The objectives of this study are: * To assess the efficacy of lanreotide given every 4 weeks in participants with advanced or metastatic paraganglioma/ pheochromocytoma. * To assess the toxicity and safety of lanreotide in participants with advanced or metastatic paraganglioma/ pheochromocytoma. * To document the effects of lanreotide on markers of biochemical activity in participants with advanced or metastatic paraganglioma/ pheochromocytoma. Primary endpoints: • Assess efficacy by estimating the tumor growth rate while a patient is enrolled on study and comparing the growth rates on lanreotide to the pre-enrolment growth rate. Secondary endpoints include measurement of: * Overall survival (OS) * Progression-free survival (PFS) * Overall response rate (ORR) according to RECIST defined as partial response (PR) + complete response (CR) * Magnitude of reduction in levels of 24-hour urinary metanephrines, catecholamines and magnitude of reduction in serum chromogranin A, evaluated every two months while enrolled on study.
Lanreotide is FDA approved for certain kinds of neuroendocrine tumors. This study seeks to determine if lanreotide is beneficial for patients with paraganglioma/ pheochromocytoma. Given the rarity of pheochromocytoma/paraganglioma that precludes the conduct of a randomized clinical trial in a timely manner, a novel method for assessing efficacy is being proposed. Efficacy will be assessed by estimating the tumor growth rate while a patient is enrolled on study and comparing the growth rates on lanreotide to the pre-enrollment growth rates. The method of analysis that will be used has been previously described. For this assessment a minimum of three tumor measurements will be required.
Study Type
INTERVENTIONAL
Allocation
NA
Purpose
TREATMENT
Masking
NONE
Enrollment
10
Participants will receive lanreotide 120 mg deep subcutaneous injection every 4 weeks (±7 days) for 52 weeks, followed by an extension phase in which all patients will continue to receive lanreotide 120 mg injection every 4 weeks (±7 days) if there is no evidence of disease progression.
Columbia University Irving Medical Center
New York, New York, United States
Cleveland Clinic
Cleveland, Ohio, United States
Rate of tumor growth
Efficacy will be assessed by measuring the tumor growth rate while a patient is enrolled on study and comparing the growth rates on lanreotide to the pre-enrollment growth rates. Tumor growth measured by a CT or MRI scan in pre-treatment, and minimum of three scans (prior to every 3rd visit, or every 12 weeks) in post-treatment.
Time frame: minimum of 32 weeks, up to 48 weeks
Overall survival (OS)
The length of time from the start of treatment that subjects with the disease are still alive.
Time frame: Observed for 48 weeks (start of treatment to end of treatment).
Overall Response Rate (ORR)
ORR is defined as the proportion of the subjects in the analysis population who have a complete response (CR) or partial response (PR). Responses are based on assessments per RECIST 1.1. Changes in only the largest diameter (unidimensional measurement) of the tumor lesions are used in the RECIST criteria. * Complete Response (CR): Disappearance of all target lesions. Any pathological lymph nodes (whether target or non-target) must have change in short axis to less than 10 mm. * Partial Response (PR): At least a 30% change in the sum of diameters of target lesions, taking as reference the baseline sum diameters.
Time frame: Minimum of 8 weeks, up to 48 weeks.
Progression-free survival
PFS is defined as the time from the first day of treatment to the first documented disease progression per RECIST 1.1 criteria and the Kaplan-Meier curve. Changes in only the largest diameter (unidimensional measurement) of the tumor lesions are used in the RECIST criteria.
Time frame: Up to 48 weeks.
Magnitude of change in analyte levels
Biochemical response of greater than 20% change in 24-hour urinary metanephrines, catecholamines and serum chromogranin A levels compared to baseline, sustained for \>12-week-period. Evaluated every two months while enrolled on study, although levels may be obtained as frequently as the investigator desires.
Time frame: Minimum of 16 weeks, up to 48 weeks.
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