Trial Outcomes & Findings for A Phase 2, Single-center, Single-arm, Open Label Trial of Vismodegib in Patients With Keratocystic Odontogenic Tumors (NCT NCT02366312)
NCT ID: NCT02366312
Last Updated: 2021-02-18
Results Overview
To evaluate the efficacy of GDC-0449 in reducing KCOT size (shrinkage) in NBCCS-associated KCOT and sporadic KCOT patients following 6 to 12 months of ingestion of 150 mg/day, up to 1 year of treatment and up to 2 years of post- treatment follow-up.
Recruitment status
COMPLETED
Study phase
PHASE2
Target enrollment
2 participants
Primary outcome timeframe
Baseline, 6 month (in treatment), 1 year (in treatment), 2 Year (post-treatment), 3 Year (post-treatment)
Results posted on
2021-02-18
Participant Flow
Subjects were recruited at the NYU Oral cancer center between mmyy and mmyy. The first participant was enrolled in mmyy and the last subject was enrolled in mmyy.
Of 3 enrolled subjects, 2 met inclusion criteria and received treatment.
Participant milestones
| Measure |
NBCCS-associated KCOT
Nevoid Basal Cell Carcinoma Syndrome (NBCCS)-associated Keratocystic Odontogenic Tumor (KCOT) Group
|
Sporadic KCOT
Sporadic Keratocystic Odontogenic Tumor (KCOT) Group
|
|---|---|---|
|
Overall Study
STARTED
|
1
|
1
|
|
Overall Study
COMPLETED
|
1
|
0
|
|
Overall Study
NOT COMPLETED
|
0
|
1
|
Reasons for withdrawal
| Measure |
NBCCS-associated KCOT
Nevoid Basal Cell Carcinoma Syndrome (NBCCS)-associated Keratocystic Odontogenic Tumor (KCOT) Group
|
Sporadic KCOT
Sporadic Keratocystic Odontogenic Tumor (KCOT) Group
|
|---|---|---|
|
Overall Study
Lost to Follow-up
|
0
|
1
|
Baseline Characteristics
A Phase 2, Single-center, Single-arm, Open Label Trial of Vismodegib in Patients With Keratocystic Odontogenic Tumors
Baseline characteristics by cohort
| Measure |
NBCCS-associated KCOT
n=1 Participants
Nevoid Basal Cell Carcinoma Syndrome (NBCCS)-associated Keratocystic Odontogenic Tumor (KCOT) Group
|
Sporadic KCOT
n=1 Participants
Sporadic Keratocystic Odontogenic Tumor (KCOT) Group
|
Total
n=2 Participants
Total of all reporting groups
|
|---|---|---|---|
|
Age, Categorical
<=18 years
|
0 Participants
n=5 Participants
|
0 Participants
n=7 Participants
|
0 Participants
n=5 Participants
|
|
Age, Categorical
Between 18 and 65 years
|
1 Participants
n=5 Participants
|
1 Participants
n=7 Participants
|
2 Participants
n=5 Participants
|
|
Age, Categorical
>=65 years
|
0 Participants
n=5 Participants
|
0 Participants
n=7 Participants
|
0 Participants
n=5 Participants
|
|
Sex: Female, Male
Female
|
0 Participants
n=5 Participants
|
0 Participants
n=7 Participants
|
0 Participants
n=5 Participants
|
|
Sex: Female, Male
Male
|
1 Participants
n=5 Participants
|
1 Participants
n=7 Participants
|
2 Participants
n=5 Participants
|
|
Race/Ethnicity, Customized
White - Not Hispanic or Latino
|
1 Participants
n=5 Participants
|
0 Participants
n=7 Participants
|
1 Participants
n=5 Participants
|
|
Race/Ethnicity, Customized
Hispanic or Latino
|
0 Participants
n=5 Participants
|
1 Participants
n=7 Participants
|
1 Participants
n=5 Participants
|
|
Race/Ethnicity, Customized
African American
|
0 Participants
n=5 Participants
|
0 Participants
n=7 Participants
|
0 Participants
n=5 Participants
|
|
Race/Ethnicity, Customized
Asian/Pacific Islander
|
0 Participants
n=5 Participants
|
0 Participants
n=7 Participants
|
0 Participants
n=5 Participants
|
|
Race/Ethnicity, Customized
Other
|
0 Participants
n=5 Participants
|
0 Participants
n=7 Participants
|
0 Participants
n=5 Participants
|
|
Region of Enrollment
United States
|
1 participants
n=5 Participants
|
1 participants
n=7 Participants
|
2 participants
n=5 Participants
|
|
Primary KCOT Tumor size at Baseline
|
3 centimeter
n=5 Participants
|
3.8 centimeter
n=7 Participants
|
2 centimeter
n=5 Participants
|
PRIMARY outcome
Timeframe: Baseline, 6 month (in treatment), 1 year (in treatment), 2 Year (post-treatment), 3 Year (post-treatment)Population: Subjects received ERIVEDGE® (vismodegib) 150 mg capsule orally once daily for 12 months.
To evaluate the efficacy of GDC-0449 in reducing KCOT size (shrinkage) in NBCCS-associated KCOT and sporadic KCOT patients following 6 to 12 months of ingestion of 150 mg/day, up to 1 year of treatment and up to 2 years of post- treatment follow-up.
Outcome measures
| Measure |
NBCCS-associated KCOT
n=1 Participants
Nevoid Basal Cell Carcinoma Syndrome (NBCCS)-associated Keratocystic Odontogenic Tumor (KCOT) Group
|
Sporadic KCOT
n=1 Participants
Sporadic Keratocystic Odontogenic Tumor (KCOT) Group
|
|---|---|---|
|
KCOT Volume (Greatest Dimension)
Baseline
|
3 centimeter
|
3.8 centimeter
|
|
KCOT Volume (Greatest Dimension)
6 month (In treatment)
|
0 centimeter
|
2.6 centimeter
|
|
KCOT Volume (Greatest Dimension)
1 Year (In treatment)
|
2.3 centimeter
|
2.0 centimeter
|
|
KCOT Volume (Greatest Dimension)
2 Year (post-treatment)
|
2.2 centimeter
|
1.9 centimeter
|
|
KCOT Volume (Greatest Dimension)
3 Year (post-treatment)
|
2.0 centimeter
|
0 centimeter
|
Adverse Events
NBCCS-associated KCOT
Serious events: 0 serious events
Other events: 1 other events
Deaths: 0 deaths
Sporadic KCOT
Serious events: 1 serious events
Other events: 1 other events
Deaths: 0 deaths
Serious adverse events
| Measure |
NBCCS-associated KCOT
n=1 participants at risk
Nevoid Basal Cell Carcinoma Syndrome (NBCCS)-associated Keratocystic Odontogenic Tumor (KCOT) Group
|
Sporadic KCOT
n=1 participants at risk
Sporadic Keratocystic Odontogenic Tumor (KCOT) Group
|
|---|---|---|
|
Blood and lymphatic system disorders
High grade Newuorendocrine Carcinoma of head/neck
|
0.00%
0/1 • Up to three years
|
100.0%
1/1 • Number of events 1 • Up to three years
|
Other adverse events
| Measure |
NBCCS-associated KCOT
n=1 participants at risk
Nevoid Basal Cell Carcinoma Syndrome (NBCCS)-associated Keratocystic Odontogenic Tumor (KCOT) Group
|
Sporadic KCOT
n=1 participants at risk
Sporadic Keratocystic Odontogenic Tumor (KCOT) Group
|
|---|---|---|
|
General disorders
Pain
|
0.00%
0/1 • Up to three years
|
100.0%
1/1 • Number of events 1 • Up to three years
|
|
Ear and labyrinth disorders
Earache
|
0.00%
0/1 • Up to three years
|
100.0%
1/1 • Number of events 1 • Up to three years
|
|
General disorders
Sore Throat
|
0.00%
0/1 • Up to three years
|
100.0%
1/1 • Number of events 1 • Up to three years
|
|
Musculoskeletal and connective tissue disorders
Cramping
|
0.00%
0/1 • Up to three years
|
100.0%
1/1 • Number of events 1 • Up to three years
|
|
Musculoskeletal and connective tissue disorders
spasm
|
100.0%
1/1 • Number of events 1 • Up to three years
|
0.00%
0/1 • Up to three years
|
|
General disorders
Allergic reaction
|
100.0%
1/1 • Number of events 1 • Up to three years
|
0.00%
0/1 • Up to three years
|
|
Musculoskeletal and connective tissue disorders
Spasm
|
100.0%
1/1 • Number of events 1 • Up to three years
|
0.00%
0/1 • Up to three years
|
|
Musculoskeletal and connective tissue disorders
cramp
|
100.0%
1/1 • Number of events 1 • Up to three years
|
0.00%
0/1 • Up to three years
|
|
Skin and subcutaneous tissue disorders
Hair loss
|
0.00%
0/1 • Up to three years
|
100.0%
1/1 • Number of events 1 • Up to three years
|
|
Gastrointestinal disorders
Pain
|
0.00%
0/1 • Up to three years
|
100.0%
1/1 • Number of events 1 • Up to three years
|
|
Nervous system disorders
Facial numbness
|
100.0%
1/1 • Number of events 1 • Up to three years
|
0.00%
0/1 • Up to three years
|
|
Musculoskeletal and connective tissue disorders
Leg Cramp
|
100.0%
1/1 • Number of events 1 • Up to three years
|
0.00%
0/1 • Up to three years
|
|
Respiratory, thoracic and mediastinal disorders
Chest Discomfort
|
100.0%
1/1 • Number of events 2 • Up to three years
|
0.00%
0/1 • Up to three years
|
|
General disorders
Cold symptoms
|
100.0%
1/1 • Number of events 2 • Up to three years
|
0.00%
0/1 • Up to three years
|
|
Gastrointestinal disorders
GI upset
|
100.0%
1/1 • Number of events 2 • Up to three years
|
0.00%
0/1 • Up to three years
|
|
Reproductive system and breast disorders
Upper Respiratory Infection
|
100.0%
1/1 • Number of events 1 • Up to three years
|
0.00%
0/1 • Up to three years
|
|
General disorders
Loss of taste
|
0.00%
0/1 • Up to three years
|
100.0%
1/1 • Number of events 1 • Up to three years
|
|
Musculoskeletal and connective tissue disorders
Toes cramping
|
0.00%
0/1 • Up to three years
|
100.0%
1/1 • Number of events 1 • Up to three years
|
|
Gastrointestinal disorders
Loose stool
|
100.0%
1/1 • Number of events 2 • Up to three years
|
0.00%
0/1 • Up to three years
|
|
Musculoskeletal and connective tissue disorders
lower extremity pain
|
0.00%
0/1 • Up to three years
|
100.0%
1/1 • Number of events 1 • Up to three years
|
|
Musculoskeletal and connective tissue disorders
Right facial pain
|
0.00%
0/1 • Up to three years
|
100.0%
1/1 • Number of events 1 • Up to three years
|
|
Nervous system disorders
Headache
|
100.0%
1/1 • Number of events 1 • Up to three years
|
0.00%
0/1 • Up to three years
|
|
Musculoskeletal and connective tissue disorders
Left sided muscle ache
|
100.0%
1/1 • Number of events 1 • Up to three years
|
0.00%
0/1 • Up to three years
|
|
Musculoskeletal and connective tissue disorders
Sacral injury
|
100.0%
1/1 • Number of events 1 • Up to three years
|
0.00%
0/1 • Up to three years
|
|
Infections and infestations
Nasopharyn lesion
|
0.00%
0/1 • Up to three years
|
100.0%
1/1 • Number of events 1 • Up to three years
|
|
Endocrine disorders
bilateral lower extremities swelling
|
0.00%
0/1 • Up to three years
|
100.0%
1/1 • Number of events 1 • Up to three years
|
Additional Information
Results disclosure agreements
- Principal investigator is a sponsor employee
- Publication restrictions are in place