Authorised Therapeutic exploratory (Phase II) Arterio-Venous Malformations

Monocentric Pilot Trial evaluating the safety and efficacy of Regorafenib in Arterio-Venous Malformations that are refractory to standard care

EU CTIS ID: 2022-501830-47-01

What this study is testing

To confirm the safety of regorafenib in patients with AVM and long-term tolerability; To evaluate the efficacy of regorafenib on signs and symptoms caused by AVMs that are refractory to standard care; To evaluate whether regorafenib treatment can alleviate signs and symptoms caused by these complex vascular anomalies that are refractory to standard care

  • Therapeutic exploratory (Phase II)

A plain-language read of the study's public EU CTIS listing. The study team confirms the details.

Who can take part

You may be able to join if

  • Inclusion is limited to adults aged at least 18 years old: 1. Patients with complex and symptomatic fast-flow vascular malformations that are refractory to standard care (surgery and/or embolization) with progression and life-threatening disease (Shobinger classification III et IV). They have to present pain as the predominant complaint with Visual Analogic Scale (VAS>4), not alleviated with standard painkiller, including morphinic derivatives. 2. Patients must have adequate bone marrow function: hemoglobin> 10,0 g/dl, neutrophils >1.500/mm³ and platelets > 100.000/mm³. 3. Patients must have the following laboratory values: • Total serum bilirubin ≤ 1.5 x ULN (or totally bilirubin ≤3 x ULN with direct bilirubin ≤ 1.5 x ULN in patients with well documented Gilbert Syndrome) • Serum alanine aminotransferase (ALT) and aspartate aminotransferase (AST) ≤ 3 x ULN (or < 5.0 x ULN if hepatic metastases are present) • Serum creatinine < 1.5 x ULN. If the serum creatinine is ≥ 1.5 x ULN, then a 24-hour Creatinine Clearance must be conducted and the result must be ≥ 60 mL/min. 4. ECOG score 0-1 5. Patients previously treated with sirolimus, alpelisib, trametinib or thalidomide could be included 6. Patients able to swallow. 7. Negative urine pregnancy test in women of childbearing potential (WOCBP) with sexual relations. 8. WOCBP (participant or non-participant partner) agree to use highly effective contraceptive method during treatment and for 8 weeks after stopping regorafenib. 9.Male (participant or non-participant partner) agree to use mandatory condom during the treatment duration and for 8 weeks after stopping regorafenib

You likely can't join if

  • As AVM can result in impaired cardiac function, congestive heart failure is allowed if stabilized. 1. New York Heart Association functional classification Grade 3-4 congestive heart failure should be excluded from this study, but remains at the discretion of the investigator as regorafenib could indirectly improve cardiac function by controlling AVM. Other significant cardiac diseases, including unstable angina pectoris, ventricular arrhythmia, valvular disease with documented compromise in cardiac function, myocardial infarction within the last 6 months, documented by persistent elevated cardiac enzymes or persistent regional wall abnormalities on assessment of LVEF function, family history of congenital long or short QT, or known history of QT/QTc prolongation of Torsades de Pointes (TdP) are excluded from this trial. 2. Impairment of Gastro-Intestinal (GI) function or GI disease that may significantly alter the absorption of regorafenib (e.g., ulcerative diseases, uncontrolled nausea, vomiting, diarrhea ≥ Grade 2, malabsorption syndrome, or small bowel resection) 3. Known hypersensitivity to drugs or metabolites from similar classes as study treatment. 4. Patient has (an)other concurrent severe and /or uncontrolled medical condition(s) that would, in the investigator’s judgement, contraindicate participation in the clinical study (e.g. acute or chronic pancreatitis, liver cirrhosis, active chronic hepatitis, severely impaired lung function with a spirometry ≤ 50% of the normal predicted value and/or O2 saturation ≤ 88% at rest, etc.) 5. Immunocompromised patients, including known seropositivity for HIV 6. Pregnant or lactating women

The study team makes the final eligibility decision.

Questions & answers

Do participants get paid in this trial?

This listing doesn't specify compensation. Many trials still reimburse travel or offer a stipend, so it's worth asking the study team when you connect.

Is it free to join, and do I need insurance?

Searching and applying through BridgeMD is free. In clinical trials the study-related treatment and visits are generally provided at no cost to you. You usually don't need insurance to take part - confirm specifics with the study team.

How long does this study last?

The listing doesn't state an exact length. The study team walks you through the schedule and number of visits before you decide to enroll.

Who can join this trial?

This study is enrolling all sexes, 18-64 years, 65+ years. The study team makes the final eligibility decision.

Explore other conditions

BridgeMD is an information and trial-matching tool - not medical advice, and not the study sponsor. Details come from EU CTIS; the study team decides eligibility.