Neoplasms

All Conditions

Rare Diseases

Pheochromocytoma

Paraganglioma

Neuroendocrine Tumors

Neuroectodermal Tumors

Neuroectodermal Tumors, Primitive

Neoplasms, Germ Cell and Embryonal

Neoplasms by Histologic Type

Neoplasms, Nerve Tissue

Paragangliomas 1

Neuroendocrine Tumor

Neuroepithelioma

to evaluate the efficiency of contrast enhanced ultrasound in assessing effectiveness of anlotinib in patients with locally advanced, metastatic, or unresectable pheochromocytoma or paraganglioma(PPGL).

CEUS is a quantitative kinetic imaging modality that can assess intravascular blood flow in PPGL tumors.

Contrast-enhanced ultrasound(CEUS)

Anli Tong

Anlotinib is a multi-target receptor tyrosine kinase inhibitor (TKI) targeting tumor angiogenesis and growth. The purpose of this study is to evaluate the efficiency of contrast enhanced ultrasound in assessing effectiveness of anlotinib in patients with locally advanced, metastatic, or unresectable pheochromocytoma or paraganglioma(PPGL).

Contrast-enhanced ultrasound (CEUS) is a non-invasive and efficient imaging technique which can observe the blood flow at the tissue perfusion level with microbubble contrast agents and obtain information about tumor perfusion. There have been several studies demonstrating the utility of CEUS for early prediction of response to neoadjuvant chemotherapy in breast cancer, pancrearic cancer as well as lymphoma. However, few studies have reported the use of CEUS for the evaluation of therapeutic response in PPGL. What quantitative parameters of CEUS can early reflect the neovascular changes after treatment with anlotinib in patients with locally advanced, metastatic, or unresectable PPGL? The aim of our study is to evaluate the parameters for CEUS imaging and the therapeutic response of PPGL before and after anlotinib therapy and to determine the most useful CEUS response parameters.

Prediction and Evaluation of Anlotinib Treatment Response Using Contrast Enhanced Ultrasound PPGL Patients

Prediction and Evaluation of Anlotinib Treatment Response Using Contrast Enhanced Ultrasound in Patients With Pheochromocytoma or Paraganglioma

Before initiation of the therapy and during anlotinib therapy, patients would receive CEUS examination. The dynamic CEUS images of each lesion were analysed with the aid of time intensity curves (TICs) drawn by QLAB software (Philips Healthcare, Andover, MA). PI measurements will be compared to clinical measurement of response to treatment taking into consideration standard response criteria including radiological imaging, biochemical (catecholamine levels) response.

Before initiation of the therapy and during anlotinib therapy, patients would receive CEUS examination. The dynamic CEUS images of each lesion were analysed with the aid of time intensity curves (TICs) drawn by QLAB software (Philips Healthcare, Andover, MA). TTP measurements will be compared to clinical measurement of response to treatment taking into consideration standard response criteria.

Peking Union Medical College Hospital

Symptoms and General Pathology

Neoplasm Metastasis

Antineoplastic Agents

All Drugs and Chemicals

Temozolomide

Antineoplastic Agents, Alkylating

Alkylating Agents

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

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

Temozolomide capsule

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.

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.

The Efficacy and Safety of Temozolomide in Patients With MPPGL

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

Defined for all patients whose tumor met the criteria of CR or PR or stable disease(SD)

Defined for all patients whose tumor met the criteria of Complete Response (CR)and Partial Response (PR)

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.

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 assessed by Common Terminology Criteria for Adverse Events

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.

Penpulimab

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.

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

A Study on the Efficacy and Safety of Penpulimab in the Treatment of Metastatic Pheochromocytoma/Paraganglioma Patients Who Fail to Other Systemic Treatment

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.

Paraganglioma, Extra-Adrenal

Patients receive anlotinib hydrochloride 12mg orally once daily on days 1-14. Courses repeat every 21 days in the absence of disease progression or unacceptable toxicity.

anlotinib hydrochloride

anli tong

This phase II trial studies how well anlotinib hydrochloride works in treating patients with metastatic pheochromocytoma or paraganglioma. Anlotinib hydrochloride may stop the growth of tumor cells by blocking some of the enzymes needed for cell growth and by blocking blood flow to the tumor.

PRIMARY OBJECTIVES:

To determine the anti-tumor activity of anlotinib hydrochloride (Objective Response rate,ORR) in patients with metastatic pheochromocytomas or paragangliomas.

SECONDARY OBJECTIVES:

I. To assess safety profile of anlotinib. II. To assess progression-free survival time. III. To assess disease control rate.

OUTLINE:

Patients receive anlotinib hydrochloride 12mg orally once daily on days 1-14. Courses repeat every 21 days in the absence of disease progression or unacceptable toxicity.

Patients undergo urine and blood sample collection, imaging examinations at baseline and periodically during study.

After completion of study therapy, patients are followed up every 3-6 months.

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

A Phase 2 Study of Anlotinib in Patients With Metastatic Pheochromocytoma or Paraganglioma

Incidence of adverse events assessed by Common Terminology Criteria for Adverse Events version 4.0

Study title	NCT ID	Conditions	Interventions	Eligibility	Organization
Prediction and Evaluation of Anlotinib Treatment Response Using Contrast Enhanced Ultrasound PPGL Patients	NCT06015061	Pheochromocytom... Ultrasonography Paraganglioma, ... Pheochromocytom...	Contrast-enhanc...	18 Years - 80 Years	Peking Union Medical College Hospital
The Efficacy and Safety of Temozolomide in Patients With MPPGL	NCT05858177	Pheochromocytom... Paraganglioma, ... Pheochromocytom...	Temozolomide ca...	18 Years - 75 Years	Peking Union Medical College Hospital
Prediction and Evaluation of Anlotinib Treatment Response Using Contrast Enhanced Ultrasound PPGL Patients	NCT06015061	Pheochromocytom... Ultrasonography Paraganglioma, ... Pheochromocytom...	Contrast-enhanc...	18 Years - 80 Years	Peking Union Medical College Hospital
The Efficacy and Safety of Penpulimab in the Treatment of Metastatic PPGL Patients Who Fail to Other Systemic Treatment	NCT05885399	Pheochromocytom... Pheochromocytom... Paraganglioma, ...	Penpulimab	15 Years - 75 Years	Peking Union Medical College Hospital
The Efficacy and Safety of Penpulimab in the Treatment of Metastatic PPGL Patients Who Fail to Other Systemic Treatment	NCT05885399	Pheochromocytom... Pheochromocytom... Paraganglioma, ...	Penpulimab	15 Years - 75 Years	Peking Union Medical College Hospital
The Efficacy and Safety of Anlotinib in Patients With Metastatic Pheochromocytoma or Paraganglioma	NCT04860700	Malignant Adren... Malignant Parag... Pheochromocytom... Paraganglioma, ... Paraganglioma, ...	anlotinib hydro...	18 Years - 75 Years	Peking Union Medical College Hospital

Spots Global Cancer Trial Database for pheochromocytoma, metastatic