von Hippel-Lindau (VHL) disease MedDRA version: 9.1 Level: LLT Classification code 10047716 Term: Von Hippel-Lindau disease
Conditions
Sponsors
ARTIC (Association pour la Recherche de Thérapeutiques Innovantes en Cancérologie)
Eligibility
Sex/Gender
All
Inclusion criteria
Inclusion criteria: 1. Patients must have genetically or clinically confirmed VHL disease and have symptoms from VHL that are no longer controllable by conventional approaches. 2. Patients must have at least one of the following lesions : • Eye : retinal hemangioblastoma that can no longer be treated by laser therapy or cryotherapy and resulting in progressive loss of vision; • CNS : cerebellar, bulbar, spinal, or cerebellopontine angle haemangioblastoma or endolymphatic sac tumor causing neurological symptoms that are not amenable to further surgery, or have recurred after a first surgery; • Kidney: multiple or bilateral tumors not accessible to conservative surgery, or tumors having recurred after surgery and/or radiofrequency ablation or advanced/metastatic RCC;. • Pancreas: inextirpable or advanced neuroendocrine tumors. 3. Patients previously treated for VHL with surgery, chemotherapy or radiotherapy are considered eligible for this study under the condition that these treatments were completed more than 4 weeks prior starting the study treatment. Previously radiated lesions will be considered as target lesions only if they demonstrate unequivocal evidence of growth upon imagery. 4. Male or female, at least 18 year-old. 5. Performance status ECOG 0-2 6. Life expectancy ? 3 months Are the trial subjects under 18? no Number of subjects for this age range: F.1.2 Adults (18-64 years) yes F.1.2.1 Number of subjects for this age range F.1.3 Elderly (>=65 years) yes F.1.3.1 Number of subjects for this age range
Exclusion criteria
Exclusion criteria: 1. Chemotherapy, radiotherapy, radiofrequency or surgery within 4 weeks prior to entering the study or not complete recovering from adverse events due to drugs administered more than 4 weeks earlier. 4. Previous treatment with sunitinib 5. NCI-CTCAE grade ? 3 hemorrhage within 4 weeks prior to study entry. 6. History of known or suspected brain metastases, spinal cord compression, carcinomatous meningitis, evidence of leptomeningeal disease (excepted leptomeningeal hemangioblastoma, according to the neurologist) on screening CT scan or MRI. 7. Any of the following within the 6 months prior to study drug administration: symptomatic congestive heart failure, myocardial infarction or coronary artery bypass, pulmonary embolism, ongoing severe or unstable angina pectoris, NCI-CTCAE grade ? 2 cardiac dysrhythmia, cerebrovascular accident or transient ischemic attack. 8. Hypertension >140/90 mmHg that cannot be controlled despite optimal antihypertensive therapy. 13. Pregnancy or breastfeeding. Patients must agree to use effective contraception during the study, including oral contraceptives, intrauterine devices, or being unable to procreate. 14. Any other malignancy within the last 3 years excepted basal cell carcinoma, in situ cervical carcinoma, squamous cell skin cancer, pT1/a bladder cancer with no evidence of recurrence during the last 12 months.
Design outcomes
Primary
| Measure | Time frame |
|---|---|
| Main Objective: To determine the objective response rate in VHL patients with advanced or untreatable tumors after initiation of treatment with sunitinib;Secondary Objective: 1) To evaluate the safety and tolerability of sunitinib in VHL patients according to the NCI-CTC criteria Version 3.0. 2) To determine time-to-event variables of survival, time to disease progression, time to response and duration of response. 3) To evaluate quality of life in VHL patients receiving sunitinib;Primary end point(s): Objective response rate (ORR) defined as the fraction of patients having received at least one dose of sunitinib with confirmed responses (complete or partial) on measurable lesion(s) according to RECIST criteria v1.0. | — |
Countries
France
Outcome results
None listed