Gastro-enteropancreatic Neuroendocrine Tumor
Conditions
Keywords
hypoglycemia
Brief summary
Pasireotide binds to somatostatin receptors sst2 and sst5, which can lead to significant hyperglycemia. The investigators would like to administer pasireotide as a treatment for refractory hypoglycemia in the setting of metastatic insulin-producing pancreatic neuro-endocrine tumor.
Interventions
DRUGPasireotide
Pasireotide will be used, in addition to diazoxide, as a medical treatment to blunt hypoglycemia in the setting of autonomous insulin secretion.
DRUGDiazoxide
Pasireotide will be used, in addition to diazoxide, as a medical treatment to blunt hypoglycemia in the setting of autonomous insulin secretion.
Sponsors
Novartis
University of Maryland, Baltimore
Study design
Allocation
NA
Intervention model
SINGLE_GROUP
Primary purpose
TREATMENT
Masking
NONE
Eligibility
Sex/Gender
MALE
Age
18 Years to 75 Years
Healthy volunteers
No
Inclusion criteria
1. Aged 18 years or older 2. Biopsy-proven (primary or metastatic lesion) metastatic neuroendocrine tumor of the gastrointestinal and pancreatic location with disease determined by CT scan or MRI 3. Patients with history of clinical syndrome symptoms (e.g. hypoglycemia) 4. Patients not controlled by treatment with currently available somatostatin analogues. 5. No evidence of significant liver disease: * Serum bilirubin ≤1.5 x ULN * INR \< 1.3 * ALT and AST ≤ 3x ULN, * Alkaline phosphatase ≤ 2.5 x ULN 6. Written informed consent obtained prior to treatment to be consistent with local regulatory requirements 7. Is suffering from a serious or life-threatening disease or condition 8. Does not have access to a comparable or satisfactory alternative treatment (i.e., comparable or satisfactory treatment is not available or does not exist) 9. Is not eligible for participation in any of the IMP's ongoing clinical trials or has recently completed a clinical trial that has been terminated and, after considering other options (e.g., trial extensions, amendments, etc.), the clinical team has determined that treatment is necessary and there are no other feasible alternatives for the patient 10. Meets any other relevant medical criteria for compassionate use of the investigational product 11. Is not being transferred from an ongoing clinical trial for which they are still eligible 12. There are meaningful human clinical data to support an assessment that the potential benefits to patient outweigh risks.
Exclusion criteria
1. Patients with a known hypersensitivity to somatostatin analogs or any component of the pasireotide LAR or s.c. formulations. 2. Patients with abnormal coagulation (PT or aPTT elevated by 30% above normal limits). 3. Patients on continuous anticoagulation therapy. Patients who were on anticoagulant therapy must complete a washout period of at least 10 days and have confirmed normal coagulation parameters before study inclusion. 4. Patients currently using warfarin / warfarin derivatives 5. Patients with symptomatic cholelithiasis. 6. Patients who are not biochemically euthyroid. Patients with known history of hypothyroidism are eligible if they are on adequate and stable replacement thyroid hormone therapy for at least 3 months. 7. QT-related
Design outcomes
Primary
| Measure | Time frame | Description |
|---|---|---|
| Hypoglycemia | up to 12 months | number of times glucose \< 70 mg/dl with and without symptoms |
Outcome results
None listed