What side effects are associated with this type of intervention?

Common treatments for Adenoid Cystic Carcinoma, particularly those similar to Regorafenib, often involve tyrosine kinase inhibitors that target tumor growth and angiogenesis. Known side effects of these treatments include hypertension, hand-foot skin reactions, fatigue, diarrhea, and liver toxicity. Other similar agents, such as sorafenib and sunitinib, also frequently cause gastrointestinal disturbances, skin reactions, and cardiovascular issues.

What prior FDA approvals exist?

As of now, there are no FDA-approved treatments specifically for Adenoid Cystic Carcinoma (ACC) that are similar to Regorafenib. Regorafenib itself is an investigational drug for ACC, known for its ability to interfere with cancer cell growth and inhibit angiogenesis. Other drugs with similar mechanisms, such as tyrosine kinase inhibitors, are being explored in clinical trials but have not yet received FDA approval for ACC.

What other types of research exist?

Promising novel alternatives to Regorafenib for Adenoid Cystic Carcinoma patients include: 1) NOTCH inhibitors such as AL101, which is currently being evaluated in a phase II trial (NCT03691207). 2) mTOR inhibitors like Everolimus, which has shown potential in reducing tumor burden. 3) Antiangiogenic kinase inhibitors targeting mutated RET kinase, such as Selpercatinib and Pralsetinib, which have demonstrated clinical activity in RET-altered cancers.

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Multi-kinase inhibitor

Regorafenib for Adenoid Cystic Carcinoma

Phase 2

Waitlist Available

Led By Alan Ho, MD, PhD

Research Sponsored by Memorial Sloan Kettering Cancer Center

Eligibility Criteria Checklist

Specific guidelines that determine who can or cannot participate in a clinical trial

Must have

Patients must have recurrent and/or metastatic disease not amenable to potentially curative surgery or radiotherapy.

ECOG performance status ≤ 2 (Karnofsky ≥ 60%,).

Must not have

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).

Active or clinically significant cardiac disease including:

Timeline

Screening 3 weeks

Treatment Varies

Follow Up 1 year

Awards & highlights

No Placebo-Only Group

Summary

This trial is testing regorafenib, a pill that can stop cancer cells from growing and reduce their blood supply, in patients with adenoid cystic carcinomas. The study aims to see if this medication can be effective for these patients.

Who is the study for?

Adults with confirmed adenoid cystic carcinoma that's recurrent or spread and not treatable by surgery or radiotherapy. They must have measurable disease, no recent severe side effects from past treatments, and be in relatively good health with a life expectancy of at least 3 months. Pregnant women can't participate, and those who can have children must use contraception.

What is being tested?

The trial is testing Regorafenib, an oral drug that may slow cancer growth. Initially given at lower than the FDA-approved dose for other cancers to reduce side effects, the dose may increase if well-tolerated after one month.

What are the potential side effects?

Regorafenib might cause high blood pressure, bleeding problems, heart issues like chest pain or heart attack within the last six months, liver problems beyond certain limits set by the study criteria.

Eligibility Criteria

Inclusion Criteria

You may be eligible if you check “Yes” for the criteria below

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My cancer has returned or spread and cannot be removed by surgery or cured with radiation.

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I can take care of myself but might not be able to do active work.

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I am 18 years old or older.

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My amylase and creatinine levels are within normal limits.

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I can swallow and keep down pills.

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I have a tumor that can be measured with scans.

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My liver function tests are within the required range.

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My blood counts meet the required levels without transfusions.

Exclusion Criteria

You may be eligible for the trial if you check “No” for criteria below:

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I am not taking strong medication inhibitors or inducers like rifampin or ketoconazole.

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I do not have any serious heart conditions.

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I have untreated heart artery disease.

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I am on medication for heart rhythm problems not managed by beta blockers or digoxin.

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I have HIV or hepatitis B/C that needs antiviral treatment.

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I currently have symptoms of interstitial lung disease.

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My high blood pressure is not controlled even with medication.

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I have not had severe bleeding in the last 4 weeks.

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I have been diagnosed with phaeochromocytoma.

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I do not have an infection that would stop me from receiving the study treatment.

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I have a wound or ulcer that hasn’t healed, but it’s not caused by cancer.

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I have had an organ transplant.

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I am not pregnant or breast-feeding.

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I have previously taken regorafenib.

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I am not using any herbal remedies like St. John's wort.

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I have a history of bleeding disorders.

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I require dialysis for kidney failure.

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I am on medication for seizures.

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I have a condition that affects how my body absorbs nutrients.

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My heart condition is not severe and is classified below NYHA Class III.

Timeline

Screening ~ 3 weeks3 visits

Treatment ~ Varies

Follow Up ~ 1 year

Screening ~ 3 weeks

Treatment ~ Varies

Follow Up ~1 year

This trial's timeline: 3 weeks for screening, Varies for treatment, and 1 year for reporting.

Treatment Details

Study Objectives

Study objectives can provide a clearer picture of what you can expect from a treatment.

Primary study objectives

best overall response rate

patients alive without disease progression

Secondary study objectives

Safety

Side effects data

From 2019 Phase 1 & 2 trial • 495 Patients • NCT02024607

82%

Diarrhoea

76%

Nausea

71%

Fatigue

57%

Vomiting

53%

Abdominal pain

30%

Neuropathy peripheral

27%

Dyspnoea

25%

Anaemia

23%

Neutrophil count decreased

23%

Constipation

22%

Weight decreased

21%

Hypokalaemia

20%

Oedema peripheral

18%

Dehydration

17%

Cough

17%

Neutropenia

17%

Pyrexia

17%

Peripheral sensory neuropathy

16%

Thrombocytopenia

15%

Platelet count decreased

15%

Back pain

13%

Mucosal inflammation

13%

Temperature intolerance

13%

Dysgeusia

13%

Chromaturia

12%

White blood cell count decreased

12%

Urinary tract infection

11%

Dizziness

11%

Depression

10%

Hyponatraemia

10%

Stomatitis

10%

Ascites

10%

Dysphagia

10%

Anxiety

Headache

Abdominal distension

Insomnia

Arthralgia

Asthenia

Pain in extremity

Alopecia

Urine ketone body present

Blood alkaline phosphatase increased

Pulmonary embolism

Lymphopenia

Leukopenia

Blood bilirubin increased

Dyspepsia

Sepsis

Palmar-plantar erythrodysaesthesia syndrome

Hypertension

Urine leukocyte esterase positive

Abdominal pain upper

Gastrooesophageal reflux disease

Proteinuria

Rash

Flatulence

Chills

Dry mouth

Myalgia

Epistaxis

Muscle spasms

Pneumonia

pelvic fracture

Haematemesis

Disease progression

Pleural effusion

Confusional state

Mental status changes

malignant neoplasm progression

Large intestine perforation

Oesophagitis

Lung abscess

embolism

pancreatic carcinoma

fall

Death

Haemorrhage intranial

Atrial fibrillation

Colitis

Enterocutaneous fistula

Gastrointestinal haemorrhage

Upper gastrointestinal haemorrhage

Chest pain

somnolence

syncope

Febrile neutropenia

Acute kidney injury

Acute respiratory failure

Hypoxia

Pneumonia aspiration

Clostridium difficile colitis

Influenza

Perirectal abscess

Salmonella sepsis

Septic shock

hip fracture

deep vein thrombosis

haematoma

pelvic venous thrombosis

Cholangitis

Ischaemic cerebral infarction

Hyperglycaemia

subdural haematoma

100%

80%

60%

40%

20%

Study treatment Arm

Napabucasin Plus FOLFOX6

Napabucasin Plus FOLFOX6 Plus Bevacizumab

Napabucasin Plus FOLFIRI Plus Bevacizumab

Napabucasin Plus Regorafenib

Napabucasin Plus FOLFIRI

Napabucasin Plus Irinotecan

Napabucasin Plus CAPOX

Awards & Highlights

No Placebo-Only Group

All patients enrolled in this study will receive some form of active treatment.

Trial Design

1Treatment groups

Experimental Treatment

Group I: patients with adenoid cystic carcinomaExperimental Treatment1 Intervention

This is a single-arm phase II study of patients with progressive, recurrent/metastatic adenoid cystic carcinoma (R/M ACC) treated with regorafenib.

Treatment

First Studied

Drug Approval Stage

How many patients have taken this drug

Regorafenib

2014

Completed Phase 2

~1630

0 / 28Eligibility criteria met

Research Highlights

Information in this section is not a recommendation. We encourage patients to speak with their healthcare team when evaluating any treatment decision.

Mechanism Of Action

Side Effect Profile

Prior Approvals

Other Research

The most common treatments for Adenoid Cystic Carcinoma (ACC) include surgery, radiation therapy, and targeted therapies such as tyrosine kinase inhibitors. Regorafenib, a tyrosine kinase inhibitor, works by interfering with cancer cell growth and reducing the formation of blood vessels around tumors, which is crucial for tumor survival and metastasis. This mechanism is particularly important for ACC patients because it targets the pathways that contribute to the aggressive nature of the cancer, potentially improving outcomes by inhibiting tumor growth and spread.

Regorafenib Reverses Temozolomide-Induced CXCL12/CXCR4 Signaling and Triggers Apoptosis Mechanism in Glioblastoma.Regorafenib sensitizes human breast cancer cells to radiation by inhibiting multiple kinases and inducing DNA damage.Z-360 Suppresses Tumor Growth in MIA PaCa-2-bearing Mice via Inhibition of Gastrin-induced Anti-Apoptotic Effects.