Neoplasms

All Conditions

Symptoms and General Pathology

Rare Diseases

Pheochromocytoma

Paraganglioma

Neoplasm Metastasis

Neuroendocrine Tumors

Neuroectodermal Tumors

Neuroectodermal Tumors, Primitive

Neoplasms, Germ Cell and Embryonal

Neoplasms by Histologic Type

Neoplasms, Nerve Tissue

Paragangliomas 1

Neuroendocrine Tumor

Neuroepithelioma

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

Anli Tong

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

Peking Union Medical College Hospital

Patients receive anlotinib hydrochloride 8-12mg orally once daily on days 1-14. Courses repeat every 21 days

Anlotinib hydrochloride

Yunying Cui

This phase II trial studies the effectiveness of anlotinib hydrochloride in the neoadjuvant therapy of locally advanced, or unresectable pheochromocytoma or paragangliom(PPGL). Anrotinib is used preoperatively in order to change unresectable tumors to resectable and reduce the high risk of surgery.

This prospective, single arm phase II study is designed to evaluate the efficacy of neoadjuvant therapy with anlotinib hydrochloride in locally advanced,or unresectable PPGL patients. Locally advanced,or unresectable PPGL patients receive anlotinib hydrochloride(10-12mg orally once daily on days 1-14, courses repeat every 21 days). Imaging examinations will be conducted after 4 courses to re-evaluate the surgical possibility. If the patient's tumor shrinks after 4 courses but is still unresectable, the patients will continue antirotinib therapy for another 4 courses.

PRIMARY OBJECTIVES:

The proportion of patients whose PPGL change from unresectable to resectable tumor.

SECONDARY OBJECTIVES:

To determine the objective response rate (ORR) . To determine the ratio of tumor shrinkage. To determine the biochemical response . To determine the R0 resection rate. To determine the Major pathological response rate (MPR). To determine the pathologic complete remission（pCR). To assess the safety of anlotinib treatment.

A Study on the Safety and Effectiveness of Anlotinib for Neoadjuvant Treatment of PPGL

A Study on the Safety and Effectiveness of Anlotinib for Neoadjuvant Treatment of Locally Advanced, or Unresectable Pheochromocytoma or Paraganglioma

The proportion of PPGL patients whose tumor change from unresectable to resectable

The proportion of decrease in the sum of total size of the target lesions after treatment compared to before treatment.

An effective response of 24hCA, MNs meant that the concentration decreaseed by more than 40% than the baseline value or decreaseed to the normal range.

The proportion of patients with surgical resection reached R0 resection

Defined as the remaining surviving tumor after surgical resection do not exceed 10% of the initial tumor tissue.

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

Study title	NCT ID	Conditions	Interventions	Eligibility	Organization	Link
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
A Study on the Safety and Effectiveness of Anlotinib for Neoadjuvant Treatment of PPGL	NCT05883085	Pheochromocytom... Paraganglioma	Anlotinib hydro...	18 Years - 75 Years	Peking Union Medical College Hospital

Spots Global Cancer Trial Database for pheochromocytoma or paraganglioma