Trial Outcomes & Findings for Regorafenib in Patients With Progressive, Recurrent/Metastatic Adenoid Cystic Carcinoma (NCT NCT02098538)
NCT ID: NCT02098538
Last Updated: 2025-09-10
Results Overview
Per Response Evaluation Criteria In Solid Tumors Criteria (RECIST v1.0) for target lesions and assessed by MRI: Complete Response (CR), Disappearance of all target lesions; Partial Response (PR), \>=30% decrease in the sum of the longest diameter of target lesions; Overall Response (OR) = CR + PR
Recruitment status
COMPLETED
Study phase
PHASE2
Target enrollment
38 participants
Primary outcome timeframe
6 months
Results posted on
2025-09-10
Participant Flow
Participant milestones
| Measure |
Patients With Adenoid Cystic Carcinoma
This is a single-arm phase II study of patients with progressive, recurrent/metastatic adenoid cystic carcinoma (R/M ACC) treated with regorafenib.
|
|---|---|
|
Overall Study
STARTED
|
38
|
|
Overall Study
COMPLETED
|
38
|
|
Overall Study
NOT COMPLETED
|
0
|
Reasons for withdrawal
Withdrawal data not reported
Baseline Characteristics
Regorafenib in Patients With Progressive, Recurrent/Metastatic Adenoid Cystic Carcinoma
Baseline characteristics by cohort
| Measure |
Patients With Adenoid Cystic Carcinoma
n=38 Participants
This is a single-arm phase II study of patients with progressive, recurrent/metastatic adenoid cystic carcinoma (R/M ACC) treated with regorafenib.
|
|---|---|
|
Age, Continuous
|
55 years
n=5 Participants
|
|
Sex: Female, Male
Female
|
19 Participants
n=5 Participants
|
|
Sex: Female, Male
Male
|
19 Participants
n=5 Participants
|
|
Race (NIH/OMB)
American Indian or Alaska Native
|
0 Participants
n=5 Participants
|
|
Race (NIH/OMB)
Asian
|
5 Participants
n=5 Participants
|
|
Race (NIH/OMB)
Native Hawaiian or Other Pacific Islander
|
0 Participants
n=5 Participants
|
|
Race (NIH/OMB)
Black or African American
|
0 Participants
n=5 Participants
|
|
Race (NIH/OMB)
White
|
33 Participants
n=5 Participants
|
|
Race (NIH/OMB)
More than one race
|
0 Participants
n=5 Participants
|
|
Race (NIH/OMB)
Unknown or Not Reported
|
0 Participants
n=5 Participants
|
|
Ethnicity (NIH/OMB)
Hispanic or Latino
|
3 Participants
n=5 Participants
|
|
Ethnicity (NIH/OMB)
Not Hispanic or Latino
|
35 Participants
n=5 Participants
|
|
Ethnicity (NIH/OMB)
Unknown or Not Reported
|
0 Participants
n=5 Participants
|
|
Region of Enrollment
United States
|
38 Participants
n=5 Participants
|
PRIMARY outcome
Timeframe: 6 monthsPer Response Evaluation Criteria In Solid Tumors Criteria (RECIST v1.0) for target lesions and assessed by MRI: Complete Response (CR), Disappearance of all target lesions; Partial Response (PR), \>=30% decrease in the sum of the longest diameter of target lesions; Overall Response (OR) = CR + PR
Outcome measures
| Measure |
Patients With Adenoid Cystic Carcinoma
n=38 Participants
This is a single-arm phase II study of patients with progressive, recurrent/metastatic adenoid cystic carcinoma (R/M ACC) treated with regorafenib.
|
|---|---|
|
Number of Participants Alive With and Without Disease Progression
Participants alive without disease progression
|
4 Participants
|
|
Number of Participants Alive With and Without Disease Progression
Participants alive with disease progression
|
6 Participants
|
|
Number of Participants Alive With and Without Disease Progression
Not evaluable
|
28 Participants
|
PRIMARY outcome
Timeframe: 6 months(BOR = CR+PR) documented by RECIST v1.1 criteria
Outcome measures
| Measure |
Patients With Adenoid Cystic Carcinoma
n=38 Participants
This is a single-arm phase II study of patients with progressive, recurrent/metastatic adenoid cystic carcinoma (R/M ACC) treated with regorafenib.
|
|---|---|
|
Best Overall Response Rate
Participants with CR+PR
|
1 Participants
|
|
Best Overall Response Rate
Participants without CR+PR
|
37 Participants
|
SECONDARY outcome
Timeframe: 1 yearSafety will be assessed in terms of AEs, laboratory data and vital sign data, which will be collected for all patients. Appropriate summaries of these data will be presented. AE will be listed individually per patient according to CTCAE version 4.0, and the number of patients experiencing each AE will be summarized. The safety population will comprise all patients who receive at least one dose of study treatment.
Outcome measures
| Measure |
Patients With Adenoid Cystic Carcinoma
n=38 Participants
This is a single-arm phase II study of patients with progressive, recurrent/metastatic adenoid cystic carcinoma (R/M ACC) treated with regorafenib.
|
|---|---|
|
Number of Participants Evaluated for Adverse Events
|
38 Participants
|
Adverse Events
Patients With Adenoid Cystic Carcinoma
Serious events: 14 serious events
Other events: 38 other events
Deaths: 28 deaths
Serious adverse events
| Measure |
Patients With Adenoid Cystic Carcinoma
n=38 participants at risk
This is a single-arm phase II study of patients with progressive, recurrent/metastatic adenoid cystic carcinoma (R/M ACC) treated with regorafenib.
|
|---|---|
|
Gastrointestinal disorders
Abdominal Pain
|
5.3%
2/38 • 1 year
|
|
Investigations
Alanine aminotransferase, increased
|
5.3%
2/38 • 1 year
|
|
Investigations
Aspartate aminotransferase, increased
|
2.6%
1/38 • 1 year
|
|
Blood and lymphatic system disorders
Anemia
|
2.6%
1/38 • 1 year
|
|
Musculoskeletal and connective tissue disorders
Back pain
|
2.6%
1/38 • 1 year
|
|
Hepatobiliary disorders
Bilirubin, increased
|
2.6%
1/38 • 1 year
|
|
Gastrointestinal disorders
Diarrhea
|
2.6%
1/38 • 1 year
|
|
Metabolism and nutrition disorders
Dehydration
|
2.6%
1/38 • 1 year
|
|
Respiratory, thoracic and mediastinal disorders
Dyspnea
|
13.2%
5/38 • 1 year
|
|
General disorders
Chills
|
2.6%
1/38 • 1 year
|
|
General disorders
Fatigue
|
5.3%
2/38 • 1 year
|
|
Injury, poisoning and procedural complications
Fall
|
2.6%
1/38 • 1 year
|
|
Metabolism and nutrition disorders
Hyponatremia
|
2.6%
1/38 • 1 year
|
|
Metabolism and nutrition disorders
Hypokalemia
|
2.6%
1/38 • 1 year
|
|
Respiratory, thoracic and mediastinal disorders
Hypoxia
|
2.6%
1/38 • 1 year
|
|
Metabolism and nutrition disorders
Hypophosphatemia
|
7.9%
3/38 • 1 year
|
|
Infections and infestations
Infections and infestations - Other, specify
|
2.6%
1/38 • 1 year
|
|
Investigations
Lymphocyte count, decreased
|
10.5%
4/38 • 1 year
|
|
Nervous system disorders
Lethargy
|
2.6%
1/38 • 1 year
|
|
General disorders
Non-cardiac chest pain
|
2.6%
1/38 • 1 year
|
|
Neoplasms benign, malignant and unspecified (incl cysts and polyps)
Neoplasms ben/mal/unk (inc cyst/polyp) Other, spec
|
2.6%
1/38 • 1 year
|
|
Gastrointestinal disorders
Oral pain
|
2.6%
1/38 • 1 year
|
|
General disorders
Pain
|
2.6%
1/38 • 1 year
|
|
Skin and subcutaneous tissue disorders
Palmar-plantar erythrodysesthesia
|
2.6%
1/38 • 1 year
|
|
Blood and lymphatic system disorders
Platelet count, decreased
|
2.6%
1/38 • 1 year
|
|
Respiratory, thoracic and mediastinal disorders
Pleural effusion
|
5.3%
2/38 • 1 year
|
|
Cardiac disorders
Pericardial effusion
|
2.6%
1/38 • 1 year
|
|
Cardiac disorders
Pericarditis
|
2.6%
1/38 • 1 year
|
|
Musculoskeletal and connective tissue disorders
Pain in extremity
|
2.6%
1/38 • 1 year
|
|
Respiratory, thoracic and mediastinal disorders
Pneumothorax
|
2.6%
1/38 • 1 year
|
|
Skin and subcutaneous tissue disorders
Rash, maculopapular
|
13.2%
5/38 • 1 year
|
|
Infections and infestations
Soft tissue infection
|
2.6%
1/38 • 1 year
|
|
Nervous system disorders
Syncope
|
2.6%
1/38 • 1 year
|
|
Surgical and medical procedures
Surgical and medical procedures - Other, specify
|
2.6%
1/38 • 1 year
|
Other adverse events
| Measure |
Patients With Adenoid Cystic Carcinoma
n=38 participants at risk
This is a single-arm phase II study of patients with progressive, recurrent/metastatic adenoid cystic carcinoma (R/M ACC) treated with regorafenib.
|
|---|---|
|
Investigations
Alanine aminotransferase, increased
|
71.1%
27/38 • 1 year
|
|
Investigations
Aspartate aminotransferase, increased
|
76.3%
29/38 • 1 year
|
|
Skin and subcutaneous tissue disorders
Alopecia
|
36.8%
14/38 • 1 year
|
|
Musculoskeletal and connective tissue disorders
Arthralgia
|
21.1%
8/38 • 1 year
|
|
Investigations
Alkaline phosphatase, increased
|
65.8%
25/38 • 1 year
|
|
Metabolism and nutrition disorders
Anorexia
|
34.2%
13/38 • 1 year
|
|
Gastrointestinal disorders
Anal ulcer
|
2.6%
1/38 • 1 year
|
|
Gastrointestinal disorders
Anal hemorrhage
|
2.6%
1/38 • 1 year
|
|
Psychiatric disorders
Anxiety
|
5.3%
2/38 • 1 year
|
|
Blood and lymphatic system disorders
Anemia
|
47.4%
18/38 • 1 year
|
|
Gastrointestinal disorders
Abdominal pain
|
23.7%
9/38 • 1 year
|
|
Investigations
Activated partial thromboplastin time prolonged
|
2.6%
1/38 • 1 year
|
|
Respiratory, thoracic and mediastinal disorders
Allergic rhinitis
|
2.6%
1/38 • 1 year
|
|
Eye disorders
Blurred vision
|
23.7%
9/38 • 1 year
|
|
Investigations
Bilirubin, increased
|
73.7%
28/38 • 1 year
|
|
Respiratory, thoracic and mediastinal disorders
Bronchopulmonary hemorrhage
|
2.6%
1/38 • 1 year
|
|
Skin and subcutaneous tissue disorders
Burn
|
2.6%
1/38 • 1 year
|
|
Musculoskeletal and connective tissue disorders
Back pain
|
21.1%
8/38 • 1 year
|
|
Eye disorders
Cataract
|
2.6%
1/38 • 1 year
|
|
Gastrointestinal disorders
Cheilitis
|
2.6%
1/38 • 1 year
|
|
Cardiac disorders
Chest pain - cardiac
|
2.6%
1/38 • 1 year
|
|
Respiratory, thoracic and mediastinal disorders
Cough
|
65.8%
25/38 • 1 year
|
|
Gastrointestinal disorders
Constipation
|
39.5%
15/38 • 1 year
|
|
General disorders
Chills
|
2.6%
1/38 • 1 year
|
|
Gastrointestinal disorders
Colitis
|
2.6%
1/38 • 1 year
|
|
Investigations
Cholesterol high
|
2.6%
1/38 • 1 year
|
|
Infections and infestations
Conjunctivitis infective
|
2.6%
1/38 • 1 year
|
|
Investigations
Creatinine increased
|
2.6%
1/38 • 1 year
|
|
Gastrointestinal disorders
Diarrhea
|
42.1%
16/38 • 1 year
|
|
Skin and subcutaneous tissue disorders
Dry skin
|
21.1%
8/38 • 1 year
|
|
Eye disorders
Dry eye
|
7.9%
3/38 • 1 year
|
|
Gastrointestinal disorders
Dyspepsia
|
18.4%
7/38 • 1 year
|
|
Gastrointestinal disorders
Dysphagia
|
26.3%
10/38 • 1 year
|
|
Respiratory, thoracic and mediastinal disorders
Dyspnea
|
39.5%
15/38 • 1 year
|
|
Psychiatric disorders
Depression
|
5.3%
2/38 • 1 year
|
|
Gastrointestinal disorders
Dry mouth
|
21.1%
8/38 • 1 year
|
|
Nervous system disorders
Dizziness
|
23.7%
9/38 • 1 year
|
|
Nervous system disorders
Dysgeusia
|
5.3%
2/38 • 1 year
|
|
Metabolism and nutrition disorders
Dehydration
|
5.3%
2/38 • 1 year
|
|
Ear and labyrinth disorders
External ear pain
|
2.6%
1/38 • 1 year
|
|
Infections and infestations
Eye infection
|
2.6%
1/38 • 1 year
|
|
General disorders
Edema limbs
|
2.6%
1/38 • 1 year
|
|
Respiratory, thoracic and mediastinal disorders
Epistaxis
|
21.1%
8/38 • 1 year
|
|
Nervous system disorders
Facial nerve disorder
|
5.3%
2/38 • 1 year
|
|
General disorders
Facial pain
|
2.6%
1/38 • 1 year
|
|
Injury, poisoning and procedural complications
Fall
|
5.3%
2/38 • 1 year
|
|
General disorders
Fever
|
21.1%
8/38 • 1 year
|
|
Eye disorders
Floaters
|
7.9%
3/38 • 1 year
|
|
General disorders
Fatigue
|
84.2%
32/38 • 1 year
|
|
Gastrointestinal disorders
Gastroesophageal reflux disease
|
7.9%
3/38 • 1 year
|
|
Musculoskeletal and connective tissue disorders
Generalized muscle weakness
|
2.6%
1/38 • 1 year
|
|
Reproductive system and breast disorders
Genital edema
|
2.6%
1/38 • 1 year
|
|
Gastrointestinal disorders
Gingival pain
|
2.6%
1/38 • 1 year
|
|
Investigations
Hemoglobin, increased
|
21.1%
8/38 • 1 year
|
|
Nervous system disorders
Headache
|
28.9%
11/38 • 1 year
|
|
Ear and labyrinth disorders
Hearing impaired
|
7.9%
3/38 • 1 year
|
|
Respiratory, thoracic and mediastinal disorders
Hoarseness
|
44.7%
17/38 • 1 year
|
|
Metabolism and nutrition disorders
Hypophosphatemia
|
57.9%
22/38 • 1 year
|
|
Vascular disorders
Hypertension
|
78.9%
30/38 • 1 year
|
|
Metabolism and nutrition disorders
Hypocalcemia
|
31.6%
12/38 • 1 year
|
|
Metabolism and nutrition disorders
Hypoglycemia
|
23.7%
9/38 • 1 year
|
|
Metabolism and nutrition disorders
Hypoalbuminemia
|
71.1%
27/38 • 1 year
|
|
Metabolism and nutrition disorders
Hyperglycemia
|
97.4%
37/38 • 1 year
|
|
Metabolism and nutrition disorders
Hypokalemia
|
15.8%
6/38 • 1 year
|
|
Endocrine disorders
Hypothyroidism
|
2.6%
1/38 • 1 year
|
|
Metabolism and nutrition disorders
Hyperkalemia
|
5.3%
2/38 • 1 year
|
|
Metabolism and nutrition disorders
Hyponatremia
|
36.8%
14/38 • 1 year
|
|
Psychiatric disorders
Insomnia
|
7.9%
3/38 • 1 year
|
|
Reproductive system and breast disorders
Irregular menstruation
|
2.6%
1/38 • 1 year
|
|
Investigations
Lipase increased
|
2.6%
1/38 • 1 year
|
|
Infections and infestations
Lung infection
|
2.6%
1/38 • 1 year
|
|
Investigations
Lymphocyte count, decreased
|
34.2%
13/38 • 1 year
|
|
Gastrointestinal disorders
Lip pain
|
2.6%
1/38 • 1 year
|
|
Musculoskeletal and connective tissue disorders
Muscle weakness lower limb
|
2.6%
1/38 • 1 year
|
|
Musculoskeletal and connective tissue disorders
Musculoskeletal & conn tissue disorder, other
|
2.6%
1/38 • 1 year
|
|
Musculoskeletal and connective tissue disorders
Myalgia
|
23.7%
9/38 • 1 year
|
|
General disorders
Malaise
|
5.3%
2/38 • 1 year
|
|
Gastrointestinal disorders
Mucositis, oral
|
15.8%
6/38 • 1 year
|
|
Infections and infestations
Mucosal infection
|
7.9%
3/38 • 1 year
|
|
Respiratory, thoracic and mediastinal disorders
Nasal congestion
|
5.3%
2/38 • 1 year
|
|
Gastrointestinal disorders
Nausea
|
39.5%
15/38 • 1 year
|
|
Musculoskeletal and connective tissue disorders
Neck pain
|
5.3%
2/38 • 1 year
|
|
Neoplasms benign, malignant and unspecified (incl cysts and polyps)
Neoplasms ben/mal/unk (inc cyst/polyp) Other, spec
|
2.6%
1/38 • 1 year
|
|
Nervous system disorders
Neuralgia
|
2.6%
1/38 • 1 year
|
|
Investigations
Neutrophil count decreased
|
2.6%
1/38 • 1 year
|
|
General disorders
Non-cardiac chest pain
|
2.6%
1/38 • 1 year
|
|
Gastrointestinal disorders
Oral cavity fistula
|
2.6%
1/38 • 1 year
|
|
Gastrointestinal disorders
Oral hemorrhage
|
15.8%
6/38 • 1 year
|
|
Gastrointestinal disorders
Oral pain
|
44.7%
17/38 • 1 year
|
|
Gastrointestinal disorders
Oral dysesthesia
|
7.9%
3/38 • 1 year
|
|
Infections and infestations
Otitis media
|
2.6%
1/38 • 1 year
|
|
General disorders
Pain
|
60.5%
23/38 • 1 year
|
|
Musculoskeletal and connective tissue disorders
Pain in extremity
|
5.3%
2/38 • 1 year
|
|
Blood and lymphatic system disorders
Platelet count, decreased
|
52.6%
20/38 • 1 year
|
|
Skin and subcutaneous tissue disorders
Pruritus
|
13.2%
5/38 • 1 year
|
|
Skin and subcutaneous tissue disorders
Palmar-plantar erythrodysesthesia syndrome
|
73.7%
28/38 • 1 year
|
|
Respiratory, thoracic and mediastinal disorders
Pleuritic pain
|
2.6%
1/38 • 1 year
|
|
Nervous system disorders
Peripheral motor neuropathy
|
2.6%
1/38 • 1 year
|
|
Respiratory, thoracic and mediastinal disorders
Pneumonitis
|
2.6%
1/38 • 1 year
|
|
Nervous system disorders
Peripheral sensory neuropathy
|
39.5%
15/38 • 1 year
|
|
Cardiac disorders
Pericarditis
|
2.6%
1/38 • 1 year
|
|
Cardiac disorders
Palpitations
|
5.3%
2/38 • 1 year
|
|
Eye disorders
Periorbital edema
|
5.3%
2/38 • 1 year
|
|
Reproductive system and breast disorders
Pelvic pain
|
2.6%
1/38 • 1 year
|
|
Nervous system disorders
Paresthesia
|
15.8%
6/38 • 1 year
|
|
Skin and subcutaneous tissue disorders
Rash, acneiform
|
5.3%
2/38 • 1 year
|
|
Skin and subcutaneous tissue disorders
Rash, maculopapular
|
44.7%
17/38 • 1 year
|
|
Renal and urinary disorders
Renal and urinary disorders - Other, specify
|
2.6%
1/38 • 1 year
|
|
Respiratory, thoracic and mediastinal disorders
Sore throat
|
7.9%
3/38 • 1 year
|
|
Skin and subcutaneous tissue disorders
Scalp pain
|
2.6%
1/38 • 1 year
|
|
Cardiac disorders
Sinus tachycardia
|
15.8%
6/38 • 1 year
|
|
Infections and infestations
Sinusitis
|
5.3%
2/38 • 1 year
|
|
Investigations
Serum amylase increased
|
5.3%
2/38 • 1 year
|
|
Skin and subcutaneous tissue disorders
Skin hyperpigmentation
|
5.3%
2/38 • 1 year
|
|
Infections and infestations
Skin infection
|
7.9%
3/38 • 1 year
|
|
Skin and subcutaneous tissue disorders
Skin ulceration
|
2.6%
1/38 • 1 year
|
|
Injury, poisoning and procedural complications
Tracheal hemorrhage
|
2.6%
1/38 • 1 year
|
|
Musculoskeletal and connective tissue disorders
Trismus
|
26.3%
10/38 • 1 year
|
|
Infections and infestations
Tooth infection
|
2.6%
1/38 • 1 year
|
|
Gastrointestinal disorders
Toothache
|
2.6%
1/38 • 1 year
|
|
Ear and labyrinth disorders
Tinnitus
|
5.3%
2/38 • 1 year
|
|
Infections and infestations
Upper respiratory infection
|
2.6%
1/38 • 1 year
|
|
Renal and urinary disorders
Urinary frequency
|
18.4%
7/38 • 1 year
|
|
Infections and infestations
Urinary tract infection
|
2.6%
1/38 • 1 year
|
|
Renal and urinary disorders
Urinary tract pain
|
5.3%
2/38 • 1 year
|
|
Vascular disorders
Vascular disorders, other
|
2.6%
1/38 • 1 year
|
|
Cardiac disorders
Ventricular tachycardia
|
2.6%
1/38 • 1 year
|
|
Gastrointestinal disorders
Vomiting
|
18.4%
7/38 • 1 year
|
|
Investigations
Weight loss
|
39.5%
15/38 • 1 year
|
|
Blood and lymphatic system disorders
White blood cell, decreased
|
34.2%
13/38 • 1 year
|
Additional Information
Dr. Alan Ho, MD, PhD
Memorial Sloan Kettering Cancer Center
Phone: 646-608-3774
Email: [email protected]
Results disclosure agreements
- Principal investigator is a sponsor employee
- Publication restrictions are in place