The Efficacy and Safety of Temozolomide in Patients With MPPGL (MPPGL)

May 4, 2023 updated by: Peking Union Medical College Hospital

The Efficacy and Safety of Temozolomide in Patients With Metastatic Pheochromocytoma or Paraganglioma

Metastatic pheochromocytoma / paraganglioma (MPP) are rare while the prognosis was poor. Temozolomide (TMZ) is a novel oral alkylation chemotherapeutic agent. TMZ has been recommended in National Comprehensive Cancer Network (NCCN) Guidelines Version 1.2019 for treating MPP patients.However, studies investigating TMZ efficacy in MPP patients are extremely limited. The largest study involved only 15 patients till date. The safety and efficacy of TMZ treatment in MPP patients need to be verified in larger studies.

Study Overview

Status

Completed

Conditions

Intervention / Treatment

Detailed Description

Patients with histologically or radiologically confirmed MPP were enrolled. TMZ was administered orally at an initial daily dose of 150 mg/m2 per day for 5 days, every 28 days. In patients with a good tolerance during the first cycle, the dose was increased to 200 mg/m2 per day for 5 days, every 28 days. Plasma normetanephrine and metanephrine (MNs), 24-hour urinary catecholamine excretion (24hCA) and neuron specific enolase (NSE) were measured at baseline and every 1-3 cycle. 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, TMZ efficacy was evaluated by 18F-fluorodeoxyglucose (18F-FDG-PET/CT). The primary endpoint was objective response rate (ORR) 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. The investigators will try to explore which patients are more suitable for TMZ treatment.

Study Type

Interventional

Enrollment (Actual)

62

Phase

  • Phase 2

Contacts and Locations

This section provides the contact details for those conducting the study, and information on where this study is being conducted.

Study Locations

  • China
    • Beijing
      • Beijing, Beijing, China, 100730
        • Peking Union Medical College Hospital

Participation Criteria

Researchers look for people who fit a certain description, called eligibility criteria. Some examples of these criteria are a person's general health condition or prior treatments.

Eligibility Criteria

Ages Eligible for Study

  • Adult
  • Older Adult

Accepts Healthy Volunteers

No

Description

Inclusion Criteria:

Patients with metastatic pheochromocytomas and paragangliomas. Subjects with Eastern Cooperative Oncology Group (ECOG) performance status of 0, 1 or 2.

Estimated life expectancy longer than 6 months. Having normal organ function as defined by hemoglobin levels ≥10 g/dL, absolute neutrophil count ≥ 1.5 x 109/L, platelet count ≥ 80 x 109/L, total bilirubin ≤1.5 institutional upper limit, aspartate aminotransferase ≤5 institutional upper limit, alanine aminotransferase ≤5 institutional upper limit, and serum creatinine <3.0 mg/dL.

Exclusion Criteria:

Didn't meet eligibility for organ function. Pregnancy or breastfeeding. Uncontrolled congestive heart failure and severe infection.

Study Plan

This section provides details of the study plan, including how the study is designed and what the study is measuring.

How is the study designed?

Design Details

  • Primary Purpose: Treatment
  • Allocation: N/A
  • Interventional Model: Single Group Assignment
  • Masking: None (Open Label)

Arms and Interventions

Participant Group / Arm
Intervention / Treatment
Other: Temozolomide
TMZ was given orally per day for 5 days, every 28 days until disease progression or intolerable toxicities.
Drug: Temozolomide capsule
Temozolomide was given orally per day for 5 days, every 28 days until disease progression or intolerable toxicities.
Other Names:
  • Temozolomide

What is the study measuring?

Primary Outcome Measures

Outcome Measure
Measure Description
Time Frame
disease control rate (DCR)
Time Frame: At the end of Cycle 3 (each cycle is 28 days)
Defined for all patients whose tumor met the criteria of CR or PR or stable disease(SD)
At the end of Cycle 3 (each cycle is 28 days)
objective response rate (ORR)
Time Frame: At the end of Cycle 3 (each cycle is 28 days)
Defined for all patients whose tumor met the criteria of Complete Response (CR)and Partial Response (PR)
At the end of Cycle 3 (each cycle is 28 days)

Secondary Outcome Measures

Outcome Measure
Measure Description
Time Frame
progression-free survival (PFS)
Time Frame: At least 1 cycle(each cycle is 28 days), up to 100 weeks,From date of randomization until the date of first documented progression or date of death from any cause, whichever came first, assessed up to 100 months
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.
At least 1 cycle(each cycle is 28 days), up to 100 weeks,From date of randomization until the date of first documented progression or date of death from any cause, whichever came first, assessed up to 100 months
biochemical response
Time Frame: At the end of Cycle 1 (each cycle is 28 days)
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
At the end of Cycle 1 (each cycle is 28 days)
Incidence of adverse events
Time Frame: At the end of Cycle 1 (each cycle is 28 days)
Incidence of adverse events assessed by Common Terminology Criteria for Adverse Events
At the end of Cycle 1 (each cycle is 28 days)

Collaborators and Investigators

This is where you will find people and organizations involved with this study.

Sponsor

Peking Union Medical College Hospital

Investigators

  • Principal Investigator: Anli Tong, Peking Union Medical College Hospital

Study record dates

These dates track the progress of study record and summary results submissions to ClinicalTrials.gov. Study records and reported results are reviewed by the National Library of Medicine (NLM) to make sure they meet specific quality control standards before being posted on the public website.

Study Major Dates

Study Start (Actual)

October 1, 2019

Primary Completion (Actual)

October 1, 2022

Study Completion (Actual)

January 1, 2023

Study Registration Dates

First Submitted

April 18, 2023

First Submitted That Met QC Criteria

May 4, 2023

First Posted (Actual)

May 15, 2023

Study Record Updates

Last Update Posted (Actual)

May 15, 2023

Last Update Submitted That Met QC Criteria

May 4, 2023

Last Verified

April 1, 2023

More Information

Terms related to this study

Keywords

Additional Relevant MeSH Terms

Other Study ID Numbers

  • 06086-03

Plan for Individual participant data (IPD)

Plan to Share Individual Participant Data (IPD)?

NO

Drug and device information, study documents

Studies a U.S. FDA-regulated drug product

No

Studies a U.S. FDA-regulated device product

No

This information was retrieved directly from the website clinicaltrials.gov without any changes. If you have any requests to change, remove or update your study details, please contact register@clinicaltrials.gov. As soon as a change is implemented on clinicaltrials.gov, this will be updated automatically on our website as well.

Clinical Trials on Pheochromocytoma, Metastatic

Clinical Trials on Temozolomide capsule

Search Similar Trials

Sponsors and Collaborators

Medical Conditions

Drug Interventions

CROs by country

CROs in Azerbaijan

Conditions

Rare Diseases

Drug Interventions

Dietary Supplements

Sponsor/Collaborators

Locations

Subscribe