Metastatic pheochromocytoma / paraganglioma (MPP) are rare while the prognosis was poor. Penpulimab is specifically an immune check-point inhibitor of PD1 and has been approved for the treatment of several malignancies.This phase II trial studies the efficacy and safety of penpulimab in the treatment of MPP patients who fail to other systemic therapy.
This was a prospective observational study. Patients with histologically or radiologically confirmed MPP and fail to other systemic therapy were enrolled. Penpulimab will be administered intravenously at a dose of 200 mg every 3 weeks. Treatment continued until the patient exhibited radiographic or clinical disease progression or unacceptable adverse events.Plasma normetanephrine and metanephrine (MNs), 24-hour urinary catecholamine excretion (24hCA) were measured at baseline and every 1-3cycle. Contrast-enhanced computed tomography(CT) of chest, abdomen and pelvis were used to assess measurable target lesions at baseline and every 3 cycles. For patients who only had bone metastases or no measurable target lesions, The efficacy was evaluated by 18F-fluorodeoxyglucose (18F-FDG-PET/CT). The primary endpoint was objective response rate (ORR) and the disease control rate (DCR) per Response Evaluation Criteria In Solid Tumors(RECIST) 1.1/PERCIST1.0. Secondary endpoints included biochemical (catecholamine levels) response rate (BRR), progression-free survival (PFS) and safety.
Study Type
INTERVENTIONAL
Allocation
NA
Purpose
TREATMENT
Masking
NONE
Enrollment
5
Penpulimab will be administered intravenously at a dose of 200 mg every 3 weeks. Treatment continued until the patient exhibited radiographic or clinical disease progression or unacceptable adverse events.
Peking Union Medical College Hospital
Beijing, China
RECRUITINGThe objective response rate (ORR)
Defined for all patients whose tumor met the criteria of Complete Response (CR)and Partial Response (PR)
Time frame: At the end of Cycle 3(each cycle is 21 days)
The disease control rate (DCR)
Defined for all patients whose tumor met the criteria of CR or PR or stable disease(SD)
Time frame: At the end of Cycle 3(each cycle is 21 days)
progression-free survival (PFS)
PFS is defined as the time from the first day of treatment to the first documented disease progression per RECIST 1.1 criteria. Changes in only the largest diameter (unidimensional measurement) of the tumor lesions are used in the RECIST criteria.
Time frame: At least 1 cycle(each cycle is 21 days)
biochemical response
An effective response of 24hCA, MNs or NSE meant that the concentration decreased by more than 40% than the baseline value or decreased to the normal range
Time frame: At the end of Cycle 3 (each cycle is 21 days)
Incidence of adverse events
Incidence of adverse events assessed by Common Terminology Criteria for Adverse Events
Time frame: At the end of Cycle 1 (each cycle is 21 days)
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