Adenoid Cystic CarcinomaPhase 2NCT02098538
Regorafenib in Patients With Progressive, Recurrent/Metastatic Adenoid Cystic Carcinoma
A Phase 2 study testing Regorafenib in people with Adenoid Cystic Carcinoma, run by Memorial Sloan Kettering Cancer Center at 5 sites in United States.
Trial summary
Regorafenib is an oral medication that can interfere with cancer cell growth and reduce the growth of blood vessels around tumors. This study will help find out if regorafenib is a useful drug for treating patients with adenoid cystic carcinomas. Read more about this study
- Who is it for?
- Ages 18 years and older
- Conditions the study is for
- Adenoid Cystic Carcinoma
- What is being tested?
- Regorafenib
- Type of treatment
- Pill or oral medicine
- Could I receive a placebo?
- No, everyone receives an active treatment
- How many people
- 38 participants
- Where
- 5 sitesUnited States
- Study dates
- Mar 2014to 7 Nov 2024
- Phase
- Phase 2
- Are there results?
- Yes, posted in the registrySummary in the doctors' view
Key requirements
Who can take part, as listed by the sponsor. Only the study team can confirm whether you are eligible, so take this list to your doctor.
- Ages 18 years and older
- All sexes
- No healthy volunteers
- Adult, Older adult
Who may be able to join
- Patients must have pathologically or cytologically confirmed adenoid cystic carcinoma. Cancers arising from non-salivary gland primary sites are allowed.
- Patients must have recurrent and/or metastatic disease not amenable to potentially curative surgery or radiotherapy.
- At least 2 weeks must have elapsed since the end of prior systemic treatment (4 weeks for bevacizumab- containing regimens) or radiotherapy with resolution of all treatment-related toxicity to NCI CTCAE Version 4.0 grade ≤1 (or tolerable grade 2) or back to baseline (except for alopecia, lymphopenia, or hypothyroidism). Any number of prior therapies for recurrent/metastatic ACC are allowed.
- Patients must have RECIST v1.1 measurable disease, defined as at least one lesion that can be accurately measured in at least one dimension (longest diameter to be recorded for nonnodal lesions and short axis for nodal lesions) as ≥ 20 mm with conventional techniques or as ≥ 10 mm with spiral CT scan.
- Patients must have documentation of a new or progressive lesion on a radiologic imaging study performed within 6 months prior to study enrollment (progression of disease over any interval is allowed) and/or new/worsening disease related symptoms within 6 months prior to study enrollment. Note: This assessment will be performed by the treating investigator. Evidence of progression by RECIST criteria is not required.
- Patients must have archival tissue from the primary tumor or metastases available for correlative studies. Either a paraffin block or twenty unstained slides are acceptable. If twenty slides are not available, a lesser amount may be acceptable after discussion with the Principal Investigator, Dr. Alan L. Ho.
Show all 21Show fewer
- Patients must agree to undergo biopsy of a malignant lesion. Biopsies do not need to be done if either the investigator or person performing the biopsy judges that no tumor is accessible for biopsy or that biopsy poses too great of a risk to the patient. Patients may also be exempt if frozen tumor tissue has been collected within 12 months of study enrollment that the Principal Investigator deems is appropriate/sufficient for analysis on this protocol.
- Age ≥ 18 years.
- ECOG performance status ≤ 2 (Karnofsky ≥ 60%,).
- Life expectancy of at least 12 weeks (3 months) as determined by the investigator.
- Life expectancy of at least 12 weeks (3 months) as determined by the investigator.
- Subjects must be able to understand and be willing to sign the written informed consent form. A signed informed consent form must be appropriately obtained prior to the conduct of any trial-specific procedure.
- Adequate bone marrow, liver and renal function as assessed by the following laboratory requirements:
- Total bilirubin ≤ 1.5 x the upper limits of normal (ULN) Alanine aminotransferase (ALT) and aspartate amino-transferase (AST) ≤ 2.5 x ULN (≤ 5 x ULN for subjects with liver involvement of their cancer)
- Alkaline phosphatase limit ≤ 2.5 x ULN (≤ 5 x ULN for subjects with liver involvement of their cancer)
- Lipase ≤ 1.5 x the ULN
- Amylase ≤ 1.5 x the ULN Serum creatinine ≤ 1.5 x the ULN or calculated creatinine clearance >60 ml/min
- Platelet count ≥ 100,000 /mm3, hemoglobin (Hb) ≥ 9 g/dL, absolute neutrophil count (ANC) ≥ 1500/mm3. Blood transfusion to meet the inclusion criteria will not be allowed.
- Women of childbearing potential must have a negative serum pregnancy test performed within 2 weeks prior to the start of study drug. Post-menopausal women (defined as no menses for at least 1 year) and surgically sterilized women are not required to undergo a pregnancy test.
- Subjects (men and women) of childbearing potential must agree to use adequate contraception beginning at the signing of the consent form until at least 3 months after the last dose of study drug. The definition of adequate contraception will be based on the judgment of the principal investigator or a designated associate.
- Subject must be able to swallow and retain oral medication.
Who cannot join
- Concurrent anti-cancer therapy (chemotherapy, definitive radiation therapy, surgery, immunotherapy, biologic therapy, or tumor embolization) other than study treatment. Concurrent therapy with bisphosphonates or denosumab for bone metastases is allowed.
- Palliative radiation to non-target lesions is also allowed.
- Prior use of regorafenib.
- Uncontrolled hypertension (systolic pressure >140 mm Hg or diastolic pressure > 90 mm Hg [NCI-CTCAE v4.0] on repeated measurement) despite optimal medical management.
- Concurrent use of another investigational drug or device therapy (i.e., outside of study treatment) during, or within 4 weeks of trial entry (signing of the informed consent form).
- Concurrent use of strong CYP3A4 inhibitors (e.g. clarithromycin, grapefruit juice, itraconazole, ketoconazole, nefazodone, posaconazole, telithromycin, and voriconazole). or strong CYP3A4 inducers (e.g. rifampin, phenytoin, carbamazepine, phenobarbital, and St. John's Wort).
Show all 32Show fewer
- Use of any herbal remedy (e.g. St. John's wort [Hypericum perforatum])
- Major surgical procedure, open biopsy, or significant traumatic injury within 28 days before start of study medication.
- Active or clinically significant cardiac disease including:
- Congestive heart failure - New York Heart Association (NYHA) > Class II.
- Active coronary artery disease that is not medically treated.
- Cardiac arrhythmias requiring anti-arrhythmic therapy other than beta blockers or digoxin.
- Unstable angina (anginal symptoms at rest), new-onset angina within 3 months before randomization, or myocardial infarction within 6 months before randomization.
- Therapeutic anticoagulation with Vitamin-K antagonists (e.g., warfarin) is not allowed if the medication dose and/or INR/PTT are not considered stable by the treating physician. If the dose and/or INR/PTT are stable, therapeutic anticoagulation with Vitamin-K antagonists is allowed with close monitoring. Anticoagulation with heparin or heparinoids is allowed.
- Evidence or history of bleeding diathesis or coagulopathy.
- Any hemorrhage or bleeding event ≥ NCI CTCAE Grade 3 within 4 weeks prior to start of study medication.
- Subjects with thrombotic, embolic, venous, or arterial events, such as cerebrovascular accident (including transient ischemic attacks) deep vein thrombosis or pulmonary embolism within 6 months of start of study treatment.
- Subjects with a past or current diagnosis of another malignancy that will interfere with conduct of the trial. Patients with past or current cancer diagnoses other than ACC are allowed to enroll if the investigator believes it will not interfere with trial conduct.
- Patients with phaeochromocytoma.
- Known history of human immunodeficiency virus (HIV) infection or current chronic or active hepatitis B or C infection requiring treatment with antiviral therapy.
- Active infection that would impair the ability of the patient to receive study treatment.
- Symptomatic metastatic brain or leptomeningeal tumors (asymptomatic or treated metastatic brain and leptomeningeal tumors are allowed).
- Presence of a non-healing wound or non-healing ulcer that is not tumor related.
- Renal failure requiring hemo-or peritoneal dialysis.
- Patients with seizure disorder requiring medication.
- Interstitial lung disease with ongoing signs and symptoms at the time of informed consent.
- History of organ allograft (including corneal transplant).
- Known or suspected allergy or hypersensitivity to any of the study drugs, study drug classes, or excipients of the formulations given during the course of this trial.
- Any malabsorption condition.
- Women who are pregnant or breast-feeding.
- Any condition which, in the investigator's opinion, makes the subject unsuitable for trial participation.
- Substance abuse, medical, psychological or social conditions that may interfere with the subject's participation in the study or evaluation of the study results.
Trial locations and contacts
5 sites in 1 country.
5 sites in 1 country
United States5 sites
Memorial Sloan Kettering Cancer Center
Basking Ridge, New Jersey
Memorial Sloan Kettering Cancer Center @ Suffolk
Commack, New York, 11725
Memorial Sloan Kettering Westchester
Harrison, New York, 10604
Memorial Sloan Kettering Cancer Center
New York, New York, 10065
Memorial Sloan Kettering at Mercy Medical Center
Rockville Centre, New York
About this study
Official title: A Phase II Study of Regorafenib in Patients With Progressive, Recurrent/Metastatic Adenoid Cystic Carcinoma
Regorafenib is an oral medication that can interfere with cancer cell growth and reduce the growth of blood vessels around tumors. This study will help find out if regorafenib is a useful drug for treating patients with adenoid cystic carcinomas. Regorafenib has been approved by the Food and Drug Administration (FDA) for use in other cancers, but remains an experimental drug that has not yet been approved for use in adenoid cystic carcinoma.
In this study, the patient will initially be treated with a dose of regorafenib that is lower than what the FDA approved for other cancers in an attempt to decrease the risk of side effects. It is possible that this lower starting dose may not be as effective as the higher FDA approved dose. If the patient does well with the lower dose for at least a month on treatment, the physician may consider increasing the dose to the FDA approved dose.
- Conditions
- Adenoid Cystic Carcinoma
- Being tested
- Regorafenib
- Keywords
- Regorafenib, 13-244
- Medical subject headings
- Carcinoma, Adenoid Cystic
What participants receive
What participants receive. In a randomised study each person is assigned to one group by chance.
RegorafenibDrug
All eligible patients will receive a starting regorafenib dose of 120 mg daily taken orally for 3 weeks in a 4-week cycle. Patients for whom regorafenib dose reduction was not performed or required may have their treatment dose increased to the FDA-approved dose of 160 mg daily orally for 3 weeks in a 4-week cycle in cycle #2 or beyond (this is not mandatory). RECIST v1.1 tumor assessments will be made at baseline (CT or MRI) and then approximately every 2 cycles (or every 8 weeks (+/- 1 week)). After 10 months, imaging will be done every 3 cycles (or every 12 weeks (+/- 1 week)). Patients may remain on study until progression of disease or unacceptable toxicity
| Study group | Role | What they receive |
|---|---|---|
| patients with adenoid cystic carcinoma | Experimental | This is a single-arm phase II study of patients with progressive, recurrent/metastatic adenoid cystic carcinoma (R/M ACC) treated with regorafenib. Receives: Drug: Regorafenib |
What is being measured
The primary outcome is the main question the study is designed to answer.
Number of Participants Alive With and Without Disease Progression
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
Measured: 6 months
Best Overall Response Rate
(BOR = CR+PR) documented by RECIST v1.1 criteria
Measured: 6 months
Secondary outcomes (1)
Number of Participants Evaluated for Adverse Events
Safety 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.
Measured: 1 year
Timeline
From the start of the study to its completion; the mark is today. Estimated dates are the sponsor’s plan.
- First posted28 Mar 2014 (estimated)
- Study startMar 2014
- Primary completion7 Nov 2024
- Study completion7 Nov 2024
Resources
- Official record on ClinicalTrials.govThe registry entry this page is built from, with its full change history.
- Posted resultsParticipant flow, outcome measures and adverse events reported by the sponsor.
- Other trials for Adenoid Cystic CarcinomaStudies of the same condition, open and under way.
- More trials run by Memorial Sloan Kettering Cancer CenterThe sponsor's other registered studies.
- What the trial phases meanHow a treatment moves from first tests in people to approval.
More details
For doctors and researchers: how the study is designed, who runs it, and its registry IDs.
Study design
- Study type
- Interventional
- Phase
- Phase 2
- Primary purpose
- Treatment
- Allocation
- Not applicable
- Intervention model
- Single group
- Masking
- None (open label)
- Enrolment
- 38 participants (actual)
Sponsor and investigators
- Lead sponsor
- Memorial Sloan Kettering Cancer Center
- Sponsor type
- University, hospital or other
- Collaborators
- Bayer
- Responsible party
- Sponsor
- Investigators
- Alan Ho, MD, PhD, Principal investigator, Memorial Sloan Kettering Cancer Center
IDs, dates and publications
- ClinicalTrials.gov ID
- NCT02098538
- Sponsor's protocol ID
- 13-244
- Status last verified
- Nov 2024
- First submitted
- 24 Mar 2014
- Last updated
- 10 Sep 2025
- Results posted
- Yes, on ClinicalTrials.gov
Study at a glance
From the registry record, last updated by the sponsor on 10 Sep 2025. Recruitment status is as the sponsor reported it: confirm with the study team.
- Official title
- A Phase II Study of Regorafenib in Patients With Progressive, Recurrent/Metastatic Adenoid Cystic Carcinoma
- ClinicalTrials.gov ID
- NCT02098538
- Acronym / sponsor ID
- 13-244
- Conditions
- Adenoid Cystic Carcinoma
- Keywords
- Regorafenib, 13-244
- Phase
- Phase 2
- Study type
- Interventional
- Recruitment status
- Completed
- Enrolment
- 38 participants (actual)
- Lead sponsor
- Memorial Sloan Kettering Cancer CenterUniversity, hospital or other
- Collaborators
- Bayer
- Study start
- Mar 2014 (actual)
- Primary completion
- 7 Nov 2024 (actual)
- Study completion
- 7 Nov 2024 (actual)
- First posted
- 28 Mar 2014 (estimated)
- Results
- Posted 10 Sep 2025
Clinical summary
The research question as the sponsor registered it. This describes what the study plans to test, not what it found.
Regorafenib is an oral medication that can interfere with cancer cell growth and reduce the growth of blood vessels around tumors. This study will help find out if regorafenib is a useful drug for treating patients with adenoid cystic carcinomas. Regorafenib has been approved by the Food and Drug Administration (FDA) for use in other cancers, but remains an experimental drug that has not yet been approved for use in adenoid cystic carcinoma.
In this study, the patient will initially be treated with a dose of regorafenib that is lower than what the FDA approved for other cancers in an attempt to decrease the risk of side effects. It is possible that this lower starting dose may not be as effective as the higher FDA approved dose. If the patient does well with the lower dose for at least a month on treatment, the physician may consider increasing the dose to the FDA approved dose.
Study design
- Primary purpose
- Treatment
- Allocation
- Not applicable
- Intervention model
- Single group
- Masking
- None (open label)
- Number of arms
- 1
| Arm | Type | Receives |
|---|---|---|
| patients with adenoid cystic carcinoma | Experimental | Drug: RegorafenibThis is a single-arm phase II study of patients with progressive, recurrent/metastatic adenoid cystic carcinoma (R/M ACC) treated with regorafenib. |
Who may qualify
- Ages
- 18 years and older
- Sex
- All
- Healthy volunteers
- Not accepted
- Age groups
- Adult, Older adult
Key inclusion criteria (21 listed)
- Patients must have pathologically or cytologically confirmed adenoid cystic carcinoma. Cancers arising from non-salivary gland primary sites are allowed.
- Patients must have recurrent and/or metastatic disease not amenable to potentially curative surgery or radiotherapy.
- At least 2 weeks must have elapsed since the end of prior systemic treatment (4 weeks for bevacizumab- containing regimens) or radiotherapy with resolution of all treatment-related toxicity to NCI CTCAE Version 4.0 grade ≤1 (or tolerable grade 2) or back to baseline (except for alopecia, lymphopenia, or hypothyroidism). Any number of prior therapies for recurrent/metastatic ACC are allowed.
- Patients must have RECIST v1.1 measurable disease, defined as at least one lesion that can be accurately measured in at least one dimension (longest diameter to be recorded for nonnodal lesions and short axis for nodal lesions) as ≥ 20 mm with conventional techniques or as ≥ 10 mm with spiral CT scan.
- Patients must have documentation of a new or progressive lesion on a radiologic imaging study performed within 6 months prior to study enrollment (progression of disease over any interval is allowed) and/or new/worsening disease related symptoms within 6 months prior to study enrollment. Note: This assessment will be performed by the treating investigator. Evidence of progression by RECIST criteria is not required.
Key exclusion criteria (32 listed)
- Concurrent anti-cancer therapy (chemotherapy, definitive radiation therapy, surgery, immunotherapy, biologic therapy, or tumor embolization) other than study treatment. Concurrent therapy with bisphosphonates or denosumab for bone metastases is allowed.
- Palliative radiation to non-target lesions is also allowed.
- Prior use of regorafenib.
- Uncontrolled hypertension (systolic pressure >140 mm Hg or diastolic pressure > 90 mm Hg [NCI-CTCAE v4.0] on repeated measurement) despite optimal medical management.
- Concurrent use of another investigational drug or device therapy (i.e., outside of study treatment) during, or within 4 weeks of trial entry (signing of the informed consent form).
Eligibility is decided by the study team after screening; this overview does not establish that a patient qualifies. Full inclusion and exclusion criteria.
Interventions
| Intervention | Type | Arms |
|---|---|---|
| RegorafenibAll eligible patients will receive a starting regorafenib dose of 120 mg daily taken orally for 3 weeks in a 4-week cycle. Patients for whom regorafenib dose reduction was not performed or required may have their treatment dose increased to the FDA-approved dose of 160 mg daily orally for 3 weeks in a 4-week cycle in cycle #2 or beyond (this is not mandatory). RECIST v1.1 tumor assessments will be made at baseline (CT or MRI) and then approximately every 2 cycles (or every 8 weeks (+/- 1 week)). After 10 months, imaging will be done every 3 cycles (or every 12 weeks (+/- 1 week)). Patients may remain on study until progression of disease or unacceptable toxicity | Drug | patients with adenoid cystic carcinoma |
Primary outcome
Number of Participants Alive With and Without Disease Progression
Time frame: 6 months
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
Best Overall Response Rate
Time frame: 6 months
(BOR = CR+PR) documented by RECIST v1.1 criteria
1 secondary outcome
Number of Participants Evaluated for Adverse Events
Time frame: 1 year
Results and safety
Loading the posted results from ClinicalTrials.gov…
Research links
- Official record on ClinicalTrials.govLast updated by the sponsor 10 Sep 2025.
- Posted resultsParticipant flow, baseline, all outcome measures and adverse events.
- Memorial Sloan Kettering Cancer CenterLink provided by the sponsor.
Related trials for Adenoid Cystic Carcinoma
RecruitingPhase 1/2
Docetaxel and SX-682 in Recurrent/Metastatic Head and Neck Squamous Cell Carcinoma, Salivary Gland Carcinoma, and Advanced Prostate Cancer
Head and Neck Cancer, Head and Neck Squamous Cell Carcinoma
RecruitingPhase 1/2
VMD-928 Monotherapy and in Combination With Pembrolizumab to Treat TrkA Overexpression Driven Solid Tumors or Lymphoma
Head and Neck Carcinoma, Adenoid Cystic Carcinoma
RecruitingObservational study
SPARE-1st: Surgery Alone vs Surgery Plus Postoperative Radiotherapy for Sinonasal Adenoid Cystic Carcinoma
Adenoid Cystic Carcinoma, Sinonasal Carcinoma
RecruitingPhase 2
Neoadjuvant Anlotinib and Epirubicin for T4 Sinonasal Adenoid Cystic Carcinoma
Adenoid Cystic Carcinoma
RecruitingObservational study
Surgery Plus Postoperative Radiotherapy for T4 Sinonasal Adenoid Cystic Carcinoma
Adenoid Cystic Carcinoma, Sinonasal Carcinoma
Referral and contact
AzTrials does not enrol participants or take referrals. Contact the study team or a site listed below; they decide who can take part. This study is not recruiting at the moment.
Alan Ho, MD, PhD principal investigator
Memorial Sloan Kettering Cancer Center
5 sites in 1 country: see every site and its status.
Questions about this trial
What is this clinical trial studying?
A Phase 2 study testing Regorafenib in people with Adenoid Cystic Carcinoma, run by Memorial Sloan Kettering Cancer Center at 5 sites in United States.
The full registered description is in the About this study section and the official record.
Who may be eligible to take part?
The sponsor lists 21 inclusion and 32 exclusion criteria. Eligibility is decided by the study team after screening, so read the full criteria and contact a listed site.
Is this clinical trial currently recruiting?
The record says: finished, as of the sponsor's last update on 10 Sep 2025. Status can change between updates, so confirm with the study contact before making plans.
Where is the study taking place?
The record lists 5 sites in United States. Whether a site is taking part right now should be confirmed with that site. See every site.
What are the possible benefits and risks?
They depend on the study and the treatment, and no study can promise a benefit. The study team explains the known risks during informed consent, before anyone joins; the record itself does not list them.
What does this phase mean?
Phase 2 studies give the treatment to a larger group to see whether it has the intended effect and to keep checking safety.
Not every study follows the same path, and some studies (observational studies, device studies) have no phase.
Are results available for this study?
Yes. Results are posted in the registry (first posted 10 Sep 2025). A summary is in the For doctors view, and the full results are on ClinicalTrials.gov.
How can a doctor refer a patient or contact the research team?
The record lists no contact. The official record names the sponsor, who can be asked directly. AzTrials does not enrol participants or take referrals.
What does the primary outcome measure?
Number of Participants Alive With and Without Disease Progression (time frame: 6 months). The study lists 2 primary outcomes in all. It is what the study was designed to measure, not a finding.
Where can I read the original study information?
Does a listing mean the treatment is approved or proven to work?
No. A listing means the study is registered. It does not show that the treatment is safe, works, or is approved by a regulator.
How often is this information updated?
AzTrials copies changed records from ClinicalTrials.gov every night and reloads the whole registry every week. The sponsor last updated this record on 10 Sep 2025.