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Study on the Safety and Effectiveness of Regorafenib for Patients with Arterio-Venous Malformations Unresponsive to Standard Treatment

Verified siteRegistered drugNo placebo

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What is this trial about?

A plain-language summary of the goals, design and what participants do

This clinical trial is focused on studying a condition known as Arterio-Venous Malformations (AVMs). AVMs are abnormal connections between arteries and veins, which can lead to various complications. The trial is investigating the use of a medication called Regorafenib, which is taken as a film-coated tablet. This medication is being tested to see if it can help patients whose AVMs have not responded to standard treatments like surgery or embolization.

The purpose of the study is to evaluate the safety and effectiveness of Regorafenib in managing symptoms caused by AVMs. Participants in the study will take the medication orally and will be monitored over a period of time to assess any changes in their condition. The study aims to determine if Regorafenib can improve symptoms such as pain and the overall quality of life for those affected by these complex vascular anomalies.

Throughout the study, participants will undergo various assessments, including questionnaires about their quality of life and evaluations of their symptoms, particularly focusing on pain. Additionally, the size of the AVM lesions will be measured using imaging techniques like MRI and other scans. The study will also explore how the medication affects participants' ability to perform daily activities. This trial is an important step in finding new ways to help those with AVMs that do not respond to existing treatments.

The research process

The trial runs in 6 steps – from screening to follow-up. Each step says what happens and what the team monitors.

Step 1
Joining the trial
Upon joining the trial, participants will be informed about the study's purpose, which is to evaluate the safety and effectiveness of regorafenib in treating arterio-venous malformations that do not respond to standard treatments.
Participants will be required to provide informed consent, confirming their understanding and willingness to participate in the trial.
Step 2
Initial assessment
An initial assessment will be conducted to ensure eligibility. This includes confirming that participants are at least 18 years old and have arterio-venous malformations that are not responsive to standard care.
Participants will undergo various tests to check blood counts, liver function, and kidney function to ensure they meet the necessary health criteria.
Step 3
Medication administration
Participants will begin taking regorafenib orally. The specific dosage, frequency, and duration of administration will be provided by the study team.
Participants will be monitored for any side effects or adverse reactions to the medication.
Step 4
Regular monitoring
Throughout the trial, participants will have regular check-ups to monitor their health and the effectiveness of the treatment.
These check-ups will include physical examinations, blood tests, and imaging studies such as MRI and ultrasound to assess the size of the malformations.
Step 5
Quality of life assessment
Participants will complete a quality of life questionnaire to evaluate any changes in symptoms, particularly pain, and overall well-being.
The questionnaire will help determine the impact of regorafenib on daily life and symptom management.
Step 6
End of trial evaluation
At the end of the trial, a final evaluation will be conducted to assess the overall effectiveness and safety of regorafenib.
Participants will receive a summary of their health status and any changes observed during the trial.

Who can join the trial?

14 criteria

Must be an adult aged at least 18 years old.
Must have complex and symptomatic fast-flow vascular malformations that do not respond to standard treatments like surgery or embolization, and have a life-threatening condition. Must experience pain that is not relieved by standard painkillers, including strong ones like morphine, with a pain score greater than 4 on a scale.
Must have adequate bone marrow function, which means:
- Hemoglobin (a protein in red blood cells) greater than 10.0 g/dl.
- Neutrophils (a type of white blood cell) greater than 1,500/mm³.
- Platelets (cells that help with blood clotting) greater than 100,000/mm³.
- Must have certain laboratory values within specific limits:
  - Total serum bilirubin (a substance made by the liver) less than or equal to 1.5 times the upper limit of normal (ULN), or less than or equal to 3 times ULN with direct bilirubin less than or equal to 1.5 times ULN if you have Gilbert Syndrome.
  - Serum alanine aminotransferase (ALT) and aspartate aminotransferase (AST) (liver enzymes) less than or equal to 3 times ULN, or less than 5 times ULN if liver metastases are present.
  - Serum creatinine (a waste product in the blood) less than 1.5 times ULN. If it is higher, a 24-hour creatinine clearance test must show a result of at least 60 mL/min.
  - Must have an ECOG score of 0-1, which means you are fully active or have some symptoms but do not need bed rest during the day.
  - Can have been previously treated with medications like sirolimus, alpelisib, trametinib, or thalidomide.
  - Must be able to swallow pills.
  - Women of childbearing potential must have a negative urine pregnancy test if they are sexually active.
  - Women of childbearing potential (participant or partner) must agree to use a highly effective contraceptive method during treatment and for 8 weeks after stopping the medication.
  - Men (participant or partner) must agree to use a condom during treatment and for 8 weeks after stopping the medication.

Who cannot join the trial?

10 criteria

Patients with other serious health conditions that could interfere with the study.
Patients who are pregnant or breastfeeding.
Patients who have had another type of cancer within the last 5 years, except for skin cancer that is not melanoma.
Patients who have had a heart attack or stroke within the last 6 months.
Patients who have uncontrolled high blood pressure.
Patients who have an active infection that requires treatment with antibiotics.
Patients who have a known allergy to the study medication or similar medications.
Patients who are currently participating in another clinical trial.
Patients who have a history of bleeding disorders.
Patients who have had major surgery within the last 4 weeks.

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Responsive
Cliniques Universitaires Saint-Luc
Woluwe-Saint-Lambert, Belgium

Trial locations

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Investigated drugs

Regorafenib is a medication used in this trial to help manage a condition called arterio-venous malformations (AVMs), which are abnormal connections between arteries and veins. These malformations can cause various symptoms and are sometimes difficult to treat with standard therapies. Regorafenib works by blocking certain proteins that help cancer cells grow and spread, and it is being tested to see if it can also reduce the symptoms and improve the condition of patients with AVMs that do not respond to usual treatments. The trial aims to confirm that regorafenib is safe for patients and to see if it can effectively alleviate the signs and symptoms associated with these complex vascular anomalies.

Regorafenib

What is already known about the treatment

Regorafenib – Regorafenib is a medication taken orally in the form of a tablet. It is currently being studied in clinical trials for its potential to treat Arterio-Venous Malformations (AVMs) that do not respond to standard treatments. The main goal of using regorafenib is to see if it can safely and effectively reduce the symptoms and complications caused by these complex blood vessel issues. At the molecular level, regorafenib works by blocking certain proteins that help cancer cells grow and spread, which may also help in managing AVMs. It is classified as a multi-kinase inhibitor, a type of drug that targets multiple pathways involved in disease progression.

Investigated diseases

Arteriovenous Malformations – Arteriovenous malformations (AVMs) are abnormal connections between arteries and veins, bypassing the capillary system. These vascular anomalies can occur in various parts of the body, including the brain, spine, and other organs. AVMs are often present at birth, although they may not cause symptoms until later in life. As they progress, AVMs can lead to a range of symptoms depending on their location, such as headaches, seizures, or neurological deficits if located in the brain. Over time, the abnormal blood flow through AVMs can cause the vessels to weaken and potentially rupture, leading to bleeding. The size and complexity of AVMs can vary, influencing the severity of symptoms and potential complications.