Von Hippel-Lindau (VHL) Disease
Conditions
Interventions
1:axitinib
Sponsors
Peking University First Hospital
Eligibility
Sex/Gender
All
Age
18 Years to 65 Years
Inclusion criteria
Inclusion criteria: 1. Subjects must provide written informed consent prior to performance of study-specific procedures or assessments, and must be willing to comply with treatment and follow up. Procedures conducted as part of the subject's routine clinical management (e.g., blood count, imaging study) and obtained prior to signing of informed consent may be utilized for screening or baseline purposes provided these procedures are conducted as specified in the protocol; 2. Genetically confirmed diagnosis of VHL or measurable disease consistent with the clinical diagnosis of VHL; 3. Measurable disease criteria: At least one measurable VHL related lesion, which is undergoing surveillance, and patient is not at immediate risk of needing intervention for this or other lesions. Biopsy is not required given the known likely etiology and natural history in the setting of a positive genetic test: 1) Brain: asymptomatic hemangioblastoma, >/= 0.5 cm; 2) Spine: asymptomatic hemangioblastoma, >/= 0.5 cm ; 3) Renal: solid mass suspicious for RCC >/= 1 cm or cystic mass (Bosniak 3-4) >/= 1 cm; 4) Pancreas: solid mass >/= 1cm and 3 cm but not considered operable; 5) Eye: asymptomatic peripapillary and/or macular hemangioblastoma, any size; 6) Adrenal: asymptomatic or controlled pheochromocytoma greater than 1cm in size. 4. Patients may have received prior VHL-related systemic therapy, provided not within 14 days or five half-lives of a drug (whichever is longer) prior to the first dose of axitinib.
Exclusion criteria
Exclusion criteria: 1. Prior malignancy. Note: Subjects who have had another non VHL related malignancy and have been disease-free for 2 years, or subjects with a history of completely resected non-melanomatous skin carcinoma are eligible. 2. Clinically significant gastrointestinal abnormalities that may increase the risk for gastrointestinal bleeding including, but not limited to: 1) Active peptic ulcer disease; 2) Known intraluminal metastatic lesion/s with risk of bleeding; 3) Inflammatory bowel disease (e.g. ulcerative colitis, Chrohn's disease), or other gastrointestinal conditions with increased risk of perforation; 4) History of abdominal fistula, gastrointestinal perforation, or intra abdominal abscess within 28 days prior to beginning study treatment. 3. Clinically significant gastrointestinal abnormalities that may affect absorption of investigational product including, but not limited to: 1) Malabsorption syndrome; 2) Major resection of the stomach or small bowel; 4. Presence of uncontrolled infection; 5. History of any one or more of the following cardiovascular conditions within the past 6 months: 1) Cardiac angioplasty or stenting; 2) Myocardial infarction; 3) Unstable angina; 4) Coronary artery bypass graft surgery; 5) Symptomatic peripheral vascular disease; 6) Class III or IV congestive heart failure, as defined by the New York Heart Association (NYHA); 6. Poorly controlled hypertension [defined as systolic blood pressure (SBP) of >/= 140 mmHg or diastolic blood pressure (DBP) of >/= 90mmHg]; 7. History of cerebrovascular accident including transient ischemic attack (TIA), pulmonary embolism or untreated deep venous thrombosis (DVT) within the past 6 months.
Design outcomes
Primary
| Measure | Time frame |
|---|---|
| availability; | — |
Countries
China
Contacts
Public ContactKan Gong
Peking University First Hospital
Outcome results
None listed