Skip to main content
OpenTrials
Completed

NCT Number: NCT05858177

The Efficacy and Safety of Temozolomide in Patients With MPPGL

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.

Completed

Looking for future studies?

Notify Me

Key information

Conditions

Pheochromocytoma, Metastatic Neoplasm Metastasis Neoplasms Neoplasms by Histologic Type Neoplasms, Germ Cell and Embryonal Neoplasms, Nerve Tissue Neoplastic Processes Neuroectodermal Tumors Neuroendocrine Tumors Paraganglioma Paraganglioma, Extra-Adrenal Paraganglioma, Malignant Pathologic Processes Pathological Conditions, Signs and Symptoms Pheochromocytoma Pheochromocytoma Malignant

Age range

18 year–75 year

Sex eligibility

All sexes

Study type

Interventional

Phase

Phase 2

Primary location

Peking Union Medical College Hospital

Beijing, Beijing Municipality, 100730, China

About this study

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.

Who can participate

Healthy volunteers accepted: No

Only the study team can determine whether someone qualifies for participation.

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.

Treatment and study plan

Temozolomide capsule

Drug

Temozolomide was given orally per day for 5 days, every 28 days until disease progression or intolerable toxicities.

Other names: Temozolomide

Primary outcomes

  1. 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)

  2. 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)

Secondary outcomes

  1. 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.

  2. 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

  3. 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

Sponsors and collaborators

Lead sponsor

Peking Union Medical College Hospital

Other

Registry information

Official study title

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

Acronym: MPPGL

Important dates

Study start
2019
Primary completion
2022
Study completion
2023
First posted
May 15, 2023
Registry last updated
May 15, 2023

OpenTrials presents study information sourced from ClinicalTrials.gov. The official registry record should be consulted for the latest information.

View the official ClinicalTrials.gov record (opens in a new tab)

This listing is for discovery and informational purposes only. It is not medical advice, does not guarantee that a study is recruiting, and does not determine eligibility. Contact the study team and a qualified healthcare professional when considering participation.

Related clinical trials

Published trials that share one or more normalized conditions with this study.