; data was collected on study for 62 months total
|
|
General disorders
Chills
|
2.9%
1/35 • Up to 15 months after start of study treatment; data was collected on study for 62 months total
|
|
General disorders
Edema face
|
5.7%
2/35 • Up to 15 months after start of study treatment; data was collected on study for 62 months total
|
|
General disorders
Edema limbs
|
2.9%
1/35 • Up to 15 months after start of study treatment; data was collected on study for 62 months total
|
|
General disorders
Fatigue
|
34.3%
12/35 • Up to 15 months after start of study treatment; data was collected on study for 62 months total
|
|
General disorders
Fever
|
2.9%
1/35 • Up to 15 months after start of study treatment; data was collected on study for 62 months total
|
|
General disorders
General disorders and administration site conditions - Other
|
2.9%
1/35 • Up to 15 months after start of study treatment; data was collected on study for 62 months total
|
|
General disorders
Pain
|
11.4%
4/35 • Up to 15 months after start of study treatment; data was collected on study for 62 months total
|
|
Infections and infestations
Papulopustular rash
|
5.7%
2/35 • Up to 15 months after start of study treatment; data was collected on study for 62 months total
|
|
Injury, poisoning and procedural complications
Bruising
|
2.9%
1/35 • Up to 15 months after start of study treatment; data was collected on study for 62 months total
|
|
Injury, poisoning and procedural complications
Fall
|
5.7%
2/35 • Up to 15 months after start of study treatment; data was collected on study for 62 months total
|
|
Investigations
Alanine aminotransferase increased
|
2.9%
1/35 • Up to 15 months after start of study treatment; data was collected on study for 62 months total
|
|
Investigations
Alkaline phosphatase increased
|
2.9%
1/35 • Up to 15 months after start of study treatment; data was collected on study for 62 months total
|
|
Investigations
Aspartate aminotransferase increased
|
2.9%
1/35 • Up to 15 months after start of study treatment; data was collected on study for 62 months total
|
|
Investigations
Creatinine increased
|
2.9%
1/35 • Up to 15 months after start of study treatment; data was collected on study for 62 months total
|
|
Investigations
Weight loss
|
34.3%
12/35 • Up to 15 months after start of study treatment; data was collected on study for 62 months total
|
|
Metabolism and nutrition disorders
Anorexia
|
34.3%
12/35 • Up to 15 months after start of study treatment; data was collected on study for 62 months total
|
|
Metabolism and nutrition disorders
Hyperglycemia
|
5.7%
2/35 • Up to 15 months after start of study treatment; data was collected on study for 62 months total
|
|
Metabolism and nutrition disorders
Hyperkalemia
|
2.9%
1/35 • Up to 15 months after start of study treatment; data was collected on study for 62 months total
|
|
Musculoskeletal and connective tissue disorders
Arthralgia
|
8.6%
3/35 • Up to 15 months after start of study treatment; data was collected on study for 62 months total
|
|
Musculoskeletal and connective tissue disorders
Arthritis
|
5.7%
2/35 • Up to 15 months after start of study treatment; data was collected on study for 62 months total
|
|
Musculoskeletal and connective tissue disorders
Back pain
|
11.4%
4/35 • Up to 15 months after start of study treatment; data was collected on study for 62 months total
|
|
Musculoskeletal and connective tissue disorders
Generalized muscle weakness
|
14.3%
5/35 • Up to 15 months after start of study treatment; data was collected on study for 62 months total
|
|
Musculoskeletal and connective tissue disorders
Muscle weakness lower limb
|
8.6%
3/35 • Up to 15 months after start of study treatment; data was collected on study for 62 months total
|
|
Musculoskeletal and connective tissue disorders
Musculoskeletal and connective tissue disorder - Other
|
5.7%
2/35 • Up to 15 months after start of study treatment; data was collected on study for 62 months total
|
|
Musculoskeletal and connective tissue disorders
Myalgia
|
65.7%
23/35 • Up to 15 months after start of study treatment; data was collected on study for 62 months total
|
|
Musculoskeletal and connective tissue disorders
Pain in extremity
|
2.9%
1/35 • Up to 15 months after start of study treatment; data was collected on study for 62 months total
|
|
Nervous system disorders
Dizziness
|
8.6%
3/35 • Up to 15 months after start of study treatment; data was collected on study for 62 months total
|
|
Nervous system disorders
Dysgeusia
|
74.3%
26/35 • Up to 15 months after start of study treatment; data was collected on study for 62 months total
|
|
Nervous system disorders
Facial muscle weakness
|
2.9%
1/35 • Up to 15 months after start of study treatment; data was collected on study for 62 months total
|
|
Nervous system disorders
Peripheral motor neuropathy
|
2.9%
1/35 • Up to 15 months after start of study treatment; data was collected on study for 62 months total
|
|
Nervous system disorders
Spasticity
|
20.0%
7/35 • Up to 15 months after start of study treatment; data was collected on study for 62 months total
|
|
Psychiatric disorders
Agitation
|
2.9%
1/35 • Up to 15 months after start of study treatment; data was collected on study for 62 months total
|
|
Psychiatric disorders
Hallucinations
|
2.9%
1/35 • Up to 15 months after start of study treatment; data was collected on study for 62 months total
|
|
Psychiatric disorders
Insomnia
|
5.7%
2/35 • Up to 15 months after start of study treatment; data was collected on study for 62 months total
|
|
Psychiatric disorders
Libido decreased
|
2.9%
1/35 • Up to 15 months after start of study treatment; data was collected on study for 62 months total
|
|
Reproductive system and breast disorders
Erectile dysfunction
|
2.9%
1/35 • Up to 15 months after start of study treatment; data was collected on study for 62 months total
|
|
Respiratory, thoracic and mediastinal disorders
Dyspnea
|
2.9%
1/35 • Up to 15 months after start of study treatment; data was collected on study for 62 months total
|
|
Respiratory, thoracic and mediastinal disorders
Epistaxis
|
2.9%
1/35 • Up to 15 months after start of study treatment; data was collected on study for 62 months total
|
|
Respiratory, thoracic and mediastinal disorders
Nasal congestion
|
11.4%
4/35 • Up to 15 months after start of study treatment; data was collected on study for 62 months total
|
|
Respiratory, thoracic and mediastinal disorders
Productive cough
|
2.9%
1/35 • Up to 15 months after start of study treatment; data was collected on study for 62 months total
|
|
Respiratory, thoracic and mediastinal disorders
Sinus disorder
|
2.9%
1/35 • Up to 15 months after start of study treatment; data was collected on study for 62 months total
|
|
Skin and subcutaneous tissue disorders
Alopecia
|
48.6%
17/35 • Up to 15 months after start of study treatment; data was collected on study for 62 months total
|
|
Skin and subcutaneous tissue disorders
Photosensitivity
|
2.9%
1/35 • Up to 15 months after start of study treatment; data was collected on study for 62 months total
|
|
Skin and subcutaneous tissue disorders
Pruritus
|
2.9%
1/35 • Up to 15 months after start of study treatment; data was collected on study for 62 months total
|
|
Skin and subcutaneous tissue disorders
Rash maculo-papular
|
2.9%
1/35 • Up to 15 months after start of study treatment; data was collected on study for 62 months total
|
|
Skin and subcutaneous tissue disorders
Skin and subcutaneous tissue disorders - Other
|
11.4%
4/35 • Up to 15 months after start of study treatment; data was collected on study for 62 months total
|
|
Skin and subcutaneous tissue disorders
Skin ulceration
|
5.7%
2/35 • Up to 15 months after start of study treatment; data was collected on study for 62 months total
|
|
Skin and subcutaneous tissue disorders
Urticaria
|
2.9%
1/35 • Up to 15 months after start of study treatment; data was collected on study for 62 months total
|
|
Surgical and medical procedures
Surgical and medical procedures - Other
|
2.9%
1/35 • Up to 15 months after start of study treatment; data was collected on study for 62 months total
|
|
Vascular disorders
Hypotension
|
2.9%
1/35 • Up to 15 months after start of study treatment; data was collected on study for 62 months total
|
Additional Information
Cagri Besirli, MD, PhD
University of Michigan Rogel Cancer Center
Phone: 734-232-8404
Email: [email protected]
Results disclosure agreements
- Principal investigator is a sponsor employee
- Publication restrictions are in place