2022-501830-47-01招募中2 期
Monocentric Pilot Trial evaluating the safety and efficacy of Regorafenib in Arterio-Venous Malformations that are refractory to standard care
试验速览
- 阶段
- 2 期
- 状态
- 招募中
- 入组人数
- 10
- 试验地点
- 1
- 主要终点
- The efficacy assessment of regorafenib will be based on these following criteria : 1) Quality of life questionnaire adapted from OVAMA questionary with self-perception of improved quality of life (in %) 2) Evaluation of symptoms focused on pain. 3) Size of the lesion on the basis of clinical evaluation and the 12-month doppler ultrasonography, MRI and arteriography.
研究概览
简要总结
*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
入排标准
- 年龄范围
- 18 years 至 65+ years（18-64 Years, 65+ Years）
- 接受健康志愿者
- 是
入选标准
- •Inclusion is limited to adults aged at least 18 years old:
- •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.
- •Patients must have adequate bone marrow function: hemoglobin> 10,0 g/dl, neutrophils >1.500/mm³ and platelets > 100.000/mm³.
- •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.
- •ECOG score 0-1
- •Patients previously treated with sirolimus, alpelisib, trametinib or thalidomide could be included
- •Patients able to swallow.
- •Negative urine pregnancy test in women of childbearing potential (WOCBP) with sexual relations.
- •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
排除标准
- •As AVM can result in impaired cardiac function, congestive heart failure is allowed if stabilized.
- •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.
- •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)
- •Known hypersensitivity to drugs or metabolites from similar classes as study treatment.
- •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.)
- •Immunocompromised patients, including known seropositivity for HIV
- •Pregnant or lactating women
结局指标
主要结局
The efficacy assessment of regorafenib will be based on these following criteria : 1) Quality of life questionnaire adapted from OVAMA questionary with self-perception of improved quality of life (in %) 2) Evaluation of symptoms focused on pain. 3) Size of the lesion on the basis of clinical evaluation and the 12-month doppler ultrasonography, MRI and arteriography.
The efficacy assessment of regorafenib will be based on these following criteria : 1) Quality of life questionnaire adapted from OVAMA questionary with self-perception of improved quality of life (in %) 2) Evaluation of symptoms focused on pain. 3) Size of the lesion on the basis of clinical evaluation and the 12-month doppler ultrasonography, MRI and arteriography.
次要结局
- A secondary endpoint will explore the efficacy of regorafenib based on Functional limitation. The evaluation of functional limitation (difficulties in performing any action of everyday life) based on a scale ranging from 0 (no limitation) to 10 (excessive limitation).
研究者
Dr Emmanuel Seront
Scientific
Cliniques Universitaires Saint-Luc
研究点 (1)
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