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The Efficacy and Safety of Penpulimab in the Treatment of Metastatic PPGL Patients Who Fail to Other Systemic Treatment

Primary Purpose

Pheochromocytoma, Metastatic, Pheochromocytoma Malignant, Paraganglioma, Malignant

Status
Recruiting
Phase
Phase 2
Locations
China
Study Type
Interventional
Intervention
Penpulimab
Sponsored by
Peking Union Medical College Hospital
About
Eligibility
Locations
Arms
Outcomes
Full info

About this trial

This is an interventional treatment trial for Pheochromocytoma, Metastatic

Eligibility Criteria

15 Years - 75 Years (Child, Adult, Older Adult)All SexesDoes not accept healthy volunteers

Inclusion Criteria: Provide written informed consent. Age 18-75 years old Eastern Cooperative Oncology Group (ECOG) performance status 0, 1, or 2. Patients with histologically or radiologically confirmed MPP and fail to other systemic therapy. Estimated life expectancy longer than 6 months. Confirmed non-pregnancy and lactation. During the entire study period and within 6 months after the last administration, the subjects and their spouses are willing to use efficient contraceptive measures. Laboratory requirements: Absolute granulocyte count (AGC) greater than 1.5 x 109/L; Platelet count greater than 80 x 109/L; Hemoglobin greater than 90g/L; Serum bilirubin less than 1.5 x upper limit of normal (ULN); --)Serum aspartate aminotransferase (AST) and alanine aminotransferase (ALT) less than 2.5 x ULN; Serum creatinine less than 1.5 x ULN or creatinine clearance (CCr)≥60ml/min; Doppler ultrasound assessment: left ventricular ejection fraction (LVEF) ≥ lower limit of normal value (50%). Exclusion Criteria: Patients who had been previously treated with anti-PD1, anti-PD-L1, or anti-PD-L2 medications were excluded from this trial. Has a diagnosis of immunodeficiency or is receiving systemic steroid therapy or any other form of immunosuppressive therapy within 14 days prior to the first dose of trial treatment. Has a known history of active TB (Bacillus Tuberculosis). Has a known history of Human Immunodeficiency Virus (HIV). Has known active Hepatitis B (e.g., HBsAg reactive) or Hepatitis C (e.g., HCV RNA [qualitative] is detected). Has an active infection requiring systemic therapy. Didn't meet eligibility for organ function. Abnormal coagulation (INR >1.5 or prothrombin time (PT) > ULN 4 seconds or APTT >1.5 ULN), bleeding tendency or being treated with thrombolytic or anticoagulant therapy. Uncontrolled congestive heart failure .

Sites / Locations

  • Peking Union Medical College HospitalRecruiting

Arms of the Study

Arm 1

Arm Type

Experimental

Arm Label

penpulimab

Arm Description

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.

Outcomes

Primary Outcome Measures

The objective response rate (ORR)
Defined for all patients whose tumor met the criteria of Complete Response (CR)and Partial Response (PR)
The disease control rate (DCR)
Defined for all patients whose tumor met the criteria of CR or PR or stable disease(SD)

Secondary Outcome Measures

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.
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
Incidence of adverse events
Incidence of adverse events assessed by Common Terminology Criteria for Adverse Events

Full Information

First Posted
May 22, 2023
Last Updated
May 31, 2023
Sponsor
Peking Union Medical College Hospital
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1. Study Identification

Unique Protocol Identification Number
NCT05885399
Brief Title
The Efficacy and Safety of Penpulimab in the Treatment of Metastatic PPGL Patients Who Fail to Other Systemic Treatment
Official Title
A Study on the Efficacy and Safety of Penpulimab in the Treatment of Metastatic Pheochromocytoma/Paraganglioma Patients Who Fail to Other Systemic Treatment
Study Type
Interventional

2. Study Status

Record Verification Date
May 2023
Overall Recruitment Status
Recruiting
Study Start Date
April 1, 2023 (Actual)
Primary Completion Date
March 1, 2025 (Anticipated)
Study Completion Date
March 1, 2025 (Anticipated)

3. Sponsor/Collaborators

Responsible Party, by Official Title
Sponsor
Name of the Sponsor
Peking Union Medical College Hospital

4. Oversight

Studies a U.S. FDA-regulated Drug Product
No
Studies a U.S. FDA-regulated Device Product
No

5. Study Description

Brief Summary
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.
Detailed Description
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.

6. Conditions and Keywords

Primary Disease or Condition Being Studied in the Trial, or the Focus of the Study
Pheochromocytoma, Metastatic, Pheochromocytoma Malignant, Paraganglioma, Malignant

7. Study Design

Primary Purpose
Treatment
Study Phase
Phase 2
Interventional Study Model
Single Group Assignment
Masking
None (Open Label)
Allocation
N/A
Enrollment
5 (Anticipated)

8. Arms, Groups, and Interventions

Arm Title
penpulimab
Arm Type
Experimental
Arm Description
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.
Intervention Type
Drug
Intervention Name(s)
Penpulimab
Intervention Description
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.
Primary Outcome Measure Information:
Title
The objective response rate (ORR)
Description
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)
Title
The disease control rate (DCR)
Description
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)
Secondary Outcome Measure Information:
Title
progression-free survival (PFS)
Description
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)
Title
biochemical response
Description
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)
Title
Incidence of adverse events
Description
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)

10. Eligibility

Sex
All
Minimum Age & Unit of Time
15 Years
Maximum Age & Unit of Time
75 Years
Accepts Healthy Volunteers
No
Eligibility Criteria
Inclusion Criteria: Provide written informed consent. Age 18-75 years old Eastern Cooperative Oncology Group (ECOG) performance status 0, 1, or 2. Patients with histologically or radiologically confirmed MPP and fail to other systemic therapy. Estimated life expectancy longer than 6 months. Confirmed non-pregnancy and lactation. During the entire study period and within 6 months after the last administration, the subjects and their spouses are willing to use efficient contraceptive measures. Laboratory requirements: Absolute granulocyte count (AGC) greater than 1.5 x 109/L; Platelet count greater than 80 x 109/L; Hemoglobin greater than 90g/L; Serum bilirubin less than 1.5 x upper limit of normal (ULN); --)Serum aspartate aminotransferase (AST) and alanine aminotransferase (ALT) less than 2.5 x ULN; Serum creatinine less than 1.5 x ULN or creatinine clearance (CCr)≥60ml/min; Doppler ultrasound assessment: left ventricular ejection fraction (LVEF) ≥ lower limit of normal value (50%). Exclusion Criteria: Patients who had been previously treated with anti-PD1, anti-PD-L1, or anti-PD-L2 medications were excluded from this trial. Has a diagnosis of immunodeficiency or is receiving systemic steroid therapy or any other form of immunosuppressive therapy within 14 days prior to the first dose of trial treatment. Has a known history of active TB (Bacillus Tuberculosis). Has a known history of Human Immunodeficiency Virus (HIV). Has known active Hepatitis B (e.g., HBsAg reactive) or Hepatitis C (e.g., HCV RNA [qualitative] is detected). Has an active infection requiring systemic therapy. Didn't meet eligibility for organ function. Abnormal coagulation (INR >1.5 or prothrombin time (PT) > ULN 4 seconds or APTT >1.5 ULN), bleeding tendency or being treated with thrombolytic or anticoagulant therapy. Uncontrolled congestive heart failure .
Central Contact Person:
First Name & Middle Initial & Last Name or Official Title & Degree
Anli Tong
Phone
13911413589
Email
tonganli@hotmail.com
Overall Study Officials:
First Name & Middle Initial & Last Name & Degree
Anli Tong
Organizational Affiliation
Peking Union Medical College Hospital
Official's Role
Principal Investigator
Facility Information:
Facility Name
Peking Union Medical College Hospital
City
Beijing
Country
China
Individual Site Status
Recruiting
Facility Contact:
First Name & Middle Initial & Last Name & Degree
Anli Tong
Phone
13911413589
Email
tonganli@hotmail.com

12. IPD Sharing Statement

Learn more about this trial

The Efficacy and Safety of Penpulimab in the Treatment of Metastatic PPGL Patients Who Fail to Other Systemic Treatment

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